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

Amyotrophic Lateral Sclerosis Clinical Trial

— REViVALS-1B  

Official title:

A 6-Month Extension Study Following Protocol VMALS-002-2 (A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05176093
• Other study ID #: VMALS-002-2b
• Status: Completed
• Phase: Phase 2
• Start date: November 14, 2021
• Est. completion date: December 29, 2022

Study information

• Verified date: January 2023
• Source: Helixmith Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: December 29, 2022
• Est. primary completion date: December 29, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Biological:
• Engensis
Lyophilized biologic to be reconstituted containing Engensis

Other:
• Placebo
Injectable Liquid

Locations

Country	Name	City	State
Korea, Republic of	Hanyang University Medical Center	Seoul
United States	Austin Neuromuscular Center	Austin	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Helixmith Co., Ltd.

Countries where clinical trial is conducted

United States,  Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	• To evaluate changes in muscle function following Engensis injections in ALS Participants	• Changes from Baseline (Study VMALS 002-2 Day 0) in Revised Amyotrophic Lateral Sclerosis Function Rating (ALSFRS-R) scores at Day 365 for Engensis compared to Placebo	Day 0 to Day 365
Other	• To evaluate muscle strength changes following Engensis injections in ALS Participants	• Change from Baseline (Study VMALS 002-2 Day 0) in muscle strength assessed bilaterally by Handheld Dynamometry (HHD) in muscles in the upper and lower extremities at Day 365 for Engensis compared to Placebo	Day 0 to Day 365
Other	• To determine whether IM administration of Engensis has effects on respiratory capacity in ALS Participants	• Change from Baseline (Study VMALS 002-2 Day 0) in Slow Vital Capacity (SVC) at Day 365 for Engensis compared to Placebo	Day 0 to Day 365
Other	• To determine whether IM administration of Engensis has effects on respiratory function in ALS Participants	• Time to tracheostomy for Engensis	Day 0 to Day 365
Other	• To determine whether IM administration of Engensis has positive effects on survival in ALS Participants	• Time to all-cause mortality compared to Placebo	Day 0 to Day 365
Other	• To evaluate Quality of Life improvement following Engensis injections in ALS Participants compared to Placebo	• Change from Baseline (Day 0) in Quality of Life (QoL) using the Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ; with 40 items, ALSAQ 40) on Day 365 for Engensis compared to Placebo	Day 0 to Day 365
Other	• To evaluate Patient Reported Outcome improvement following Engensis injections in ALS Participants compared to Placebo	• Patient Global Impression of Change (PGIC) at Day 365 for Engensis compared to Placebo	Day 365
Other	• To evaluate Clinician Reported Outcome improvement following Engensis injections in ALS Participants compared to Placebo	• Clinical Global Impression of Change (CGIC) at Day 365 for Engensis compared to Placebo	Day 365
Primary	• To evaluate the long-term safety of intramuscular (IM) injections of Engensis in Participants with Amyotrophic Lateral Sclerosis (ALS)	• Incidence of adverse events (AE) for Engensis compared to Placebo	Day 0 to Day 365