First in Human (FIH) Study of ALN-SOD in Adult Participants with Amyotrophic Lateral Sclerosis Associated with Mutation in the SOD1 Gene (SOD1-ALS)
- Conditions
- Amyotrophic lateral sclerosis (ALS)
- Registration Number
- 2023-510344-20-00
- Lead Sponsor
- Regeneron Pharmaceuticals Inc.
- Brief Summary
To evaluate the safety and tolerability of single and repeated IT ALN-SOD administration in participants with ALS associated with mutation in the SOD1 gene (SOD1-ALS)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Not specified
- Target Recruitment
- 13
Weakness attributable to ALS and a SOD1 mutation that has been previously described as associated with ALS or is considered likely to cause ALS, as defined in the protocol.
Slow vital capacity (SVC) ≥50% predicted value based on age, gender and height, measured in upright position.
Body Mass Index (BMI) ≤35 kg/m2 at time of screening.
If participants are taking riluzole, edaravone, or AMX0035, they must be on a stable dose for at least 4 weeks prior to initial dosing visit and are expected to remain at that dose until the end of the study.
Platelet count >50,000/microliter.
Has normal blood pressure readings, as defined in the protocol.
Other protocol defined inclusion criteria apply
Concurrent participation in another interventional clinical trial.
Has had a tracheostomy.
Has dementia, as assessed by the investigator.
Has uncontrolled psychiatric disease, including psychosis, active or recent suicidal ideation, untreated major depression, in the past 30 days.
Has a medical history of brain or spinal disease/injury that would interfere with the lumbar puncture (LP) process, CSF circulation or safety assessment, as defined in the protocol.
Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter.
Presents any concern to the study investigator that might confound the results of the study or poses an additional risk to the participant by their participation in the study.
Was hospitalized (ie, >24 hours) for any reason other than ALS within 30 days of the screening visit.
Other protocol defined exclusion criteria apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Primary Outcome Measures
Name Time Method Incidence of treatment-emergent adverse event (TEAEs) in participants treated with ALN-SOD Incidence of treatment-emergent adverse event (TEAEs) in participants treated with ALN-SOD
Severity of TEAEs in participants treated with ALN-SOD Severity of TEAEs in participants treated with ALN-SOD
- Secondary Outcome Measures
Name Time Method Incidence of anti-drug antibodies (ADAs) to ALN-SOD in serum Incidence of anti-drug antibodies (ADAs) to ALN-SOD in serum
Titer of ADAs to ALN-SOD in serum Titer of ADAs to ALN-SOD in serum
Concentration of ALN-SOD in plasma Concentration of ALN-SOD in plasma
Concentration of ALN-SOD in CSF Concentration of ALN-SOD in CSF
Concentration of SOD1 protein in cerebrospinal fluid (CSF) Concentration of SOD1 protein in cerebrospinal fluid (CSF)
Change in concentration of SOD1 protein in CSF Change in concentration of SOD1 protein in CSF
Concentration of neurofilament light chain (NfL) in plasma Concentration of neurofilament light chain (NfL) in plasma
Change in concentration of NfL in plasma Change in concentration of NfL in plasma
Concentration of NfL in CSF Concentration of NfL in CSF
Change in concentration of NfL in CSF Change in concentration of NfL in CSF
Trial Locations
- Locations (14)
Macquarie University
🇦🇺Sydney, New South Wales, Australia
Concord Repatriation General Hospital
🇦🇺Concord, New South Wales, Australia
Sunshine Coast University Hospital
🇦🇺Birtinya, Queensland, Australia
University of Alberta Hospital, Edmonton, Division of Neurology
🇨🇦Edmonton, Alberta, Canada
University Hospital - London Health Sciences Centre
🇨🇦London, Ontario, Canada
Sunnybrook Research Institute
🇨🇦Toronto, Ontario, Canada
Montreal Neurological Institute and Hospital
🇨🇦Montreal, Quebec, Canada
Hokkaido University Hospital
🇯🇵Sapporo, Hokkaido, Japan
Tokushima University Hospital
🇯🇵Tokushima, Tokushima, Japan
Toho University Omori Medical Center
🇯🇵Ōta-ku, Tokyo, Japan
Scroll for more (4 remaining)Macquarie University🇦🇺Sydney, New South Wales, Australia