Trial Outcomes & Findings for NAD+ Precursor Supplementation in Friedreich's Ataxia (NCT NCT04817111)

NCT ID: NCT04817111

Last Updated: 2023-07-17

Results Overview

Safety will be monitored through collection of laboratory assessments (CBC, complete metabolic profile, 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. We report the proportion of individuals who had at least one treatment-emergent adverse event of Grade 1 or higher.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 7 participants
• Primary outcome timeframe: 14 Days
• Results posted on: 2023-07-17

Participant Flow

Participant milestones

Measure	Open Label - MIB-626 MIB-626 MIB-626: Two (2) 500 mg Tablets, By Mouth, Daily
Overall Study STARTED	7
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

NAD+ Precursor Supplementation in Friedreich's Ataxia

Baseline characteristics by cohort

Measure	Open Label - MIB-626 n=7 Participants MIB-626 MIB-626: Two (2) 500 mg Tablets, By Mouth, Daily
Age, Continuous	26 years n=93 Participants
Sex: Female, Male Female	2 Participants n=93 Participants
Sex: Female, Male Male	5 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	7 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants

PRIMARY outcome

Timeframe: 14 Days

Safety will be monitored through collection of laboratory assessments (CBC, complete metabolic profile, 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. We report the proportion of individuals who had at least one treatment-emergent adverse event of Grade 1 or higher.

Outcome measures

Measure	Open Label - MIB-626 n=7 Participants MIB-626 MIB-626: Two (2) 500 mg Tablets, By Mouth, Daily
Percentage of Individuals With Treatment-emergent Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 5.0.	4 Participants

SECONDARY outcome

Timeframe: Change from baseline to 14 days.

Population: 1/7 individuals was unable to complete an MRI scan.

Measure the within-participant change in PCr/ATP ratio before and after treatment with MIB-626.

Outcome measures

Measure	Open Label - MIB-626 n=6 Participants MIB-626 MIB-626: Two (2) 500 mg Tablets, By Mouth, Daily
Cardiac 31-Phosphorus-Magnetic Resonance Spectroscopy (MRS): Phosphocreatine (PCr)/Adenosine Tri-Phosphate (ATP) Ratio	-0.445 PCr to ATP ratio Interval -0.6025 to -0.0925

SECONDARY outcome

Timeframe: Change from baseline to 14 days.

Population: This secondary outcome measure could not be collected for any participant because insufficient MRI scanning time remained during the visits after main outcomes were collected.

Assess the within-participant change in skeletal muscle post-exercise CrCEST recovery (an index of skeletal muscle mitochondrial oxidative phosphorylation capacity).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Change from baseline to 14 days.

Assess within-participant changes in grip strength (via hand grip dynamometry) before and after treatment with MIB-626.

Outcome measures

Measure	Open Label - MIB-626 n=7 Participants MIB-626 MIB-626: Two (2) 500 mg Tablets, By Mouth, Daily
Grip Strength	-0.65 kg Interval -2.625 to -0.075

SECONDARY outcome

Timeframe: Change from baseline to 14 days.

Measure the concentration of NAD+ in whole blood before and after treatment with MIB-626.

Outcome measures

Measure	Open Label - MIB-626 n=6 Participants MIB-626 MIB-626: Two (2) 500 mg Tablets, By Mouth, Daily
Concentration of Nicotinamide Adenine Dinucleotide (NAD+) in Whole Blood	49.9 micro-molar Interval 42.0 to 54.0

Adverse Events

Open Label - MIB-626

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Open Label - MIB-626 n=7 participants at risk MIB-626 MIB-626: Two (2) 500 mg Tablets, By Mouth, Daily
Gastrointestinal disorders Belching	14.3% 1/7 • Number of events 1 • 14 days
Gastrointestinal disorders Constipation	14.3% 1/7 • Number of events 1 • 14 days
Musculoskeletal and connective tissue disorders Back pain	14.3% 1/7 • Number of events 1 • 14 days
Musculoskeletal and connective tissue disorders Neck pain	14.3% 1/7 • Number of events 1 • 14 days
Psychiatric disorders Insomnia	14.3% 1/7 • Number of events 1 • 14 days

Additional Information

Dr. Shana McCormack

Children's Hospital of Philadelphia

Phone: 215-590-3174

Email: mccormacks1@email.chop.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60