History of Changes for Study: NCT04615923

HEALEY ALS Platform Trial - Regimen D Pridopidine

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Study Record Versions

Version	Submitted Date	Changes
1	October 29, 2020	None (earliest Version on record)
2	December 25, 2020	Recruitment Status, Study Status, Contacts/Locations and Oversight
3	January 22, 2022	Recruitment Status and Study Status
4	April 27, 2022	Study Status
5	August 15, 2022	Study Status and Study Design
6	September 18, 2022	Study Status
7	May 15, 2023	Study Status
8	June 30, 2023	Quality Control Review has not concluded Returned: July 24, 2023 Recruitment Status, Outcome Measures, Study Status, Document Section
9	August 12, 2023	Study Status, Adverse Events, Outcome Measures

Comparison Format:

Merged
Side-by-Side

Study Identification


Unique Protocol ID:	2019P003518D
Brief Title:	HEALEY ALS Platform Trial - Regimen D Pridopidine
Official Title:	HEALEY ALS Platform Trial - Regimen D Pridopidine
Secondary IDs:

Study Status


Record Verification:	August 2022
Overall Status:	Active, not recruiting
Study Start:	December 18, 2020
Primary Completion:	June 15, 2022 [Actual]
Study Completion:	March 2023 [Anticipated]

First Submitted:	October 29, 2020
First Submitted that Met QC Criteria:	October 29, 2020
First Posted:	November 4, 2020 [Actual]

Last Update Submitted that Met QC Criteria:	August 15, 2022
Last Update Posted:	August 17, 2022 [Actual]

Sponsor/Collaborators


Sponsor:	Merit E. Cudkowicz, MD
Responsible Party:	Sponsor-Investigator Investigator: Merit E. Cudkowicz, MD Official Title: Chief, Neurology Department Affiliation: Massachusetts General Hospital
Collaborators:	Prilenia Therapeutics

Oversight


U.S. FDA-regulated Drug:	Yes
U.S. FDA-regulated Device:	No
Data Monitoring:	Yes

Study Description

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.

Conditions


Conditions:	Amyotrophic Lateral Sclerosis
Keywords:	ALS Placebo-Controlled Double-Blind Regimen Specific Appendix Lou Gehrig's Disease Pridopidine Prilenia Therapeutics

Study Design


Study Type:	Interventional
Primary Purpose:	Treatment
Study Phase:	Phase 2/Phase 3
Interventional Study Model:	Parallel Assignment
Number of Arms:	2
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Allocation:	Randomized
Enrollment:	163 [Actual]

Arms and Interventions

Arms	Assigned Interventions
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

Outcome Measures


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.

Eligibility


Minimum Age:	18 Years
Maximum Age:
Sex:	All
Gender Based:
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). 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). 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. Participants with known history of long QT syndrome or a first degree relative with this condition. Participants using prohibited medications within the 4 weeks prior to the Regimen Specific Screening Visit, as detailed in section 5.9. Participants using the following medications at the time of the Regimen Specific Screening Visit: Nuedexta - at a dosage higher than 20 mg dextromethorphan + 10 mg quinidine BID Citalopram - at a dosage higher than 20 mg/day Escitalopram - at a dosage higher than 10 mg/day Participants with a known allergy to any ingredient of the study intervention (pridopidine, silicified microcrystalline cellulose, and magnesium stearate).

Contacts/Locations


Study Officials:	Merit Cudkowicz, MD Principal Investigator Massachusetts General Hospital
Locations:	United States, Massachusetts
	Healey Center for ALS at Mass General Boston, Massachusetts, United States, 02114

IPDSharing


Plan to Share IPD:	No

References


Citations:
Links:
Available IPD/Information: