Clinical trial NCT06527222: A Study of Ranolazine in ALS
Official title: Ranolazine in ALS: Safety, and Effect on Cramps, Function and Quality of Life.
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Motor Neuron Disease
Lou Gehrig's Disease
Placebo-Controlled
Lead sponsor: University of Kansas Medical Center
Sponsor class: OTHER
Collaborators: name: ALS Association
First posted: 2024-07-30
Enrollment: 72
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate safety, effect on cramps, function and quality of life of ranolazine versus placebo for the treatment of ALS.

Detailed description: A prospective, multi center, double-blind, placebo-controlled, parallel group study of 2 doses of ranolazine (500 mg and 1000 mg twice daily) compared to placebo in patients with ALS.

Approximately 72 adults with ALS will be enrolled into the study in the United States at approximately 7 ALS treatment sites.

Participants will take oral ranolazine or placebo twice daily, attend a minimum of 5 onsite research visits, and 4 remote research visits.

The study is estimated to last 28 weeks for each participant.

Eligibility criteria: Inclusion Criteria:

* 18 years or older
* Diagnosed with clinically definite, possible, probably, or lab-supported probable ALS per revised El Escorial criteria
* Breathing assessment called forced vital capacity (FVC) greater than or equal to 50%.
* Able to swallow pills at the start of the study and expected to for the length of the study.
* If on ALS modifying medications must be on a stable dose at least 30 days.
* Experiencing 4 or more cramps per week during a 2-week screening period.

Exclusion Criteria:

* Disease duration \< 5 years
* Tracheostomy invasive ventilation, or noninvasive ventilation of more than 12 hours/day
* Pregnant or lactating, adults unable to consent, and prisoners
* Taking ranolazine or investigational drug or has received an investigational drug within 30 days (or 5 half-lives for drugs, whichever is longer) prior to screening
* Medically uncontrolled comorbidities (heart, liver, kidney disease)
* Baseline QTc interval prolongation \>450 ms for men/ \>470 ms for women, history of long QT syndrome, or medications which prolong the QT interval
* Participation in an experimental drug trial less than 30 days before screening
* Patients have to be on a stable dosage of any medications used to treat muscle cramps for ≥30 days or have been off these medications ≥30 days prior to randomization.

Primary outcomes:
measure: Frequency of Treatment-Emergent Adverse Events
timeFrame: Up to 28 weeks
measure: Tolerability of treatment assignment
timeFrame: Up to 28 weeks
measure: Muscle cramp frequency
timeFrame: Up to 28 weeks
measure: Muscle cramp severity
timeFrame: Up to 28 weeks
measure: Muscle cramps impact on quality of life
timeFrame: Up to 28 weeks
measure: Safety Lab Cystatin C
timeFrame: Up to 28 weeks
measure: Safety Lab Estimated Glomerular Filtration Rate (eGFR)
timeFrame: Up to 28 weeks
measure: Safety Lab Alanine Transaminase (ALT)
timeFrame: Up to 28 weeks
measure: Safety Lab Aspartate Transferase (AST)
timeFrame: Up to 28 weeks
measure: Safety Lab Alkaline Phosphatase (ALP)
timeFrame: Up to 28 weeks
measure: Safety Lab Total Bilirubin
timeFrame: Up to 28 weeks
Secondary outcomes:
measure: Muscle strength
timeFrame: Up to 28 weeks
measure: ALS Functional Rating Scale-Revised (ALSFRS-R)
timeFrame: Up to 28 weeks
measure: Forced Vital Capacity (FVC)
timeFrame: Up to 28 weeks
measure: Serum neurofilament light
timeFrame: Up to 28 weeks
measure: Lymphocyte Mitochondrial Function
timeFrame: Up to 28 weeks
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Jacksonville
country: United States
facility: Mayo Clinic Florida
state: Florida
city: Fairway
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Wichita
country: United States
facility: University of Kansas Medical Center: Wichita
state: Kansas
city: Columbia
country: United States
facility: University of Missouri Health Care
state: Missouri
city: Columbus
country: United States
facility: The Ohio State University
state: Ohio
city: Milwaukee
country: United States
facility: The Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: University of Kansas Medical Center
name: Jeffrey Statland, MD
role: PRINCIPAL_INVESTIGATOR
References: