A Phase 1/2a Open-Label Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early efficacy of Q-Cells® transplantation in subjects with ALS. For each of the two dose levels, transplantation of Q-Cells® into the lumbar and cervical spinal cord will be evaluated sequentially. Subjects will be blinded to side of treatment in cohorts receiving unilateral transplants. The study will be conducted at sites with extensive clinical experience with the care of patients with ALS.

Disease:

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

Study Type:

Interventional Trial

Study Category:

Stem Cell

Study Status:

Active, not yet recruiting

Phase:

Phase II

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

N/A

Clinicaltrials.gov ID (11 digit #):

NCT02478450

Neals Affiliated?

No

Coordinating Center Contact Information


.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early efficacy of Q-Cells® transplantation in subjects with ALS. For each of the two dose levels, transplantation of Q-Cells® into the lumbar and cervical spinal cord will be evaluated sequentially. Subjects will be blinded to side of treatment in cohorts receiving unilateral transplants. The study will be conducted at sites with extensive clinical experience with the care of patients with ALS.

The study is planned to enroll up to 12 subjects over 18 months. Each subject receives a single time point administration of Q-Cells®: with 5 (unilateral administration) or 10 (bilateral administration) transplantation foci targeted to anterior horn in either the lumbar or cervical spinal cord.

The study consists of Screening, Pre-operative/Treatment, and Post-treatment study periods. The study data will be assessed for safety and efficacy after the last subject has completed the 9-month study visit. Following the 9-month study period, subjects who consent will continue to be followed for safety and efficacy long-term in a separate protocol.

Study Sponsor:

Q Therapeutics, Inc.

Participant Duration:

Estimated Enrollment:

30

Estimated Study Start Date:

05/01/2017

Estimated Study Completion Date:

12/30/2020

Posting Last Modified Date:

03/01/2017

Date Study Added to alsconsortium.org:

08/18/2015
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    -Ability to understand the purpose and risks of the study and provided signed and dated informed consent and authorization to collect and use protected health information (PHI) in accordance with national and local subject privacy regulations.
    -Live within reasonable driving distance of study center (<3 hours)
    -Have a caregiver willing/able to assist in the transportation and care required by study participation
    -Subject is 18 - 80 years of age (inclusive) on day of Screening Visit.
    -Subject is diagnosed with sporadic or familial ALS within the past 48 months.
    -Subject meets the laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria.
    -Subject has an upright FVC ≥65% of predicted value for age, height, and gender at screening.
    -Subject has not taken riluzole for at least 30 days prior to Visit 1, or has been on a stable dose of riluzole for at least 30 days prior to Visit 1. (Riluzole-naïve subjects are permitted in the study.)
    -Subject is medically able to undergo the study procedures and physically able to adhere to the visit schedule at the time of study entry.
    -For women of child bearing capacity, negative pregnancy test within 2 weeks of surgery.
    -Males and females should agree to practice effective birth control during study participation.

    Exclusion Criteria:
    -Subject in whom causes of neuromuscular weakness other than ALS have not been practically excluded.
    -Subject with significant cognitive impairment, clinical dementia, or major psychiatric illness including psychosis, bipolar disease, major depression, as determined by the DSM-V.
    -Subject with a diagnosis of other neurodegenerative diseases (e.g., Parkinson's disease, Alzheimer's disease). Any subject suffering with medical conditions that impair nerve or muscle function (e.g., notable peripheral neuropathy, metabolic muscle disease).
    -Subject with a clinically significant history of unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active malignancy or infectious disease or other medically significant illness.
    -History of spine surgery or anatomic variation incompatible with route of administration (as determined by neurosurgeon).
    -Severe cervical or lumbar stenosis, cord compression, or cervical or lumbar myelopathy.
    -Abnormal flow voids on the surface of the spinal cord suggestive of arteriovenous malformation (AVM).
    -Any evidence of CNS malignancy or CNS lesions as defined by imaging studies of the CNS (MRI of brain and spinal cord).
    -Uncontrolled hypertension (Systolic BP>160mmHg and/or Diastolic BP >100mmHg) or history of thrombotic events or poorly controlled medical conditions that, in the opinion of the site investigator and/or surgeon, increase risk of surgery.
    -Subjects who cannot undergo MRI examination because of the presence of a pacemaker, an implanted defibrillator or certain other implanted electronic or metallic devices, or who have been or might have been exposed to metal fragments, or any reason the subject cannot undergo an MRI routinely for the duration of the trial.
    -Subject with clinically significant abnormal clinical laboratory values, as determined by the Investigator at the screening visit (Visit 1).
    -Subjects who are immune compromised or who have a condition contraindicated to treatment with immunosuppression agents (e.g, tuberculosis, latent infection).
    -Subject with aspartate aminotransferase (AST) or alanine aminotransferase (ALT) value >3.0 times the upper limit of normal at the screening visit (Visit 1).
    -Subject with a history of alcohol or drug abuse or dependence within 1 year of screening visit (Visit 1), per DSM-V criteria
    -Subject unlikely to comply with study requirements, as determined by Investigator.
    -Subject who has been exposed to any other experimental agent (off-label use or investigational) within 30 days of screening visit (Visit 1). Biologic agents may need additional time for washout and should be discussed with the sponsor.
    -Subject with pre-existing anti-human leukocyte antigen (HLA) class I or class II antibodies directed against the Q-Cells®, as determined by panel reactive antibody (PRA) assay.
    -Allergy to treatment or any of its constituents (e.g., chicken eggs), or allergy to any of the co-administered immunosuppressants or any of their excipients.
    -Any contraindication to Tacrolimus, mycophenolate mofetil, or prednisone as determined by investigator.

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