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Why We Started Power

We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

Bask
Bask GillCEO at Power
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      Clear All
      Why We Started Power

      We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

      Bask
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

      60 Genetic Disorders Trials Near You

      Power is an online platform that helps thousands of Genetic Disorders patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

      Learn More About Power
      No Placebo
      Highly Paid
      Stay on Current Meds
      Pivotal Trials (Near Approval)
      Breakthrough Medication

      AOC 1001 for Myotonic Dystrophy

      Columbus, Ohio
      This trial is testing a new medicine called AOC 1001 to see if it is safe and effective for adults with a muscle disease called Myotonic Dystrophy Type 1. The medicine is given through an IV, and researchers want to know if it helps muscles work better.

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Breastfeeding, New Conditions, Others

      37 Participants Needed

      EDIT-301 for Sickle Cell Disease

      Columbus, Ohio
      This trial is testing a new treatment called EDIT-301 for people aged 12 to 50 with severe sickle cell disease. The treatment uses the patient's own stem cells, which are changed in a lab and then put back into their body to help fight the disease.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:12 - 50

      Key Eligibility Criteria

      Disqualifiers:HLA-matched Donor, Prior HSCT, Vasculopathy, Malignancy, Others
      Must Not Be Taking:Hydroxyurea, Voxelotor, Crizanlizumab, L-glutamine

      45 Participants Needed

      Gene Therapy for Hemophilia A

      Columbus, Ohio
      This trial tests a new gene therapy combined with anti-inflammatory medication for patients with severe hemophilia A. The treatment aims to help their bodies produce a necessary clotting protein and reduce inflammation.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Liver Disease, Hepatitis B, Hepatitis C, Others
      Must Be Taking:Prophylactic Corticosteroids

      22 Participants Needed

      Gene Therapy for Rett Syndrome

      Columbus, Ohio
      This trial tests NGN-401, a gene therapy using a virus to deliver a healthy gene, in female children with Rett syndrome. It aims to improve brain cell function by providing the correct version of a missing or faulty gene.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:3 - 10
      Sex:Female

      Key Eligibility Criteria

      Disqualifiers:Normal Hand Function, Other Conditions, Others
      Must Be Taking:Anti-epileptics

      33 Participants Needed

      Cell Therapy for Duchenne Muscular Dystrophy

      Cincinnati, Ohio
      This trial tests a cell therapy called CAP-1002 in boys and young men with Duchenne muscular dystrophy. The therapy involves giving special cells through an IV to help improve muscle function. The goal is to see if this treatment can repair or regenerate damaged muscles.
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:10+
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Low LVEF, High BMI, Others
      Must Be Taking:Glucocorticoids

      104 Participants Needed

      Genomic Risk Assessment for Genetic Disorders

      Cincinnati, Ohio
      The eMERGE Network embraces the opportunity to use new methods in genomic medicine, information science, and research participant engagement to identify people at very high risk for specific diseases and recommend individualized approaches to prevention and care. The investigators will conduct a prospective study, with diverse and underserved participants, across ten eMERGE study sites to evaluate clinical implementation of a Genome Informed Risk Assessment (GIRA) tool that combines genetic, family history, and clinical risk information from participants.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:3 - 75

      Key Eligibility Criteria

      Disqualifiers:Transplant, Transfusion, Research Staff, Others

      26877 Participants Needed

      Combination Therapy for Soft Tissue Sarcoma

      Cincinnati, Ohio
      The study participant has been diagnosed with non-rhabdomyosarcoma (NRSTS). Primary Objectives Intermediate-Risk * To estimate the 3-year event-free survival for intermediate-risk patients treated with ifosfamide, doxorubicin, pazopanib, surgery, and maintenance pazopanib, with or without RT. * To characterize the pharmacokinetics of pazopanib and doxorubicin in combination with ifosfamide in intermediate-risk participants, to assess potential covariates to explain the inter- and intra-individual pharmacokinetic variability, and to explore associations between clinical effects and pazopanib and doxorubicin pharmacokinetics. High-Risk * To estimate the maximum tolerated dose (MTD) and/or the recommended phase 2 dosage (RP2D) of selinexor in combination with ifosfamide, doxorubicin, pazopanib, and maintenance pazopanib in high-risk participants. * To characterize the pharmacokinetics of selinexor, pazopanib and doxorubicin in combination with ifosfamide in high-risk participants, to assess potential covariates to explain the inter- and intra-individual pharmacokinetic variability, and to explore associations between clinical effects and selinexor, pazopanib and doxorubicin pharmacokinetics. Secondary Objectives * To estimate the cumulative incidence of primary site local failure and distant metastasis-free, disease-free, event-free, and overall survival in participants treated on the risk-based treatment strategy defined in this protocol. * To define and describe the CTCAE Grade 3 or higher toxicities, and specific grade 1-2 toxicities, in low- and intermediate-risk participants. * To study the association between radiation dosimetry in participants receiving radiation therapy and the incidence and type of dosimetric local failure, normal adjacent tissue exposure, and musculoskeletal toxicity. * To evaluate the objective response rate (complete and partial response) after 3 cycles for high-risk patients receiving the combination of selinexor with ifosfamide, doxorubicin, pazopanib, and maintenance pazopanib. * To assess the relationship between the pharmacogenetic variation in drug-metabolizing enzymes or drug transporters and the pharmacokinetics of selinexor, pazopanib, and doxorubicin in intermediate- or high-risk patients. Exploratory Objectives * To explore the correlation between radiographic response, pathologic response, survival, and toxicity, and tumor molecular characteristics, as assessed through next-generation sequencing (NGS), including whole genome sequencing (WGS), whole exome sequencing (WES), and RNA sequencing (RNAseq). * To explore the feasibility of determining DNA mutational signatures and homologous repair deficiency status in primary tumor samples and to explore the correlation between these molecular findings and the radiographic response, survival, and toxicity of patients treated on this protocol. * To explore the feasibility of obtaining DNA methylation profiling on pretreatment, post-induction chemotherapy, and recurrent (if possible) tumor material, and to assess the correlation with this and pathologic diagnosis, tumor control, and survival outcomes where feasible. * To explore the feasibility of obtaining high resolution single-cell RNA sequencing of pretreatment, post-induction chemotherapy, and recurrent (if possible) tumor material, and to characterize the longitudinal changes in tumor heterogeneity and tumor microenvironment. * To explore the feasibility of identifying characteristic alterations in non-rhabdomyosarcoma soft tissue sarcoma in cell-free DNA (cfDNA) in blood as a non-invasive method of detecting and tracking changes during therapy, and to assess the correlation of cfDNA and mutations in tumor samples. * To describe cardiovascular and musculoskeletal health, cardiopulmonary fitness among children and young adults with NRSTS treated on this protocol. * To investigate the potential prognostic value of serum cardiac biomarkers (high-sensitivity cardiac troponin I (hs-cTnI), N-terminal pro B-type natriuretic peptide (NT-Pro-BNP), serial electrocardiograms (EKGs), and serial echocardiograms in patients receiving ifosfamide, doxorubicin, and pazopanib, with or without selinexor. * To define the rates of near-complete pathologic response (\>90% necrosis) and change in FDG PET maximum standard uptake value (SUVmax) from baseline to week 13 in intermediate risk patients with initially unresectable tumors treated with induction pazopanib, ifosfamide, and doxorubicin, and to correlate this change with tumor control and survival outcomes. * To determine the number of high-risk patients initially judged unresectable at diagnosis that are able to undergo primary tumor resection after treatment with ifosfamide, doxorubicin, selinexor, and pazopanib. * To identify the frequency with which assessment of volumes of interest (VOIs) of target lesions would alter RECIST response assessment compared with standard linear measurements.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:< 30

      Key Eligibility Criteria

      Disqualifiers:CNS Metastases, Uncontrolled Hypertension, Others
      Must Not Be Taking:CYP3A4 Substrates, Inhibitors, Inducers

      139 Participants Needed

      IC14 for Arrhythmogenic Cardiomyopathy

      Cleveland, Ohio
      The goal of this clinical trial is to test IC14 (atibuclimab) in patients with arrhythmogenic cardiomyopathy (ACM) and who have an implantable cardoverter/defibrillator in place. ACM is also called arrhythmogenic right ventricular dysplasia (ARV) or arrhythmogenic right ventricular cardiomyopathy (ARVC). The main questions the study aims to answer are the effect of treatment on blood markers of inflammation, safety, and pharmacokinetics. There will also be measurements of myocardial imaging of C-C chemokine receptor type 2 (CCR2+) immune cells (optional), monitoring of cardiac arrhythmias using the patient's pre-existing intracardiac cardioverter/defibrillator (ICD) and a Holter monitor, electrocardiogram (ECG), echocardiogram (ECHO), and blood tests. Results will be compared to baseline; there is no inactive placebo treatment group. Participants will be asked to undergo screening and baseline testing, then receive 4 intravenous infusions with blood measurements before and after the infusion (including 24, 48, and 72 hours and 7, 14, and 28 days). Participants will be offered specialized scanning of the heart muscle, and will be asked to provide recordings from their ICD, undergo Holter monitoring twice, and have electrocardiograms (ECG), echocardiograms (ECHO) and blood tests.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2

      Key Eligibility Criteria

      Disqualifiers:Myocardial Infarction, Heart Failure, Others
      Must Not Be Taking:Antiarrhythmics, High-dose Corticosteroids, DMARDS

      5 Participants Needed

      AI Chatbot for Prenatal Disorder

      Cleveland, Ohio
      The goal of this study is to ensure that pregnant patients have the resources and support needed to access Prenatal Screening \& Diagnostic Testing (PS\&D) in an informed and evidence-based fashion by developing an innovative digital tool to support patients' decision-making and contributing fundamental knowledge to advance science in a way that promotes patients' access to new prenatal applications of genomic science and technology. Our central hypothesis is that, by focusing on patient engagement as a key driver to improve patient outcomes, the use of an evidence-based artificial-intelligence (AI) powered patient engagement tool will increase patients' ability to seek information and structure a decision-making process that, in turn, increases informed decisions about PS\&D and decreases decisional conflict associated with those decisions. Using data from NEST (Ensuring Patients Informed Access to NIPT \[non-invasive prenatal testing\]), the investigators designed the next iteration of NEST, a point-of care shared decision-making tool powered by artificial intelligence (AI) to provide a personalized and dynamic decision support tool: Obstetric Prenatal Genetic Testing Engagement Solution (OPUS). OPUS is an AI-enabled healthcare chatbot (a computer program capable of processing and simulating human conversation) that provides patients with personalized information and decision-making support at different stages of the PS\&D pathway. It functions using a series of questions contained in the NEST with a branching logic sequence of questions and answers based on the responses to and from the patient, using a conversational and adaptable interaction. It also contains nested tiers of information, ranging from introductory to detailed information about patient engagement, health literacy, the different PS\&D options, and resources to learn about insurance coverage for PS\&D. OPUS was designed to be accessed by patients with different technological resources and preferences, using a cell phone, a mobile device, or a computer.

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased
      Age:18 - 50
      Sex:Female

      Key Eligibility Criteria

      Disqualifiers:Under 18, Not Pregnant, Others

      600 Participants Needed

      Neonate WGS Testing for Genetic Disorders

      Pittsburgh, Pennsylvania
      The purpose of this study is to understand how the use of whole genome sequencing (WGS) may be able to increase the speed with which a diagnosis is made for patients in an intensive care unit population. This is not an assessment of a new device, test, or technology. This project is an investigation of the utility of this technology in clinical care when compared to standard of care testing. The study will look at the ability to more quickly diagnose a patient (time to diagnosis and efficacy of testing) as compared to standard of care testing. The study will also look at the impact of WGS on patient outcomes and cost of clinical care.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Unphased
      Age:< 1

      Key Eligibility Criteria

      Disqualifiers:Known Diagnosis, Major Anomaly, Infection, Others

      400 Participants Needed

      Gene Therapy for Leber Congenital Amaurosis

      Ann Arbor, Michigan
      The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A\>G in intron 26 of the CEP290 gene ("LCA10-IVS26").
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:3+

      Key Eligibility Criteria

      Disqualifiers:Other Mutations, Infections, Steroid Response, Others
      Must Not Be Taking:Corticosteroids

      34 Participants Needed

      Gene Therapy for Fabry Disease

      Pittsburgh, Pennsylvania
      This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2

      Key Eligibility Criteria

      Disqualifiers:Kidney Transplant, HIV, Diabetes, Others
      Must Be Taking:ERT

      18 Participants Needed

      Gene Therapy for Krabbe Disease

      Ann Arbor, Michigan
      This trial tests a single dose of a virus carrying a healthy gene in patients with severe forms of Krabbe disease. The virus helps by delivering the healthy gene to the patient's cells. Gene therapy has shown promise in extending survival in previous studies.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:< 18

      Key Eligibility Criteria

      Disqualifiers:HIV, GvHD, Infections, Others
      Must Not Be Taking:Immunosuppressive Drugs

      9 Participants Needed

      Genome Sequencing for Newborn Screening

      Royal Oak, Michigan
      This research study is exploring the use of genomic sequencing in the newborn period to screen healthy babies for current and future health risks. The study will enroll a diverse cohort of 500 healthy infants and their parents from Boston, MA; New York City, NY; and Birmingham, AL. A small blood sample will be collected from each infant, and whole genome sequencing will be performed in 1/2 of the cohort following a randomized controlled trial design. 3 months later, the randomization status and sequencing results will be shared with parents and pediatricians. Investigators will study the medical, behavioral, and economic outcomes of genomic sequencing to better understand how this technology can be implemented in outpatient primary care settings.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased
      Age:0 - 12

      Key Eligibility Criteria

      Disqualifiers:Unwilling To Share Genomic Reports, Others

      500 Participants Needed

      Family Health History Education for Hereditary Diseases in the Black Community

      Flint, Michigan
      The understanding, utilization and uptake of Family Health History is essential to the prevention of health disparities in the African American community. Creating a culturally tailored Family Health History tool, co-developed by members of the African American community will inform, educate and empower African Americans about health issues related to their family genealogy. Applying the knowledge gained via Family Health Histories to increase preventative behaviors including screenings thus linking people to needed health services to prevent the onset of disease and illness.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased

      Key Eligibility Criteria

      Disqualifiers:Non English Speaking, Non African American, Others

      100 Participants Needed

      Wheelchair Skills Training for Mobility Impairments

      Grand Rapids, Michigan
      This trial tests a training program called Skills on Wheels, which helps children with physical disabilities learn to use manual wheelchairs on their own. The goal is to make them more confident and safe in using their wheelchairs, and to improve their social skills and participation in activities.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:5 - 17

      Key Eligibility Criteria

      Disqualifiers:Unsafe Wheelchair, Safety Concerns, Others

      4 Participants Needed

      Powered Wheelchair Standing Devices for Developmental Delay

      Grand Rapids, Michigan
      For children who use a power wheelchair, a powered wheelchair standing device (PWSD) may be considered for daily use. A PWSD allows a child to electronically move between sitting and standing and can be driven in either position. Existing published PWSD research in pediatrics is limited to boys with Duchenne muscular dystrophy (DMD).(1, 2) While these studies provide some insights into PWSD use in boys with DMD, they do not reflect PWSD use in children with other conditions. The purpose of this exploratory study is to determine the feasibility of a research protocol exploring use of a PWSD in children who have neurodevelopmental conditions other than DMD.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:5 - 17

      Key Eligibility Criteria

      Disqualifiers:Duchenne Muscular Dystrophy, Others

      4 Participants Needed

      CRISPR-Cas9 Gene Editing for Beta Thalassemia

      Chicago, Illinois
      This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2, 3
      Age:12 - 35

      Key Eligibility Criteria

      Disqualifiers:Prior Allo-HSCT, Sickle Cell, Others

      59 Participants Needed

      Dupilumab for Itchy Skin Diseases

      Chicago, Illinois
      Severe itch is a common symptom of many genetic skin disorders and leads to a negative impact on patient quality of life. The investigators hypothesize that: a) intervention with dupilumab will improve itch in patients with pruritic genetic inflammatory skin disorders, even those not recognized to be Th2-driven; and b) the administration of dupilumab will be well-tolerated, regardless of underlying genetic skin disorder. The total clinical study duration will be 26 months (104 Weeks). The treatment period will include a 16-week open-label phase and a 20-month long-term extension phase for those who qualify and wish to continue.
      No Placebo Group
      Prior Safety Data

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Early Phase 1
      Age:6+

      Key Eligibility Criteria

      Disqualifiers:Ichthyosis Vulgaris, Active Infections, Malignancies, Others
      Must Not Be Taking:Immunosuppressives, Biologics, Retinoids, Others

      30 Participants Needed

      ECUR-506 for Ornithine Transcarbamylase Deficiency

      Chicago, Illinois
      Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build-up excess levels of ammonia in their blood, potentially resulting in devastating consequences, including cumulative and irreversible neurological damage, coma and death. The severe form of the condition emerges shortly after birth and is more common in boys than girls. This is a Phase 1/2/3, open-label, multicenter, safety, efficacy, and dose finding study of ECUR-506 in male babies with neonatal onset OTC deficiency. The primary objective of this study is to evaluate the safety, tolerability, and efficacy of up to three dose levels of ECUR-506 following intravenous (IV) administration of a single dose.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:24 - 7
      Sex:Male

      Key Eligibility Criteria

      Disqualifiers:Hypoxic Ischemic Encephalopathy, Liver Failure, Others
      Must Not Be Taking:Gene Therapy

      8 Participants Needed

      Why Other Patients Applied

      "My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."

      HZ
      Arthritis PatientAge: 78

      "I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."

      WR
      Obesity PatientAge: 58

      "As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."

      IZ
      Healthy Volunteer PatientAge: 38

      "I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."

      AG
      Paralysis PatientAge: 50

      "I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."

      ID
      Pancreatic Cancer PatientAge: 40
      Match to a Genetic Disorders Trial

      Gene Therapy (TSHA-102) for Rett Syndrome

      Chicago, Illinois
      This trial is testing a new gene therapy called TSHA-102 in adult women with Rett syndrome. The therapy aims to fix the genetic problems causing the disorder by adding healthy genes to their cells. Researchers will study its safety and effectiveness over several years. TSHA-102 is a gene therapy aimed at addressing the genetic deficiencies in Rett syndrome by adding healthy MECP2 genes to the cells.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:6 - 21
      Sex:Female

      Key Eligibility Criteria

      Disqualifiers:Other Neurodevelopmental Disorders, Brain Injury, Atypical Rett, Others

      15 Participants Needed

      L-Phenylalanine for Phenylketonuria Carriers

      Guelph, Ontario
      This is a clinical intervention study of PKU carriers (cases) and non-carriers (controls). Upon completing the informed consent process, participants will complete baseline measures of chronic mental health prior to the intervention (PHQ-9, GAD-7, BIS-11). Participants will attend the Human Nutraceutical Research Unit (HNRU) at the University of Guelph, fasted, and first undergo baseline measures of cognition and acute mental health (mood) and provide samples or saliva, urine and dried blood spots to evaluate phenylalanine (Phe), tyrosine (Tyr) and their metabolites (PAH pathway functioning) as well as for genetic testing of the PAH gene. Participants will also complete a brief questionnaire which will include age, sex, ethnicity, income, weight and height (measured using a stadiometer and calibrated weigh scale), and confirmation that participants arrived to the lab fasted (i.e. have only had water to drink and no other foods/ beverages prior to analyses). Blood pressure and heart rate will also be measured at baseline. Following baseline tests, participants will consume a pure L-Phe supplement dosed at 100 mg/kg mixed with 125 mL of water and 125mL of orange juice. Blood pressure and heart rate will be repeated at 1-hour post-L-Phe consumption. Two-hours postprandial, participants will repeat the cognitive tests and acute mental health (mood) assessment, blood pressure and heart rate measurement and provide follow-up saliva, urine and dried blood spot samples. Participants will also be asked to report any side effects they experienced with the L-Phe consumption.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased

      Key Eligibility Criteria

      Disqualifiers:PKU, Neurodegenerative Conditions, Hypertension, Others
      Must Not Be Taking:Monoamine Oxidase Inhibitors

      50 Participants Needed

      Long-Term DCR-PHXC Treatment for Primary Hyperoxaluria

      Hamilton, Ontario
      This trial aims to provide ongoing access to the treatment DCR-PHXC for patients who were part of earlier studies and their younger siblings. It will also check if the treatment is safe and effective over an extended period. The treatment is intended for people with a condition called Primary Hyperoxaluria (PH). DCR-PHXC is designed to inhibit the enzyme responsible for the final step of oxalate production.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:6+

      Key Eligibility Criteria

      Disqualifiers:Renal Transplant, Hepatic Transplant, Dialysis, Others

      75 Participants Needed

      VX-670 for Myotonic Dystrophy

      Winston-Salem, North Carolina
      The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:18 - 64

      Key Eligibility Criteria

      Disqualifiers:Illness, Clinical Condition, Others

      36 Participants Needed

      Gene Therapy for Chronic Granulomatous Disease

      Bethesda, Maryland
      Background: Chronic granulomatous disease (CGD) is a genetic disorder. People with CGD are missing a gene that affects their white blood cells. White cells are part of the immune system, and people with GCD are vulnerable to many infections. Researchers want to test a new treatment to replace the missing gene that may be safer than the current treatment for CGD. Objective: To test a new type of gene therapy in people with CGD. Eligibility: People aged 3 years or older with CGD. Design: Participants will undergo apheresis: Blood will be collected through a tube attached to a needle inserted in a vein; the blood will run through a machine that separates certain cells (stem cells); the remaining blood will be returned to the body through a second needle. The participant s stem cells will be modified in a laboratory to add the gene they are missing. Participants will stay in the hospital for about 40 days. For the first 10 days, they will undergo many exams, including imaging scans and tests of their heart and lung function. They will receive drugs to prepare their bodies for the gene therapy. They will receive a "central line": A hollow tube will be inserted into a vein in the chest, with a port opening above the skin. This port will be used to draw blood and administer drugs without the need for new needle sticks. For the gene therapy, each participant s own modified stem cells will be put into their body through the port. Participants will have 8 follow-up visits over 3 years.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 1, 2
      Age:3 - 65

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, HIV, Hepatitis, Anemia, Others
      Must Not Be Taking:Gamma-interferon

      10 Participants Needed

      CRISPR Therapy (CTX001) for Thalassemia

      Toronto
      This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:2 - 11

      Key Eligibility Criteria

      Disqualifiers:Prior HSCT, Sickle Cell Variant, Others

      15 Participants Needed

      mRNA-3745 for Glycogen Storage Disease

      Toronto, Ontario
      This trial is testing a new treatment called mRNA-3745, which uses messenger RNA to help the body produce a missing protein. It is aimed at adults and children with Glycogen Storage Disease Type Ia (GSD1a). The study will check if the treatment is safe and how well it works.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:6+

      Key Eligibility Criteria

      Disqualifiers:Solid Organ Transplant, Diabetes, Others

      45 Participants Needed

      Genome Sequencing for Inherited Disorders

      Toronto, Ontario
      Individually rare genetic diseases are collectively common, and affect many Canadian families. Making the right diagnosis is both important and challenging. Healthcare providers and families often remain in the dark for too long, limited by the scope and speed of current genetic testing. The goal of this clinical trial is to learn if performing genome sequencing (a comprehensive genetic test) as soon as a rare genetic disease is suspected is more effective than usual care, where a person waits to see a genetics specialist and then typically gets offered more targeted testing. Researchers will compare a "genome-sequencing first" approach to the standard-of-care in individuals who were referred to the Genetics Clinic at either SickKids or CHEO and recently had their referral accepted by the clinic. The main questions this clinical trial aims to answer are: 1. Are there more and faster diagnoses with a "genome sequencing first" approach compared to standard-of-care? 2. What do patients, families, and healthcare providers think about a "genome sequencing first" approach compared to standard-of-care? 3. What is the financial impact of a "genome sequencing first" approach compared to standard-of-care on the healthcare system? Participants will be asked to: * Let us review their medical records. * Complete up to 5 questionnaires over the course of the study. * Give a blood sample for clinical genome sequencing (if in the genome sequencing first group). This study aims to provide the robust evidence needed to improve care pathways for rare disease diagnosis in Canada. The findings also promise to help translate new genetic technologies into the clinic. Earlier diagnosis is a key first step towards personalized care, targeted treatments, and better outcomes.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Unphased
      Age:< 18

      Key Eligibility Criteria

      Disqualifiers:Clinical Diagnosis, Urgent Referral, Previous Sequencing, Others

      200 Participants Needed

      Fluoxetine for Genetic Disorder

      Toronto, Ontario
      This is a single patient study of oral powdered fluoxetine to target developmental outcomes in a child with KCNC1-related disorder. This trial will be conducted at Holland Bloorview Kids Rehabilitation Hospital over 32 to 42 weeks, using a quasi experimental ABA phase design (placebo-fluoxetine-placebo) with randomized and blinded active treatment start and stop moments.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Unphased
      Age:< 18

      Key Eligibility Criteria

      Disqualifiers:Hypersensitivity To Fluoxetine, Long QT, Others
      Must Not Be Taking:MAOIs, SSRIs, Tricyclics, Others

      1 Participants Needed

      CTX001 for Sickle Cell Disease and Thalassemia

      Nashville, Tennessee
      This trial tests a new treatment where a patient's own blood stem cells are modified to fix faulty genes. It targets patients with severe blood disorders who need frequent transfusions. The goal is to help their bodies produce healthy blood cells. Recent advances in treatment methods expand the potentially curative options for patients.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:12 - 35

      Key Eligibility Criteria

      Disqualifiers:Prior HSCT, Active Infection, Others

      26 Participants Needed

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      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?
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      Frequently Asked Questions

      How much do Genetic Disorders clinical trials pay?

      Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

      How do Genetic Disorders clinical trials work?

      After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Genetic Disorders trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Genetic Disorders is 12 months.

      How do I participate in a study as a "healthy volunteer"?

      Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

      What does the "phase" of a clinical trial mean?

      The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

      Do I need to be insured to participate in a Genetic Disorders medical study?

      Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

      What are the newest Genetic Disorders clinical trials?

      Most recently, we added N-Acetylcysteine for Cornelia de Lange Syndrome, Antisense Oligonucleotide Treatment for Leukodystrophy and Transcranial Direct Current Stimulation for Dementia to the Power online platform.