Pridopidine in Amyotrophic Lateral Sclerosis (EAP 2)

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Available

Administrative data

• NCT number: NCT06069934
• Other study ID #: PL101-ALS501
• Status: Available

Study information

• Verified date: June 2024
• Source: Prilenia
• Contact: Victoria Wang
• Phone: +1-857-282
• Email: vjwang[@]mgh.harvard.edu
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: Available
• Enrollment: 0
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• Pridopidine
Pridopidine 45 mg once 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)

Locations

Country	Name	City	State
Puerto Rico	CHALS-CCT Program, UPR-MSC	San Juan
United States	University of Colorado Anschutz Medical Campus	Aurora	Colorado
United States	National Neuromuscular Research Institute	Austin	Texas
United States	Saint Alphonsus Regional Medical Center	Boise	Idaho
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Lahey Hospital Medical Center	Burlington	Massachusetts
United States	University of North Carolina at Chapel Hill	Chapel Hill	North Carolina
United States	(CAMC) Charleston Area Medical Center	Charleston	West Virginia
United States	Northwestern University	Chicago	Illinois
United States	The University of Chicago	Chicago	Illinois
United States	OhioHealth	Columbus	Ohio
United States	Texas Neurology	Dallas	Texas
United States	UT Southwestern Medical Center	Dallas	Texas
United States	Essentia Health	Duluth	Minnesota
United States	Nova Southeastern University	Fort Lauderdale	Florida
United States	Hackensack University Medical Center	Hackensack	New Jersey
United States	Nerve and Muscle Center of Texas	Houston	Texas
United States	UC Irvine	Irvine	California
United States	UW Health University Hospital	Madison	Wisconsin
United States	Hospital for Special Care	New Britain	Connecticut
United States	Barrow Neurological Institute	Phoenix	Arizona
United States	Virginia Commonwealth University	Richmond	Virginia
United States	Mayo Clinic Rochester	Rochester	Minnesota
United States	Washington University	Saint Louis	Missouri
United States	California Pacific Medical Center - Forbes Norris MDA/ALS Research Center	San Francisco	California
United States	Stony Brook University Hospital	Stony Brook	New York
United States	SUNY Upstate	Syracuse	New York
United States	University of South Florida	Tampa	Florida
United States	Georgetown University Medical Center	Washington	District of Columbia
United States	University of Massachusetts Chan Medical School	Worcester	Massachusetts

Sponsors (4)

Lead Sponsor	Collaborator
Prilenia	Massachusetts General Hospital, National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)

Countries where clinical trial is conducted

United States,  Puerto Rico,