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HEALEY ALS Platform Trial - Regimen D Pridopidine

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ClinicalTrials.gov Identifier: NCT04615923
Recruitment Status : Enrolling by invitation
First Posted : November 4, 2020
Last Update Posted : December 29, 2020
Sponsor:
Collaborator:
Prilenia Therapeutics
Information provided by (Responsible Party):
Merit E. Cudkowicz, MD, Massachusetts General Hospital

Brief Summary:

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.

Regimen D will evaluate the safety and efficacy of a single study drug, pridopidine, in participants with ALS.


Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Pridopidine Drug: Matching Placebo Phase 2 Phase 3

Detailed Description:

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683.

Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen.

If a participant is randomized to Regimen D Pridopidine, the participant will complete a screening visit to assess additional Regimen D eligibility criteria. Once Regimen D eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active pridopidine or matching placebo.

Regimen D will enroll by invitation, as participants may not choose to enroll in Regimen D. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen D.

For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: HEALEY ALS Platform Trial - Regimen D Pridopidine
Actual Study Start Date : December 18, 2020
Estimated Primary Completion Date : March 2022
Estimated Study Completion Date : September 2022


Arm Intervention/treatment
Experimental: Pridopidine
Pridopidine is administered orally twice daily for 24 weeks.
Drug: Pridopidine

Administration: Oral

Dose: 45mg twice daily


Placebo Comparator: Matching Placebo
Matching placebo is administered orally twice daily for 24 weeks.
Drug: Matching Placebo

Administration: Oral

Dose: one capsule twice daily





Primary Outcome Measures :
  1. Disease Progression [ Time Frame: 24 Weeks ]
    Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.


Secondary Outcome Measures :
  1. Bulbar Function in Participants with Bulbar Dysfunction [ Time Frame: 24 Weeks ]
    Change in bulbar function over time in participants with bulbar dysfunction at baseline as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) bulbar subdomain. Each question is scored from 4 (normal) to 0 (no ability), with a maximum total score of 12 and a minimum total score of 0 for the bulbar subdomain. Patients with higher scores have more bulbar function.

  2. Bulbar Function in all Randomized Participants [ Time Frame: 24 Weeks ]
    Change in bulbar function over time in all randomized participants as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) bulbar subdomain. Each question is scored from 4 (normal) to 0 (no ability), with a maximum total score of 12 and a minimum total score of 0 for the bulbar subdomain. Patients with higher scores have more bulbar function.

  3. Speech [ Time Frame: 24 Weeks ]
    Change in speech over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) speech subdomain. The speech question is scored from 4 (normal) to 0 (no ability), with a maximum total score of 4 and a minimum total score of 0. Patients with higher scores have better speech.

  4. Respiratory Function [ Time Frame: 24 Weeks ]
    Change in respiratory function over time as measured by Slow Vital Capacity (SVC).

  5. Bulbar Function in Participants with Rapid pre-baseline Progression [ Time Frame: 24 Weeks ]
    Change in bulbar function over time in participants with rapid pre-baseline progression as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) bulbar subdomain. Each question is scored from 4 (normal) to 0 (no ability), with a maximum total score of 12 and a minimum total score of 0 for the bulbar subdomain. Patients with higher scores have more bulbar function.

  6. Time to Bulbar Dysfunction [ Time Frame: 24 Weeks ]
    Time from baseline to the first observed bulbar dysfunction as measured by an ALS Functional Rating Scale-Revised (ALSFRS-R) bulbar subdomain score of less than 12.

  7. Muscle Strength [ Time Frame: 24 Weeks ]
    Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).

  8. Survival [ Time Frame: 24 Weeks ]
    Comparison of rate of occurrence between groups.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).

Exclusion Criteria:

  • The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).

    1. Participants with a confirmed prolonged Fridericia-corrected QT (QTcF) interval (defined as a QTcF interval of >450 ms for men and >470 ms for women).
    2. Participants with clinically significant heart disease, clinically significant history of arrhythmia, symptomatic or uncontrolled atrial fibrillation despite treatment, or asymptomatic sustained ventricular tachycardia, or presence of left bundle branch block.
    3. Participants with known history of long QT syndrome or a first degree relative with this condition.
    4. Participants using prohibited medications within the 4 weeks prior to the Regimen Specific Screening Visit, as detailed in section 5.9.
    5. Participants using the following medications at the time of the Regimen Specific Screening Visit:

      1. Nuedexta - at a dosage higher than 20 mg dextromethorphan + 10 mg quinidine BID
      2. Citalopram - at a dosage higher than 20 mg/day
      3. Escitalopram - at a dosage higher than 10 mg/day
    6. Participants with a known allergy to any ingredient of the study intervention (pridopidine, silicified microcrystalline cellulose, and magnesium stearate).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04615923


Locations
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United States, Massachusetts
Healey Center for ALS at Mass General
Boston, Massachusetts, United States, 02114
Sponsors and Collaborators
Merit E. Cudkowicz, MD
Prilenia Therapeutics
Investigators
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Principal Investigator: Merit Cudkowicz, MD Massachusetts General Hospital
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Responsible Party: Merit E. Cudkowicz, MD, Chief, Neurology Department, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT04615923    
Other Study ID Numbers: 2019P003518D
First Posted: November 4, 2020    Key Record Dates
Last Update Posted: December 29, 2020
Last Verified: December 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Merit E. Cudkowicz, MD, Massachusetts General Hospital:
ALS
Placebo-Controlled
Double-Blind
Regimen Specific Appendix
Lou Gehrig's Disease
Pridopidine
Prilenia Therapeutics
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases