Amyotrophic Lateral SclerosisPhase 2NCT06527222
A Study of Ranolazine in ALS
A Phase 2 study testing Ranolazine in people with Amyotrophic Lateral Sclerosis, run by Swathy Chandrashekhar, MBBS at 6 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Ranolazine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 72 participants
- Where
- 6 sitesUnited States
- Study dates
- 29 Apr 2025to Jul 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
University of Missouri Health Care
Columbia, Missouri, 65212
Amanda Heider, 573-884-6852, ahmc4@umsystem.edu
William D Arnold, MD
- Recruiting
The Ohio State University
Columbus, Ohio, 43210
Atchayah Balakrishna, Atchayah.Balakrishna@osumc.edu
Kaneshia Hives, Kaneshia.Hives@osumc.edu
- Recruiting
University of Kansas Medical Center
Fairway, Kansas, 66205
Nick Staudenmier, BS, 913-945-9922, nstaudenmier@kumc.edu
Katie Lillig, BS, 913-945-9932, Kjennens2@kumc.edu
- Recruiting
Mayo Clinic Florida
Jacksonville, Florida, 32224
Huy B. Tran, 904-953-4503, Tran.Huy1@mayo.edu
Bjorn E Oskarsson, MD
- Recruiting
University of California, San Francisco
San Francisco, California, 94143
Michael Han, 415-353-7381, Michael.Han@ucsf.edu
Neuromuscular.Research@ucsf.edu
- Recruiting
University of Kansas Medical Center: Wichita
Wichita, Kansas, 67214
KUMC CTSU Wichita, 316-293-1833, clinicaltrialunit@kumc.edu
Hannah Hancock, hhancock@kumc.edu
About this study
Official title: Ranolazine in ALS: Safety, and Effect on Cramps, Function and Quality of Life.
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.
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Ranolazine, Placebo
- Keywords
- ALS, Motor Neuron Disease, Lou Gehrig's Disease, Placebo-Controlled
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RanolazineDrug
Also called Ranexa
500mg twice daily
RanolazineDrug
Also called Ranexa
1000 mg twice daily
PlaceboDrug
Ranolazine placebo twice daily
| Study group | Role | What they receive |
|---|---|---|
| Ranolazine low dose | Experimental | Participants receive Ranolazine 500mg orally twice daily for 24 weeks. Receives: Drug: Ranolazine |
| Ranolazine high dose | Experimental | Participants receive Ranolazine 1000mg orally twice daily for 24 weeks. Receives: Drug: Ranolazine |
| Placebo | Placebo comparator | Participants receive Ranolazine placebo orally twice daily for 24 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Treatment-Emergent Adverse Events
Patient report and medical records will be used to document adverse events and severe adverse events. Adverse events and severe adverse events will be assessed by the investigator and reported as needed for safety.
Measured: Up to 28 weeks
Tolerability of treatment assignment
Tolerability will be measured by percentage of patients who complete the treatment assignment. Dose limiting toxicities will be determined by individual with an adverse event necessitating stopping.
Measured: Up to 28 weeks
Muscle cramp frequency
Muscle cramp frequency will be measured numerically with a reporting period of 7 days.
Changes will be compared from baseline to week 28 utilizing a modified Qualitative, Patient-Centered Assessment of Muscle Cramp Impact and Severity questionnaire.
Measured: Up to 28 weeks
Muscle cramp severity
Muscle cramp severity will be measured by a score of 1-10 (1 being a very mild muscle cramp and 10 being the most severe cramp you ever experienced). Changes will be compared from baseline to week 28 utilizing a modified Qualitative, Patient-Centered Assessment of Muscle Cramp Impact and Severity questionnaire.
Measured: Up to 28 weeks
Muscle cramps impact on quality of life
Effect of muscle cramps on quality of life will be measured with three patient reported yes or no questions (Yes indicating an impact on quality of life or no indicating no impact on quality of life). Changes will be compared from baseline to week 28 utilizing a modified Qualitative, Patient-Centered Assessment of Muscle Cramp Impact and Severity questionnaire
Measured: Up to 28 weeks
Safety Lab Cystatin C
Patient safety measured with lab value Cystatin C in mg/L.
Measured: Up to 28 weeks
Safety Lab Estimated Glomerular Filtration Rate (eGFR)
Patient safety measured with lab value eGFR in mL/min/1.73.
Measured: Up to 28 weeks
Safety Lab Alanine Transaminase (ALT)
Patient safety measured with lab value ALT in IU/L.
Measured: Up to 28 weeks
Safety Lab Aspartate Transferase (AST)
Patient safety measured with lab value AST in IU/L.
Measured: Up to 28 weeks
Safety Lab Alkaline Phosphatase (ALP)
Patient safety measured with lab value ALP in IU/L.
Measured: Up to 28 weeks
Safety Lab Total Bilirubin
Patient safety measured with lab value total bilirubin in mg/dL.
Measured: Up to 28 weeks
Secondary outcomes (5)
Muscle strength
Change in muscle strength over time as measured by hand grip and hand-held dynamometry (HHD) in pounds.
Measured: Up to 28 weeks
ALS Functional Rating Scale-Revised (ALSFRS-R)
Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Measured: Up to 28 weeks
Forced Vital Capacity (FVC)
Change in respiratory function as measured by Forced Vital Capacity (FVC) in liters.
Measured: Up to 28 weeks
Serum neurofilament light
Changes in serum neurofilament light measured from baseline to end of treatment assignment. Data will be collected to determine differences between placebo and Ranolazine treatment groups at completion of the study.
Measured: Up to 28 weeks
Lymphocyte Mitochondrial Function
Change in mitochondrial function in lymphocytes measured from baseline to the end of treatment assignment. Data will be collected to determine differences between placebo and Ranolazine treatment groups at completion of the study
Measured: Up to 28 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2024
- Study start29 Apr 2025
- Primary completionJul 2028 (estimated)
- Study completionJul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Swathy Chandrashekhar, MBBS
- Sponsor type
- University, hospital or other
- Collaborators
- ALS Association
- Responsible party
- Sponsor-investigator, Swathy Chandrashekhar, MBBS, University of California, San Francisco
- Investigators
- Jeffrey Statland, MD, Principal investigator, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06527222
- Sponsor's protocol ID
- 24-43175
- Status last verified
- Jun 2026
- First submitted
- 25 Apr 2024
- Last updated
- 8 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ranolazine in ALS: Safety, and Effect on Cramps, Function and Quality of Life.
- ClinicalTrials.gov ID
- NCT06527222
- Acronym / sponsor ID
- 24-43175
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Motor Neuron Disease, Lou Gehrig's Disease, Placebo-Controlled
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- Swathy Chandrashekhar, MBBSUniversity, hospital or other
- Collaborators
- ALS Association
- Study start
- 29 Apr 2025 (actual)
- Primary completion
- Jul 2028 (estimated)
- Study completion
- Jul 2028 (estimated)
- First posted
- 30 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Ranolazine low dose | Experimental | Drug: RanolazineParticipants receive Ranolazine 500mg orally twice daily for 24 weeks. |
| Ranolazine high dose | Experimental | Drug: RanolazineParticipants receive Ranolazine 1000mg orally twice daily for 24 weeks. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants receive Ranolazine placebo orally twice daily for 24 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (8 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RanolazineAlso: Ranexa500mg twice daily | Drug | Ranolazine low dose |
| RanolazineAlso: Ranexa1000 mg twice daily | Drug | Ranolazine high dose |
| PlaceboRanolazine placebo twice daily | Drug | Placebo |
Primary outcome
Frequency of Treatment-Emergent Adverse Events
Time frame: Up to 28 weeks
Patient report and medical records will be used to document adverse events and severe adverse events. Adverse events and severe adverse events will be assessed by the investigator and reported as needed for safety.
Tolerability of treatment assignment
Time frame: Up to 28 weeks
Tolerability will be measured by percentage of patients who complete the treatment assignment. Dose limiting toxicities will be determined by individual with an adverse event necessitating stopping.
Muscle cramp frequency
Time frame: Up to 28 weeks
Muscle cramp frequency will be measured numerically with a reporting period of 7 days. Changes will be compared from baseline to week 28 utilizing a modified Qualitative, Patient-Centered Assessment of Muscle Cramp Impact and Severity questionnaire.
Muscle cramp severity
Time frame: Up to 28 weeks
Muscle cramp severity will be measured by a score of 1-10 (1 being a very mild muscle cramp and 10 being the most severe cramp you ever experienced). Changes will be compared from baseline to week 28 utilizing a modified Qualitative, Patient-Centered Assessment of Muscle Cramp Impact and Severity questionnaire.
Muscle cramps impact on quality of life
Time frame: Up to 28 weeks
Effect of muscle cramps on quality of life will be measured with three patient reported yes or no questions (Yes indicating an impact on quality of life or no indicating no impact on quality of life). Changes will be compared from baseline to week 28 utilizing a modified Qualitative, Patient-Centered Assessment of Muscle Cramp Impact and Severity questionnaire
Safety Lab Cystatin C
Time frame: Up to 28 weeks
Patient safety measured with lab value Cystatin C in mg/L.
Safety Lab Estimated Glomerular Filtration Rate (eGFR)
Time frame: Up to 28 weeks
Patient safety measured with lab value eGFR in mL/min/1.73.
Safety Lab Alanine Transaminase (ALT)
Time frame: Up to 28 weeks
Patient safety measured with lab value ALT in IU/L.
Safety Lab Aspartate Transferase (AST)
Time frame: Up to 28 weeks
Patient safety measured with lab value AST in IU/L.
Safety Lab Alkaline Phosphatase (ALP)
Time frame: Up to 28 weeks
Patient safety measured with lab value ALP in IU/L.
Safety Lab Total Bilirubin
Time frame: Up to 28 weeks
Patient safety measured with lab value total bilirubin in mg/dL.
5 secondary outcomes
Muscle strength
Time frame: Up to 28 weeks
ALS Functional Rating Scale-Revised (ALSFRS-R)
Time frame: Up to 28 weeks
Forced Vital Capacity (FVC)
Time frame: Up to 28 weeks
Serum neurofilament light
Time frame: Up to 28 weeks
Lymphocyte Mitochondrial Function
Time frame: Up to 28 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jeffrey Statland, MD principal investigator
University of Kansas Medical Center
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ranolazine in people with Amyotrophic Lateral Sclerosis, run by Swathy Chandrashekhar, MBBS at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Katie Lillig, BS (913-945-9932, Kjennens2@kumc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of Treatment-Emergent Adverse Events (time frame: Up to 28 weeks). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jun 2026.