Clinical trial NCT05176093: A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Helixmith Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2022-01-04
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Detailed description: VMALS-002-2b was a safety extension study following VMALS-002-2, which was a Phase 2a, double-blind, randomized, placebo-controlled, multicenter study designed to assess the safety of intramuscular administration of Engensis in subjects with Amyotrophic Lateral Sclerosis.Following completion of the Day 180 visit in VMALS-002-2 and signing of written informed consent, subjects were enrolled in this approximately 180-day extension study, with subjects completing combined participation with a Day 365 visit. Assessments to be conducted during this extension study were as follows: vital signs, weight and height, complete physical examination, 12-lead electrocardiogram, a record of all concomitant medications and procedures, clinical laboratory assessments, Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, handheld dynamometry, slow vital capacity, Amyotrophic Lateral Sclerosis Assessment Questionnaire, 40 questions, Patient Global Impression of Change, and Clinical Global Impression of Change. Assessments of treatment-emergent adverse events, treatment-emergent serious adverse events, adverse events of special interest, and clinically significant laboratory values continued from the Day 180 visit of Study VMALS-002-2 through the VMALS-002-2b extension study to Day 365.

Eligibility criteria: 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

Primary outcomes:
measure: Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term
timeFrame: Day 0 to Day 365
Secondary outcomes:
Other outcomes:
measure: To Evaluate Changes in Muscle Function Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants
timeFrame: Day 0 and Day 365
measure: To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants
timeFrame: Day 0 and Day 365
measure: To Determine Whether Intramuscular Administration of Engensis Has Effects on Respiratory Capacity in Amyotrophic Lateral Sclerosis Participants
timeFrame: Day 0, Day 240, Day 300 and Day 365
measure: To Determine if Intramuscular Engensis Has Effects on Respiratory Function in Amyotrophic Lateral Sclerosis Participants - Tracheostomy
timeFrame: Day 0 to Day 365
measure: To Determine if Intramuscular Administration of Engensis Has Positive Effects on Survival in Amyotrophic Lateral Sclerosis Participants - All-Cause Mortality
timeFrame: Day 0 to Day 365
measure: To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo
timeFrame: Day 0, Day 240 and Day 365
measure: To Evaluate Patient Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo
timeFrame: Day 240 and Day 365
measure: To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo
timeFrame: Day 240 and Day 365
Locations:
city: Austin
country: United States
facility: Austin Neuromuscular Center
state: Texas
city: Seoul
country: Korea, Republic of
facility: Hanyang University Medical Center
Study officials:
References: