Clinical trial NCT06069934: A Second Intermediate-Size Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (Pridopidine EAP 2)
Official title: Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (PL101-ALS501)
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic Lateral Sclerosis
Pridopidine
Lou Gehrig's disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Cognition Disorders
Neurocognitive Disorders
Mental Disorders
Lead sponsor: Prilenia
Sponsor class: INDUSTRY
Collaborators: name: Massachusetts General Hospital
name: National Institute of Neurological Disorders and Stroke (NINDS)
name: National Institutes of Health (NIH)
First posted: 2023-10-06
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT

Brief summary: 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: 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.

Eligibility criteria: 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Barrow Neurological Institute
state: Arizona
city: La Jolla
country: United States
facility: University of California, San Diego Health
state: California
city: Orange
country: United States
facility: UC Irvine
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Center - Forbes Norris MDA/ALS Research Center
state: California
city: Aurora
country: United States
facility: University of Colorado Anschutz Medical Campus
state: Colorado
city: New Britain
country: United States
facility: Hospital for Special Care
state: Connecticut
city: Fort Lauderdale
country: United States
facility: Nova Southeastern University
state: Florida
city: Jacksonville
country: United States
facility: Mayo Clinic Jacksonville
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Kansas City
country: United States
facility: The University of Kansas Medical Center
state: Kansas
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Burlington
country: United States
facility: Lahey Hospital Medical Center
state: Massachusetts
city: Worcester
country: United States
facility: University of Massachusetts Chan Medical School
state: Massachusetts
city: Duluth
country: United States
facility: Essentia Health
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic Rochester
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: New York
country: United States
facility: Columbia University
state: New York
city: Stony Brook
country: United States
facility: Stony Brook University Hospital
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate
state: New York
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Columbus
country: United States
facility: OhioHealth
state: Ohio
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Philadelphia
country: United States
facility: Lewis Katz School of Medicine at Temple University
state: Pennsylvania
city: Dallas
country: United States
facility: Texas Neurology
state: Texas
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: San Juan
country: Puerto Rico
facility: CHALS-CCT Program, UPR-MSC
Study officials:
References: