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11 Friedreichs Ataxia Trials
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.
ASP2016 + Prednisolone for Friedreich Ataxia
Cincinnati, OhioFriedreich 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
Trial Phase:Phase 1
Age:18 - 40
Sex:All
14 Participants Needed
Gene Therapy for Cardiomyopathy in Friedreich's Ataxia
Philadelphia, PennsylvaniaThis 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
Sex:All
8 Participants Needed
Omaveloxolone for Friedreich's Ataxia
Philadelphia, PennsylvaniaIn 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 4 different groups based on their age:
* Group 1A: 12 to 15 years old, taking 150 milligrams (mg) of BIIB141
* Group 1B: 12 to 15 years old, taking a dose of BIIB141 based on the data from Group 1A
* Group 2: 6 to 11 years old, taking a dose of BIIB141 based on Group 1 data
* Group 3: 2 to 5 years old, taking a dose of BIIB141 based on Group 2 data
* During Part 2, participants from Part 1 will take BIIB141 once in the study research center. Their dose will be based on what they could safely take during Part 1. 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
Sex:All
Key Eligibility Criteria
Disqualifiers:Uncontrolled Diabetes, Heart Disease, Liver Disease, Others
Must Not Be Taking:CYP3A4 Inhibitors, CYP3A4 Inducers
20 Participants Needed
CTI-1601 for Friedreich's Ataxia
Columbus, OhioThis 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
Age:18+
Sex:All
75 Participants Needed
Omaveloxolone for Friedreich's Ataxia
Columbus, OhioThis 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
Sex:All
172 Participants Needed
Nomlabofusp for Friedreich's Ataxia
Chevy Chase, MarylandThe 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
Sex:All
Key Eligibility Criteria
Disqualifiers:Compound Heterozygous FRDA, Cardiac Condition, Others
Must Not Be Taking:Omaveloxolone
30 Participants Needed
Vatiquinone for Friedreich Ataxia
Philadelphia, PennsylvaniaThis 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
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:All
Sex:All
130 Participants Needed
NAD+ Precursor (NR) + Exercise for Ataxia
Philadelphia, PennsylvaniaRandomized, 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
Sex:All
80 Participants Needed
Gene Therapy for Cardiomyopathy in Friedreich's Ataxia
New York, New YorkThe 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
Sex:All
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 YorkTo 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
Age:18 - 75
Sex:All
60 Participants Needed
Respiratory Training for Friedreich's Ataxia
Gainesville, FloridaThis 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:Recruiting
Trial Phase:Unphased
Age:18 - 65
Sex:All
8 Participants Needed
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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 Nomlabofusp for Friedreich's Ataxia, ASP2016 + Prednisolone for Friedreich Ataxia and Respiratory Training for Friedreich's Ataxia to the Power online platform.