Health Trials

First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)

ConditionsAmyotrophic Lateral Sclerosis (ALS)Mutation in the Superoxide Dismutase-1 (SOD1) Gene
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorRegeneron Pharmaceuticals

About this trial

This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people.

The aim of the study is to see how safe and tolerable the study drug is.

The study is looking at several other research questions, including:

* The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as Cerebrospinal Fluid (CSF) * How much study drug is in the blood and in the CSF, at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects) * What effects the study drug has on ALS symptoms

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Sequential

Treatments tested in this trial

  • ALN-SOD

    Drug

    Administered per the protocol

  • Diluent

    Other intervention

    Administered per the protocol

  • Placebo (PB)

    Drug

    Administered per the protocol

Treatment groups

54 Participants
are divided into 6 treatment groups

6

Treatment groups

See each treatment group below.

Group A: Cohort 1 - Dose Level 1Experimental treatment 3 interventions
Group B: Cohort 2 - Dose Level 2Experimental treatment 3 interventions
Group C: Cohort 3 - Dose Level 3Experimental treatment 3 interventions
Group D: Cohort 4 - Dose Level 4Experimental treatment 3 interventions
Group E: Cohort 5 (Optional) - ≤ Dose Level 5Experimental treatment 3 interventions
Group F: Selected Dose Expansion Cohort(s)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of Treatment-Emergent Adverse Event (TEAEs) in participants treated with ALN-SOD

Time frame
At week 4 and through week 228
2

Severity of TEAEs in participants treated with ALN-SOD

Time frame
At week 4 and through week 228

Secondary outcomes

1

Concentration of Neurofilament Light chain (NfL) in plasma over time

Time frame
Up to approximately week 228
2

Change in concentration of NfL in plasma over time

Time frame
Up to approximately week 228
3

Concentration of SOD1 protein in Cerebrospinal Fluid (CSF) over time

Time frame
Up to approximately week 228
4

Change in concentration of SOD1 protein in CSF over time

Time frame
Up to approximately week 228

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Regeneron Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
Apply now