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A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis (REViVALS-1B)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05176093
Recruitment Status : Active, not recruiting
First Posted : January 4, 2022
Last Update Posted : November 7, 2022
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Biological: Engensis Other: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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)
Actual Study Start Date : November 14, 2021
Estimated Primary Completion Date : January 2023
Estimated Study Completion Date : February 2023


Arm Intervention/treatment
Active Comparator: 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
Biological: Engensis
Lyophilized biologic to be reconstituted containing Engensis

Placebo Comparator: 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
Other: Placebo
Injectable Liquid




Primary Outcome Measures :
  1. • To evaluate the long-term safety of intramuscular (IM) injections of Engensis in Participants with Amyotrophic Lateral Sclerosis (ALS) [ Time Frame: Day 0 to Day 365 ]
    • Incidence of adverse events (AE) for Engensis compared to Placebo


Other Outcome Measures:
  1. • To evaluate changes in muscle function following Engensis injections in ALS Participants [ Time Frame: Day 0 to Day 365 ]
    • 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

  2. • To evaluate muscle strength changes following Engensis injections in ALS Participants [ Time Frame: Day 0 to Day 365 ]
    • 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

  3. • To determine whether IM administration of Engensis has effects on respiratory capacity in ALS Participants [ Time Frame: Day 0 to Day 365 ]
    • Change from Baseline (Study VMALS 002-2 Day 0) in Slow Vital Capacity (SVC) at Day 365 for Engensis compared to Placebo

  4. • To determine whether IM administration of Engensis has effects on respiratory function in ALS Participants [ Time Frame: Day 0 to Day 365 ]
    • Time to tracheostomy for Engensis

  5. • To determine whether IM administration of Engensis has positive effects on survival in ALS Participants [ Time Frame: Day 0 to Day 365 ]
    • Time to all-cause mortality compared to Placebo

  6. • To evaluate Quality of Life improvement following Engensis injections in ALS Participants compared to Placebo [ Time Frame: Day 0 to Day 365 ]
    • 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

  7. • To evaluate Patient Reported Outcome improvement following Engensis injections in ALS Participants compared to Placebo [ Time Frame: Day 365 ]
    • Patient Global Impression of Change (PGIC) at Day 365 for Engensis compared to Placebo

  8. • To evaluate Clinician Reported Outcome improvement following Engensis injections in ALS Participants compared to Placebo [ Time Frame: Day 365 ]
    • Clinical Global Impression of Change (CGIC) at Day 365 for Engensis compared to Placebo



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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05176093


Locations
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United States, Texas
Austin Neuromuscular Center
Austin, Texas, United States, 78759
Korea, Republic of
Hanyang University Medical Center
Seoul, Korea, Republic of, 04763
Sponsors and Collaborators
Helixmith Co., Ltd.
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Responsible Party: Helixmith Co., Ltd.
ClinicalTrials.gov Identifier: NCT05176093    
Other Study ID Numbers: VMALS-002-2b
First Posted: January 4, 2022    Key Record Dates
Last Update Posted: November 7, 2022
Last Verified: November 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases