CompletedNot Applicable

NAD+ and Exercise in FA

United States75 participantsStarted 2020-09-03

Plain-language summary

Randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on Peak VO2 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 (Peak VO2) in FA. A key secondary objective is to measure the effect of combination administration (NR + exercise) on glucose homeostasis (Si) in FA.

Who can participate

Age range

10 Years – 40 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Molecular diagnosis of Friedrich's Ataxia (FA).
. Males and Females, Age 10 to 40 years (inclusive).
. Girls, 11 years of age and older, must have a negative urine/serum pregnancy test and must use an acceptable method of contraception, including abstinence, a barrier method (diaphragm or condom), Depo-Provera, or an oral contraceptive, for the duration of the study.
. Not currently meeting exercise guidelines as outlined by The Physical Activity Guidelines for Americans.
. Cardiac echocardiogram or cardiac MRI, performed within 1 year of enrollment, showing an LVEF \> 45%
. ECG, performed within 1 year of enrollment, without clinically significant arrhythmia.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Within-Participant Change in Peak V02 (Maximal Oxygen Uptake on Cardiopulmonary Exercise Testing)

Timeframe: Baseline to 12 Weeks

Trial details

NCT IDNCT04192136

SponsorChildren's Hospital of Philadelphia

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-02-07

Results submitted2026-01-22

View on ClinicalTrials.gov More Friedreich Ataxia 1 trials

Plain-language summary

Who can participate

Age range

10 Years – 40 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Molecular diagnosis of Friedrich's Ataxia (FA).
. Males and Females, Age 10 to 40 years (inclusive).
. Girls, 11 years of age and older, must have a negative urine/serum pregnancy test and must use an acceptable method of contraception, including abstinence, a barrier method (diaphragm or condom), Depo-Provera, or an oral contraceptive, for the duration of the study.
. Not currently meeting exercise guidelines as outlined by The Physical Activity Guidelines for Americans.
. Cardiac echocardiogram or cardiac MRI, performed within 1 year of enrollment, showing an LVEF \> 45%
. ECG, performed within 1 year of enrollment, without clinically significant arrhythmia.

NAD+ and Exercise in FA

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

NAD+ and Exercise in FA

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Exclusion criteria