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| ID | Type | Description | Link |
|---|---|---|---|
| MIB-626-201 | Other Identifier | Metro International Biotech, LLC |
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| Name | Class |
|---|---|
| Children's Hospital of Philadelphia | OTHER |
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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.
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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open Label - MIB-626 | Other | MIB-626 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MIB-626 | Drug | Two (2) 500 mg Tablets, By Mouth, Daily |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Individuals With Treatment-emergent Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 5.0. | 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. | 14 Days |
| Measure | Description | Time Frame |
|---|---|---|
| Cardiac 31-Phosphorus-Magnetic Resonance Spectroscopy (MRS): Phosphocreatine (PCr)/Adenosine Tri-Phosphate (ATP) Ratio | Measure the within-participant change in PCr/ATP ratio before and after treatment with MIB-626. | Change from baseline to 14 days. |
| Post-Exercise Creatine Chemical Exchange Saturation Transfer (CrCEST) Magnetic Resonance Imaging (MRI) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Shana E McCormack, MD | Children's Hospital of Philadelphia | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27812541 | Background | DeBrosse C, Nanga RPR, Wilson N, D'Aquilla K, Elliott M, Hariharan H, Yan F, Wade K, Nguyen S, Worsley D, Parris-Skeete C, McCormick E, Xiao R, Cunningham ZZ, Fishbein L, Nathanson KL, Lynch DR, Stallings VA, Yudkoff M, Falk MJ, Reddy R, McCormack SE. Muscle oxidative phosphorylation quantitation using creatine chemical exchange saturation transfer (CrCEST) MRI in mitochondrial disorders. JCI Insight. 2016 Nov 3;1(18):e88207. doi: 10.1172/jci.insight.88207. | |
| Background | Bagga P, Wilson N., DeBrosse D., Hariharan H., Reddy R., editor. In vivo detection of NAD+ in human calf muscle at 7T using 28-channel knee volume coil. International Society for Magnetic Resonance in Medicine; 2019; Montreal, Canada. | ||
| 27648458 |
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IPD may be available with appropriate regulatory approvals.
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| ID | Title | Description |
|---|---|---|
| FG000 | Open Label - MIB-626 | MIB-626 MIB-626: Two (2) 500 mg Tablets, By Mouth, Daily |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Open Label - MIB-626 | MIB-626 MIB-626: Two (2) 500 mg Tablets, By Mouth, Daily |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Individuals With Treatment-emergent Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 5.0. | 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. | Posted | Count of Participants | Participants | 14 Days |
|
14 days
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Open Label - MIB-626 | MIB-626 MIB-626: Two (2) 500 mg Tablets, By Mouth, Daily |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Belching | Gastrointestinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Shana McCormack | Children's Hospital of Philadelphia | 215-590-3174 | mccormacks1@email.chop.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 6, 2022 | Jun 21, 2023 | Prot_003.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 6, 2022 | Jun 21, 2023 | SAP_004.pdf |
| ICF | No | No | Yes | Informed Consent Form | Jun 21, 2022 | Oct 26, 2022 | ICF_000.pdf |
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| ID | Term |
|---|---|
| D005621 | Friedreich Ataxia |
| ID | Term |
|---|---|
| D013132 | Spinocerebellar Degenerations |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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Open Label, 14 Days (+/- 2 Days)
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Assess the within-participant change in skeletal muscle post-exercise CrCEST recovery (an index of skeletal muscle mitochondrial oxidative phosphorylation capacity). |
| Change from baseline to 14 days. |
| Grip Strength | Assess within-participant changes in grip strength (via hand grip dynamometry) before and after treatment with MIB-626. | Change from baseline to 14 days. |
| Concentration of Nicotinamide Adenine Dinucleotide (NAD+) in Whole Blood | Measure the concentration of NAD+ in whole blood before and after treatment with MIB-626. | Change from baseline to 14 days. |
| Background |
| Patel M, Isaacs CJ, Seyer L, Brigatti K, Gelbard S, Strawser C, Foerster D, Shinnick J, Schadt K, Yiu EM, Delatycki MB, Perlman S, Wilmot GR, Zesiewicz T, Mathews K, Gomez CM, Yoon G, Subramony SH, Brocht A, Farmer J, Lynch DR. Progression of Friedreich ataxia: quantitative characterization over 5 years. Ann Clin Transl Neurol. 2016 Jul 25;3(9):684-94. doi: 10.1002/acn3.332. eCollection 2016 Sep. |
| years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Units |
|---|
| Counts |
|---|
| Participants |
|
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| Secondary | Cardiac 31-Phosphorus-Magnetic Resonance Spectroscopy (MRS): Phosphocreatine (PCr)/Adenosine Tri-Phosphate (ATP) Ratio | Measure the within-participant change in PCr/ATP ratio before and after treatment with MIB-626. | 1/7 individuals was unable to complete an MRI scan. | Posted | Median | Inter-Quartile Range | PCr to ATP ratio | Change from baseline to 14 days. |
|
|
|
|
| Secondary | Post-Exercise Creatine Chemical Exchange Saturation Transfer (CrCEST) Magnetic Resonance Imaging (MRI) | Assess the within-participant change in skeletal muscle post-exercise CrCEST recovery (an index of skeletal muscle mitochondrial oxidative phosphorylation capacity). | 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. | Posted | Change from baseline to 14 days. |
|
|
| Secondary | Grip Strength | Assess within-participant changes in grip strength (via hand grip dynamometry) before and after treatment with MIB-626. | Posted | Median | Inter-Quartile Range | kg | Change from baseline to 14 days. |
|
|
|
|
| Secondary | Concentration of Nicotinamide Adenine Dinucleotide (NAD+) in Whole Blood | Measure the concentration of NAD+ in whole blood before and after treatment with MIB-626. | Posted | Median | Inter-Quartile Range | micro-molar | Change from baseline to 14 days. |
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| 0 |
| 7 |
| 0 |
| 7 |
| 4 |
| 7 |
| Constipation | Gastrointestinal disorders | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Neck pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Insomnia | Psychiatric disorders | Systematic Assessment |
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| D009422 | Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028361 | Mitochondrial Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |