Trial Outcomes & Findings for Safety and Efficacy of Ranolazine for the Treatment of Amyotrophic Lateral Sclerosis (NCT NCT03472950)

NCT ID: NCT03472950

Last Updated: 2024-12-09

Results Overview

Measured as any drug-related serious adverse event, or drug-related adverse event necessitating study withdrawal. If a dose has less than 33% DLTs it will be considered tolerable.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 14 participants
• Primary outcome timeframe: Up to Week 12
• Results posted on: 2024-12-09

Participant Flow

Participant milestones

Measure	Ranolazine 500mg Participants will take Ranolazine 500mg twice daily for up to 4 weeks. Ranolazine 500 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).	Ranolazine 1000mg Participants will take Ranolazine 1000mg twice daily for up to 4 weeks. Ranolazine 1000 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).
Overall Study STARTED	6	8
Overall Study COMPLETED	6	4
Overall Study NOT COMPLETED	0	4

Reasons for withdrawal

Measure	Ranolazine 500mg Participants will take Ranolazine 500mg twice daily for up to 4 weeks. Ranolazine 500 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).	Ranolazine 1000mg Participants will take Ranolazine 1000mg twice daily for up to 4 weeks. Ranolazine 1000 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).
Overall Study Lost to Follow-up	0	1
Overall Study Adverse Event	0	3

Baseline Characteristics

Safety and Efficacy of Ranolazine for the Treatment of Amyotrophic Lateral Sclerosis

Baseline characteristics by cohort

Measure	Ranolazine 500mg n=6 Participants Participants will take Ranolazine 500mg twice daily for up to 4 weeks. Ranolazine 500 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).	Ranolazine 1000mg n=8 Participants Participants will take Ranolazine 1000mg twice daily for up to 4 weeks. Ranolazine 1000 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).	Total n=14 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants	6 Participants n=7 Participants	11 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Age, Continuous	53 years n=5 Participants	55 years n=7 Participants	54 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants	6 Participants n=7 Participants	11 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian	6 Participants n=5 Participants	8 Participants n=7 Participants	14 Participants n=5 Participants
Race/Ethnicity, Customized Non Hispanic	6 Participants n=5 Participants	8 Participants n=7 Participants	14 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants	8 participants n=7 Participants	14 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to Week 12

Measured as any drug-related serious adverse event, or drug-related adverse event necessitating study withdrawal. If a dose has less than 33% DLTs it will be considered tolerable.

Outcome measures

Measure	Ranolazine 500mg n=6 Participants Participants will take Ranolazine 500mg twice daily for up to 4 weeks. Ranolazine 500 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).	Ranolazine 1000mg n=8 Participants Participants will take Ranolazine 1000mg twice daily for up to 4 weeks. Ranolazine 1000 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).
Dose Limiting Toxicities (DLT)	1 Participants	2 Participants

SECONDARY outcome

Timeframe: Weekly for 12 weeks

The percent change in cramp frequency: average daily cramp frequency, comparing week 12-baseline

Outcome measures

Measure	Ranolazine 500mg n=6 Participants Participants will take Ranolazine 500mg twice daily for up to 4 weeks. Ranolazine 500 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).	Ranolazine 1000mg n=8 Participants Participants will take Ranolazine 1000mg twice daily for up to 4 weeks. Ranolazine 1000 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).
Percent Change in Cramp Frequency	-39.98 Percent Change in Cramp Frequency Standard Deviation 23.94	-68.38 Percent Change in Cramp Frequency Standard Deviation 27.74

SECONDARY outcome

Timeframe: Weekly for 12 weeks

Percent change in average weekly cramp severity (1 being a very mild muscle cramp and 10 being the most severe cramp you ever experienced)

Outcome measures

Measure	Ranolazine 500mg n=6 Participants Participants will take Ranolazine 500mg twice daily for up to 4 weeks. Ranolazine 500 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).	Ranolazine 1000mg n=8 Participants Participants will take Ranolazine 1000mg twice daily for up to 4 weeks. Ranolazine 1000 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).
Percentage Change in Average Weekly Cramp Severity	-38.68 Percentage change in average weekly cram Standard Deviation 19.46	-54.05 Percentage change in average weekly cram Standard Deviation 39.94

SECONDARY outcome

Timeframe: Weekly for 12 weeks

Outcome measures

Measure	Ranolazine 500mg n=6 Participants Participants will take Ranolazine 500mg twice daily for up to 4 weeks. Ranolazine 500 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).	Ranolazine 1000mg n=8 Participants Participants will take Ranolazine 1000mg twice daily for up to 4 weeks. Ranolazine 1000 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).
Change in Nocturnal Awakenings Per Week, Comparing Week 12 to Baseline	-1.67 Number of nights awakened Standard Deviation 2.09	-4.57 Number of nights awakened Standard Deviation 6.80

SECONDARY outcome

Timeframe: Up to week 12

Outcome measures

Measure	Ranolazine 500mg n=6 Participants Participants will take Ranolazine 500mg twice daily for up to 4 weeks. Ranolazine 500 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).	Ranolazine 1000mg n=8 Participants Participants will take Ranolazine 1000mg twice daily for up to 4 weeks. Ranolazine 1000 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).
Muscle Fasciculations Count	33 Muscle fasciculations count Standard Deviation 25	58 Muscle fasciculations count Standard Deviation 25

Adverse Events

Ranolazine 500mg

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Ranolazine 1000mg

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Ranolazine 500mg n=6 participants at risk Participants will take Ranolazine 500mg twice daily for up to 4 weeks. Ranolazine 500 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).	Ranolazine 1000mg n=8 participants at risk Participants will take Ranolazine 1000mg twice daily for up to 4 weeks. Ranolazine 1000 MG: Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).
Gastrointestinal disorders Constipation	16.7% 1/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	50.0% 4/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Gastrointestinal disorders Gastroesophageal reflux	16.7% 1/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	25.0% 2/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Ear and labyrinth disorders Dizziness	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Gastrointestinal disorders Indigestion	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Number of events 2 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Infections and infestations Dental Abscess	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Infections and infestations Sinusitis	16.7% 1/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	0.00% 0/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Infections and infestations Upper respiratory infection	50.0% 3/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Infections and infestations Influenza	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	25.0% 2/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
General disorders Drowsiness	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	25.0% 2/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Psychiatric disorders Depression	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Gastrointestinal disorders Nausea	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Nervous system disorders Headache	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Gastrointestinal disorders Vomiting	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Musculoskeletal and connective tissue disorders Upper extremity weakness	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Musculoskeletal and connective tissue disorders Decreased muscle tone	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Renal and urinary disorders Urine odor	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.
Musculoskeletal and connective tissue disorders Worsening muscle cramps	0.00% 0/6 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.	12.5% 1/8 • Adverse event data was collected for 12 weeks. This began at baseline visit through the week 12 visit.

Additional Information

Jeffrey Statland, MD

University of Kansas Medical Center

Phone: 913-945-9933

Email: Jstatland@kumc.edu

Results disclosure agreements

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