Trial Outcomes & Findings for An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead) (NCT NCT05267574)

NCT ID: NCT05267574

Last Updated: 2024-05-28

Results Overview

Number of participants with AEs and severity

• Recruitment status: TERMINATED
• Study phase: PHASE2/PHASE3
• Target enrollment: 155 participants
• Primary outcome timeframe: Baseline through study termination, an average of 12.1 months
• Results posted on: 2024-05-28

Participant Flow

Participant milestones

Measure	REN001 100 mg once daily REN001: Once daily dosing
Overall Study STARTED	155
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	155

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

Baseline characteristics by cohort

Measure	REN001 n=155 Participants 100 mg once daily REN001: Once daily dosing
Age, Customized 18-25 years	7 Participants n=5 Participants
Age, Customized 26-45 years	53 Participants n=5 Participants
Age, Customized 46-64 years	78 Participants n=5 Participants
Age, Customized >= 65 years	17 Participants n=5 Participants
Sex: Female, Male Female	103 Participants n=5 Participants
Sex: Female, Male Male	52 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	148 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	6 Participants n=5 Participants
Race/Ethnicity, Customized Asian	4 Participants n=5 Participants
Race/Ethnicity, Customized White	144 Participants n=5 Participants
Race/Ethnicity, Customized Not Reported	5 Participants n=5 Participants
Race/Ethnicity, Customized Other	2 Participants n=5 Participants
Region of Enrollment North America	5 participants n=5 Participants
Region of Enrollment Europe	133 participants n=5 Participants
Region of Enrollment Australia	17 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline through study termination, an average of 12.1 months

Number of participants with AEs and severity

Outcome measures

Measure	REN001 n=155 Participants 100 mg once daily REN001: Once daily dosing
Number of Participants With Mild, Moderate, Severe TEAEs, TEAEs Leading to Study Discontinuation, All TEAEs and All TESAEs Mild TEAEs	68 participants
Number of Participants With Mild, Moderate, Severe TEAEs, TEAEs Leading to Study Discontinuation, All TEAEs and All TESAEs Moderate TEAEs	50 participants
Number of Participants With Mild, Moderate, Severe TEAEs, TEAEs Leading to Study Discontinuation, All TEAEs and All TESAEs Severe TEAEs	5 participants
Number of Participants With Mild, Moderate, Severe TEAEs, TEAEs Leading to Study Discontinuation, All TEAEs and All TESAEs TEAEs leading to study drug discontinuation	1 participants
Number of Participants With Mild, Moderate, Severe TEAEs, TEAEs Leading to Study Discontinuation, All TEAEs and All TESAEs Total TEAEs	123 participants
Number of Participants With Mild, Moderate, Severe TEAEs, TEAEs Leading to Study Discontinuation, All TEAEs and All TESAEs Total TESAEs	12 participants

SECONDARY outcome

Timeframe: Study Termination

Number of participants

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Month 24

Distance walked in meters

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Month 24

The MFIS is a 21-item scale to describe the impact of fatigue on physical, cognitive, and psychosocial functioning. The questionnaire includes 9 physical, 10 cognitive, and 2 psychosocial items with each item scored between 0=Never and 4=Almost Always

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Month 24

The PGIS is a 2-item questionnaire to describe the severity of fatigue and muscle symptoms. Each item is scored as Absent, Mild, Moderate, Severe, or Very Severe

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Month 24

The BPI is a 15-item questionnaire to describe severity of pain and its interference on functioning. The questionnaire includes 4 pain severity items each scored between 0=No Pain and 10= Pain, and 7 pain interference items each scored between 0=Does not Interfere and 10=Completely Interferes

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Month 24

The PROMIS Short Form - FACIT Fatigue 13a is a 13-item questionnaire to describe fatigue and its impact upon daily activities and function. Each item is scored between 1=Not At All and 5=Very Much

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Month 24

The SF-36 is a 36-item questionnaire to describe health status and quality of life. The questionnaire includes 8 domains (physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions). Items are scored, summed into domains, and transformed into a scale of 0-100

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Month 24

The WPAI:SHP is a 6-item questionnaire to describe impairment in work and activities due to a certain disease. Items are scored, summed, and transformed into a scale of 0-100%

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Month 24

The PGIC is a 2-item questionnaire to describe the change in fatigue and muscle symptoms since starting the study. Each item is scored as Very Much Worse, Moderately Worse, Minimally Worse, No Change, Minimally Improved, Moderately Improved, or Very Much Improved

Outcome measures

Outcome data not reported

Adverse Events

REN001

Serious events: 12 serious events

Other events: 123 other events

Deaths: 0 deaths

Serious adverse events

Measure	REN001 n=155 participants at risk 100 mg once daily REN001: Once daily dosing
Cardiac disorders Cardiac Failure	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Cardiac disorders Cardio-respiratory arrest	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Cardiac disorders Supraventricular tachycardia	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Cardiac disorders Ventricular Fibrillation	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Nervous system disorders Cerebrovascular Accident	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Nervous system disorders Migraine	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Nervous system disorders Radiculopathy	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Infections and infestations COVID-19	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Infections and infestations Campylobacter gastroenteritis	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Injury, poisoning and procedural complications Compression fracture	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Injury, poisoning and procedural complications Fall	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Injury, poisoning and procedural complications Lower limb fracture	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Eye disorders Cataract	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Gastrointestinal disorders Intestinal Pseudo-obstruction	0.65% 1/155 • Up to 48 months Regular Investigator assessment
Metabolism and nutrition disorders Lactic acidosis	0.65% 1/155 • Up to 48 months Regular Investigator assessment

Other adverse events

Measure	REN001 n=155 participants at risk 100 mg once daily REN001: Once daily dosing
Infections and infestations COVID-19	16.1% 25/155 • Up to 48 months Regular Investigator assessment
Infections and infestations Upper respiratory tract	6.5% 10/155 • Up to 48 months Regular Investigator assessment
Infections and infestations Influenza	5.8% 9/155 • Up to 48 months Regular Investigator assessment
Gastrointestinal disorders Constipation	5.2% 8/155 • Up to 48 months Regular Investigator assessment
General disorders Fatigue	7.7% 12/155 • Up to 48 months Regular Investigator assessment
Nervous system disorders Headache	11.0% 17/155 • Up to 48 months Regular Investigator assessment
Musculoskeletal and connective tissue disorders Myalgia	7.7% 12/155 • Up to 48 months Regular Investigator assessment
Musculoskeletal and connective tissue disorders Pain in extremity	5.8% 9/155 • Up to 48 months Regular Investigator assessment
Skin and subcutaneous tissue disorders Dry skin	5.2% 8/155 • Up to 48 months Regular Investigator assessment
Metabolism and nutrition disorders Vitamin D deficiency	5.2% 8/155 • Up to 48 months Regular Investigator assessment
Eye disorders Refraction disorder	6.5% 10/155 • Up to 48 months Regular Investigator assessment

Additional Information

Alex Dorenbaum, MD, Chief Medical Officer

Reneo Pharmaceuticals, Inc.

Phone: (415) 328-9115

Email: adorenbaum@reneopharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee An Investigator shall not publish any data (poster, abstract, paper, etc.) without having consulted with the Sponsor in advance.
• Publication restrictions are in place

Restriction type: OTHER