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

NCT05176093 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: VMALS-002-2b
• Study Type: interventional
• Target: 8
• Locations: 2 countries
• Sites: 2

Brief Summary

The purpose of this study is to evaluate the long-term safety of intramuscular administration of Engensis in Participants with Amyotrophic Lateral Sclerosis 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, treatment emergent serious adverse events, adverse events of special interest, and the clinically significant laboratory values.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term

  To evaluate the long-term safety of intramuscular injections of Engensis in Participants with Amyotrophic Lateral Sclerosis in more than 2 Participants by System Organ Class and Preferred Term (Safety Analysis Population)

  Day 0 to Day 365

Other Outcomes (8)

• To Evaluate Changes in Muscle Function Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants

  Day 0 and Day 365

• To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants

  Day 0 and Day 365

• To Determine Whether Intramuscular Administration of Engensis Has Effects on Respiratory Capacity in Amyotrophic Lateral Sclerosis Participants

  Day 0, Day 240, Day 300 and Day 365

Study Arms (2)

Engensis

ACTIVE COMPARATOR

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

Biological: Engensis

Placebo

PLACEBO COMPARATOR

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

Other: Placebo

Interventions

Engensis BIOLOGICAL

Lyophilized biologic to be reconstituted containing Engensis

Engensis

Placebo OTHER

Injectable Liquid

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• \- None

Sponsors & Collaborators

• Helixmith Co., Ltd.: lead

Study Sites (2)

Austin Neuromuscular Center

Austin, Texas, 78759, United States

Location

Hanyang University Medical Center

Seoul, 04763, South Korea

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Jinsub Lee, PhD.
• Organization: Helixmith Co., Ltd.

jinsub.lee@helixmith.com

+82-10-8256-0439

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 5, 2021
• First Posted: January 4, 2022
• Study Start: November 14, 2021
• Primary Completion: December 29, 2022
• Study Completion: August 23, 2024
• Last Updated: October 6, 2025
• Results First Posted: May 29, 2025

Record last verified: 2025-08

Locations

US (1); KR (1)