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62 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 PowerAOC 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
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
MAS825 for Autoinflammatory Diseases
Cincinnati, Ohio
This trial is testing a new medication called MAS825 in patients with rare genetic conditions that don't respond well to typical treatments. The study will take several years to evaluate the medication's effectiveness and safety. The goal is to see if MAS825 can help manage these conditions by targeting the underlying genetic problems.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:0 - 100
Key Eligibility Criteria
Disqualifiers:HIV, Hepatitis B/C, Tuberculosis, Others
Must Not Be Taking:Anti-rejection, Immunomodulatory
17 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
Ultevursen for Retinitis Pigmentosa
Ann Arbor, Michigan
The purpose of this Phase 2b study is to evaluate the safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:8+
Key Eligibility Criteria
Disqualifiers:Non-exon 13 Mutations, Other Retinal Diseases, Unstable CME, Others
Must Not Be Taking:Carbonic Anhydrase Inhibitors
81 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
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:Recruiting
Age:5 - 17
Key Eligibility Criteria
Disqualifiers:Not Listed
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
Gene Therapy for Rett Syndrome
Chicago, Illinois
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
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:4 - 10
Sex:Female
Key Eligibility Criteria
Disqualifiers:Normal Hand Function, Other Conditions, Others
Must Be Taking:Anti-epileptics
14 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
Why Other Patients Applied
"I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."
FF
ADHD PatientAge: 31
"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
"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 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
"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
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
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
Antibiotics and Bleach Baths for Eczema
Bethesda, Maryland
Background:
- Atopic dermatitis, or eczema, is a chronic skin disorder. Patients sometimes have infections with S. aureus bacteria. Researchers want to study how eczema treatments affect the number and the type of bacteria on the skin.
Objectives:
- To study the effect of eczema treatments on skin bacteria.
Eligibility:
* Individuals between 2 and 25 years of age who have moderate to severe atopic dermatitis.
* Healthy volunteers between 18 and 40 years of age with no history of eczema.
Design:
* Participants will be screened with a physical exam and medical history. Research samples will be collected. Skin biopsies may also be performed.
* All participants will be assigned to one of several study groups.
* Healthy volunteers must not have taken antibiotics in the year before the start of the study.
* All participants will have regular study visits during their 1-year participation. More research samples will be collected at these visits.
* Healthy volunteers may be asked to come in for a one-time follow up after the 1 year mark.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:2 - 50
Key Eligibility Criteria
Disqualifiers:Pregnancy, Immunodeficiency, Chronic Illnesses, Others
Must Not Be Taking:Antibiotics, Corticosteroids, CAM Agents
130 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
Clinical Decision Support for Genetic Disorders
Nashville, Tennessee
This study will evaluate the effectiveness of SIGHT as a clinical support system to prompt provider/patient discussion and shared decision making regarding the need for genetic testing in the form of a chromosomal microarray. Identifying patients at high predicted probability of needing a test in clinical settings will be examined to determine if it decreases the duration of time to testing and increases diagnostic yield. SIGHT requires only data already collected in routine clinical encounters and is calculated prior to a clinical visit at VUMC.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:1 - 20
Key Eligibility Criteria
Disqualifiers:Age, Prior Chromosomal Microarray
1000 Participants Needed
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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 GillCEO at Power
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, Sepofarsen for Leber Congenital Amaurosis and Gene Therapy for Spastic Paraplegia to the Power online platform.
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