A Second Intermediate-Size Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (Pridopidine EAP 2)

NCT ID: NCT06069934

Last Updated: 2025-05-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NO_LONGER_AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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Protocol PL101-ALS501: This EAP will provide access to pridopidine for up to 200 patients with ALS who are ineligible for clinical trials.

Detailed Description

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ALS: Pridopidine will be given at a dose of 45 mg twice daily p.o. (or via feeding tube) over a 2-week up-titration period; followed by pridopidine 45 mg twice daily p.o. (or via feeding tube) for the remainder of the treatment period (through Week 104).

Each patient will be followed for 2 years with regularly scheduled visits. The screening and baseline visits will be performed in person; subsequent visits may occur in person or remotely. Recommended in-person visits will occur at Weeks 12, 52, 78, and end of treatment (Week 104 or early termination). If the patient is unable to complete the visits in person, these visits may also be completed remotely.

Conditions

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Amyotrophic Lateral Sclerosis

Interventions

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Pridopidine

Pridopidine 45 mg hard gelatin capsules once daily p.o. (or via feeding tube)

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Sporadic or familial ALS.
* Patient does not qualify for clinical trials of pridopidine or as per site investigator's opinion, and is not medically or geographically suitable for other clinical trials.
* Capable of providing informed consent and complying with study procedures, in the site investigator's opinion.
* Patient has established care with a physician at a specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the EAP.
* Pridopidine naive patients must have a life expectancy of at least 6 months in the site investigator's opinion.

Exclusion Criteria

* Confirmed prolonged Fridericia-corrected QT (QTcF) interval (\>450 ms for men; \>470 ms for women).
* Clinically significant heart disease, clinically significant history of arrhythmia, symptomatic or uncontrolled atrial fibrillation despite treatment, asymptomatic sustained ventricular tachycardia, or left bundle branch block.
* Known history of long QT syndrome or a first degree relative with long QT syndrome.
* Use of prohibited medications within the 4 weeks prior to baseline.
* Use of Nuedexta (\>20 mg dextromethorphan and \>10 mg quinidine twice daily); citalopram \>20 mg/day; escitalopram \>10 mg/day.
* Known allergy to pridopidine or any of the exipients (silicified microcrystalline cellulose, magnesium stearate).
* History of any clinically significant or unstable medical condition or laboratory abnormality that, based on site investigator's judgment, may interfere with assessment of the study objectives.
* Female who is pregnant or nursing or who plans to get pregnant during the course of the EAP.
* Female of child-bearing potential or male unwilling or unable to use accepted methods of birth control.
* Use of investigational treatments for ALS (as part of participation in a clinical trial or another EAP) within 5 half-lives (if known) or 30 days (whichever is longer) prior to screening (other than pridopidine).
* Patient receives or has received any gene or cell-based therapy.
* Active cancer or history of cancer, except for basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.
* Patients who chose to take experimental medications and/or supplements, and for whom this is the only reason they are not eligible for trials.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Massachusetts General Hospital

OTHER

Sponsor Role collaborator

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role collaborator

Prilenia

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Barrow Neurological Institute

Phoenix, Arizona, United States

Site Status

University of California, San Diego Health

La Jolla, California, United States

Site Status

UC Irvine

Orange, California, United States

Site Status

California Pacific Medical Center - Forbes Norris MDA/ALS Research Center

San Francisco, California, United States

Site Status

University of Colorado Anschutz Medical Campus

Aurora, Colorado, United States

Site Status

Hospital for Special Care

New Britain, Connecticut, United States

Site Status

Nova Southeastern University

Fort Lauderdale, Florida, United States

Site Status

Mayo Clinic Jacksonville

Jacksonville, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

Site Status

The University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Lahey Hospital Medical Center

Burlington, Massachusetts, United States

Site Status

University of Massachusetts Chan Medical School

Worcester, Massachusetts, United States

Site Status

Essentia Health

Duluth, Minnesota, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Mayo Clinic Rochester

Rochester, Minnesota, United States

Site Status

Washington University

St Louis, Missouri, United States

Site Status

Columbia University

New York, New York, United States

Site Status

Stony Brook University Hospital

Stony Brook, New York, United States

Site Status

SUNY Upstate

Syracuse, New York, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

OhioHealth

Columbus, Ohio, United States

Site Status

Thomas Jefferson University

Philadelphia, Pennsylvania, United States

Site Status

Lewis Katz School of Medicine at Temple University

Philadelphia, Pennsylvania, United States

Site Status

Texas Neurology

Dallas, Texas, United States

Site Status

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

CHALS-CCT Program, UPR-MSC

San Juan, , Puerto Rico

Site Status

Countries

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United States Puerto Rico

Other Identifiers

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PL101-ALS501

Identifier Type: -

Identifier Source: org_study_id

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