Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

Overview

Official title

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral Sclerosis

Brief title

Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

Registration type

ClinicalTrials.gov

NCT number

NCT07322003

Status

Recruiting

Study category

Interventional, Drug trial, Genetic, Presymptomatic

Sponsor / center

Prilenia

Research center

Emory ALS Center

Collaborators

Ferrer Internacional S.A.

Study type

Interventional study

Phase

Phase 3

Enrollment

500

Start date

02-01-2026

Primary completion date

03-XX-2028

Estimated / actual completion date

03-XX-2029

Last source update

09-22-2026

What is being studied?

This is a Phase 3, randomized study consisting of a double-blind placebo-controlled (DBPC) period followed by an open-label extension (OLE) to evaluate the efficacy and safety of pridopidine administered orally at a dose of 45 mg twice a day in adult participants with early and rapidly progressing ALS. Standard of care treatments (e.g. riluzole, edaravone and Nuedexta) will be allowed as long as participants are on a stable dose for at least 4 weeks prior to dosing. In the DBPC period, participants will be randomized in a 3:2 ratio to the pridopidine and placebo arms. In the DBPC period, participants will receive pridopidine or placebo for 48 weeks. In the OLE period, all participants will receive pridopidine for 48 weeks, while maintaining the blind to their original randomization for both the participant as well as the Investigator and other clinical staff. The total study duration per participant will be 102 weeks including screening and follow up. Throughout the study, participants will be assessed through on-site clinic visits and virtual visits (via telephone).

Intervention(s)

Center-specific eligibility information

Emory lists oral medication, vital capacity greater than 60%, disease onset fewer than 18 months before screening, ability to swallow oral medication, and a 48-week double-blind period with an optional open-label extension.

Who may be eligible?

This is a simplified summary. The official study team or research center determines eligibility.

Population / disease status

Presymptomatic gene carrier, Diagnosed ALS

Diagnosis / conditions

Amyotrophic Lateral Sclerosis

Age

18 Years to 80 Years

Disease duration

≤ 18 months

Respiratory criteria

The official eligibility criteria include language relevant to this topic; review the full criteria below.

ALSFRS-R criteria

Not stated in the official study record.

Genetic criteria

The official eligibility criteria include language relevant to this topic; review the full criteria below.

Medication requirements

The official eligibility criteria include language relevant to this topic; review the full criteria below.

Major inclusion and exclusion criteria from the official record

Key Inclusion Criteria:

* Definite ALS or Probable ALS using the El Escorial criteria.
* Symptom onset of ≤18 months at screening.
* Slow vital capacity (SVC) greater or equal to 60% predicted.
* Treatment Research Initiative to Cure ALS (TRICALS) Risk Profile Calculator score, based on the European Network for the Cure of ALS (ENCALS) survival prediction model, in the range of -6 to -2, inclusive, at screening.
* Able to swallow a capsule.

Key Exclusion Criteria:

* Presence of tracheostomy or permanent assisted ventilation.
* 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.
* Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent and participate in the study.
* Clinically significant and/or unstable medical condition (other than ALS) that may either pose a clinically meaningful risk to the participant and/or to study completion.
* Use of medications that prolong QT interval.
* Previous treatment with pridopidine, gene therapy, or antisense oligonucleotides.
* Confirmed mutation in the SOD1, FUS or C9orf72 gene.
* Pregnancy.

Genetics

Gene-specific study?

Yes

Gene or variant

C9orf72, SOD1

Genetic test required?

Not stated in the official study record.

Known carrier required?

Not stated

Confirmed pathogenic variant required?

Not stated in the official study record.

Presymptomatic carriers eligible?

Yes

At-risk relative eligible?

Not stated

Genetic test provided?

Not stated in the official study record.

Genetic counseling provided?

Not stated in the official study record.

Results returned / offered?

Not stated

Family history required?

Not stated in the official study record.

Study design

Randomized?

Not stated in the official study record.

Allocation

Not stated in the official study record.

Intervention model

Not stated in the official study record.

Masking

Not stated in the official study record.

Placebo

Yes

Primary purpose

Not stated in the official study record.

Endpoints

Primary outcome measures

• Change from baseline through Week 26 and Week 48 in the Revised ALS Functional Rating Scale (ALSFRS-R) total score adjusted for mortality

Secondary outcome measures

• Overall survival at Week 96
• Change from baseline through Week 26 and Week 48 in speaking rate as measured by quantitative speech assessment in the clinic
• Change from baseline through Week 48 in intelligibility of speech by quantitative speech assessment in the clinic.
• Change from baseline through Week 48 in percent predicted slow vital capacity (SVC)
• Change from baseline through Week 48 in the Bulbar subdomain of the ALSFRS-R
• Change from baseline through Week 48 in the Amyotrophic Lateral Sclerosis Assessment Questionnaire - 40 (ALSAQ-40)

A biomarker outcome should not automatically be interpreted as a clinical outcome.

Locations

Contact

Research-center contact: Wanda Sanchez · wanda.sanchez@emory.edu

Central contact: Prilenia Medical Information · MedInfo@prilenia.com · 857-574-5755