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RNA-based Therapy

ALN-SOD for ALS

Phase 1
Recruiting
Research Sponsored by Regeneron Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Has normal blood pressure readings
Weakness attributable to ALS and a SOD1 mutation associated with ALS or likely to cause ALS
Must not have
Uncontrolled psychiatric disease including psychosis, active or recent suicidal ideation, untreated major depression in the past 30 days
Hospitalization (>24 hours) for any reason other than ALS within 30 days of the screening visit
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years

Summary

This trial is testing a new drug, ALN-SOD, on people with a specific type of amyotrophic lateral sclerosis (ALS) caused by a gene mutation called SOD1. The main

Who is the study for?
This trial is for adults with ALS caused by a SOD1 gene mutation. Participants should have an SVC of at least 50% the predicted value, a BMI of 35 or less, and stable doses if taking specific ALS medications. They must not have low platelet counts or abnormal blood pressure.
What is being tested?
The study tests ALN-SOD, an experimental drug for 'SOD1-ALS'. It's the first time this drug is used in humans to evaluate safety and effects on ALS symptoms, biomarkers in blood/CSF, drug levels over time, and potential antibody development against it.
What are the potential side effects?
Since this is a first-in-human study for ALN-SOD, specific side effects are unknown but may include immune reactions like antibodies against the drug which could reduce effectiveness or cause other side effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My blood pressure is within the normal range.
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I have weakness due to ALS and a genetic mutation linked to it.
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My lung function test shows at least 50% of the expected value for my age, gender, and height.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I do not have uncontrolled mental health issues like psychosis or recent severe depression.
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I have not been hospitalized for reasons other than ALS in the last 30 days.
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I have a tracheostomy.
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I have no brain or spinal issues that affect spinal taps or safety assessments.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

4Treatment groups
Experimental Treatment
Group I: Cohort 4 (Optional) ≤ High DoseExperimental Treatment3 Interventions
Placebo during double-blind treatment period
Group II: Cohort 3 - High DoseExperimental Treatment3 Interventions
Placebo during double-blind treatment period
Group III: Cohort 2 - Mid DoseExperimental Treatment3 Interventions
Placebo during double-blind treatment period
Group IV: Cohort 1 - Low DoseExperimental Treatment3 Interventions
Placebo during double-blind treatment period
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo (PB)
2004
Completed Phase 2
~90

Find a Location

Who is running the clinical trial?

Regeneron PharmaceuticalsLead Sponsor
671 Previous Clinical Trials
386,461 Total Patients Enrolled
Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals
284 Previous Clinical Trials
255,301 Total Patients Enrolled
~28 spots leftby Apr 2029
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