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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?

      13 Friedreichs Ataxia Trials Near You

      Power is an online platform that helps thousands of Friedreichs Ataxia 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

      SGT-212 Gene Therapy for Friedreich's Ataxia

      Columbus, Ohio
      The purpose of this study is to evaluate the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212. It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA. All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.
      No Placebo Group

      Trial Details

      Trial Status:Not Yet Recruiting
      Trial Phase:Phase 1
      Age:18 - 40

      Key Eligibility Criteria

      Disqualifiers:Antibodies AAV9, BMI ≥33, Diabetes, Others

      10 Participants Needed

      ASP2016 + Prednisolone for Friedreich Ataxia

      Cincinnati, Ohio
      Friedreich Ataxia is a rare condition that causes damage to the nervous system and muscles. People with Friedreich Ataxia have difficulty walking, lose sensation in their arms and legs, and have slurred speech. It can also affect the heart and many people with Friedrich Ataxia develop serious heart problems. Friedreich Ataxia is a genetic condition which means a faulty gene is passed down through families. This type of gene therapy treats a genetic condition by providing a healthy copy of the gene. At the time this study started, there was no approved treatment for heart problems in people with Friedreich Ataxia. In this study, ASP2016 is being tested in humans for the first time. The people taking part are adults with Friedreich Ataxia who have heart problems. The main aims of the study are to check the safety of ASP2016 and how people cope with (tolerate) ASP2016. ASP2016 is given as a slow injection into a vein. This is called an infusion. People will also take tablets of a medicine called prednisolone. This is taken to stop the immune system interfering with ASP2016. Each person in the study will be given 1 single infusion of ASP2016. Different small groups will receive lower or higher doses of ASP2016. Each person will stay overnight in the clinic for at least 1 night after their infusion. For the first few months, people will visit the clinic regularly. There may be the option of home visits by a study nurse at some visits. At the 6-month and 12-month visits extra tests, procedures, and scans will be done. One of these is an ECHO (echocardiogram) scan. This is like an ultrasound scan for the heart. Another is an endomyocardial biopsy. A tiny piece of their heart tissue is removed (biopsy). A flexible hollow tube (catheter) goes into the blood vessels up to the heart. Then, a small device on the end of the catheter takes a tiny piece of heart tissue (about the size of a pencil tip). Another is a cardiac MRI. This takes pictures of the inside of the heart using a powerful magnet. Another is a cardiopulmonary exercise test (CPET). This involves moving a specially designed set of bicycle pedals using hands and arms. This will check how the lungs, heart and muscles are affected during exercise. After the 12-month visit, people will visit the clinic every few months for up to a few years.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Age:18 - 40

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      14 Participants Needed

      Gene Therapy for Cardiomyopathy in Friedreich's Ataxia

      Philadelphia, Pennsylvania
      This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2
      Age:18 - 50

      Key Eligibility Criteria

      Disqualifiers:Diabetes, Liver Dysfunction, Infections, Others
      Must Not Be Taking:Corticosteroids, Immunosuppressives

      8 Participants Needed

      Omaveloxolone for Friedreich's Ataxia

      Philadelphia, Pennsylvania
      In this study, researchers will learn more about BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedrich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 is processed in the body of children and teens who are 2 to 15 years old. The main question researchers want to answer in this study is: * How does the body process BIIB141 in children and teens? * How many participants have medical problems during the study? * Are there any changes in the participants' overall health during the study? * Are there any changes in the participants' heart health? * Are there any changes in how the participants move through puberty? Puberty is the time in someone's life when their body changes from a child to an adult. This study will be done as follows: * Participants will be screened to see if they can join the study. The screening period will be up to 14 days, after which participants will check into their study research center. * There are 2 parts to this study. During Part 1, participants will take a single dose of BIIB141. Participants will be in 1 of 7 different groups based on their age: * Group A1: 12 to 15 years old, taking 150 milligrams (mg) of BIIB141 * Group A2: 12 to 15 years old, taking a dose of BIIB141 based on the data from Group A1 * Group B1: 7 to 11 years old, taking a dose of BIIB141 based on Group A1 data * Group C1: 2 to 6 years old, taking a dose of BIIB141 based on Groups A1, A2, and B1 data * Group A3: 12 to 15 years old, taking a dose of BIIB141 based on Groups A1, A2, and B1 data * Group B2: 7 to 11 years old, taking a dose of BIIB141 based on Groups A1, A2, and B1 data * Group C2: 2 to 6 years old, taking a dose of BIIB141 based on Group A1, A2, A3, B1, B2, and C1 data. * During Part 2, participants from Part 1 will take BIIB141 once in the study research center. Cohort A1 will take 150 mg of BIIB141. Dose of Cohorts A2 and B1 will be based on data from Cohort A1, dose of Cohorts C1, A3 and B2 will be based on data from Cohorts A1, A2 and B1, while Cohort C2's dose will be based on all the other groups. Participants will then take it once a day at home. * After leaving the study research center in Part 2, participants will return for tests at Week 4, Week 12, Week 24, and then every 24 weeks. Participants will also be contacted by telephone at Week 2, Week 8, and Week 18. * Participants will be in this study for up to 240 weeks.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1
      Age:2 - 15

      Key Eligibility Criteria

      Disqualifiers:Uncontrolled Diabetes, Heart Disease, Liver Disease, Others
      Must Not Be Taking:CYP3A4 Inhibitors, CYP3A4 Inducers

      35 Participants Needed

      CTI-1601 for Friedreich's Ataxia

      Columbus, Ohio
      This is an open-label extension (OLE) study designed to evaluate the long-term safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of subcutaneous (SC) administration of CTI-1601, also known as nomlabofusp, in subjects with Friedreich's ataxia (FRDA). The objectives of this OLE study are: * To evaluate the safety of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA * To evaluate the PK of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA * To evaluate the effect of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA on: * Tissue FXN concentrations * Clinical evaluations of FRDA * Gene Expression and select lipids
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 2

      Key Eligibility Criteria

      Disqualifiers:Cardiac Condition, Investigational Drug, Others
      Must Not Be Taking:Amiodarone, Erythropoietin, Etravirine, Others

      75 Participants Needed

      Omaveloxolone for Friedreich's Ataxia

      Columbus, Ohio
      This trial is testing omaveloxolone, a drug that may help people with Friedreich's ataxia by boosting their body's natural defenses against cell damage. The study focuses on patients with this genetic condition because they have weakened antioxidant systems. Omaveloxolone works by activating a protein that helps protect cells from damage. It aims to improve cell function and reduce harmful effects in people with Friedreich's ataxia.

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2
      Age:16 - 40

      Key Eligibility Criteria

      Disqualifiers:Diabetes, Heart Disease, Infections, Others
      Must Not Be Taking:Cytochrome P450, P-glycoprotein

      172 Participants Needed

      Nomlabofusp for Friedreich's Ataxia

      Chevy Chase, Maryland
      The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1
      Age:2 - 17

      Key Eligibility Criteria

      Disqualifiers:Compound Heterozygous FRDA, Cardiac Condition, Others
      Must Not Be Taking:Omaveloxolone

      30 Participants Needed

      Omaveloxolone for Friedreich's Ataxia

      Philadelphia, Pennsylvania
      In this study, researchers will learn more about the effects and safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedreich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 works in the body and about its safety in children and teens who are 2 to 15 years old. The main questions researchers want to answer in this study are: * How does BIIB141 affect the participants' FA symptoms balance and stability? * How many participants have medical problems during the study? * Are there any changes in the participants' overall health during the study? * Are there any changes in the participants' heart health? * Are there any changes in how the participants move through puberty? Puberty is the time in someone's life when their body changes from a child to an adult. Researchers will also learn more about: \- How the body processes BIIB141 in children and teens This study will be done as follows: * Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which participants will check into their study research center. * There are 2 parts in this study. During Part 1, participants will take either BIIB141 or a placebo once a day. * In Part 1, participants will take BIIB141 or the placebo in a study research center on Day 1, and then at in-person visits at Week 4, Week 12, Week 26, and Week 52. On all other days, they will take BIIB141 or the placebo at home. Part 1 lasts up to 52 weeks. * During Part 2, participants from Part 1 will either continue taking BIIB141 or start it if they were taking the placebo. Part 2 will last up to 104 weeks. * In Part 1, participants will have up to 10 visits to their study research center and a phone call at Week 2. In Part 2, participants will have visits at Weeks 4, 8,12, 26, and every 26 weeks after that until they leave the study, and a phone call at Week 2. There will be a final phone call to check on the participants' health 31 days after their last dose. * Each participant will be in the study for up to about 3 years
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:2 - 15

      Key Eligibility Criteria

      Disqualifiers:Diabetes, Heart Disease, Others

      255 Participants Needed

      Vatiquinone for Friedreich Ataxia

      Philadelphia, Pennsylvania
      This trial is testing the long-term safety and effectiveness of vatiquinone in people with Friedreich ataxia who have already used this medication. The goal is to ensure it remains safe and helpful over an extended period. Idebenone, a predecessor to vatiquinone, has been used in Friedreich's ataxia patients and shown positive effects on cardiac hypertrophy.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Other Study, Pregnancy, Breastfeeding

      130 Participants Needed

      NAD+ Precursor (NR) + Exercise for Ataxia

      Philadelphia, Pennsylvania
      Randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on VO2max and Si in Friedreich's Ataxia (FA). The primary objective of this research is to measure the effect of combination administration (NR + exercise) on aerobic capacity (VO2max) in FA. A key secondary objective is to measure the effect of combination administration (NR + exercise) on glucose homeostasis (Si) in FA.

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:10 - 40

      Key Eligibility Criteria

      Disqualifiers:Diabetes, Anemia, Kidney Disease, Others
      Must Not Be Taking:Statins, Supraphysiologic Steroids

      80 Participants Needed

      Gene Therapy for Cardiomyopathy in Friedreich's Ataxia

      New York, New York
      The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA). AAVrh.10hFXN is a serotype rh.10 adeno-associated virus gene transfer vector coding for Frataxin (FXN). The drug is administered intravenously. This is a phase 1, open label, dose escalation study with a total of 25 participants.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1
      Age:18 - 50

      Key Eligibility Criteria

      Disqualifiers:Diabetes, Heart Failure, Hypertension, Others
      Must Not Be Taking:Corticosteroids, Immunosuppressives

      25 Participants Needed

      Seated Exercise for Neurological Disorders

      White Plains, New York
      To assess the impact of a 12-week virtual seated physical intervention on cardiovascular health and wellness in people with chronic neurological impairments (CNI).
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased

      Key Eligibility Criteria

      Disqualifiers:Cognitive Impairment, Medical Issues, Others

      60 Participants Needed

      Respiratory Training for Friedreich's Ataxia

      Gainesville, Florida
      This is a prospective interventional study of patients with Friedreich's Ataxia that receive respiratory strength training for a period of 12 weeks with two research visits at the beginning and at the end of the study period. Visits include swallowing evaluation with fiberoptic endoscopic evaluation of swallowing, pulmonary function testing, surface electromyography and patient surveys.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:18 - 65

      Key Eligibility Criteria

      Disqualifiers:Allergy To Lidocaine, Others
      Must Not Be Taking:Antibiotics, Corticosteroids, Neuromuscular Agents

      8 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

      "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

      "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 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
      Match to a Friedreichs Ataxia Trial

      Know someone looking for new options?
      Spread the word

      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?
      Match to a Trial
      Match to a Trial

      Frequently Asked Questions

      How much do Friedreichs Ataxia 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 Friedreichs Ataxia 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 Friedreichs Ataxia 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 Friedreichs Ataxia 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 Friedreichs Ataxia 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 Friedreichs Ataxia clinical trials?

      Most recently, we added SGT-212 Gene Therapy for Friedreich's Ataxia, Omaveloxolone for Friedreich's Ataxia and Nomlabofusp for Friedreich's Ataxia to the Power online platform.