Healey Amyotrophic Lateral Sclerosis Platform Trial: A Randomized, Double-Blind, Placebo-Controlled Platform Study: IC14 Arm

Study Purpose:

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS

Disease:

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

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not yet enrolling

Phase:

Phase II

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

Merit E. Cudkowicz, MD Massachusetts General Hospital

Clinicaltrials.gov ID (11 digit #):

NCT04390386

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital
Lindsay Pothier / .(JavaScript must be enabled to view this email address) / 617-643-5582
.(JavaScript must be enabled to view this email address) Boston, Massachusetts 02115 United States

Full Study Summary:

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial.

In this trial, multiple investigational products for ALS will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen.

The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting.

Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized in a 3:1 ratio to either study drug or placebo.

The IC14 regimen is not yet active in this trial.

New regimens will be continuously added as new investigational products become available. The HEALEY ALS Platform Trial will enroll additional participants as each new regimen is available.

Study Sponsor:

Implicit Bioscience

Participant Duration:

Estimated Enrollment:

480

Estimated Study Start Date:

12/01/2019

Estimated Study Completion Date:

12/31/2021

Posting Last Modified Date:

05/28/2020

Date Study Added to alsconsortium.org:

05/28/2020
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    70

    Min Vital Capacity (% predicted normal):

    50

    Time since Symptom Onset:

    <36 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    • Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
    • Age 18 years or older.
    • Capable of providing informed consent and complying with study procedures, in the SI's opinion.
    • Time since onset of weakness due to ALS ≤ 36 months at the time of the Master Protocol Screening Visit.
    • SVC ≥ 50% of predicted capacity for age, height, and sex at the time of the Master Protocol Screening Visit.
    • Participants must either not take riluzole or be on a stable dose of riluzole for ≥ 30 days prior to the Master Protocol Screening Visit. Riluzole-naïve participants are permitted in the study.
    • Participants must either not take edaravone or have completed at least one cycle of edaravone prior to the Master Protocol Screening Visit. Edaravone-naïve participants are permitted in the study.
    • Participants must have the ability to swallow pills and liquids at the time of the Master Protocol Screening Visit and, in the SI's opinion, have the ability to swallow for the duration of the study.
    • Geographically accessible to the site.

    Exclusion Criteria:

    • Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to SI's judgment (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, or clinically significant laboratory abnormality or EKG changes).
    • Lab abnormalities include, but are not limited to: Hemoglobin < 10 g/dL, White Blood Cells < 3.0 x 103/mm3, Neutrophils, Absolute ≤ 1000/mm3, Eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter), low platelet counts (< 150 x 109 per liter), alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal (ULN), eGFR < 30 mL/min/1.73m2, thyroid-stimulating hormone (TSH) levels >10 mIU/L or <0.01 mIU/L.
    • Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.
    • Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.
    • Use of investigational treatments for ALS (off-label use or active participation in a clinical trial) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Master Protocol Screening Visit.
    • Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).
    • If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception for the duration of the trial and for 3 months, or longer as specified in each RSA, after discontinuing study treatment.
    • If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 3 months, or longer as specified in each RSA, after discontinuing study treatment.
    • Anything that would place the participant at increased risk or preclude the participant's full compliance with or completion of the study, in the SI's opinion.
    • If a participant is being re-screened, the disqualifying condition has not been resolved, or the mandatory wash-out duration has not occurred.
    • For those participating in the optional CSF collection, contraindication to undergoing a lumbar puncture (LP) in the SI's opinion. Participants undergoing the LP must not be currently taking anticoagulation medications such as warfarin that would be a contraindication to LP; aspirin and non-steroidal anti-inflammatories are allowed.

  • Site Contact Information

    Massachusetts General Hospital
    Boston, Massachusetts 02115
    United States