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Intrathecal Administration of Autologous Mesenchymal Stem Cell-derived Neural Progenitors (MSC-NP) in Progressive Multiple Sclerosis

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ClinicalTrials.gov Identifier: NCT03355365
Recruitment Status : Recruiting
First Posted : November 28, 2017
Last Update Posted : November 5, 2018
Sponsor:
Information provided by (Responsible Party):
Tisch Multiple Sclerosis Research Center of New York

Brief Summary:
This is a Phase II, Double-Blind, Placebo-Controlled, Randomized, Cross-over Study designed to determine efficacy of multiple intrathecal administrations of autologous mesenchymal stem cell-derived neural progenitor cells (MSC-NP) compared to placebo in patients with progressive multiple sclerosis. Efficacy will be measured through assessment of disability outcomes. Study participants will receive six intrathecal injections of culture-expanded autologous MSC-NPs at two month intervals in one year and six lumbar punctures as placebo treatment in a second year.

Condition or disease Intervention/treatment Phase
Multiple Sclerosis Biological: intrathecal MSC-NP injection Other: lumbar puncture placebo control Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Care Provider)
Primary Purpose: Treatment
Official Title: Autologous, Bone Marrow-Derived Mesenchymal Stem Cell-Derived Neural Progenitor Cells (MSC-NP), Expanded Ex Vivo; Administered Intrathecally
Actual Study Start Date : September 21, 2018
Estimated Primary Completion Date : November 2020
Estimated Study Completion Date : November 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: lumbar puncture with MSC-NP injection
Intrathecal MSC-NP injection
Biological: intrathecal MSC-NP injection
repeated intrathecal injection of autologous MSC-NP cells

Placebo Comparator: lumbar puncture with no injection
Lumbar puncture placebo control
Other: lumbar puncture placebo control
repeated lumbar puncture without injection




Primary Outcome Measures :
  1. Efficacy: Expanded Disability Status Scale (EDSS) Plus composite score [ Time Frame: 27 months from first treatment or placebo ]
    Changes in disability assessed based composite score of EDSS, timed 25 foot walk (T25FW), and nine hole peg test (9HPT) (EDSS-Plus). Improvement will be defined by at least one of the following three measures: ≥0.5 improvement in EDSS, ≥20% improvement in T25FW, and ≥20% improvement in 9HPT in either dominant or non-dominant upper limb. Scores will be compared individually between baseline and post-treatment. Assessments will be made at baseline and 3 months after the 6th treatment/placebo in each crossover group (month 0, 13, and 27).


Secondary Outcome Measures :
  1. Safety: incidence and severity of adverse events [ Time Frame: 27 months from first treatment or placebo ]
    Incidence and severity of adverse events in MSC-NP treatment group compared to placebo group

  2. Efficacy: bladder function [ Time Frame: 27 months from first treatment or placebo ]
    Degree of bladder dysfunction assessed by urodynamics assessment at baseline and 3 months after the 6th treatment placebo in each crossover group (month 0, 13, and 27).

  3. Efficacy: Multiple sclerosis functional composite (MSFC) [ Time Frame: 27 months from first treatment or placebo ]
    Changes in disability assessed by MSFC assessed at baseline and 3 months after the 6th treatment/placebo in each crossover group (month 0, 13, and 27).



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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of MS as defined by the McDonald criteria
  • Diagnosis of primary progressive or secondary progressive MS
  • Between the ages of 18-65 years
  • Significant disability shown by an Expanded Disability Status Score (EDSS) of greater than or equal to 3.0, and less than or equal to 6.5, that was not acquired within the last 12 months.
  • Stable disease state as evidenced by a lack of gadolinium-enhancing lesions on an MRI and by a stable MRI disease burden (number of T2 lesions and size of lesions) in the last six months and no significant change in EDSS (1 point or more) in the last 12 months
  • Must agree to undergo four MRIs: at the time of enrollment, after year 1, after year 2, and after year 3
  • Patients either within the geographical area or who are able to arrange reliable travel during the study period

Exclusion Criteria:

  • EDSS greater than 6.5
  • Duration of Disease >15 years at time of screening
  • Change of disease modifying agent < 6 months prior to beginning treatment. Additionally, no changes in disease modifying agent will be made during the course of the study.
  • Change in MS symptom management treatment < 6 months prior to beginning treatment. Additionally, no changes in MS symptom management treatments will be made during the course of the study, unless there has been clinical improvement, in which case, a patient may discontinue a medication.
  • Start of any new orthotic device or durable medical equipment < 6 months prior to beginning treatment or during the course of the study (patients may discontinue use of these devices during the course of the study if they show clinical improvement).
  • All patients who have ever been on Lemtrada (alemtuzumab)
  • All patients who have had any prior stem cell treatments, including HSCT
  • Pregnant or nursing mothers, or any woman intending to become pregnant in the next three years
  • All patients will have screening blood tests done. Only patients whose values are in the normal range as determined by the laboratory norms based on age and sex will be allowed to participate. Exceptions may be made for borderline normal laboratory values manifesting no clinical symptoms at the discretion of the Principal Investigator.
  • Use of systemic chemotherapeutic or anti-mitotic medications within three months of study start date due to the possibility of interference with bone marrow procedure
  • Any patients with a history of or with active malignancy
  • Use of steroids within three months of the study start date, as this would suggest an active disease state
  • History of cirrhosis due to increased risk of central nervous system (CNS) infection
  • Significantly uncontrolled hypertension because of increased risk for stroke or CNS hemorrhage.
  • Patients with active thyroid disease resulting in hyperthyroidism or hypothyroidism (Only well controlled patients with labs in the normal range will be included) because of hormone influence on cell growth
  • History of central nervous system infection or immunodeficiency syndromes due to increased risk of CNS infection
  • Preexisting blood disease (such as bone marrow hypoplasia, leukopenia, thrombocytopenia, or significant anemia) due to invasive nature of bone-marrow aspiration
  • Previous or current history of a coagulation disorder
  • Any metal in the body, which is contraindicated for MRI studies
  • Patients with alcohol or other substance abuse problems that may affect stem cell growth; habitual drug (including marijuana and nicotine) abusers, will be excluded from the study
  • Other major disease that, in the opinion of the Principal Investigator, would preclude participation in the study
  • Patients with Hepatitis B (HBV), Hepatitis C (HCV), syphilis, HIV-1, or HIV-2.
  • Any evidence of significant cognitive dysfunction based on a screening history and physical examination because it would preclude giving a truly informed consent
  • Patients who are enrolled in another clinical trial for MS treatment or who have received any study drug/biologics within the last 6 months. Additionally, while in the trial, patients may not enroll in any other clinical trial for MS or any other condition.
  • Patients who are anticipated to have difficultly accessing the intrathecal space related to scoliosis, obesity, or any other relevant factors determined by the PI.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03355365


Contacts
Contact: Saud A Sadiq, MD, FAAN 212 265 8070 blevine@tischms.org
Contact: Beth Levine, RN, BA, BSN 212 265 8070 blevine@tischms.org

Locations
United States, New York
Beth Levine Recruiting
New York, New York, United States, 10075
Contact: Beth Levine    212-265-8070    blevine@tischms.org   
Principal Investigator: Saud A. Sadiq, MD         
Sponsors and Collaborators
Tisch Multiple Sclerosis Research Center of New York
Investigators
Principal Investigator: Saud A Sadiq, MD, FAAN Tisch MS Research Center of New York

Additional Information:
Publications:
Responsible Party: Tisch Multiple Sclerosis Research Center of New York
ClinicalTrials.gov Identifier: NCT03355365     History of Changes
Other Study ID Numbers: TISCHMS-MSCNP-002
First Posted: November 28, 2017    Key Record Dates
Last Update Posted: November 5, 2018
Last Verified: November 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Tisch Multiple Sclerosis Research Center of New York:
Mesenchymal Stem Cells
Neural Progenitors
Autologous
Bone Marrow
Multiple Sclerosis

Additional relevant MeSH terms:
Sclerosis
Multiple Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases