ACT-1004-1239 for Progressive multiple sclerosis

Inclusion criteria

• {"criterion_text":"- Male or female, aged ≥18 to ≤55 years"}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Absence of clinical relapse within 6 months prior to Screening"}
• {"criterion_text":"- Expanded disability status scale (EDSS) score ≥ 2.0 to ≤ 6.0"}
• {"criterion_text":"- If currently on disease-modifying therapy (DMT) (except B-cell depleting therapies), it should be stable and initiated ≥ 6 months prior to Screening."}
• {"criterion_text":"- 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

• {"criterion_text":"- Inability to comply with MRI scanning or undergo lumbar punctures"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- 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)"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- 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)"}
• {"criterion_text":"- Receipt of a live (or live attenuated) vaccine within 6 weeks prior to randomization (i.e., 2 weeks prior to Visit 2)"}
• {"criterion_text":"- Treatment with B-cell depleting therapies (including but not limited to ocrelizumab, ofatumumab, cladribine, mitoxantrone) within 6 months prior to Screening"}
• {"criterion_text":"- Female participants: pregnant, lactating or planning to become pregnant during the trial (i.e., until 30 days after permanent discontinuation of trial intervention)"}

Primary endpoints

• {"endpoint_text":"- Change from baseline to Week 24 in myelin water fraction (MWF) of the corpus callosum","definition_or_measurement_approach":"Measured by magnetic resonance imaging (MRI)"}

Secondary endpoints

• {"endpoint_text":"- Change from baseline to Week 24 in visual evoked potential (VEP) P100 latency","definition_or_measurement_approach":"Electrophysiological measure (VEP P100 latency) assessed as change from baseline to Week 24"}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Biochemical concentration in CSF measured at Week 12 and Week 24 and normalized to labeled body water at Week 4"}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Biochemical concentration in CSF measured at Week 12 and Week 24 and normalized to labeled body water at Week 4"}
• {"endpoint_text":"- 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©])","definition_or_measurement_approach":"Safety monitoring of AEs, SAEs, and AEs of special interest including C-SSRS assessments"}
• {"endpoint_text":"- AEs leading to premature discontinuation of trial intervention","definition_or_measurement_approach":"Recording of adverse events that result in participant discontinuation"}
• {"endpoint_text":"- Change from baseline to all assessed time points during the trial in: – vital signs – body weight – laboratory variables – electrocardiogram (ECG)","definition_or_measurement_approach":"Serial assessments of vital signs, body weight, laboratory variables and ECG at all planned time points"}
• {"endpoint_text":"- Treatment-emergent marked abnormalities for: – vital signs – clinical laboratory variables – ECG","definition_or_measurement_approach":"Identification and reporting of marked abnormalities in vital signs, lab variables and ECG during treatment"}

Netherlands

Earliest CTIS Part Ii Submission Date

04-12-2025

Latest Decision Or Authorization Date

08-12-2025

Processing Time Days

4

Number Of Sites

1

Number Of Participants

32

Primary sponsor

Full Name

Idorsia Pharmaceuticals Ltd.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Switzerland

Third parties

• {"country":"Germany","full_name":"DATAMAP-Gesellschaft fuer Datenmanagement Datenanalyse und Datenpraesentation mbH","duties_or_roles":"ISAC support","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Italy","full_name":"Neuroquantic S.r.l.s.","duties_or_roles":"VEP Central Reader","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Netherlands","full_name":"Leids Universitair Medisch Centrum (LUMC)","duties_or_roles":"Analyze microbiology, chemistry, heamatology labs","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Slovakia","full_name":"SanaClis s.r.o.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"Ardena Bioanalysis B.V.","duties_or_roles":"analyze PK samples and CSF","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"Centre for Human Drug Research","duties_or_roles":"study design/support (in writing of the protocol); Creation of the eCRF, Provide datasets for analysis, creation of the SAP and Final Study report.","organisation_type":"Laboratory/Research/Testing facility"}