BridgeMD
A double-blind, randomized, placebo-controlled trial to test the efficacy, safety and tolerability of Dimethyl Fumarate in Friedreich Ataxia (DMF-FA-201).
EU CTIS ID: 2022-503016-16-00
What this study is testing
To test the effect of DMF on FXN transcription and frataxin protein in FRDA patients.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Molecular diagnosis of Friedreich Ataxia with a homozygous GAA expansion
- Age ≥12 years
- Body weight ≥30 Kg
- Patients able to read and sign the informed consent
You likely can't join if
- Treatment with DMF in the previous 12 months
- Positive history of alcohol or drug abuse in the past 2 years, except for medical use of cannabis
- Hypersensitivity to DMF or any other component of the study drug
- Patients not able to comply with the study
- For female patients (Sexually not active, hysterectomized, sterilized, menopause patients are excluded from the following criteria): - Pregnancy, or - Breastfeeding, or - Inadequate contraception
- Treatment with Idebenone, coenzyme Q10, or any other vitamin supplements in the previous 30 days
See the full eligibility criteria
- Molecular diagnosis of Friedreich Ataxia with a homozygous GAA expansion
- Age ≥12 years
- Body weight ≥30 Kg
- Patients able to read and sign the informed consent
- Treatment with DMF in the previous 12 months
- Positive history of alcohol or drug abuse in the past 2 years, except for medical use of cannabis
- Hypersensitivity to DMF or any other component of the study drug
- Patients not able to comply with the study
- For female patients (Sexually not active, hysterectomized, sterilized, menopause patients are excluded from the following criteria): - Pregnancy, or - Breastfeeding, or - Inadequate contraception
- Treatment with Idebenone, coenzyme Q10, or any other vitamin supplements in the previous 30 days
- Patients in treatment with any other not allowed drug
- Any Cardiac and/or Renal and/or Hepatic disease judged as clinically significant by the investigator (any abnormal and clinically non significant cardiac disease associated with Friedreich Ataxia is not an exclusion criteria)
- Any clinically significant ECG abnormalities that may interfere with the study
- Any abnormal and clinically significant laboratory exams at screening visit that may interfere with the trial
- Any acute disease that could interfere with the study, as judged by the investigator
- Patient positive to the Human Immunodeficiency Virus (HIV) or Hepatitis B or C test
- Patients with a positive history of neoplasia, with the only exception of a completely excided basal cell carcinoma
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years, 65+ years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.