Friedreich's Ataxia
Conditions
Brief summary
The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia
Detailed description
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.
Interventions
DRUGRT001
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.
DRUGPlacebo
The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001.
Sponsors
Biojiva LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Male or female 12 to 50 years of age 2. Medical history consistent with the symptoms of FRDA at ≤ 25 years of age 3. Detection of biallelic pathogenic variants in frataxin gene (FXN) 4. Ambulatory (with or without assistive device) and capable of performing other assessments/evaluations. 5. Must be able to walk 25 feet during the timed 1-minute walk Key
Exclusion criteria
1. Received treatment with other experimental therapies within the last 30 days prior to the first dose 2. Previously participated in the RT001 trial 3. Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed) 4. History of malignancies (other than basal cell carcinomas) 5. Inability to complete CPET protocol 6. Female who is breastfeeding or has a positive pregnancy test 7. History of uncontrolled diabetes mellitus (Type 1 or 2)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) | 11 months | To measure the change in MVO2 from baseline to Month 11 using CPET |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in the Timed 1 Minute Walk Distance | 11 months | Change from baseline Distance walked in 1 minute at 11 months |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| RT001 * 9 capsules daily (8.64 g total dose) given as 3 capsules three times a day (TID) with meals for the first month of treatment.
* Six capsules daily (5.76 g total dose) given as 3 capsules (BID) with breakfast, and 3 capsules with dinner after the first month of treatment
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. | 33 |
| Placebo * 9 capsules daily (8.64 g total dose) given as 3 capsules three times a day (TID) with meals for the first month of treatment.
* Six capsules daily (5.76 g total dose) given as 3 capsules (BID) with breakfast, and 3 capsules with dinner after the first month of treatment
Placebo: The placebo product is composed of encapsulated USP safflower oil. The placebo capsules are identical in appearance and size to RT001. | 32 |
| Total | 65 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 3 |
| Overall Study | Withdrawal by Subject | 7 | 9 |
Baseline characteristics
| Characteristic | Placebo | Total | RT001 |
|---|---|---|---|
| Age, Categorical <=18 years | 13 Participants | 30 Participants | 17 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 19 Participants | 35 Participants | 16 Participants |
| Age, Continuous | 21.4 years STANDARD_DEVIATION 9.99 | 19.7 years STANDARD_DEVIATION 7.99 | 18.0 years STANDARD_DEVIATION 5.02 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants | 2 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 1 Participants | 3 Participants | 2 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) White | 29 Participants | 59 Participants | 30 Participants |
| Region of Enrollment United States | 32 participants | 65 participants | 33 participants |
| Sex: Female, Male Female | 19 Participants | 33 Participants | 14 Participants |
| Sex: Female, Male Male | 13 Participants | 32 Participants | 19 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 33 | 0 / 32 |
| other Total, other adverse events | 18 / 33 | 19 / 32 |
| serious Total, serious adverse events | 1 / 33 | 1 / 32 |
Outcome results
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
Time frame: 11 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| RT001 | Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) | 0.05 ml O2/min/kg | Standard Error 0.04 |
| Placebo | Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) | 0.08 ml O2/min/kg | Standard Error 0.04 |
p-value: 0.5858Mixed Models Analysis
Secondary
Change From Baseline in the Timed 1 Minute Walk Distance
Change from baseline Distance walked in 1 minute at 11 months
Time frame: 11 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| RT001 | Change From Baseline in the Timed 1 Minute Walk Distance | -193.83 inches | Standard Error 73.16 |
| Placebo | Change From Baseline in the Timed 1 Minute Walk Distance | -220.42 inches | Standard Error 78.27 |
p-value: 0.8061Mixed Models Analysis