ALSPhase 2NCT03472950
Safety and Efficacy of Ranolazine for the Treatment of Amyotrophic Lateral Sclerosis
A Phase 2 study testing Ranolazine 500 MG and Ranolazine 1000 MG in people with ALS, run by University of Kansas Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- ALS
- What is being tested?
- Ranolazine 500 MG and Ranolazine 1000 MG
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Jun 2018to 9 Dec 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Kansas Medical Center
Kansas City, Kansas, 66160
About this study
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.
Read the detailed description
Amyotrophic Lateral Sclerosis (ALS) is a progressive debilitating and fatal neurodegenerative disease involving the motor neurons in the primary motor cortex, corticospinal tracts, brainstem and spinal cord with 5,000 newly diagnosed patients per year in the USA. There is a pressing need for additional therapies, as the only two FDA-approved drugs for ALS, riluzole and edaravone, showed prolongation of median survival of only two to three months and only a modest benefit in daily functioning, respectively. The ability to identify FDA approved drugs which can be repurposed to ALS, and which may slow disease progression, alleviate symptoms, or prolong survival will have an immediate positive impact of the lives of patients with ALS and their family members. Hypothesis: Ranolazine, an FDA approved drug for angina which inhibits the late Na+ current and intracellular Ca2+ accumulation may be neuroprotective in ALS by reducing neuronal hyperexcitability, may slow disease progression and reduce cramp frequency.
- Conditions
- ALS
- Being tested
- Ranolazine 500 MG, Ranolazine 1000 MG
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ranolazine 500 MGDrug
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 1000 MGDrug
Ranolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen).
| Study group | Role | What they receive |
|---|---|---|
| Ranolazine 500mg | Experimental | Participants will take Ranolazine 500mg twice daily for up to 4 weeks. Receives: Drug: Ranolazine 500 MG |
| Ranolazine 1000mg | Experimental | Participants will take Ranolazine 1000mg twice daily for up to 4 weeks. Receives: Drug: Ranolazine 1000 MG |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to Week 12
Secondary outcomes (4)
Percent Change in Cramp Frequency
The percent change in cramp frequency: average daily cramp frequency, comparing week 12-baseline
Measured: Weekly for 12 weeks
Percentage Change in Average Weekly Cramp Severity
Percent change in average weekly cramp severity (1 being a very mild muscle cramp and 10 being the most severe cramp you ever experienced)
Measured: Weekly for 12 weeks
Change in Nocturnal Awakenings Per Week, Comparing Week 12 to Baseline
Measured: Weekly for 12 weeks
Muscle Fasciculations Count
Measured: Up to week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2018
- Study start11 Jun 2018
- Primary completion11 Dec 2020
- Study completion9 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for ALSStudies of the same condition, open and under way.
- More trials run by University of Kansas Medical CenterThe sponsor's other registered studies.
- 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Gilead Sciences
- Responsible party
- Principal investigator, Jeffrey Statland, University of Kansas Medical Center
- Investigators
- Jeffrey Statland, MD, Principal investigator, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03472950
- Sponsor's protocol ID
- STUDY00141491
- Status last verified
- Dec 2024
- First submitted
- 1 Mar 2018
- Last updated
- 9 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Ranolazine for the Treatment of Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT03472950
- Acronym / sponsor ID
- STUDY00141491
- Conditions
- ALS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Collaborators
- Gilead Sciences
- Study start
- 11 Jun 2018 (actual)
- Primary completion
- 11 Dec 2020 (actual)
- Study completion
- 9 Dec 2022 (actual)
- First posted
- 21 Mar 2018 (actual)
- Results
- Posted 9 Dec 2024
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 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.
Detailed description
Amyotrophic Lateral Sclerosis (ALS) is a progressive debilitating and fatal neurodegenerative disease involving the motor neurons in the primary motor cortex, corticospinal tracts, brainstem and spinal cord with 5,000 newly diagnosed patients per year in the USA. There is a pressing need for additional therapies, as the only two FDA-approved drugs for ALS, riluzole and edaravone, showed prolongation of median survival of only two to three months and only a modest benefit in daily functioning, respectively. The ability to identify FDA approved drugs which can be repurposed to ALS, and which may slow disease progression, alleviate symptoms, or prolong survival will have an immediate positive impact of the lives of patients with ALS and their family members. Hypothesis: Ranolazine, an FDA approved drug for angina which inhibits the late Na+ current and intracellular Ca2+ accumulation may be neuroprotective in ALS by reducing neuronal hyperexcitability, may slow disease progression and reduce cramp frequency.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ranolazine 500mg | Experimental | Drug: Ranolazine 500 MGParticipants will take Ranolazine 500mg twice daily for up to 4 weeks. |
| Ranolazine 1000mg | Experimental | Drug: Ranolazine 1000 MGParticipants will take Ranolazine 1000mg twice daily for up to 4 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (8 listed)
- 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)
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 |
|---|---|---|
| Ranolazine 500 MGRanolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen). | Drug | Ranolazine 500mg |
| Ranolazine 1000 MGRanolazine is an FDA approved drug for angina (ongoing chest pain or pressure that is felt when the heart does not get enough oxygen). | Drug | Ranolazine 1000mg |
Primary outcome
Dose Limiting Toxicities (DLT)
Time frame: Up to Week 12
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.
4 secondary outcomes
Percent Change in Cramp Frequency
Time frame: Weekly for 12 weeks
Percentage Change in Average Weekly Cramp Severity
Time frame: Weekly for 12 weeks
Change in Nocturnal Awakenings Per Week, Comparing Week 12 to Baseline
Time frame: Weekly for 12 weeks
Muscle Fasciculations Count
Time frame: Up to week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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. This study is not recruiting at the moment.
Jeffrey Statland, MD principal investigator
University of Kansas Medical Center
1 site 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 500 MG and Ranolazine 1000 MG in people with ALS, run by University of Kansas Medical Center at 1 site 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 4 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: finished, as of the sponsor's last update on 9 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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?
Yes. Results are posted in the registry (first posted 9 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose Limiting Toxicities (DLT) (time frame: Up to Week 12). 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 9 Dec 2024.