Testing a new drug for ALS caused by SOD1 gene mutations

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial is investigating an experimental drug called ALN-SOD in adults with amyotrophic lateral sclerosis (ALS) linked to mutations in the superoxide dismutase-1 (SOD1) gene. It is the first time this drug will be administered to humans, focusing on its safety and tolerability. The study will also evaluate the drug's effects on specific biomarkers in the blood and cerebrospinal fluid, its concentration in these fluids over time, and the body's immune response to the drug. Additionally, the trial aims to assess any changes in ALS symptoms resulting from the treatment.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

1. Weakness attributable to ALS and a SOD1 variant that has been previously described as associated with ALS or is considered likely to cause ALS, as defined in the protocol
2. Slow Vital Capacity (SVC) ≥50% predicted value based on age, gender and height, measured in upright position
3. Body Mass Index (BMI) ≤35 kg/m2 at time of screening
4. If participants are taking riluzole or edaravone, 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
5. Platelet count \>50,000/microliter
6. Has normal blood pressure readings, as defined in the protocol

Key Exclusion Criteria:

1. Concurrent participation in another interventional clinical trial
2. Has had a tracheostomy
3. Has dementia, as assessed by the investigator
4. Has uncontrolled psychiatric disease, including psychosis, active or recent suicidal ideation, untreated major depression, in the past 30 days
5. 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
6. Presence of an implanted shunt for the drainage of CSF or an implanted Central Nervous System (CNS) catheter
7. 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
8. Was hospitalized (ie, \>24 hours) for any reason other than ALS within 30 days of screening
9. Has received treatment with tofersen within 6 months prior to screening

NOTE: Other protocol defined inclusion / exclusion criteria apply

Where it's running

• Concord Repatriation General Hospital
  Concord, New South Wales, Australia Recruiting
• Macquarie University
  Sydney, New South Wales, Australia Recruiting
• Sunshine Coast University Hospital
  Birtinya, Queensland, Australia Recruiting
• KU Leuven
  Leuven, Vlaams-Brabant, Belgium Recruiting
• University of Alberta Hospital, Edmonton, Division of Neurology
  Edmonton, Alberta, Canada Recruiting
• University Hospital - London Health Sciences Centre
  London, Ontario, Canada Recruiting

Study contacts

• Study coordinator: Clinical Trials Administrator
• Email: clinicaltrials@regeneron.com
• Phone: 844-734-6643

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.