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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01933802
• Date of registration: 23/08/2013
• Prospective Registration: Yes
• Primary sponsor: Tisch Multiple Sclerosis Research Center of New York
• Public title: Intrathecal Administration of Autologous Mesenchymal Stem Cell-derived Neural Progenitors (MSC-NP) in Patients With Multiple Sclerosis
• Scientific title: 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
• Date of first enrolment: April 2014
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01933802
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Saud A Sadiq, MD
• Affiliation: Tisch MS Research Center of New York

Key inclusion & exclusion 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

- Previous or current history of a coagulation disorder

- Any metal in the body, which is contraindicated for MRI studies

- Allergy to any of the antibiotics used in this study, e.g. tobramycin, vancomycin, or
gentamicin

- Patients with alcohol or other substance abuse problems

- Other major disease that, in the opinion of the Principal Investigator, would preclude
participation in the study

- Patients with HBV, 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



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Multiple Sclerosis

Intervention(s)

Biological: intrathecal administration of autologous MSC-NP

Primary Outcome(s)

Number of participants with adverse events . [Time Frame: 30 months]

Number of participants with adverse events [Time Frame: 9 months]

Secondary Outcome(s)

Preliminary evaluation of efficacy [Time Frame: 9 months]

Secondary ID(s)

IND 13889

TISCHMS-MSCNP-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors