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

Browse trials for

• Ataxia 295
• Cerebellar Ataxia 212
• Friedreich Ataxia 107

Ataxia

295 studies on the registry are indexed under Ataxia; 51 are open to participants now.

This study's enrollment of 19 is below the median of 26 across 216 interventional studies indexed under Ataxia.

Lead sponsor

Retrotope, Inc. is the lead sponsor of 6 studies on the registry; none are open to participants now.

Study arms

• Experimental
  RT001, oral, 1.8 g/day

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

  Drug: Low dose cohort · Drug: High dose cohort

• Experimental
  RT001, oral, 9 g/day

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

  Drug: Low dose cohort · Drug: High dose cohort

Interventions

• DrugLow dose cohort

  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)

• DrugHigh dose cohort

  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)

Primary outcomes

1. Number of Patients With Adverse Events

   Time frame: 28 days

Secondary outcomes

1. 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

2. 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

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

   TMax measured for the low and high dose cohorts

   Time frame: 24 hours

4. 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

   Time frame: Day 28-Day 31 (3 days)

5. Pharmacokinetics - Terminal Half-life Estimation 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 T1/2 at 28 days was determined from these curves

   Time frame: Day 28-Day 31 (3 days)

6. Change From Baseline at 28 Days in the Timed 25 Foot Walk (T25FW)

   The T25FW is a quantitative mobility and leg function performance test based on a timed 25-foot walk. T25FW was measured at baseline and at 28 days. These data were compared.

   Time frame: 28 days

7. Change From Baseline at 28 Days in the Friedreich Ataxia Rating Scale (FARS) - Neurological Score (Minimum Score 0, Maximum Score 125, Lower is Better)

   The FARS-neurological rating scale specifically developed and validated for Friedreich's Ataxia. The FARS-Neurological included evaluations of the neurological signs that specifically reflect neural substrates affected in patients with FA. Based on a neurological examination bulbar (11 points), upper limb coordination (36 points), lower limb coordination (16 points), peripheral nervous system (26 points), and upright stability (36 points) functions were assessed for individual sub-scores (11, 36, 16, 26, and 36) with a maximum score of 125 (Friedreich's Ataxia Study Group, Subramony et al., 2005, Lynch et al., 2006). FARS-Neurologic examinations were conducted by a qualified physician or health professional trained in the use of the FARS format. A lower score is better. The minimum score is 0, the maximum score is 125.

   Time frame: 28 days

8. Change From Baseline at 28 Days in Peak Workload for the Treated Population vs. the Comparator Population

   Peak workload was measured using cardiopulmonary exercise testing at baseline and after 28 days of treatment. The results of treatment were compared to baseline examination.

   Time frame: 28 days

Baseline characteristics