Friedreich Ataxia 1ExRx in FANCT04192136
NAD+ and Exercise in FA
A study testing Nicotinamide Riboside and Exercise Intervention in people with Friedreich Ataxia 1, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 to 40 years
- Conditions the study is for
- Friedreich Ataxia 1
- What is being tested?
- Nicotinamide Riboside and Exercise Intervention
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 75 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Sep 2020to 31 Oct 2025
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
- Children and Adolescents should do 60 minutes (1 hour) or more of moderate-to-vigorous physical activity daily.
- As a part of their physical activity, children and adolescents should include muscle-strengthening physical activity on at least 3 days a week.
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- Adults should do at least 150 minutes (2 hours and 30 minutes) to 300 minutes (5 hours) a week of moderate-intensity, or 75 minutes (1 hour and 15 minutes) to 150 minutes (2 hours and 30 minutes) a week of vigorous-intensity aerobic physical activity.
- Adults should also do muscle-strengthening activities of moderate or greater intensity that involve all major muscle groups on 2 or more days a week.
- 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.
- Weight > 24 kg
- Parental/guardian permission (informed consent) and if appropriate, child assent.
Who cannot join
- Known sensitivity to NR.
- Concurrent use of any medications, including statins, likely to increase risk of NR toxicity.
- HgbA1c > 8.5% and/or Diabetes Mellitus (DM) requiring insulin or insulin secretagogue.
- Use of supraphysiologic steroids.
- Laboratory abnormalities that indicate clinically significant anemia or bleeding risk. (Hemoglobin < 10 g/dL or Platelets < 100K)
- Laboratory abnormalities that indicate clinically significant kidney disease using serum creatinine and Modification of Diet in Renal Disease (MDRD) equation. (Estimated Glomerular Filtration Rate (eGFR) < 60 ml/min/1.73 m2)
Show all 27Show fewer
- Laboratory abnormalities that indicate clinically significant liver disease. (Aspartate Aminotransferase (AST)/Serum Glutamic Oxaloacetic Transaminase (SGOT) 3.0 x Upper Limit of Normal and/or Alanine Aminotransferase (ALT)/Serum Glutamic Pyruvic Transaminase (SGPT) 3.0 x Upper Limit of Normal)
- Uncontrolled and persistent arrhythmias that are felt to be clinically significant.
- Known history of moderate or severe left ventricular systolic dysfunction (Left Ventricular Ejection Fraction (LVEF) < 45%)
- Standard contraindications to exercise testing.
- Inability to sit and pedal unassisted in a cycle ergometry chair, at a cadence of at least 55 rotations per minute (rpm) during unloaded warm up, in a cycle ergometry chair and complete a maximal Cardio Pulmonary Exercise Test (CPET)
- Inability to sit and pedal unassisted in a recumbent tricycle.
- Any contraindication to MRI. Including:
- Any intra-luminal implant, filter, stent or valve replacement
- Any type of life assist device, pump, or prosthetic
- Any vascular clip or clamp
- Any surgically placed clips or clamps or bands on visceral organs
- Any intracranial implants of any type other than dental fillings
- Any non-removable piercings, jewelry, or medicinal patch
- Any personal history of intraocular injury or fragment in or around the orbit that cannot be cleared through radiologic examination.
- Any personal history of bullet, shrapnel, or stabbing wounds that cannot be cleared through radiologic evaluation.
- Inability to lie flat in the MRI scanner for 60-90 minutes.
- participants who cannot complete the MRI will not be excluded from participation in the remainder of the study procedures if they meet those inclusion and exclusion criteria
- Use of any investigational agent within 4 weeks of enrollment, except open-label extension phase.
- Females: pregnant, lactating, or planning to become pregnant during their participation.
- Any medical condition, in the opinion of the investigator that will interfere with the safe completion of the study.
- Parents/guardians or participants who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: NAD+ Precursor Supplementation With Exercise Training to Increase Aerobic Capacity in Friedreich's Ataxia
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.
Read the detailed description
Friedreich's Ataxia (FA) is a progressive neurodegenerative disease affecting 1 in 50,000 individuals in the U.S. Currently, there is no approved treatment.
There is a critical knowledge gap regarding the best ways to intervene to increase aerobic capacity (Peak VO2 on exercise testing) in FA. Exercise is the most potent known stimulus for increasing muscle mass and mitochondrial oxidative phosphorylation (OXPHOS) capacity, increasing Peak VO2, and increasing insulin sensitivity (Si), however, it has not been studied in FA. One adaptation seen in exercised muscles is an increase in muscle nicotinamide adenine dinucleotide (NAD+), a cofactor required for glycolytic and mitochondrial adenosine triphosphate (ATP) production. In skeletal- and cardiac muscle-specific frataxin (FXN) knock-out animals, NAD+ precursors rescued cardiac function to near-normal, additionally highlighting its translational potential in FA. Nicotinamide riboside (NR) is a NAD+ precursor currently available as a dietary supplement (Tru Niagen ®, ChromaDex, Irvine CA) that is expected to be safe and well-tolerated in adults and children. The central hypothesis is that exercise + NR will increase skeletal muscle mitochondrial OXPHOS and increase muscle mass to increase Peak VO2 in FA. The investigators expect that exercise + NR will also increase Si in this cohort.
- Conditions
- Friedreich Ataxia 1
- Being tested
- Nicotinamide Riboside, Placebo, Exercise Intervention
- Medical subject headings
- Friedreich Ataxia 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nicotinamide RibosideDietary supplement
Investigators will use (Good Manufacturing Process) GMP-grade 300 mg capsules of the dietary supplement nicotinamide riboside (ChromaDex, Irvine CA).
NR is distributed by ChromaDex, Inc., Irvine, CA. NR is available as 300 mg capsules.
The dietary supplement will be re-labeled by the Hospital of the University of Pennsylvania (HUP) Investigational Drug Service according to FDA regulations, including subject and physician name and NR or Placebo 300 mg capsules.
PlaceboDietary supplement
The matched placebo will contain the same excipients without the active supplement and is generally recognized as safe. The placebo will be covered in an identical capsule (NR will be covered in the same capsule).
Exercise InterventionOther
The exercise program consists of at-home training sessions: 3 aerobic sessions per week on the in-home bike trainer, and 2 resistance exercise sessions per week using resistance bands.
On aerobic training days, subjects will be given instruction on a recumbent Catrike trainer, titrated such that they spend 20 minutes at their target heart rate as determined by baseline Exercise Stress Test (EST) (60-80% of their heart rate at peak VO2) and as measured by their wearable device.
On resistance training days, subjects will be given instructions to complete circuits of resistance exercises. Resistance training intensity (as determined by graded resistance bands) will be maintained at 60% of pre-training maximal voluntary contraction for each muscle group.
| Study group | Role | What they receive |
|---|---|---|
| Nicotinamide Riboside (NR) | Experimental | Investigators will use Good Manufacturing Process (GMP)-grade 300 mg capsules of the dietary supplement nicotinamide riboside (ChromaDex, Irvine CA). Investigators dose based on body weight, and monitor for adverse effects (AEs).
For individuals with weight > 72 kg: 900 mg po qd x 12 wks.
For individuals with weight > 48 kg and ≤ 72 kg: 600 mg po qd x 12 wks.
For individuals with weight 24 ≤ 48 kg: 300 mg po qd x 12 wks. Receives: Dietary Supplement: Nicotinamide Riboside |
| Placebo | Placebo comparator | Matched placebo will contain the same excipients without the active supplement and is generally recognized as safe. The placebo will be covered in an identical capsule (NR will be covered in the same capsule). Doses of placebo will match the body weight schema for dosing of NR. Investigators dose based on body weight, and monitor for adverse effects (AEs).
For individuals with weight > 72 kg: 900 mg po qd x 12 wks.
For individuals with weight > 48 kg and ≤ 72 kg: 600 mg po qd x 12 wks.
For individuals with weight 24 ≤ 48 kg: 300 mg po qd x 12 wks. Receives: Dietary Supplement: Placebo |
| Exercise Intervention and NR | Experimental | The exercise program consists of at-home training sessions: 3 aerobic sessions per week on the in-home bike trainer, and 2 resistance exercise sessions per week using resistance bands. All sessions will begin with stretching and brief aerobic warm-up exercise on the in-home bike trainer. On resistance training days, subjects will be given instructions to complete circuits of resistance exercises.
Participants in this arm will receive both the Exercise Intervention and the NR. Receives: Dietary Supplement: Nicotinamide Riboside; Other: Exercise Intervention |
| Exercise Intervention and Placebo | Experimental | The exercise program consists of at-home training sessions: 3 aerobic sessions per week on the in-home bike trainer, and 2 resistance exercise sessions per week using resistance bands. All sessions will begin with stretching and brief aerobic warm-up exercise on the in-home bike trainer. On resistance training days, subjects will be given instructions to complete circuits of resistance exercises.
Participants in this arm will receive both the Exercise Intervention and the Placebo. Receives: Dietary Supplement: Placebo; Other: Exercise Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Within-Participant Change in Peak V02 (Maximal Oxygen Uptake on Cardiopulmonary Exercise Testing)
Peak V02 will be assessed by completion of an incremental symptom-limited cardio-pulmonary Exercise Stress Test (EST) on a recumbent leg cycle ergometer.
The index is based on the change in peak V02 given in liters per minute (L/min), and a higher value indicates greater oxygen uptake.
Measured: Baseline to 12 Weeks
Secondary outcomes (1)
Within-Participant Change in Whole Body Insulin Sensitivity (Si)
Whole Body Insulin Sensitivity (Si) will be assessed by completion of a stable isotope tracer-enhanced Oral Glucose Tolerance Test (OGTT). Samples will be collected at 10 time points for analysis.
The unit of measure is based on the change insulin values given in microunits per milliliter (μU/mL) and those of glucose, in milligrams per deciliter, and a higher value indicates greater insulin sensitivity. The whole body insulin sensitivity index (WBISI), also known as the Matsuda index or composite ISI, is dimensionless (unitless).
Measured: Baseline to 12 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2019
- Study start3 Sep 2020
- Primary completion7 Feb 2025
- Study completion31 Oct 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Friedreich Ataxia 1Studies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Factorial
- Model description
- Randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 75 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04192136
- Sponsor's protocol ID
- 19-016634
- Other IDs
- R01HL149722
- Acronym
- ExRx in FA
- Status last verified
- Mar 2026
- First submitted
- 6 Dec 2019
- Last updated
- 11 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Lin KY, Bucha A, McSweeney K, Wade KL, Karaj A, Tamaroff J, O'Malley S, Chung NM, Cilenti NA, Wanner J, Adzika GK, Mesaros C, Blair IA, Rojsajjakul T, Serai S, Farmer J, Bryant K, Lu Y, Harhay MO, Weber DR, Paridon SM, Seifert EL, Putt ME, Zamani P, Baur JA, Lynch DR, McCormack SE. Safety and efficacy of individualised exercise and NAD+ precursor supplementation in patients with Friedreich's ataxia in the USA: a single-centre, 2 x 2 factorial, randomised controlled trial. Lancet Neurol. 2026 May;25(5):469-481. doi: 10.1016/S1474-4422(26)00082-7.
PubMed 42009009
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- NAD+ Precursor Supplementation With Exercise Training to Increase Aerobic Capacity in Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT04192136
- Acronym / sponsor ID
- ExRx in FA / 19-016634
- Conditions
- Friedreich Ataxia 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 75 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 3 Sep 2020 (actual)
- Primary completion
- 7 Feb 2025 (actual)
- Study completion
- 31 Oct 2025 (actual)
- First posted
- 10 Dec 2019 (actual)
- Results
- Posted 11 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Friedreich's Ataxia (FA) is a progressive neurodegenerative disease affecting 1 in 50,000 individuals in the U.S. Currently, there is no approved treatment.
There is a critical knowledge gap regarding the best ways to intervene to increase aerobic capacity (Peak VO2 on exercise testing) in FA. Exercise is the most potent known stimulus for increasing muscle mass and mitochondrial oxidative phosphorylation (OXPHOS) capacity, increasing Peak VO2, and increasing insulin sensitivity (Si), however, it has not been studied in FA. One adaptation seen in exercised muscles is an increase in muscle nicotinamide adenine dinucleotide (NAD+), a cofactor required for glycolytic and mitochondrial adenosine triphosphate (ATP) production. In skeletal- and cardiac muscle-specific frataxin (FXN) knock-out animals, NAD+ precursors rescued cardiac function to near-normal, additionally highlighting its translational potential in FA. Nicotinamide riboside (NR) is a NAD+ precursor currently available as a dietary supplement (Tru Niagen ®, ChromaDex, Irvine CA) that is expected to be safe and well-tolerated in adults and children. The central hypothesis is that exercise + NR will increase skeletal muscle mitochondrial OXPHOS and increase muscle mass to increase Peak VO2 in FA. The investigators expect that exercise + NR will also increase Si in this cohort.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
- Model description
- Randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise.
| Arm | Type | Receives |
|---|---|---|
| Nicotinamide Riboside (NR) | Experimental | Dietary Supplement: Nicotinamide RibosideInvestigators will use Good Manufacturing Process (GMP)-grade 300 mg capsules of the dietary supplement nicotinamide riboside (ChromaDex, Irvine CA). Investigators dose based on body weight, and monitor for adverse effects (AEs). For individuals with weight > 72 kg: 900 mg po qd x 12 wks. For individuals with weight > 48 kg and ≤ 72 kg: 600 mg po qd x 12 wks. For individuals with weight 24 ≤ 48 kg: 300 mg po qd x 12 wks. |
| Placebo | Placebo comparator | Dietary Supplement: PlaceboMatched placebo will contain the same excipients without the active supplement and is generally recognized as safe. The placebo will be covered in an identical capsule (NR will be covered in the same capsule). Doses of placebo will match the body weight schema for dosing of NR. Investigators dose based on body weight, and monitor for adverse effects (AEs). For individuals with weight > 72 kg: 900 mg po qd x 12 wks. For individuals with weight > 48 kg and ≤ 72 kg: 600 mg po qd x 12 wks. For individuals with weight 24 ≤ 48 kg: 300 mg po qd x 12 wks. |
| Exercise Intervention and NR | Experimental | Dietary Supplement: Nicotinamide Riboside, Other: Exercise InterventionThe exercise program consists of at-home training sessions: 3 aerobic sessions per week on the in-home bike trainer, and 2 resistance exercise sessions per week using resistance bands. All sessions will begin with stretching and brief aerobic warm-up exercise on the in-home bike trainer. On resistance training days, subjects will be given instructions to complete circuits of resistance exercises. Participants in this arm will receive both the Exercise Intervention and the NR. |
| Exercise Intervention and Placebo | Experimental | Dietary Supplement: Placebo, Other: Exercise InterventionThe exercise program consists of at-home training sessions: 3 aerobic sessions per week on the in-home bike trainer, and 2 resistance exercise sessions per week using resistance bands. All sessions will begin with stretching and brief aerobic warm-up exercise on the in-home bike trainer. On resistance training days, subjects will be given instructions to complete circuits of resistance exercises. Participants in this arm will receive both the Exercise Intervention and the Placebo. |
Who may qualify
- Ages
- 10 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- 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%
Key exclusion criteria (27 listed)
- Known sensitivity to NR.
- Concurrent use of any medications, including statins, likely to increase risk of NR toxicity.
- HgbA1c > 8.5% and/or Diabetes Mellitus (DM) requiring insulin or insulin secretagogue.
- Use of supraphysiologic steroids.
- Laboratory abnormalities that indicate clinically significant anemia or bleeding risk. (Hemoglobin < 10 g/dL or Platelets < 100K)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nicotinamide RibosideInvestigators will use (Good Manufacturing Process) GMP-grade 300 mg capsules of the dietary supplement nicotinamide riboside (ChromaDex, Irvine CA). NR is distributed by ChromaDex, Inc., Irvine, CA. NR is available as 300 mg capsules. The dietary supplement will be re-labeled by the Hospital of the University of Pennsylvania (HUP) Investigational Drug Service according to FDA regulations, including subject and physician name and NR or Placebo 300 mg capsules. | Dietary supplement | Exercise Intervention and NR, Nicotinamide Riboside (NR) |
| PlaceboThe matched placebo will contain the same excipients without the active supplement and is generally recognized as safe. The placebo will be covered in an identical capsule (NR will be covered in the same capsule). | Dietary supplement | Exercise Intervention and Placebo, Placebo |
| Exercise InterventionThe exercise program consists of at-home training sessions: 3 aerobic sessions per week on the in-home bike trainer, and 2 resistance exercise sessions per week using resistance bands. On aerobic training days, subjects will be given instruction on a recumbent Catrike trainer, titrated such that they spend 20 minutes at their target heart rate as determined by baseline Exercise Stress Test (EST) (60-80% of their heart rate at peak VO2) and as measured by their wearable device. On resistance training days, subjects will be given instructions to complete circuits of resistance exercises. Resistance training intensity (as determined by graded resistance bands) will be maintained at 60% of pre-training maximal voluntary contraction for each muscle group. | Other | Exercise Intervention and NR, Exercise Intervention and Placebo |
Primary outcome
Within-Participant Change in Peak V02 (Maximal Oxygen Uptake on Cardiopulmonary Exercise Testing)
Time frame: Baseline to 12 Weeks
Peak V02 will be assessed by completion of an incremental symptom-limited cardio-pulmonary Exercise Stress Test (EST) on a recumbent leg cycle ergometer. The index is based on the change in peak V02 given in liters per minute (L/min), and a higher value indicates greater oxygen uptake.
1 secondary outcome
Within-Participant Change in Whole Body Insulin Sensitivity (Si)
Time frame: Baseline to 12 Weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42009009Lin KY, Bucha A, McSweeney K, Wade KL, Karaj A, Tamaroff J, O'Malley S, Chung NM, Cilenti NA, Wanner J, Adzika GK, Mesaros C, Blair IA, Rojsajjakul T, Serai S, Farmer J, Bryant K, Lu Y, Harhay MO, Weber DR, Paridon SM, Seifert EL, Putt ME, Zamani P, Baur JA, Lynch DR, McCormack SE. Safety and efficacy of individualised exercise and NAD+ precursor supplementation in patients with Friedreich's ataxia in the USA: a single-centre, 2 x 2 factorial, randomised controlled trial. Lancet Neurol. 2026 May;25(5):469-481. doi: 10.1016/S1474-4422(26)00082-7.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Nicotinamide Riboside and Exercise Intervention in people with Friedreich Ataxia 1, run by Children's Hospital of Philadelphia at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Within-Participant Change in Peak V02 (Maximal Oxygen Uptake on Cardiopulmonary Exercise Testing) (time frame: Baseline to 12 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04192136, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Mar 2026.