Stem Cells for Research in Motor Neuron Disease

Study Purpose:

The purpose of this study is to obtain skin biopsies from subjects with MND or pre-symptomatic familial ALS (fALS) and reprogram the fibroblasts from these samples into induced pluripotent stem (iPS) cells. The properties of the derived neurons will be compared to gain insights into potential causes of MND.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS), Hereditary Spastic Paraplegia (HSP), Healthy Volunteer, Healthy Volunteer with a Family History of ALS

Study Type:

Observational Study

Study Category:

Biomarkers/Imaging

Study Status:

Enrolling

Phase:

Not Applicable

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

James D. Berry, MD, MPH (Massachusetts General Hospital)

Clinicaltrials.gov ID (11 digit #):

Neals Affiliated?

Yes

Coordinating Center Contact Information

Massachusetts General Hospital
Connor Freeh / .(JavaScript must be enabled to view this email address) / 617-726-4282
.(JavaScript must be enabled to view this email address) 165 Cambridge Street
Suite 600
Boston, Massachusetts 02114 United States

Full Study Summary:

Neurodegenerative diseases are diseases in which nerve cells of the brain and spinal cord die. There is a need to understand the cause of these diseases and to develop treatments. While we do have animal models of these diseases, they are very limited. We are in need of models that more closely match what happens in humans. Luckily, recent advancements in stem cell technology have allowed us to create a person™s own nerve cells by taking a skin biopsy or blood sample. This study wants to use this new technology to make models for neurodegenerative diseases. We hope this will give us a better understanding of the diseases, enable us to use the cells for drug screening, and in the future, develop treatments.

Study Sponsor:

Department of Neurology Funds

Participant Duration:

This is a one time study visit at MGH that lasts about 1 to 3 hours.

Estimated Enrollment:

200

Estimated Study Start Date:

02/28/2011

Estimated Study Completion Date:

11/02/2016

Posting Last Modified Date:

04/04/2018

Date Study Added to alsconsortium.org:

11/02/2016
  • More Information

    Parking will be covered for study participants. No other compensation is available.

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    If you are taking anti-coagulant or anti-platelet medications (blood thinners), with the exception of aspirin, or have a history of keloid formation, you will not be able to take part in this study. To minimize the likelihood of an allergic reaction to the anesthetic, you cannot take part in this study if you are allergic to Lidocaine. Subjects undergoing the voluntary lumbar puncture (LP) must be medically able to undergo a LP as determined by a Study Investigator (ie: no bleeding disorder, allergy to local anesthetics, a skin infection at or near the LP site, or evidence of high intracranial pressure).

  • Site Contact Information

    Massachusetts General Hospital
    Connor Freeh / .(JavaScript must be enabled to view this email address) / 617-726-4282
    165 Cambridge Street
    Boston, Massachusetts 02114
    United States