A Phase II, Open-label, Dose Escalation and Safety Study of Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis

Study Purpose:

The study is to determine the feasibility, safety, toxicity, and maximum tolerated (safe) dose of human spinal derived neural stem cell transplantation for the treatment of Amyotrophic Lateral Sclerosis (ALS).

Disease:

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

Study Type:

Interventional Trial

Study Category:

Stem Cell

Study Status:

Closed

Phase:

Phase II

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

Neuralstem Inc.

Clinicaltrials.gov ID (11 digit #):

NCT01730716

Neals Affiliated?

Yes

Coordinating Center Contact Information

Emory University
.(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address) Atlanta, Georgia 30322 United States

Full Study Summary:

The study is to determine the feasibility, safety, toxicity, and maximum tolerated (safe) dose of human spinal derived neural stem cell transplantation for the treatment of Amyotrophic Lateral Sclerosis (ALS).

Study Sponsor:

Neuralstem Inc.

Participant Duration:

Patients will be followed postoperatively for 24 months.

Estimated Enrollment:

18

Estimated Study Start Date:

04/30/2013

Estimated Study Completion Date:

11/30/2016

Posting Last Modified Date:

08/25/2017

Date Study Added to alsconsortium.org:

06/03/2013
  • More Information

    The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years. For more information, visit: https://clinicalt...CT01730716&rank=1

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    60

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


    Inclusion Criteria:
    1. Have the ability to understand the requirements of the study, provide written informed consent, understand and provide written authorization for the use and disclosure of Protected Health Information (PHI) [per Health Insurance Portability and Accountability Act (HIPAA) Privacy Ruling] and comply with the study procedures.
    2. Subjects with sporadic or familial ALS, meeting the definition of laboratory-supported probable, probable or definite ALS according to the World Federation of Neurology El Escorial Criteria (Appendix A). At the time of enrollment subjects should be within 24 months of symptom onset.
    3. Age 18 years or older.
    4. Females must have a negative serum pregnancy test and practice an acceptable method of contraception or be of non-childbearing potential (post-menopausal for at least 2 years or surgically sterile [hysterectomy, oophorectomy or surgical sterilization]).
    5. Geographic accessibility to the study center and the ability to travel to the clinic for study visits.
    6. Presence of a willing and able caregiver.
    7. Medically able to undergo lumbar and/or cervical laminectomy or laminoplasty as determined by the site Principal Investigator and neurosurgeon.
    8. Medically able to tolerate the immunosuppression regimen consisting of basiliximab, tacrolimus, mycophenolate mofetil, prednisone and methylprednisolone as determined by the site PI.
    9. Agrees to the visit schedule as outlined in the informed consent.
    10. Not taking riluzole (Rilutek®) or on a stable dose for ≥ 30 days.
    11. Vital capacity ≥ 60% of predicted normal for age, height and gender measured in the seated position and ≥50% in supine position during the 7 days prior to surgery.
    12. Ambulatory subjects with extremity weakness and/or spasticity due to ALS. Patients undergoing lumbar surgery must have demonstrable weakness or spasticity in one or both lower extremities. Patients undergoing cervical surgery must have demonstrable weakness or spasticity in one or both upper extremities, with at least antigravity strength. Subjects must have normal neck extensor and flexor strength.

    Exclusion Criteria:

    1. Etiology of paraplegia or weakness is due to causes other than ALS.
    2. A positive result on the Panel Reactive Antibody (PRA) test, with the presence of specific HLA antibodies matching the HLA DNA profile of the donor cells.
    3. Any known immunodeficiency syndrome.
    4. Receipt of any investigational drug, device or biologic within 30 days of surgery.
    5. Any concomitant medical disease or condition limiting the safety to participate:
    A. Coagulopathy
    B. Active uncontrolled infection
    C. Hypotension requiring vasopressor therapy
    D. Previous spinal surgery that the neurosurgeon deems to be an obstacle to the planned transplantation
    E. Skin breakdown over the site of surgery
    F. Malignancy (except for non-melanoma skin cancer)
    G. Spinal stenosis severe enough to preclude surgery (as determined by the neurosurgeon)
    H. Pre-existing kyphosis by preoperative MRI or X-ray
    I. Less than 5/5 grade in neck extension and strength test at the time of surgery.
    6. Creatinine >1.5, liver function tests (SGOT/SGPT, Bilirubin, Alk Phos) > 2x upper limit of normal, hematocrit/hemoglobin < 30/10, total WBC < 4000, uncontrolled hypertension (systolic > 180 or diastolic > 100) or uncontrolled diabetes (defined as hemoglobin A1C >8), evidence of GI bleeding by hemoccult test, tuberculosis (TB test: PPD), serologic evidence of current infection with a hepatitis virus or human immunodeficiency virus (HIV).
    7. Presence of any of the following conditions:
    A. Current drug abuse or alcoholism
    B. Unstable medical conditions
    C. Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening
    8. Any condition or ALS disease phenotype that the site PI feels may interfere with participation in the study or in the interpretation of study endpoints.
    9. Any condition that the neurosurgeon feels may pose complications for the surgery.
    10. Known hypersensitivity to basiliximab, tacrolimus, mycophenolate mofetil, prednisone or methylprednisolone.
    11. Inability to provide informed consent as determined by the site PI.
    12. Inadequate family or caregiver support as determined by the site PI.

  • Site Contact Information

    University of Michigan
    Ann Arbor, Michigan 48109
    United States

    Emory University
    Atlanta, Georgia 30322
    United States

    Massachusetts General Hospital
    Boston, Massachusetts 02114
    United States