A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis

Phase 2

Completed

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Biological: Engensis; Other: Placebo

• Registration Number: NCT05176093
• Lead Sponsor: Helixmith Co., Ltd.

Brief Summary

The purpose of this study is to evaluate the long-term safety of intramuscular (IM) administration of Engensis in Participants with Amyotrophic Lateral Sclerosis (ALS) who were previously randomized, received treatment, and completed the Day 180 Visit of Study VMALS-002-2. Safety will be assessed by incidences of treatment-emergent adverse events (TEAEs), treatment emergent serious adverse events (TESAEs), adverse events of special interest (AESIs), and the clinically significant laboratory values. See the table below for additional, exploratory endpoints.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-11-05
• Study Start: 2021-11-14
• Study First Submitted QC: 2021-12-15
• Study First Posted: 2022-01-04
• Primary Completion: 2022-12-29
• Study Completion: 2022-12-29
• Status Verified: 2023-01
• Last Update Submitted: 2023-01-31
• Last Update Posted: 2023-02-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 8

Inclusion Criteria

• Participants who complete the Day 180 Visit in VMALS-002-2 are eligible to enroll in this extension study, VMALS-002-2b.

Exclusion Criteria

• None

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Engensis	Engensis	Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo	Placebo	Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart

Primary Outcome Measures

Name	Time	Method
• To evaluate the long-term safety of intramuscular (IM) injections of Engensis in Participants with Amyotrophic Lateral Sclerosis (ALS)	Day 0 to Day 365	• Incidence of adverse events (AE) for Engensis compared to Placebo

Trial Locations

Locations (2)

Austin Neuromuscular Center; 🇺🇸 Austin, Texas, United States

Hanyang University Medical Center; 🇰🇷 Seoul, Korea, Republic of