NCT04817111

NAD+ Precursor Supplementation in Friedreich's Ataxia

A Phase 2a Study of NAD+ Precursor Supplementation in Friedreich's Ataxia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Metro International Biotech, LLC · Industry
• Sex: All
• Age: 18 Years – 64 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The primary focus for this protocol is safety and tolerability. We will systematically assess for adverse events using a safety monitoring uniform report form. We will also use cardiac 31-Phosphorus-Magnetic Resonance Spectroscopy (MRS) to measure the Phosphocreatine(PCr)/Adenosine triphosphate (ATP)- γ ratio before and after treatment with MIB-626. In addition, if time permits we will use proton (1H)-MRS to measure skeletal muscle nicotinamide adenine dinucleotide (NAD+) before and after treatment.

Conditions

• Friedreich Ataxia

Interventions

Type	Name	Description
DRUG	MIB-626	Two (2) 500 mg Tablets, By Mouth, Daily

Timeline

• Start date: 2021-05-17
• Primary completion: 2022-05-19
• Completion: 2022-05-19
• First posted: 2021-03-25
• Last updated: 2023-07-17
• Results posted: 2023-07-17

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study