HEALEY ALS Platform Trial - Regimen D Pridopidine

Merit E. Cudkowicz, MD

Status and phase: Completed; Phase 3; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Pridopidine; Drug: Matching Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT04615923

2019P003518D

Details and patient eligibility

About

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.

Full 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.

Enrollment: 163 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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).

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

163 participants in 2 patient groups, including a placebo group

Pridopidine

Experimental group

Description:

Pridopidine is administered orally twice daily for 24 weeks.

Treatment:

Drug: Pridopidine

Matching Placebo

Placebo Comparator group

Description:

Matching placebo is administered orally twice daily for 24 weeks.

Treatment:

Drug: Matching Placebo