Phase 2
N=19
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
Friedreich's Ataxia
Bottom Line
View on ClinicalTrials.gov: NCT02445794 ↗Enrolled (actual)
19
Serious AEs
5.3%
Results posted
Nov 2020
Primary outcome: Primary: Number of Patients With Adverse Events — 5; 2; 5; 2 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Low dose cohort (Drug); High dose cohort (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Biojiva LLC
- Primary completion
- Jun 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients With Adverse Events |
5; 2; 5; 2 | — |
| SECONDARY Pharmacokinetics - Area Under the Concentration-time Curve After a Single Dose |
524; 915 | — |
| SECONDARY Pharmacokinetics - Maximum Observed Plasma Concentration After a Single Dose |
35.9; 58.6 | — |
| SECONDARY Pharmacokinetics - Time to Reach Maximum Plasma Concentration After a Single Dose |
6.05; 8.0 | — |
| SECONDARY Pharmacokinetics - Maximum Observed Plasma Concentration After Final Dose on Day 28 |
91; 359.9 | — |
| SECONDARY Pharmacokinetics - Terminal Half-life Estimation After Final Dose on Day 28 |
42.3; 30.3 | — |
| SECONDARY Change From Baseline at 28 Days in the Timed 25 Foot Walk (T25FW) |
.503; -1.87; -7.475; .608 | — |
| SECONDARY Change From Baseline at 28 Days in the Friedreich Ataxia Rating Scale (FARS) - Neurological Score (Minimum Score 0, Maximum Score 125, Lower is Better) |
-6.22; -6.47; -3.3; -2 | — |
| SECONDARY Change From Baseline at 28 Days in Peak Workload for the Treated Population vs. the Comparator Population |
.08; -.08 | .008 sig |
Summary
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT02445794). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.