MedPath

Pridopidine in Amyotrophic Lateral Sclerosis (EAP 2)

Conditions
Amyotrophic Lateral Sclerosis
Registration Number
NCT06069934
Lead Sponsor
Prilenia
Brief Summary

This EAP will provide access to pridopidine for up to 200 patients with ALS who are ineligible for clinical trials. Pridopidine will be given at a dose of 45 mg twice daily p.o. (or via feeding tube).

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.

Detailed Description

Not available

Recruitment & Eligibility

Status
NO_LONGER_AVAILABLE
Sex
All
Target Recruitment
Not specified
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.
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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.
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Study & Design

Study Type
EXPANDED_ACCESS
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (40)

University of Alabama at Birmingham

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Birmingham, Alabama, United States

Barrow Neurological Institute

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Phoenix, Arizona, United States

University of California, San Diego Health

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La Jolla, California, United States

UC Irvine

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Orange, California, United States

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

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San Francisco, California, United States

University of Colorado Anschutz Medical Campus

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Aurora, Colorado, United States

Hospital for Special Care

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New Britain, Connecticut, United States

Georgetown University Medical Center

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Washington, District of Columbia, United States

Nova Southeastern University

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Fort Lauderdale, Florida, United States

University of South Florida

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Tampa, Florida, United States

Saint Alphonsus Regional Medical Center

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Boise, Idaho, United States

Northwestern University

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Chicago, Illinois, United States

The University of Chicago

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Chicago, Illinois, United States

University of Iowa Hospitals and Clinics

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Iowa City, Iowa, United States

The University of Kansas Medical Center

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Kansas City, Kansas, United States

Massachusetts General Hospital

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Boston, Massachusetts, United States

Lahey Hospital Medical Center

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Burlington, Massachusetts, United States

University of Massachusetts Chan Medical School

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Worcester, Massachusetts, United States

Essentia Health

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Duluth, Minnesota, United States

University of Minnesota

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Minneapolis, Minnesota, United States

Mayo Clinic Rochester

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Rochester, Minnesota, United States

Washington University

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Saint Louis, Missouri, United States

Hackensack University Medical Center

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Hackensack, New Jersey, United States

SUNY Upstate

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Syracuse, New York, United States

Stony Brook University Hospital

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Stony Brook, New York, United States

University of North Carolina at Chapel Hill

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Chapel Hill, North Carolina, United States

Duke University

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Durham, North Carolina, United States

OhioHealth

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Columbus, Ohio, United States

Providence ALS Clinic

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Portland, Oregon, United States

Thomas Jefferson University

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Philadelphia, Pennsylvania, United States

Lewis Katz School of Medicine at Temple University

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Philadelphia, Pennsylvania, United States

Austinl Neuromuscular Center

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Austin, Texas, United States

Texas Neurology

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Dallas, Texas, United States

UT Southwestern Medical Center

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Dallas, Texas, United States

Nerve and Muscle Center of Texas

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Houston, Texas, United States

Virginia Commonwealth University

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Richmond, Virginia, United States

(CAMC) Charleston Area Medical Center

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Charleston, West Virginia, United States

UW Health University Hospital

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Madison, Wisconsin, United States

Medical College of Wisconsin

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Milwaukee, Wisconsin, United States

CHALS-CCT Program, UPR-MSC

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San Juan, Puerto Rico

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