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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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      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?

      31 Inherited Disorder Trials Near You

      Power is an online platform that helps thousands of Inherited Disorder 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

      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

      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

      Cannabidiol Gel for Fragile X Syndrome

      Cincinnati, Ohio
      This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of Cannabidiol administered as ZYN002 for the treatment of children, adolescent, and young adult patients with Fragile X Syndrome (FXS). Eligible participants will participate in up to an 18-week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to \< 30 years will be eligible to participate.
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:3 - 29

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Drug Abuse, Cardiovascular Disease, Others
      Must Be Taking:Antiseizure, Psychoactive

      250 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

      Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders

      Pittsburgh, Pennsylvania
      The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:2 - 55

      Key Eligibility Criteria

      Disqualifiers:Active Malignancy, Severe Aplastic Anemia, Others

      100 Participants Needed

      Natural History Study for Retinitis Pigmentosa

      Pittsburgh, Pennsylvania
      This is natural history study of rods and cones degenerations in patients with Retinitis Pigmentosa (RP) caused by pathogenic mutations in RHO, PDE6a or PDE6b gene mutations.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased

      Key Eligibility Criteria

      Disqualifiers:Other Gene Mutations, Ocular Disorders, Pregnancy

      82 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

      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

      Gene Therapy for Canavan Disease

      Chicago, Illinois
      The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.
      No Placebo Group

      Trial Details

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

      Key Eligibility Criteria

      Disqualifiers:Anti-AAV9 Antibodies, Prior Gene Therapy, Others
      Must Not Be Taking:Immunosuppressants, Anti-epileptics

      26 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

      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

      EIM Testing for Neuromuscular Disorders

      Bethesda, Maryland
      Background: \- Electrical impedance myography (EIM) is a new technique being studied to see if it is helpful in evaluating muscle disorders and nerve disorders. EIM looks at how a mild, painless electrical current travels through muscles. Researchers want to gain experience in using the EIM device. They will collect information on the results of using it on people with and without nerve and muscle diseases, and compare that with information from other standard tests. First, they will test the device on healthy people. Then they will test people with a variety of neuromuscular diseases. Because the test is noninvasive and not painful, researchers will test both children and adults. Objectives: \- To gain experience using the EIM muscle testing device. Eligibility: * Healthy volunteers at least 2 years old. * Individuals at least 2 years old who have neuromuscular disease. Design: * Participants will be screened with a medical history and physical exam. * Participants will have one 2-3 hour clinic visit. Researchers may request follow-up visits. * Participants will be tested with the EIM device. The device and small electrodes will be placed on their skin. An electric current will pass through the device, but the participants will not feel this. * Participants may have an ultrasound test. A gel will be put on their skin, and a device will be moved over the skin. * Participants may have a nerve test. Electrodes will be placed on their skin, and they will feel a small shock. * Participants may have a test where a thin needle is inserted in their muscle.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased
      Age:2+

      Key Eligibility Criteria

      Disqualifiers:Diabetes, Kidney Disease, Liver Disease, Others

      275 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

      Educational Video for Childhood Cancer

      Philadelphia, Pennsylvania
      This trial aims to develop and test an informational video about genetic testing for children and adolescents newly diagnosed with cancer or tumors. The video explains why both cancer tissue and normal tissue are tested, helping families understand the process and its benefits. By providing this information, the trial seeks to support families and improve their experience during a challenging time.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:12+

      Key Eligibility Criteria

      Disqualifiers:Not Meeting Inclusion Criteria

      359 Participants Needed

      eHealth Disclosure for Genetic Disorder Results

      Philadelphia, Pennsylvania
      The goal of this hybrid type 1 effectiveness-implementation study is to evaluate and compare different ways of delivering genetic research results to participants. The main questions the study aims to answer are: * Is sharing actionable genetic research results with participants through a multimedia patient-informed eHealth intervention (e.g. patient portal) no worse than sharing results by telephone or videoconference with a genetic counselor? * Will research participants access an eHealth educational intervention or chatbot education to learn about research results being offered and the option to decline learning their individual research results and how frequently participants choose to decline actionable research results? * Who benefits less and more from digital intervention with return of actionable research results and what barriers exist to using these tools for return of research results outside this study? Participants in the biobank will be offered digital tools to learn about research results being offered and the option to decline receiving these results. Those who don't decline and have an actionable result will be randomly assigned to receive their results with a genetic counselor or through an eHealth portal. Participants will complete surveys before and after receipt of results to understand patient experiences with these methods of education and return of results to determine if digital tools can be used to help ensure more patients get access to research results which could impact their health.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Unphased

      Key Eligibility Criteria

      Disqualifiers:Deceased, Prior Genetic Result, Others

      2500 Participants Needed

      Rifampin for Genetic Disorders Related to High Calcium Levels

      Philadelphia, Pennsylvania
      This study evaluates the efficacy of rifampin in the treatment of hypercalcemia and/or hypercalciuria in participants with at least one inactivating mutation of the CYP24A1 gene. Eligible subjects will receive rifampin for a total of 16 weeks during this study.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:6 - 65

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Breastfeeding, Hepatic Disease, Others
      Must Not Be Taking:CYP3A4 Inhibitors

      60 Participants Needed

      CD45RA Depleted Stem Cell Addback for Leukemia

      Philadelphia, Pennsylvania
      The major morbidities of allogeneic hematopoietic stem cell transplant (HSCT) using donors that are not human leukocyte antigen (HLA) matched siblings are graft vs host disease (GVHD) and life- threatening infections. T cell receptor alpha beta (TCRαβ) T lymphocyte depletion and CD19+ B lymphocyte depletion of alternative donor hematopoietic stem cell (HSC) grafts is effective in preventing GVHD, but immune reconstitution may be delayed, increasing the risk of infections. The central hypothesis of this study is that an addback of CD45RO memory T lymphocytes, derived from a fraction of the original donor peripheral stem cell product depleted of CD45RA naïve T lymphocytes, will accelerate immune reconstitution and help decrease the risk of infections in TCRab/CD19 depleted PSCT.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2
      Age:1 - 25

      Key Eligibility Criteria

      Disqualifiers:Hodgkin Lymphoma, Non-Burkitt Lymphoma, Others
      Must Not Be Taking:Alemtuzumab

      100 Participants Needed

      Fludarabine RIC for Bone Marrow Failure Syndrome

      Philadelphia, Pennsylvania
      This is a pilot study to determine whether fludarabine-based reduced intensity conditioning (RIC) regimens facilitate successful donor engraftment of patients with acquired aplastic anemia (AA) and Inherited bone marrow failure (iBMF) syndromes undergoing Matched related donor bone marrow transplant (MRD-BMT).
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Early Phase 1
      Age:< 22

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Infections, Myelodysplastic Syndrome, Others

      25 Participants Needed

      Cord Blood Transplant for Blood Cancers

      New York, New York
      This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:< 21

      Key Eligibility Criteria

      Disqualifiers:Advanced Metabolic Disease, CNS Leukemic, Others

      31 Participants Needed

      Why Other Patients Applied

      "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 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

      "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 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

      "I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."

      ZS
      Depression PatientAge: 51
      Match to a Inherited Disorder Trial

      Genetic Counseling for Inherited Disorders

      Minneapolis, Minnesota
      The goal of the GC-PRO study is to try to make genetic counseling better for people of all backgrounds. We are asking participants to complete two surveys and to allow audio recording of their genetic counseling visit. The purpose of the study is to understand whether trying different ways of doing genetic counseling will lead to better experiences for patients. The research team is also working with partners from the Somali, Latino/Hispanic, Black/African American, and Hmong communities to make sure the research is being done in a way that will benefit underserved communities.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased

      Key Eligibility Criteria

      Disqualifiers:Unable To Provide Consent, Others

      994 Participants Needed

      Stem Cell Transplantation for Osteopetrosis

      Minneapolis, Minnesota
      This trial tests a treatment using busulfan and fludarabine to prepare patients with certain genetic disorders for a bone marrow transplant. The goal is to safely replace their bone marrow with healthy donor cells by carefully monitoring drug levels. Busulfan is used to clear out bone marrow cells before the transplant, and fludarabine is being tested as a less harmful alternative to another drug.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:< 55

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Myeloablative Chemotherapy, Infections, Others

      100 Participants Needed

      Reduced Intensity Stem Cell Transplant for Blood Disorders

      Minneapolis, Minnesota
      This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) in patients with non-malignant or malignant diseases. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HCT.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased
      Age:< 55

      Key Eligibility Criteria

      Disqualifiers:Previous Irradiation, Pregnant, HIV Positive, Others

      30 Participants Needed

      Genetic Counseling for Genetic Disorders

      Boston, Massachusetts
      To learn about patient barriers to accessing genetic medicine, we will analyze anonymous posts from a membership-based online community \[Inspire.com\], and investigate how these barriers differ for various populations. We will then test whether these barriers can be addressed by providing online access to a genetic counselor to answer patient questions for one group of patients (virtual advisory board group) and compare to that of a control group who does not have access to a genetic counselor (virtual peer-to-peer board group).
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased

      Key Eligibility Criteria

      Disqualifiers:Not Member Of Inspire.com, Others

      70 Participants Needed

      Decitabine + Filgrastim for Acute Myeloid Leukemia

      Boston, Massachusetts
      The purpose of this study is to examine if it is feasible to administer decitabine and filgrastim after allogenic hematopoietic stem cell transplant (HCT) in children and young adults with myelodysplastic syndrome, acute myeloid leukemia and related myeloid disorders, and if the treatment is effective in preventing relapse after HCT. The names of the study drugs involved in this study are: * Decitabine (a nucleoside metabolic inhibitor) * Filgrastim (a recombinant granulocyte colony-stimulating factor (G-CSF)
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:1 - 39

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Illness, FLT3/ITD Mutations, Others

      37 Participants Needed

      Motivational Interviewing for High Cholesterol

      Duluth, Minnesota
      This trial aims to help patients with suspected Familial Hypercholesterolemia (FH) by using motivational interviews. These interviews educate patients about their health risks and encourage them to inform their family members. The goal is to improve disease management and early detection in relatives.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:2 - 75

      Key Eligibility Criteria

      Disqualifiers:Pregnancy, Prisoners, Untreated Hypothyroidism, Others
      Must Be Taking:Lipid-lowering

      300 Participants Needed

      Genome Sequencing for Genetic Disorders

      San Diego, California
      The goal of this clinical trial is to test a new method for newborn screening using whole genome sequencing, called BeginNGS. Parents will be approached to provide informed consent to enroll their newborns in prenatal, postnatal, and outpatient settings. The main questions this study aims to answer are: What is the utility of BeginNGS as compared to state newborn screening? What is the acceptability and feasibility of BeginNGS as compared to state newborn screening? What is the cost effectiveness of BeginNGS as compared to state newborn screening? Enrolled newborns will have a blood sample taken and will receive the BeginNGS test. Newborns will have also had the state newborn screening test.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased
      Age:1 - 28

      Key Eligibility Criteria

      Disqualifiers:Mother Under 18, Wards Of State, Non-US Address, Ill Neonates, Others

      10000 Participants Needed

      Antisense Oligonucleotide Treatment for Leukodystrophy

      San Diego, California
      This current study is aimed for the treatment of an individual participant with a form of TUBB4A-related leukodystrophy with hypomyelination.
      No Placebo Group

      Trial Details

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

      Key Eligibility Criteria

      Disqualifiers:Lumbar Puncture Contraindication, Others
      Must Not Be Taking:Investigational Drugs

      1 Participants Needed

      Personalized Antisense Therapy for Genetic Disorder

      San Diego, California
      This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single pediatric participant with SCN2A associated developmental epileptic encephalopathy
      No Placebo Group

      Trial Details

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

      Key Eligibility Criteria

      Disqualifiers:Others
      Must Not Be Taking:Investigational Drugs

      1 Participants Needed

      Gene Therapy for Hemophilia A

      Los Angeles, California
      This trial is testing a gene therapy called valoctocogene roxaparvovec in patients with severe haemophilia A who have developed antibodies against standard treatments. The therapy aims to provide a working version of the gene needed for proper blood clotting.
      No Placebo Group

      Trial Details

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

      Key Eligibility Criteria

      Disqualifiers:Active Infection, Immunosuppressive Disorder, Renal Dysfunction, Liver Dysfunction, Others
      Must Not Be Taking:Immune Tolerance Therapy

      10 Participants Needed

      12

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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
      Learn More About Trials
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      Frequently Asked Questions

      How much do Inherited Disorder 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 Inherited Disorder 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 Inherited Disorder 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 Inherited Disorder 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 Inherited Disorder 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 Inherited Disorder clinical trials?

      Most recently, we added Antisense Oligonucleotide Treatment for Leukodystrophy, Genome Sequencing for Inherited Disorders and CD45RA Depleted Stem Cell Addback for Leukemia to the Power online platform.