A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis

Study Purpose:

The purpose of this study is to evaluate the safety of intramuscular (IM) administration of Engensis in Participants with Amyotrophic Lateral Sclerosis (ALS) as compared to Placebo. Safety will be assessed by incidences of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), injection site reactions (ISRs) and other adverse events of special interest (AESIs), and the clinically significant laboratory values after injections of Engensis compared to Placebo. Exploratory endpoints include assessment of muscle function using the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) and ALSFRS-R subscores for Fine and Gross Motor Function; muscle strength by quantitative testing using handheld dynamometry (HHD) and the Accurate Test of Limb Isometric Strength (ATLIS) where available; quality of life using the ALS Assessment Questionnaire (ALSAQ-40); patient global impression of change (PGIC), clinical global impression of change (CGIC), and clinical global impression of severity (CGIS); and evaluation of lung function using Slow Vital Capacity (SVC). Muscle biopsies will be performed during the study for future biomarker analyses.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Gene Therapy

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT04632225

Neals Affiliated?

No

Coordinating Center Contact Information

Helixmith Co., Ltd.
Jennifer Guzman, PhD / .(JavaScript must be enabled to view this email address) / 724-396-7345
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Helixmith Co., Ltd.

Participant Duration:

Estimated Enrollment:

18

Estimated Study Start Date:

01/27/2021

Estimated Study Completion Date:

12/31/2022

Posting Last Modified Date:

03/17/2021

Date Study Added to alsconsortium.org:

12/15/2020
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    20

    Maximum Age:

    80

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    1. Clinically definite or probably Amyotrophic Lateral Sclerosis (ALS) or laboratory-supported probable ALS as defined in the revised El Escorial/Airlie House diagnostic criteria
    2. The site of onset of ALS symptoms is a limb and experiencing symptoms of lower motor dysfunction (e.g., weakness, atrophy, cramps, poor circulation, etc.) with upper motor neuron symptoms (e.g., weakness, brisk reflexes, spasticity)
    3. Diagnosis of ALS within the past 3 years, as reported by the Participant at Screening
    4. Slow Vital Capacity (SVC) ≥60% of predicted value at Screening
    5. Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) ≥30 at Screening
    6. Not taking riluzole, or on a stable dose (defined as no noted toxicities) for at least 30 days prior to Screening and throughout the study
    7. Not taking edaravone or on a maintenance cycle for at least 30 days prior to Screening and throughout the study
    8. For females of childbearing potential, a negative urine pregnancy test at Screening and on Day 0
    9. Male Participants and their female partners must agree to use double-barrier contraception during the study or provide proof of postmenopausal state (minimum 1 year) or surgical sterility
    10. Male Participants must not donate sperm during the study
    11. Female Participants must be nonpregnant, nonlactating, and either postmenopausal for at least 1 year, or surgically sterile for at least 3 months, or agree to use double-barrier contraception from 28 days prior to randomization (Day 0) and/or their last confirmed menstrual period prior to study randomization (whichever is longer) until the end of the study
    12. Capable of complying and willing to comply with the requirements and restrictions in the informed consent form and this protocol
    13. Willing to forgo new ALS treatments, approved or experimental, for at least 6 months following randomization

    Exclusion Criteria:

    1. Progressive or degenerative neurological disorder such as Alzheimer's disease, Parkinson's disease, vascular dementia, multiple sclerosis, or other neurological disorders felt by the Investigator to preclude participation
    2. Symptomatic peripheral artery disease (PAD) or PAD requiring revascularization and/or that may interfere with the conduct of the study
    3. Requires tracheotomy ventilation or noninvasive ventilation related to bulbar function
    4. Evidence by physical examination, history, or laboratory evaluation of significant concomitant disease with a life expectancy of <6 months at Screening
    5. INR values >2.0
    6. Platelet count <100,000/mL
    7. Inflammatory disorder of the blood vessels (inflammatory angiopathy or vasculitis, such as Buerger's disease)
    8. Active infection (chronic infection or severe active infection that may compromise the Participant's wellbeing or participation in the study in the Investigator's judgment)
    9. Chronic inflammatory disease (e.g., Crohn's disease, rheumatoid arthritis)
    10. Positive human immunodeficiency virus (HIV) or human T-cell lymphotrophic virus (HTLV) test at Screening
    11. Active hepatitis B as determined by hepatitis B core antibody (IgG and IgM; HBcAb), antibody to hepatitis B surface antigen (HBsAb), and hepatitis B surface antigen (HBsAg) at Screening
    12. Active hepatitis C, as determined by hepatitis C antibodies (Anti-HCV) and a hepatitis C qualitative or quantitative viral RNA test
    13. Immunosuppression due to underlying disease (e.g., rheumatoid arthritis, systemic lupus erythematosus) or to currently receiving immunosuppressive drugs, (e.g., chemotherapy, corticosteroids) or to radiation therapy
    14. Stroke or myocardial infarction within 3 months prior to Screening
    15. Active deep vein thrombosis
    16. Recent history (<5 years) or presence of cancer except basal cell carcinoma or squamous cell carcinoma of the skin that was excised and has shown no evidence of recurrence for at least 1 year
    17. Participants requiring chronic oral or injectable steroids and unwilling to refrain form taking these drugs for the duration of the study
    18. Major psychiatric disorder diagnosed in the past 6 months that have been stabilized or in the Investigator's opinion would not allow the patient to participate in the scheduled procedures
    19. Use of an investigational drug or treatment in past 30 days or previous participation in a clinical study with Engensis
    20. Stem cell treatment in the 6 months prior to Screening
    21. Malignancy within five years prior to first dose of study medication unless treated definitively without evidence of recurrence

  • Site Contact Information

    Austin Neuromuscular Center
    Michael Chiodo / .(JavaScript must be enabled to view this email address) / 512-920-0140 ext 210
    Austin, Texas 78756
    United States