Phase 2
Completed N=7
NAD+ Precursor Supplementation in Friedreich's Ataxia
Source: ClinicalTrials.gov NCT04817111 ↗Enrolled (actual)
7
Serious AEs
0.0%
Results posted
Jul 2023
Primary outcomePrimary: Percentage of Individuals With Treatment-emergent Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 5.0. — 4 Participants
Summary
The primary objective is to test the safety and tolerability of short-term therapy with a nicotinamide adenine dinucleotide (NAD+) precursor (MIB-626) in adults with Friedreich's Ataxia (FA) without overt heart failure and with a left ventricular ejection fraction ≥ 40%. A key secondary objective is to test the effects of MIB-626 on cardiac and skeletal muscle bioenergetics.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Individuals With Treatment-emergent Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 5.0. |
4 | — |
| SECONDARY Cardiac 31-Phosphorus-Magnetic Resonance Spectroscopy (MRS): Phosphocreatine (PCr)/Adenosine Tri-Phosphate (ATP) Ratio |
-0.445 | 0.18 |
| SECONDARY Post-Exercise Creatine Chemical Exchange Saturation Transfer (CrCEST) Magnetic Resonance Imaging (MRI) |
— | — |
| SECONDARY Grip Strength |
-0.65 | 0.21 |
| SECONDARY Concentration of Nicotinamide Adenine Dinucleotide (NAD+) in Whole Blood |
49.9 | 0.03 sig |
Eligibility Criteria
Inclusion Criteria
- Molecular diagnosis of Friedreich's Ataxia (FA).
- Males and females, ages 18 years to (great than or equal to) 8.5% and or Diabetes Mellitus (DM) requiring insulin or insulin secretagogue.
- Kidney disease (Estimated Glomerular Filtration Rate (eGFR) 3 x Upper Limit of Normal)
- Severe co-existing cardiac disease (Ejection Fraction (EF) < 40%, known arrhythmia) as demonstrated by an echocardiogram within 12 months of screening.
- Any contraindication to MRI, including spinal rods (related to unknown safety considerations for cardiac 31-Phosphorus -MRS).
- Use of any investigational agent within 4 weeks of enrollment.
- Females: Pregnant/lactating or planning to become pregnant during their participation.
- Any medical condition that, in the opinion of the investigator, will interfere with the safe completion of the study.
Data sourced from ClinicalTrials.gov (NCT04817111). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.