Status:
COMPLETED
NAD+ Precursor Supplementation in Friedreich's Ataxia
Lead Sponsor:
Metro International Biotech, LLC
Collaborating Sponsors:
Children's Hospital of Philadelphia
Conditions:
Friedreich Ataxia
Eligibility:
All Genders
18-64 years
Phase:
PHASE2
Brief 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 o...
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-M...
Eligibility Criteria
Inclusion
- Molecular diagnosis of Friedreich's Ataxia (FA).
- Males and females, ages 18 years to \< 65 years.
Exclusion
- Known sensitivity to nicotinamide-containing compounds.
- Concurrent use of Vitamin B3 supplements and/or any medications likely to increase risk of MIB-626 toxicity.
- HgbA1c \> (great than or equal to) 8.5% and or Diabetes Mellitus (DM) requiring insulin or insulin secretagogue.
- Kidney disease (Estimated Glomerular Filtration Rate (eGFR) \< 60 ml/min/1.73 m2) using serum creatinine and MDRD equation. The eGFR levels will be calculated using the Modified Diet in Renal Disease Study (MDRD) equation, which is the equation used by the Children's Hospital Of Philadelphia laboratory.
- Liver disease (Aspartate Aminotransferase (AST)/Alanine Aminotransferase (ALT) \> 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.
Key Trial Info
Start Date :
May 17 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 19 2022
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT04817111
Start Date
May 17 2021
End Date
May 19 2022
Last Update
July 17 2023
Active Locations (1)
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1
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104