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Study of Safety, Tolerability, and Biological Activity of LAM-002A in C9ORF72-Associated Amyotrophic Lateral Sclerosis

NCT05163886 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2025-06-22

No results posted yet for this study

Summary

This is a clinical trial to evaluate the safety, tolerability, and biological effect of LAM-002A in adults with C9ORF72-associated ALS (C9ALS).

Conditions

Interventions

DRUG

LAM-002A

LAM-002A (apilimod dimesylate) is formulated in capsules containing 25 mg of apilimod dimesylate. The capsule is Swedish orange, size 0.

OTHER

Placebo

Microcrystalline cellulose in Swedish orange, size 0 capsules.

Sponsors & Collaborators

  • OrphAI Therapeutics

    lead INDUSTRY

Principal Investigators

  • Suma Babu, M.D. · Massachusetts General Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-12-23
Primary Completion
2023-01-11
Completion
2025-05-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

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 NCT05163886 on ClinicalTrials.gov