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The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia
This is a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, tolerability, in subjects with FRDA following the oral administration of study drug (active or placebo capsules). Sixty eligible patients will undergo various assessments at different time points during the the study. The study duration is 13 months which includes screening, treatment and safety follow up phone call.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RT001 | Experimental |
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| Placebo | Placebo Comparator |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RT001 | Drug | RT001 is encapsulated 9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester, which is a site specific (C11) di-deutero synthetic homologue of LA ethyl ester. Each capsule contains 960 mg of RT001. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) | To measure the change in MVO2 from baseline to Month 11 using CPET | 11 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Timed 1 Minute Walk Distance | Change from baseline Distance walked in 1 minute at 11 months | 11 months |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Peter Milner, MD | Chief Medical Officer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Collaborative Neuroscience Network, LLC | Long Beach | California | 90806 | United States | ||
| UCLA |
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| ID | Title | Description |
|---|---|---|
| FG000 | RT001 |
RT001: RT001 is encapsulated 9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester, which is a site specific (C11) di-deutero synthetic homologue of LA ethyl ester. Each capsule contains 960 mg of RT001. |
| FG001 | Placebo |
Placebo: The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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| ID | Title | Description |
|---|---|---|
| BG000 | RT001 |
RT001: RT001 is encapsulated 9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester, which is a site specific (C11) di-deutero synthetic homologue of LA ethyl ester. Each capsule contains 960 mg of RT001. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| 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 | Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) | To measure the change in MVO2 from baseline to Month 11 using CPET | Posted | Mean | Standard Error | ml O2/min/kg | 11 months |
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12 months
Adverse event reporting was conducted in the ITT population.
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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 | RT001 |
RT001: RT001 is encapsulated 9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester, which is a site specific (C11) di-deutero synthetic homologue of LA ethyl ester. Each capsule contains 960 mg of RT001. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| non-cardiac chest pain | Cardiac disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| nausea | Gastrointestinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Mark G. Midei, MD, VP Medical Affairs | Retrotope | 4103715357 | mark@retrotope.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Nov 3, 2021 | Apr 6, 2022 | Prot_SAP_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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| ID | Term |
|---|---|
| C000634027 | RT001 |
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| Placebo | Drug | The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001. |
|
| Los Angeles |
| California |
| 90095 |
| United States |
| University of Florida | Gainesville | Florida | 32608 | United States |
| USF Ataxia Research Center | Tampa | Florida | 33612 | United States |
| University of Iowa Stead Family Children's Hospital | Iowa City | Iowa | 52242 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| BG001 | Placebo |
Placebo: The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Participants |
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| Secondary | Change From Baseline in the Timed 1 Minute Walk Distance | Change from baseline Distance walked in 1 minute at 11 months | Posted | Mean | Standard Error | inches | 11 months |
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|
|
| 0 |
| 33 |
| 1 |
| 33 |
| 18 |
| 33 |
| EG001 | Placebo |
Placebo: The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001. | 0 | 32 | 1 | 32 | 19 | 32 |
| Loss of consciousness | Nervous system disorders | Systematic Assessment | Subject had loss of consciousness due to diabetic ketoacidosis |
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| diarrhea | Gastrointestinal disorders | Systematic Assessment |
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| Falls | Injury, poisoning and procedural complications | Systematic Assessment |
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| headache | Nervous system 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 |