Health Trials

Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

ConditionAmyotrophic Lateral Sclerosis
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18-80
SponsorPrilenia

About this trial

The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine. The main question it aims to answer is:

Does pridopidine slow disease progression of ALS?

Researchers will compare pridopidine to a placebo (a look-alike substance that contains no drug) to see if pridopidine works to treat ALS.

Participants will:

Take pridopidine or a placebo by mouth every day for 48 weeks. Afterwards, all participants will take pridopidine for another 48 weeks.

Visit the clinic once every 1-3 months for checkups and tests

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Pridopidine

    Drug

    Pridopidine hard gelatin capsule.

  • Placebo

    Drug

    Placebo hard gelatin capsule.

Treatment groups

500 Participants
are divided into 2 treatment groups
Group A: PridopidineExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

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

The ALSFRS-R encompasses 12 questions grouped into 4 domains to assess bulbar symptoms, limb and trunk functionality, respiratory symptoms, and the need for percutaneous endoscopic gastrostomy (PEG), non-invasive ventilation, or tracheostomy with invasive ventilation. Each function is scored from 0 (no ability) to 4 (normal), with a minimum total score of 0 and maximum total score of 48. Higher scores indicate better functioning.

Time frame
Baseline to Week 48

Secondary outcomes

1

Overall survival at Week 96

Time to death in days from baseline as assessed at the end of open-label extension (OLE) period

Time frame
Baseline to Week 96
2

Change from baseline through Week 26 and Week 48 in speaking rate as measured by quantitative speech assessment in the clinic

Time frame
Baseline to Week 48
3

Change from baseline through Week 48 in intelligibility of speech by quantitative speech assessment in the clinic.

Time frame
Baseline to Week 48.
4

Change from baseline through Week 48 in percent predicted slow vital capacity (SVC)

SVC measures the maximal amount of air exhaled in a relaxed expiration from full inspiration to residual volume. SVC is assessed in the sitting position with a spirometer.

Time frame
Baseline to Week 48

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Prilenia

Lead sponsor

Ferrer Internacional S.A.

Collaborator

Source ClinicalTrials.gov
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