A trial to assess the efficacy and safety of ACT-1004-1239 in adults with progressive multiple sclerosis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Idorsia Pharmaceuticals Ltd.

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

3 Mar 2026 → ongoing

Decision date (initial)

2025-12-08

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Idorsia Pharmaceuticals Ltd

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the effect of ACT-1004-1239, compared with placebo, on remyelination in participants with progressive MS, as measured by magnetic resonance imaging (MRI)

Secondary objectives 2

1. To evaluate the effect of ACT-1004-1239, compared with placebo, on remyelination in participants with progressive MS, using electrophysiological and biochemical measures
2. To assess the safety and tolerability of ACT-1004-1239 in participants with progressive MS

Conditions and MedDRA coding

Progressive multiple sclerosis (MS)

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Male or female, aged ≥18 to ≤55 years
2. Diagnosis of primary or secondary progressive MS according to the 2013 revisions of clinical course of MS and the 2017 revisions of the McDonald criteria by an MS- expert neurologist prior to Screening.
3. Absence of clinical relapse within 6 months prior to Screening
4. Expanded disability status scale (EDSS) score ≥ 2.0 to ≤ 6.0
5. If currently on disease-modifying therapy (DMT), it should be stable for ≥ 6 months prior to Screening
6. For participants of childbearing potential: – Agreement to undertake monthly urine or serum pregnancy tests during the trial and up to 30 days after discontinuation of trial intervention – Agreement to use a highly effective method of contraception from Screening up to 30 days after discontinuation of trial intervention

Exclusion criteria 7

1. Inability to comply with MRI scanning or undergo lumbar punctures
2. Known history or presence of other neurologic or systemic autoimmune disorders that are assessed by the investigator to potentially interfere with the collection of data or safety of the participant
3. History of cancer within the last 5 years, including solid tumors and hematological malignancies (except basal cell, in situ squamous cell carcinomas of the skin, and in situ carcinoma of the cervix of the uterus that have been excised and resolved)
4. Active bacterial, viral, fungal, mycobacterial infection or any major episode of infection requiring hospitalization or treatment with intravenous antibiotics within 4 weeks prior to Screening or with oral antibiotics within 2 weeks prior to Screening
5. Not able or willing to stop treatment with moderate or strong CYP3A4 inhibitors or inducers from at least 4 weeks prior to randomization (i.e., at Visit 2)
6. Receipt of a live (or live attenuated) vaccine within 6 weeks prior to randomization (i.e., 2 weeks prior to Visit 2)
7. Female participants: pregnant, lactating or planning to become pregnant during the trial (i.e., until 30 days after permanent discontinuation of trial intervention)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline to Week 24 in myelin water fraction (MWF) of the corpus callosum

Secondary endpoints 7

1. Change from baseline to Week 24 in visual evoked potential (VEP) P100 latency
2. Concentrations of an undisclosed molecule (commercially confidential information) in CSF at Week 12 and at Week 24, normalized to labeled body water at Week 4
3. Concentrations of an undisclosed molecule (commercially confidential information) in CSF at Week 12 and at Week 24, normalized to labeled body water at Week 4
4. Treatment-emergent adverse events (AEs), serious AEs, and AEs of special interest (e.g., suicidal ideation and/or behavior based on the Columbia Suicide Severity Rating Scale [C-SSRS©])
5. AEs leading to premature discontinuation of trial intervention
6. Change from baseline to all assessed time points during the trial in: – vital signs – body weight – laboratory variables – electrocardiogram (ECG)
7. Treatment-emergent marked abnormalities for: – vital signs – clinical laboratory variables – ECG

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Idorsia Pharmaceuticals Ltd.

Sponsor organisation

Idorsia Pharmaceuticals Ltd.

Address

Hegenheimermattweg 91

City

Allschwil

Postcode

4123

Country

Switzerland

Scientific contact point

Organisation

Idorsia Pharmaceuticals Ltd.

Contact name

Idorsia Clinical Trials Information

Public contact point

Organisation

Idorsia Pharmaceuticals Ltd.

Contact name

Idorsia Clinical Trials Information

Third parties 6

Locations

1 EU/EEA country · 1 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 6 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