Amyotrophic Lateral Sclerosis study: ALN-SOD vs Diluent
Official title First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
ClinicalTrials.gov ID: NCT06351592
What this study is testing
What is ALN-SOD?
An investigational drug being studied as a potential treatment for SOD1-related amyotrophic lateral sclerosis (ALS).
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study will check how safe and tolerable ALN‑SOD is and look at its effects on blood and spinal fluid markers, drug levels, antibody production, and ALS symptoms.
- Who it's for
- Adults who have ALS that is caused by a mutation in the SOD1 gene.
- What's involved
- Participants will receive the study drug and have blood and spinal fluid samples taken, along with regular health checks and symptom assessments.
- Time commitment
- The study lasts several months and requires multiple clinic visits for drug administration, blood draws, spinal fluid sampling, and health evaluations.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- 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 or edaravone, they must be on a stable dose for at least 4 weeks prior to initial dosing visit and are expected...
- Platelet count \>50,000/microliter
You likely can't join if
- Concurrent participation in another treatment 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...
- Presence of an implanted shunt for the drainage of CSF or an implanted Central Nervous System (CNS) catheter
See the full eligibility criteria
- 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
- 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 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
- Platelet count \>50,000/microliter
- Has normal blood pressure readings, as defined in the protocol Key
- Concurrent participation in another treatment 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 screening
- Has received treatment with tofersen within 6 months prior to screening NOTE: Other protocol defined inclusion / exclusion criteria apply
The study team makes the final eligibility decision.
Where it is enrolling
- Concord, New South Wales, Australia
- Sydney, New South Wales, Australia
- Birtinya, Queensland, Australia
- Leuven, Vlaams-Brabant, Belgium
- Edmonton, Alberta, Canada
- London, Ontario, Canada
- Toronto, Ontario, Canada
- Montreal, Quebec, Canada
- Ulm, Baden-Wurttemberg, Germany
- Sapporo, Hokkaido, Japan
- Tokushima, Tokushima, Japan
- Ōta-ku, Tokyo, Japan
- Kyoto, Japan
- Krakow, Lesser Poland Voivodeship, Poland
- Zabrze, Silesian Voivodeship, Poland
- Warsaw, Poland
- Seoul, South Korea
- Umeå, Noorland, Sweden
- Huddinge, Stockholm County, Sweden
- Kaohsiung City, Taiwan
+ 1 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Concord, New South Wales, Australia; Sydney, New South Wales, Australia; Birtinya, Queensland, Australia; Leuven, Vlaams-Brabant, Belgium; Edmonton, Alberta, Canada; London, Ontario, Canada and 15 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.