Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS (PREVAiLS)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ferrer Internacional S.A., Prilenia Therapeutics B.V.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

12 May 2026 → ongoing

Decision date (initial)

2026-04-27

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Prilenia Therapeutics BV and Ferrer Internacional, S.A.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Therapy

To evaluate the effect of pridopidine on disease progression in participants with ALS

Secondary objectives 6

1. To evaluate the effect of pridopidine on survival
2. To evaluate the impact of pridopidine on speech
3. To evaluate the effect of pridopidine on the decline in respiratory function
4. To evaluate the effect of pridopidine on the decline in bulbar function
5. To evaluate the effect of pridopidine on quality of life
6. To evaluate the safety and tolerability of pridopidine in participants with ALS

Conditions and MedDRA coding

Amyotrophic Lateral Sclerosis

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Age 18 to 80 years (both inclusive), at the time of signing the informed consent
2. Definite ALS or Probable ALS using the El Escorial criteria
3. Symptom onset of ≤18 months at screening
4. SVC greater or equal to 60% predicted
5. 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

Exclusion criteria 4

1. Presence of tracheostomy or permanent assisted ventilation
2. 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. 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
4. 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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline through Week 26 in the ALSFRS-R total score adjusted for mortality. Change from baseline through Week 48 in the ALSFRS-R total score adjusted for mortality.

Secondary endpoints 6

1. Overall survival at Week 96
2. Change from baseline through Week 26, Week 48 in Speaking rate as measured by quantitative speech assessment in the clinic. Change from baseline through Week 48 in Intelligibility of speech as measured by quantitative speech assessment in the clinic.
3. Change from baseline through Week 48 in percent predicted slow vital capacity
4. Change from baseline through Week 48 in the Bulbar subdomain of the ALSFRS-R
5. Change from baseline through Week 48 in the ALSAQ-40
6. Incidence, nature, and severity of treatment emergent adverse events, adverse events of special interest, and serious adverse events. Incidence and shifts of clinically significant abnormalities in ECG, laboratory tests, and vital signs. Change from baseline in C-SSRS. Tolerability.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ferrer Internacional S.A.

Sponsor organisation

Ferrer Internacional S.A.

Address

Avinguda Diagonal 549

City

Barcelona

Postcode

08029

Country

Spain

Scientific contact point

Organisation

Ferrer Internacional S.A.

Contact name

Ferrer MedInfor

Public contact point

Organisation

Ferrer Internacional S.A.

Contact name

Ferrer MedInfor

Prilenia Therapeutics B.V.

Sponsor organisation

Prilenia Therapeutics B.V.

Address

Gooimeer 2/35

City

Naarden

Postcode

1411 DC

Country

Netherlands

Scientific contact point

Organisation

Prilenia Therapeutics B.V.

Contact name

Medical Information

Public contact point

Organisation

Prilenia Therapeutics B.V.

Contact name

Medical Information

Third parties 12

Sponsor responsibilities

Article 77 compliance

Ferrer Internacional S.A.

Contact point sponsor

Ferrer Internacional S.A.

Article 77 implementation

Ferrer Internacional S.A.

Locations

9 EU/EEA countries · 30 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 126 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications