Phase 2
N=14
Safety and Efficacy of Ranolazine for the Treatment of Amyotrophic Lateral Sclerosis
ALS
Bottom Line
View on ClinicalTrials.gov: NCT03472950 ↗Enrolled (actual)
14
Serious AEs
0.0%
Results posted
Dec 2024
Primary outcome: Primary: Dose Limiting Toxicities (DLT) — 1; 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ranolazine 500 MG (Drug); Ranolazine 1000 MG (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- University of Kansas Medical Center
- Primary completion
- Dec 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Dose Limiting Toxicities (DLT) |
1; 2 | — |
| SECONDARY Percent Change in Cramp Frequency |
-39.98; -68.38 | — |
| SECONDARY Percentage Change in Average Weekly Cramp Severity |
-38.68; -54.05 | — |
| SECONDARY Change in Nocturnal Awakenings Per Week, Comparing Week 12 to Baseline |
-1.67; -4.57 | — |
| SECONDARY Muscle Fasciculations Count |
33; 58 | — |
Summary
The purpose of this research study is to evaluate the safety and effectiveness of Ranolazine, and how well it is tolerated in patients with Amyotrophic Lateral Sclerosis (ALS). Ranolazine is an FDA approved drug that is used for decreasing chest pain.
Eligibility Criteria
Inclusion Criteria
- Patients with clinically definite, probable, laboratory supported probable, or possible ALS per revised El Escorial criteria
- Cramp frequency greater than 4 cramps per week during 2 week run in
- ALS functional rating scale-revised (ALSFRS-R) score of greater than 24
- Able to lie on back for study procedures
Exclusion Criteria
- Tracheostomy invasive ventilation, or use of non-invasive ventilation greater than 12 hours per day
- Pregnant or lactating
- Participation in a prior experimental drug trial less than 30 days prior to screening
- Patients taking ranolazine
- Patients taking medications which are contraindicated for use with ranolazine such as strong CYP3 inhibitors (ketoconazole, clarithromycin, nelfinavir), and CYP3 inducers (rifampin, phenobarbital)
- Patients with clinically significant medical comorbidities (hepatic, renal, cardiac, etc)
- Patients with baseline QT interval prolongation on Electrocardiography (ECG)
- Patients pre-disposed to secondary QT prolongation for other health conditions like family history of congenital long QT syndrome, heart failure, bradycardia, or cardiomyopathies
Data sourced from ClinicalTrials.gov (NCT03472950). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.