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

NCT03472950 | Completed Phase 2 Results FDA Drug

• 1 other identifier: STUDY00141491
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

ALS

Outcome Measures

Primary Outcomes (1)

• Dose Limiting Toxicities (DLT)

  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.

  Up to Week 12

Secondary Outcomes (4)

• Percent Change in Cramp Frequency

  Weekly for 12 weeks

• Percentage Change in Average Weekly Cramp Severity

  Weekly for 12 weeks

• Change in Nocturnal Awakenings Per Week, Comparing Week 12 to Baseline

  Weekly for 12 weeks

• Muscle Fasciculations Count

  Up to week 12

Study Arms (2)

Ranolazine 500mg

EXPERIMENTAL

Participants will take Ranolazine 500mg twice daily for up to 4 weeks.

Drug: Ranolazine 500 MG

Ranolazine 1000mg

EXPERIMENTAL

Participants will take Ranolazine 1000mg twice daily for up to 4 weeks.

Drug: Ranolazine 1000 MG

Interventions

Ranolazine 500 MG DRUG

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 500mg

Ranolazine 1000 MG DRUG

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Kansas Medical Center: lead
• Gilead Sciences: collaborator

Study Sites (1)

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

MeSH Terms

Interventions

Ranolazine

Intervention Hierarchy (Ancestors)

Acetanilides; Anilides; Amides; Organic Chemicals; Aniline Compounds; Amines; Piperazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Jeffrey Statland, MD
• Organization: University of Kansas Medical Center

Jstatland@kumc.edu

913-945-9933

Study Officials

• Jeffrey Statland, MD

  University of Kansas Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: March 1, 2018
• First Posted: March 21, 2018
• Study Start: June 11, 2018
• Primary Completion: December 11, 2020
• Study Completion: December 9, 2022
• Last Updated: December 9, 2024
• Results First Posted: December 9, 2024

Record last verified: 2024-12

Locations

US (1)