A trial to assess the efficacy and safety of ACT-1004-1239 in adults w
A single-center, randomized, double blind, placebo-controlled, parallel-group, proof-of-concept trial to assess the efficacy and safety of ACT-1004-1239 in adults with progressive multiple sclerosis
What it found
No result yet.
What was tested
Treatment tested
- ACT-1004-1239
Placebo
- ACT-1004-1239 matching placebo
What it measured
Main measures
Change from baseline to Week 24 in myelin water fraction (MWF) of the corpus callosum
Myelin repair
Other measures (6)
Change from baseline to Week 24 in visual evoked potential (VEP) P100 latency
Myelin repairConcentrations of an undisclosed molecule (commercially confidential information) in CSF at Week 12 and at Week 24, normalized to labeled body water at Week 4
Blood or spinal fluid markersTreatment-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©])
SafetyAEs leading to premature discontinuation of trial intervention
Change from baseline to all assessed time points during the trial in: – vital signs – body weight – laboratory variables – electrocardiogram (ECG)
SafetyTreatment-emergent marked abnormalities for: – vital signs – clinical laboratory variables – ECG
Safety
Who could take part
Progressive MS · 18 to 64
Show the full criteria
Inclusion criteria
- Male or female, aged ≥18 to ≤55 years - 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. - Absence of clinical relapse within 6 months prior to Screening - Expanded disability status scale (EDSS) score ≥ 2.0 to ≤ 6.0 - If currently on disease-modifying therapy (DMT), it should be stable for ≥ 6 months prior to Screening - 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
- Inability to comply with MRI scanning or undergo lumbar punctures - 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 - 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) - 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 - 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) - Receipt of a live (or live attenuated) vaccine within 6 weeks prior to randomization (i.e., 2 weeks prior to Visit 2) - Female participants: pregnant, lactating or planning to become pregnant during the trial (i.e., until 30 days after permanent discontinuation of trial intervention)
As registered. Criteria can change, so ask the trial team or your neurologist.
Contact details on the registryAbout this trial
In the sponsor's words
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)
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History
Started
Mar 2026
Finished
Feb 2028