NCT04745299

Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients with ALS

A Double-blind, Randomized, Multicenter, Placebo-Controlled, Parallel, Phase III Clinical Trial to Evaluate the Efficacy and Safety of Lenzumestrocel(Neuronata-R® Inj.) in Patients with Amyotrophic Lateral Sclerosis

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 115 (estimated)
• Sponsor: Corestemchemon, Inc. · Industry
• Sex: All
• Age: 25 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

ALSUMMIT is a double-blind, randomized, placebo-controlled, multi-center, parallel, phase III clinical trial to evaluate and confirm the efficacy and long-term safety of repeated Lenzumestrocel (Neuronata-R® inj.) treatment.

Detailed description

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by selective and progressive loss of motor neurons. Disease progression leads to death within 2-4 years, but there exists no definite treatment so far. Based on phase I/II clinical trial(NCT01363401), twice intrathecal autologous bone marrow-derived mesenchymal stem cells (Lenzumestrocel) injections showed significant therapeutic benefit lasting at least six months with safety in patients with ALS. Additionally, the switch from pro- to anti-inflammatory conditions, which was indicated from the inverse correlation between TGF-β1 and MCP-1 levels after Lenzumestrocel injections in the good responder, has been considered a plausible beneficial action mechanism. This study is designed to investigate the following. First, to reconfirm and evaluate the long-term efficacy of twice injections (single cycle) of Lenzumestrocel, group 1 will receive a single cycle injection with a 26-day interval. Second, to evaluate the long-term safety and efficacy of Lenzumestrocel repeated injections, group 2 will receive a single cycle injection a 26-day apart followed by three times injections every three-month interval. Group 3 will receive comparator injections.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
BIOLOGICAL	Lenzumestrocel	Single cycle administration group : injections twice in a 26-day interval Multiple administration group : injections twice in a 26-day interval followed by repeated three times injections every three months
DRUG	Riluzole	concomitant administration of Riluzole to all groups, except subjects to whom Riluzole administration is deemed impossible owing to adverse events as determined by medical experts
DRUG	Placebo Comparator	Single cycle administration group: Placebo comparator is injected three times every three months after injection of Lenzumestrocel twice in a 26-day interval. Control group: Placebo comparator is injected twice in a 26-day interval followed by repeated three times injcections every three months

Timeline

• Start date: 2021-03-23
• Primary completion: 2024-12-31
• Completion: 2026-05-03
• First posted: 2021-02-09
• Last updated: 2025-01-10

Locations

5 sites across 1 country: South Korea

Regulatory

• FDA-regulated drug study