NCT05163886

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

A Phase IIa Trial to Evaluate the Safety, Tolerability, and Biological Activity of LAM-002A (Apilimod Dimesylate Capsules) in C9ORF72-Associated Amyotrophic Lateral Sclerosis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: OrphAI Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

This is a Phase 2a, multicenter, randomized, double-blind, placebo-controlled, biomarker-driven clinical trial evaluating the safety, tolerability, and biological effect of LAM-002A in approximately 12 adults with C9ORF72-associated ALS (C9ALS). In Part A of the study, approximately twelve C9ALS participants will receive either standard of care plus LAM-002A or standard of care and placebo (randomized 2:1) for the first 12 weeks of the study (Core Study). LAM-002A will be administered as oral capsules 125 mg BID (250 mg total daily dose). The LAM-002A dose may be reduced to 100 mg BID (200 mg total daily dose) if expected gastrointestinal side effects develop. Participants who complete the first 12 weeks of the Core Study will be eligible to receive active drug (LAM-002A capsules at maximum tolerated dose of 125 or 100 mg BID) for the remainder of the study (open-label extension \[OLE\]) up to Week 24. In Part B of the study, participants who complete Part A Week 24 on study drug, will be offered the opportunity to opt-in to continue the open label extension for additional 36 weeks starting at the week 24 visit. Participants who elect not to roll over into the Treatment period B will complete the Day 168 visit, followed a final safety telephone call visit at week 28 to complete study participation. Participants who opt-in to the open label extension for an additional 36 weeks will have their end of study safety telephone call at week 64.

Conditions

• Amyotrophic Lateral Sclerosis
• ALS

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2021-12-23
• Primary completion: 2023-01-11
• Completion: 2025-05-31
• First posted: 2021-12-20
• Last updated: 2025-06-22

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study