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Study of AROSOD-1 in Adult Participants With Amyotrophic Lateral Sclerosis (ALS)

NCT05949294 · Status: WITHDRAWN · Phase: PHASE1 · Type: INTERVENTIONAL

Last updated 2024-04-17

No results posted yet for this study

Summary

In this Phase 1 adult symptomatic patients with amyotrophic lateral sclerosis (ALS) carrying a superoxide dismutase 1 (SOD1) gene mutation thought to be causative of ALS, will receive single ascending doses of ARO-SOD1 administered by intrathecal (IT) infusion. The study is primarily intended to evaluate safety, but will also evaluate the effect of ARO-SOD1 on SOD1 cerebrospinal fluid (CSF) levels as a biomarker of pharmacodynamic (PD) effect, therefore lumbar punctures will be required at timepoints throughout the study. After each participant has completed their individual final visit, participants whose SOD1 CSF levels have recovered to a satisfactory level may rescreen and enroll into higher dose cohorts; or if unable or unwilling to rescreen may enroll into an open-label study to be added by amendment when supported by nonclinical data for multidose administration.

Conditions

Interventions

DRUG

ARO-SOD1 Injection

single doses of ARO-SOD1 Injection by IT infusion

Sponsors & Collaborators

  • Arrowhead Pharmaceuticals

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-01-31
Primary Completion
2025-03-31
Completion
2025-03-31
FDA Drug
Yes

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05949294 on ClinicalTrials.gov