Trial Outcomes & Findings for A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia (NCT NCT04102501)

NCT ID: NCT04102501

Last Updated: 2022-05-27

Results Overview

To measure the change in MVO2 from baseline to Month 11 using CPET

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 65 participants
• Primary outcome timeframe: 11 months
• Results posted on: 2022-05-27

Participant Flow

Participant milestones

Measure	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.	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.
Overall Study STARTED	33	32
Overall Study 11 Month Visit	25	20
Overall Study COMPLETED	25	20
Overall Study NOT COMPLETED	8	12

Reasons for withdrawal

Measure	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.	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.
Overall Study Withdrawal by Subject	7	9
Overall Study Adverse Event	1	3

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	RT001 n=33 Participants * 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.	Placebo n=32 Participants * 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 n=65 Participants Total of all reporting groups
Age, Categorical <=18 years	17 Participants n=5 Participants	13 Participants n=7 Participants	30 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	16 Participants n=5 Participants	19 Participants n=7 Participants	35 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	18.0 years STANDARD_DEVIATION 5.02 • n=5 Participants	21.4 years STANDARD_DEVIATION 9.99 • n=7 Participants	19.7 years STANDARD_DEVIATION 7.99 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants	19 Participants n=7 Participants	33 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants	13 Participants n=7 Participants	32 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	30 Participants n=5 Participants	29 Participants n=7 Participants	59 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Region of Enrollment United States	33 participants n=5 Participants	32 participants n=7 Participants	65 participants n=5 Participants

PRIMARY outcome

Timeframe: 11 months

To measure the change in MVO2 from baseline to Month 11 using CPET

Outcome measures

Measure	RT001 n=25 Participants Change from Baseline in MVO2 at 11 months	Placebo n=20 Participants Change from Baseline in MVO2 at 11 months
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	0.08 ml O2/min/kg Standard Error 0.04

SECONDARY outcome

Timeframe: 11 months

Change from baseline Distance walked in 1 minute at 11 months

Outcome measures

Measure	RT001 n=25 Participants Change from Baseline in MVO2 at 11 months	Placebo n=20 Participants Change from Baseline in MVO2 at 11 months
Change From Baseline in the Timed 1 Minute Walk Distance	-193.83 inches Standard Error 73.16	-220.42 inches Standard Error 78.27

Adverse Events

RT001

Serious events: 1 serious events

Other events: 18 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Measure	RT001 n=33 participants at risk * 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.	Placebo n=32 participants at risk * 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.
Cardiac disorders non-cardiac chest pain	3.0% 1/33 • Number of events 1 • 12 months Adverse event reporting was conducted in the ITT population.	0.00% 0/32 • 12 months Adverse event reporting was conducted in the ITT population.
Nervous system disorders Loss of consciousness	0.00% 0/33 • 12 months Adverse event reporting was conducted in the ITT population.	3.1% 1/32 • Number of events 1 • 12 months Adverse event reporting was conducted in the ITT population.

Other adverse events

Measure	RT001 n=33 participants at risk * 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.	Placebo n=32 participants at risk * 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.
Gastrointestinal disorders nausea	12.1% 4/33 • Number of events 4 • 12 months Adverse event reporting was conducted in the ITT population.	21.9% 7/32 • Number of events 7 • 12 months Adverse event reporting was conducted in the ITT population.
Gastrointestinal disorders diarrhea	9.1% 3/33 • Number of events 3 • 12 months Adverse event reporting was conducted in the ITT population.	15.6% 5/32 • Number of events 5 • 12 months Adverse event reporting was conducted in the ITT population.
Injury, poisoning and procedural complications Falls	21.2% 7/33 • Number of events 7 • 12 months Adverse event reporting was conducted in the ITT population.	15.6% 5/32 • Number of events 5 • 12 months Adverse event reporting was conducted in the ITT population.
Nervous system disorders headache	12.1% 4/33 • Number of events 4 • 12 months Adverse event reporting was conducted in the ITT population.	6.2% 2/32 • Number of events 2 • 12 months Adverse event reporting was conducted in the ITT population.

Additional Information

Mark G. Midei, MD, VP Medical Affairs

Retrotope

Phone: 4103715357

Email: mark@retrotope.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place