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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
Children's Hospital of Philadelphia
Information provided by (Responsible Party):
Shana McCormack, Children's Hospital of Philadelphia

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 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.

Condition or disease Intervention/treatment Phase
Friedreich Ataxia Drug: MIB-626 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Open Label, 14 Days (+/- 2 Days)
Masking: None (Open Label)
Primary Purpose: Other
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

Arm Intervention/treatment
Open Label - MIB-626
Drug: MIB-626
Two (2) 500 mg Tablets, By Mouth, Daily

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 64 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Molecular diagnosis of Friedreich's Ataxia (FA).
  • Males and females, ages 18 years to < 65 years.

Exclusion Criteria:

  • 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.

Information from the National Library of Medicine

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

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United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Metro International Biotech, LLC
Children's Hospital of Philadelphia
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Principal Investigator: Shana E McCormack, MD Children's Hospital of Philadelphia
  Study Documents (Full-Text)

Documents provided by Shana McCormack, Children's Hospital of Philadelphia:
Informed Consent Form  [PDF] October 20, 2021

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Responsible Party: Shana McCormack, Attending Physician, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT04817111    
Other Study ID Numbers: 19-016525
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.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases