Ranolazine for ALS

Recruiting at 7 trial locations

Overseen By Katie Lillig, BS

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Jeffrey Statland

Must not be taking: Ranolazine, QT prolonging drugs

Disqualifiers: Pregnancy, Heart disease, Liver disease, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 5

Duration: 24 weeks

72 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness of the medication ranolazine for people with ALS, also known as Lou Gehrig’s disease. The researchers aim to determine if ranolazine can safely reduce muscle cramps and improve daily function and quality of life compared to a placebo. Participants may receive a placebo, a low dose, or a high dose of ranolazine. Individuals diagnosed with ALS who experience frequent muscle cramps might be suitable candidates for this study. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, providing participants an opportunity to contribute to important ALS research.

Do I need to stop my current medications to join the trial?

You may need to stop certain medications, especially if they are investigational drugs or affect the QT interval. If you are on ALS modifying medications or medications for muscle cramps, you must be on a stable dose for at least 30 days before joining the trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ranolazine is generally safe for people with ALS (amyotrophic lateral sclerosis). In a previous study, participants took up to 2000 mg per day without major problems. The most common side effects involved the stomach, such as nausea or an upset stomach, but these were not severe for most people. Ranolazine is also used for other conditions, like angina (chest pain), which provides additional insight into its safety.

In this trial, participants will take either a low dose (500 mg) or a high dose (1000 mg) of ranolazine twice a day. Earlier studies suggest these doses are safe. However, as with any treatment, monitoring for possible side effects remains important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ALS?

Ranolazine is unique for ALS treatment because it targets sodium channels in nerve cells, which may help reduce nerve damage associated with the disease. Current ALS treatments, like riluzole and edaravone, have different mechanisms, focusing on slowing disease progression and reducing oxidative stress, respectively. Researchers are excited about Ranolazine because it offers a novel approach that could complement existing therapies, potentially enhancing overall treatment effectiveness for people with ALS. Additionally, the exploration of both low and high-dose options may provide insights into the optimal dosage needed for maximum benefit.

What evidence suggests that ranolazine might be an effective treatment for ALS?

Research suggests that ranolazine might help reduce muscle cramps in people with ALS (Amyotrophic Lateral Sclerosis). In previous studies where participants knew they were taking the drug, those who took up to 2000 mg of ranolazine daily experienced fewer cramps. Most tolerated the drug well, although some reported stomach issues. In this trial, participants will receive either a low dose (500 mg), a high dose (1000 mg) of ranolazine, or a placebo. Researchers believe ranolazine protects nerve cells by calming overactive nerves, which might help slow the disease's progression. While more research is needed to confirm these benefits, early results are promising for improving the quality of life in ALS patients. ^{1 2 3 4 5}

Who Is on the Research Team?

Jeffrey Statland

Principal Investigator

University of Kansas Medical Center

Are You a Good Fit for This Trial?

This trial is for individuals with ALS, a type of motor neuron disease. Participants should meet specific health criteria to join but the exact inclusion and exclusion details are not provided here.

Inclusion Criteria

Breathing assessment called forced vital capacity (FVC) greater than or equal to 50%

I have been diagnosed with ALS according to specific criteria.

I have had 4 or more cramps per week in the last 2 weeks.

Exclusion Criteria

Participation in an experimental drug trial less than 30 days before screening

I do not have a long QT interval or take medications that could prolong it.

My condition was diagnosed less than 5 years ago.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Ranolazine or placebo orally twice daily for 24 weeks

24 weeks

5 visits (in-person), 4 visits (remote)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ranolazine

Trial Overview The study is testing the safety and effectiveness of Ranolazine compared to a placebo in treating ALS. It will look at how well it reduces cramps, maintains function, and improves quality of life.

How Is the Trial Designed?

Group I: Ranolazine low dose Experimental Treatment 1 Intervention

Participants receive Ranolazine 500mg orally twice daily for 24 weeks.

Group II: Ranolazine high dose Experimental Treatment 1 Intervention

Participants receive Ranolazine 1000mg orally twice daily for 24 weeks.

Group III: Placebo Placebo Group 1 Intervention

Participants receive Ranolazine placebo orally twice daily for 24 weeks.

Who Is Running the Clinical Trial?

Jeffrey Statland

Lead Sponsor

Trials

2

Recruited

100+

Swathy Chandrashekhar, MD

Lead Sponsor

Trials

1

Recruited

70+

Swathy Chandrashekhar, MBBS

Lead Sponsor

ALS Association

Collaborator

Trials

46

Recruited

17,500+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35466411/

Open-label pilot study of ranolazine for cramps in amyotrophic ... Ranolazine was well tolerated in ALS up to 2000 mg/day, with gastrointestinal side effects being the most frequent. Ranolazine reduced cramp frequency and ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06527222

Study Details | NCT06527222 | A Study of Ranolazine in ALS 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.

3. neurology.org neurology.org/doi/10.1212/WNL.90.15_supplement.P4.450

Study Design and Preliminary Results of Safety ... Our hypothesis is that ranolazine may be neuroprotective in ALS by reducing neuronal hyperexcitability, may slow disease progression, reduce cramp frequency, ...

4. neals.org neals.org/als-trials/nct03472950

Safety and Efficacy of Ranolazine for the Treatment ... 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 ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9197965/

Open-label Pilot Study of Ranolazine for Cramps in ... Ranolazine was well tolerated in ALS up to 2000 mg daily with gastrointestinal side effects being the most frequent. Ranolazine reduced cramp frequency and ...