NAD+ Precursor Supplementation in Friedreich's Ataxia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04817111|
Recruitment Status : Active, not recruiting
First Posted : March 25, 2021
Last Update Posted : October 28, 2022
- Study Details
- Tabular View
- Results Submitted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|Friedreich Ataxia||Drug: MIB-626||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Open Label, 14 Days (+/- 2 Days)|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2a Study of NAD+ Precursor Supplementation in Friedreich's Ataxia|
|Actual Study Start Date :||May 17, 2021|
|Actual Primary Completion Date :||May 19, 2022|
|Estimated Study Completion Date :||May 19, 2023|
Open Label - MIB-626
Two (2) 500 mg Tablets, By Mouth, Daily
- Incidence of treatment-emergent adverse events as assessed by Common Terminology Criteria for Adverse Events version 5.0. [ Time Frame: 14 Days ]Safety will be monitored through collection of laboratory assessments (CBC, CMP, lipid profile, HbA1c), vital signs (heart rate, blood pressure), and ECG, all of which will be reviewed for clinically relevant abnormalities, and standardized assessment of symptoms.
- Cardiac 31-Phosphorus-MRS: PCr/ATP Ratio [ Time Frame: Change from baseline to 14 days. ]Measure the within-participant change in PCr/ATP-γ ratio before and after treatment with MIB-626.
- Post-Exercise CrCEST [ Time Frame: Change from baseline to 14 days. ]Assess the within-participant change in skeletal muscle post-exercise CrCEST recovery (an index of skeletal muscle mitochondrial oxidative phosphorylation capacity) and the within-participant change skeletal muscle NAD+ via 1H-MRS before and after treatment with MIB-626.
- Grip Strength [ Time Frame: Change from baseline to 14 days. ]Assess within-participant changes in grip strength (via hand grip dynamometry) before and after treatment with MIB-626.
- Concentration of NAD+ in Whole Blood [ Time Frame: Change from baseline to 14 days. ]Measure the concentration of NAD+ (and associated metabolites) in whole blood before and after treatment with MIB-626.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years to 64 Years (Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Molecular diagnosis of Friedreich's Ataxia (FA).
- Males and females, ages 18 years to < 65 years.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04817111
|United States, Pennsylvania|
|The Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Shana E McCormack, MD||Children's Hospital of Philadelphia|
Documents provided by Shana McCormack, Children's Hospital of Philadelphia:
|Responsible Party:||Shana McCormack, Attending Physician, Children's Hospital of Philadelphia|
|Other Study ID Numbers:||
MIB-626-201 ( Other Identifier: Metro International Biotech, LLC )
|First Posted:||March 25, 2021 Key Record Dates|
|Last Update Posted:||October 28, 2022|
|Last Verified:||October 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Plan Description:||IPD may be available with appropriate regulatory approvals.|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Nervous System Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn