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

Biojiva LLC19 enrolled

Overview

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

Study RT001-002 is a randomized, double-blind, controlled, ascending dose study to evaluate the safety, tolerability, pharmacokinetic, disease state, and exploratory endpoints in patients with Friedreich's ataxia after oral administration. The study includes 2 dose levels of RT001.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Friedreich's Ataxia

Interventions

Low dose cohortDRUG

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

High dose cohortDRUG

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

Eligibility

Sex: ALLMin age: 18 YearsMax age: 50 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Male or female 18 to 50 years of age 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: 1. Received treatment with other experimental therapies within the last 30 days prior to the first dose 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

Locations (2)

Collaborative Neuroscience Network, LLC

Long Beach, California, United States

University of South Florida

Tampa, Florida, United States

Outcomes

Primary Outcomes

Number of Patients With Adverse Events

Time frame: 28 days

Secondary Outcomes

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

AUC 0-24 hours post-dose (Hours -1.0 to -0.5 (pre-breakfast, pre-dose), 0.5 (± 5 min), 1 (± 5 min), 1.5 (± 5 min), 2 (± 10 min), 4 (± 10 min) (pre-lunch), 6 (± 10 min), 8 (± 10 min), 12 (± 10 min), and 16 (± 30 min) Day 2: 24 hours following dosing on Day 1 (± 30 min; pre-breakfast, pre-dose) was measured for the low and high dose cohorts after a single dose of RT001

Time frame: 24 hours

Pharmacokinetics - Maximum Observed Plasma Concentration After a Single Dose

Plasma levels were measured for the following time points: Day 1: Hours -1.0 to -0.5 (pre-breakfast, pre-dose), 0.5 (± 5 min), 1 (± 5 min), 1.5 (± 5 min), 2 (± 10 min), 4 (± 10 min) (pre-lunch), 6 (± 10 min), 8 (± 10 min), 12 (± 10 min), and 16 (± 30 min) Day 2: 24 hours following dosing on Day 1 (± 30 min; pre-breakfast, pre-dose) PK curves were constructed from these data and CMax measured on the curves for the low and high dose cohorts

Time frame: 24 hours

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

TMax measured for the low and high dose cohorts

Time frame: 24 hours

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

After 28 days of dosing, the final dose of RT001 was administered, and PK samples were obtained at the following timepoints (all timepoints refer to final dose on Day 28): Day 28: Hours -1.0 to -0.5 (pre-breakfast, pre-dose on Day 28), 0.5 (± 5 min), 1 (± 5 min), 1.5 (± 5 min), 2 (± 10 min), 4 (± 10 min) (pre-lunch), 6 (± 10 min), 8 (± 10 min), 12 (± 10 min), and 16 (± 30 min) Day 29: Hours 24 (± 30 min; pre-breakfast) and 32 (± 30 min) hours following final dose on Day 28 Day 30: 48 hours following final dose on Day 28 (± 30 min; pre-breakfast) Day 31: 72 hours following final dose on Day 28 (± 30 min; pre-breakfast) PK curves were constructed, and CMax at 28 days was determined from these curves

Data from ClinicalTrials.gov

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