A First in Human Study of RT001 in Patients With Friedreich's Ataxia

NCT02445794 | Completed Phase 1 Results

• 1 other identifier: RT001-002
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.

Conditions

Friedreich's Ataxia

Outcome Measures

Primary Outcomes (1)

• Number of Patients With Adverse Events

  28 days

Secondary Outcomes (8)

• Pharmacokinetics - Area Under the Concentration-time Curve After a Single Dose

  24 hours

• Pharmacokinetics - Maximum Observed Plasma Concentration After a Single Dose

  24 hours

• Pharmacokinetics - Time to Reach Maximum Plasma Concentration After a Single Dose

  24 hours

• Pharmacokinetics - Maximum Observed Plasma Concentration After Final Dose on Day 28

  Day 28-Day 31 (3 days)

• Pharmacokinetics - Terminal Half-life Estimation After Final Dose on Day 28

  Day 28-Day 31 (3 days)

Study Arms (2)

RT001, oral, 1.8 g/day

EXPERIMENTAL

RT001, oral, 1.8 g QD for 28 days or matching comparator

Drug: Low dose cohort; Drug: High dose cohort

RT001, oral, 9 g/day

EXPERIMENTAL

RT001, oral, 4.5 g BID for 28 days or matching comparator

Drug: Low dose cohort; Drug: High dose cohort

Interventions

Low dose cohort DRUG

RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 900 mg of RT001.

Also known as: RT001 1.8 g/d (2 capsule per day), RT001 comparator 1.8 g/d (2 capsule per day)

RT001, oral, 1.8 g/day; RT001, oral, 9 g/day

High dose cohort DRUG

RT001 comparator is encapsulated non-deuterated linoleic acid ethyl ester.

Also known as: RT001 9.0 g/d (9 capsule per day), RT001 comparator 9.0 g/d (9 capsule per day)

RT001, oral, 1.8 g/day; RT001, oral, 9 g/day

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female 18 to 50 years of age
• Medical history consistent with the symptoms of FRDA at ≤ 25 years of age
• Homozygous for GAA repeat expansions in the Frataxin gene in the affected range for FRDA
• FARS-Neurological score of 20-90 points
• Ambulatory (with or without assistive device) and capable of performing assessments/evaluations
• Body Mass Index ≤ 29.9 kg/m2
• Agrees to dietary restrictions and agrees to receive calls from a dietary coach
• Signed the informed consent form prior to entry into the study
• Agrees to spend the required number of overnight clinic days
• Able to provide the necessary repeated blood samples

You may not qualify if:

• Received treatment with other experimental therapies within the last 30 days prior to the first dose
• Known point mutation in the FXN gene
• History of malignancies (other than basal cell carcinomas)
• Impaired renal function at screening
• Alanine transaminase (ALT) or aspartate transaminase (AST) laboratory values \> 2 x upper limit of normal (ULN) at screening
• 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
• Female who is breastfeeding or has a positive pregnancy test
• 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
• Unwilling or unable to comply with the requirements of the protocol
• Clinically significant cardiac abnormalities at screening that, in the opinion of the Investigator, would make the patient unsuitable for enrollment
• Diabetes mellitus (Type 1 or 2)
• Suicidal ideation as determined by the Columbia-Suicide Severity Rating Scale
• History, within the last 2 years, of alcohol abuse, significant mental illness, or physical opioid dependence
• Cannot adhere to the dietary guidance required to be followed by the protocol
• Cannot take the medication due to impairment in swallowing capsules

Sponsors & Collaborators

• Biojiva LLC: lead

Study Sites (2)

Collaborative Neuroscience Network, LLC

Long Beach, California, 90806, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

Cohort Studies; RT001

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Epidemiologic Studies; Epidemiologic Study Characteristics; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Results Point of Contact

• Title: Frederic Heerinckx/VP Clinical Operations
• Organization: Retrotope, Inc

frederic@retrotope.com

650-437-0700

Study Officials

• Curtis Scribner, MD

  Biojiva LLC

  STUDY DIRECTOR

• Theresa Zesiewicz, MD

  USF Ataxia Research Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 6, 2015
• First Posted: May 15, 2015
• Study Start: August 1, 2015
• Primary Completion: June 1, 2016
• Study Completion: July 1, 2016
• Last Updated: November 27, 2020
• Results First Posted: November 27, 2020

Record last verified: 2020-09

Locations

US (2)