Phase 1 Safety Study of Autologous Bone Marrow-derived Mesenchymal Stem Cell-derived Neural Progenitor Cells (MSC-NP), Expanded Ex Vivo, Administered Intrathecally in Patients With Multiple Sclerosis

Tisch Multiple Sclerosis Research Center of New York1 site in 1 country20 target enrollmentApril 2014

ConditionsMultiple Sclerosis

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Multiple Sclerosis
Sponsor: Tisch Multiple Sclerosis Research Center of New York
Enrollment: 20
Locations: 1
Primary Endpoint: Number of participants with adverse events .
Status: Completed
Last Updated: 8 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

The study is an open-label, phase I clinical trial designed to evaluate the safety and tolerability of intrathecal administration of autologous mesenchymal stem cell-derived neural progenitor cells (MSC-NP) in patients with progressive multiple sclerosis. Study participants will receive three intrathecal injections of culture-expanded autologous MSC-NPs at three month intervals.

Registry

clinicaltrials.gov

Start Date

April 2014

End Date

March 2017

Last Updated

8 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Tisch Multiple Sclerosis Research Center of New York

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Diagnosis of MS as defined by the McDonald criteria
•Diagnosis of primary progressive or secondary progressive MS
•Between the ages of 18-70 years
•Significant disability shown by an Expanded Disability Status Score (EDSS) of 3.0 or greater 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 MRIs at the time of enrollment, 2 months after the first treatment, and 27 months after the last treatment
•Live in northern New Jersey, southern New York, or southwestern Connecticut during the study period, or patients must be able to arrange reliable travel accommodations to be present for every study visit if they live farther away.

Exclusion Criteria

•Pregnant or nursing mothers or any woman intending to become pregnant in the next three years
•All patients will have pre-study liver function tests, PT/PPT, platelets, hematocrit, and renal function laboratory 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.
•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 a highly active disease state
•History of cirrhosis due to increased risk of central nervous system (CNS) infection
•Poorly controlled hypertension because of increased risk for stroke or CNS hemorrhage. Specifically, any patient with a systolic blood pressure value of ≥ 145 mm/Hg or a diastolic blood pressure value of ≥ 95 mm/Hg will be excluded from study participation.
•History of thyroid disorders or other endocrine disorders 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

Outcomes

Primary Outcomes

Number of participants with adverse events .

Time Frame: 30 months

The co-primary objective will be to observe long term safety of the treatment 6 months and 30 months following the last treatment.

Number of participants with adverse events

Time Frame: 9 months

The primary objective of the study is to assess the safety and tolerability of intrathecal therapy with autologous MSCNPs in MS. Number of participants with adverse events will be documented 1 day, 1 week, 1 month and 2 months post treatment for three treatments (followup for third treatment is 3 months instead of 2 months).