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

May 14, 2025 updated by: Prilenia

Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (PL101-ALS501)

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

Study Overview

Status

No longer available

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Puerto Rico
      • San Juan, Puerto Rico, 00935
        • CHALS-CCT Program, UPR-MSC
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • University of Alabama at Birmingham
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Barrow Neurological Institute
    • California
      • La Jolla, California, United States, 92037-0886
        • University of California, San Diego Health
      • Orange, California, United States, 92868
        • UC Irvine
      • San Francisco, California, United States, 94109
        • California Pacific Medical Center - Forbes Norris MDA/ALS Research Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado Anschutz Medical Campus
    • Connecticut
      • New Britain, Connecticut, United States, 06053
        • Hospital for Special Care
    • Florida
      • Fort Lauderdale, Florida, United States, 33314
        • Nova Southeastern University
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic Jacksonville
      • Tampa, Florida, United States, 33612
        • University of South Florida
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • The University of Kansas Medical Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
      • Burlington, Massachusetts, United States, 08105
        • Lahey Hospital Medical Center
      • Worcester, Massachusetts, United States, 01655
        • University of Massachusetts Chan Medical School
    • Minnesota
      • Duluth, Minnesota, United States, 55805
        • Essentia Health
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic Rochester
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
    • New York
      • New York, New York, United States, 10032
        • Columbia University
      • Stony Brook, New York, United States, 11794
        • Stony Brook University Hospital
      • Syracuse, New York, United States, 13210
        • SUNY Upstate
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University
    • Ohio
      • Columbus, Ohio, United States, 43214
        • OhioHealth
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Thomas Jefferson University
      • Philadelphia, Pennsylvania, United States, 19140
        • Lewis Katz School of Medicine at Temple University
    • Texas
      • Dallas, Texas, United States, 75390
        • UT Southwestern Medical Center
      • Dallas, Texas, United States, 75206
        • Texas Neurology
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria Amyotrophic Lateral Sclerosis (ALS):

  • 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 ALS:

  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Prilenia

Collaborators

Massachusetts General Hospital
National Institute of Neurological Disorders and Stroke (NINDS)
National Institutes of Health (NIH)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

September 27, 2023

First Submitted That Met QC Criteria

September 27, 2023

First Posted (Actual)

October 6, 2023

Study Record Updates

Last Update Posted (Actual)

May 18, 2025

Last Update Submitted That Met QC Criteria

May 14, 2025

Last Verified

April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PL101-ALS501

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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