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A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04102501
Recruitment Status : Completed
First Posted : September 25, 2019
Results First Posted : May 27, 2022
Last Update Posted : May 27, 2022
Sponsor:
Information provided by (Responsible Party):
Retrotope, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Friedreich's Ataxia
Interventions Drug: RT001
Drug: Placebo
Enrollment 65
Recruitment Details  
Pre-assignment Details  
Arm/Group Title RT001 Placebo
Hide Arm/Group Description
  • 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.
  • 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.
Period Title: Overall Study
Started 33 32
11 Month Visit 25 20
Completed 25 20
Not Completed 8 12
Reason Not Completed
Withdrawal by Subject             7             9
Adverse Event             1             3
Arm/Group Title RT001 Placebo Total
Hide Arm/Group Description
  • 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.
  • 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.

 Total of all reporting groups
Overall Number of Baseline Participants 33 32 65
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants 32 participants 65 participants
<=18 years
17
  51.5%
13
  40.6%
30
  46.2%
Between 18 and 65 years
16
  48.5%
19
  59.4%
35
  53.8%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 33 participants 32 participants 65 participants
18.0  (5.02) 21.4  (9.99) 19.7  (7.99)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants 32 participants 65 participants
Female
14
  42.4%
19
  59.4%
33
  50.8%
Male
19
  57.6%
13
  40.6%
32
  49.2%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants 32 participants 65 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
2
   6.3%
2
   3.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
30
  90.9%
29
  90.6%
59
  90.8%
More than one race
2
   6.1%
1
   3.1%
3
   4.6%
Unknown or Not Reported
1
   3.0%
0
   0.0%
1
   1.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 33 participants 32 participants 65 participants
33 32 65
1.Primary Outcome
Title Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET)
Hide Description To measure the change in MVO2 from baseline to Month 11 using CPET
Time Frame 11 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RT001 Placebo
Hide Arm/Group Description:
Change from Baseline in MVO2 at 11 months
Change from Baseline in MVO2 at 11 months
Overall Number of Participants Analyzed 25 20
Mean (Standard Error)
Unit of Measure: ml O2/min/kg
0.05  (0.04) 0.08  (0.04)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection RT001, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5858
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline in the Timed 1 Minute Walk Distance
Hide Description Change from baseline Distance walked in 1 minute at 11 months
Time Frame 11 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RT001 Placebo
Hide Arm/Group Description:
Change from baseline in Timed 1 minute Walk distance
Change from baseline in the timed 1 minute walk distance
Overall Number of Participants Analyzed 25 20
Mean (Standard Error)
Unit of Measure: inches
-193.83  (73.16) -220.42  (78.27)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection RT001, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8061
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Time Frame 12 months
Adverse Event Reporting Description Adverse event reporting was conducted in the ITT population.
 
Arm/Group Title RT001 Placebo
Hide Arm/Group Description
  • 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.
  • 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.
All-Cause Mortality
RT001 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/33 (0.00%)      0/32 (0.00%)    
Hide Serious Adverse Events
RT001 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/33 (3.03%)      1/32 (3.13%)    
Cardiac disorders     
non-cardiac chest pain †  1/33 (3.03%)  1 0/32 (0.00%)  0
Nervous system disorders     
Loss of consciousness † [1]  0/33 (0.00%)  0 1/32 (3.13%)  1
†
Indicates events were collected by systematic assessment
[1]
Subject had loss of consciousness due to diabetic ketoacidosis
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
RT001 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   18/33 (54.55%)      19/32 (59.38%)    
Gastrointestinal disorders     
nausea †  4/33 (12.12%)  4 7/32 (21.88%)  7
diarrhea †  3/33 (9.09%)  3 5/32 (15.63%)  5
Injury, poisoning and procedural complications     
Falls †  7/33 (21.21%)  7 5/32 (15.63%)  5
Nervous system disorders     
headache †  4/33 (12.12%)  4 2/32 (6.25%)  2
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Mark G. Midei, MD, VP Medical Affairs
Organization: Retrotope
Phone: 4103715357
EMail: mark@retrotope.com
Layout table for additonal information
Responsible Party: Retrotope, Inc.
ClinicalTrials.gov Identifier: NCT04102501    
Other Study ID Numbers: RT001-006
First Submitted: September 23, 2019
First Posted: September 25, 2019
Results First Submitted: April 6, 2022
Results First Posted: May 27, 2022
Last Update Posted: May 27, 2022