Clinical trial NCT04615923: HEALEY ALS Platform Trial - Regimen D Pridopidine
Official title: HEALEY ALS Platform Trial - Regimen D Pridopidine
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Placebo-Controlled
Double-Blind
Regimen Specific Appendix
Lou Gehrig's Disease
Pridopidine
Prilenia Therapeutics
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Prilenia
First posted: 2020-11-04
Enrollment: 163
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility 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).

Primary outcomes:
measure: Disease Progression as Assessed by the ALSFRS-R Total Score
timeFrame: Baseline to 24 Weeks
measure: Mortality Event Rate
timeFrame: Baseline to 24 Weeks
Secondary outcomes:
measure: Change in Bulbar Function in Participants With Bulbar Dysfunction at Baseline
timeFrame: Baseline to 24 Weeks
measure: Bulbar Function in All Randomized Participants
timeFrame: Baseline to 24 Weeks
measure: Respiratory Function
timeFrame: Baseline to 24 Weeks
measure: Bulbar Function in Participants With Rapid Pre-baseline Progression
timeFrame: Baseline to 24 Weeks
measure: Time to Bulbar Decline
timeFrame: Baseline to 24 Weeks
measure: Muscle Strength
timeFrame: Baseline to 24 Weeks
measure: Number of Participants That Experienced Death or Death Equivalent
timeFrame: Baseline to 24 Weeks
Other outcomes:
Locations:
city: Boston
country: United States
facility: Healey Center for ALS at Mass General
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Merit Cudkowicz, MD
role: PRINCIPAL_INVESTIGATOR
References: