A First in Human Study of RT001 in Patients With Friedreich's Ataxia
NCT ID: NCT02445794
Last Updated: 2020-11-27
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
19 participants
INTERVENTIONAL
2015-08-31
2016-07-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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RT001, oral, 1.8 g/day
RT001, oral, 1.8 g QD for 28 days or matching comparator
Low dose cohort
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 900 mg of RT001.
High dose cohort
RT001 comparator is encapsulated non-deuterated linoleic acid ethyl ester.
RT001, oral, 9 g/day
RT001, oral, 4.5 g BID for 28 days or matching comparator
Low dose cohort
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 900 mg of RT001.
High dose cohort
RT001 comparator is encapsulated non-deuterated linoleic acid ethyl ester.
Interventions
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Low dose cohort
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 900 mg of RT001.
High dose cohort
RT001 comparator is encapsulated non-deuterated linoleic acid ethyl ester.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Medical history consistent with the symptoms of FRDA at ≤ 25 years of age
3. Homozygous for GAA repeat expansions in the Frataxin gene in the affected range for FRDA
4. FARS-Neurological score of 20-90 points
5. Ambulatory (with or without assistive device) and capable of performing assessments/evaluations
6. Body Mass Index ≤ 29.9 kg/m2
7. Agrees to dietary restrictions and agrees to receive calls from a dietary coach
8. Signed the informed consent form prior to entry into the study
9. Agrees to spend the required number of overnight clinic days
10. Able to provide the necessary repeated blood samples
Exclusion Criteria
2. Known point mutation in the FXN gene
3. History of malignancies (other than basal cell carcinomas)
4. Impaired renal function at screening
5. Alanine transaminase (ALT) or aspartate transaminase (AST) laboratory values \> 2 x upper limit of normal (ULN) at screening
6. Known hepatitis B surface antigen (HBsAg)-positive, or known or suspected active hepatitis C infection, or is known to be human immunodeficiency virus (HIV) positive
7. Female who is breastfeeding or has a positive pregnancy test
8. Male participant or female participant of child bearing potential, who is sexually active and unwilling/unable to use a medically acceptable and effective double barrier birth control method throughout the study
9. Unwilling or unable to comply with the requirements of the protocol
10. Clinically significant cardiac abnormalities at screening that, in the opinion of the Investigator, would make the patient unsuitable for enrollment
11. Diabetes mellitus (Type 1 or 2)
12. Suicidal ideation as determined by the Columbia-Suicide Severity Rating Scale
13. History, within the last 2 years, of alcohol abuse, significant mental illness, or physical opioid dependence
14. Cannot adhere to the dietary guidance required to be followed by the protocol
15. Cannot take the medication due to impairment in swallowing capsules
18 Years
50 Years
ALL
No
Sponsors
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Biojiva LLC
INDUSTRY
Responsible Party
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Principal Investigators
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Curtis Scribner, MD
Role: STUDY_DIRECTOR
Biojiva LLC
Theresa Zesiewicz, MD
Role: PRINCIPAL_INVESTIGATOR
USF Ataxia Research Center
Locations
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Collaborative Neuroscience Network, LLC
Long Beach, California, United States
University of South Florida
Tampa, Florida, United States
Countries
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Other Identifiers
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RT001-002
Identifier Type: -
Identifier Source: org_study_id