search
Back to results

Pridopidine in Amyotrophic Lateral Sclerosis (EAP 2)

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
Pridopidine
Sponsored by
Prilenia
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Amyotrophic Lateral Sclerosis focused on measuring Amyotrophic Lateral Sclerosis, Pridopidine, Lou Gehrig's disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All Sexes

Inclusion Criteria: Sporadic or familial ALS. Patient does not qualify for clinical trials of pridopidine or other clinical trials for the treatment of ALS at the enrolling site Capable of providing informed consent and complying with study procedures Patient has established care with a physician at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the EAP Life expectancy of at least 6 months. 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 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 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 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.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    September 27, 2023
    Last Updated
    September 27, 2023
    Sponsor
    Prilenia
    Collaborators
    Massachusetts General Hospital, National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)
    search

    1. Study Identification

    Unique Protocol Identification Number
    NCT06069934
    Brief Title
    Pridopidine in Amyotrophic Lateral Sclerosis (EAP 2)
    Official Title
    A Second Intermediate-Size Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (Pridopidine EAP 2)
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    September 2023
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Prilenia
    Collaborators
    Massachusetts General Hospital, National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)

    4. Oversight

    5. Study Description

    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 4, 12, 28, 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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Amyotrophic Lateral Sclerosis
    Keywords
    Amyotrophic Lateral Sclerosis, Pridopidine, Lou Gehrig's disease

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Pridopidine
    Intervention Description
    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)

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Eligibility Criteria
    Inclusion Criteria: Sporadic or familial ALS. Patient does not qualify for clinical trials of pridopidine or other clinical trials for the treatment of ALS at the enrolling site Capable of providing informed consent and complying with study procedures Patient has established care with a physician at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the EAP Life expectancy of at least 6 months. 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 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 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 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.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Jennifer Kulesa-Kelley
    Phone
    1-857-282
    Ext
    9430
    Email
    Jkulesa-kelley@mgh.harvard.edu
    First Name & Middle Initial & Last Name or Official Title & Degree
    NN

    12. IPD Sharing Statement

    Learn more about this trial

    Pridopidine in Amyotrophic Lateral Sclerosis (EAP 2)

    We'll reach out to this number within 24 hrs