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

NCT ID: NCT03822858

Last Updated: 2025-08-17

Basic Information

• Recruitment Status: AVAILABLE
• Study Classification: EXPANDED_ACCESS

Brief Summary

To give expanded access to intrathecal autologous MSC-NP treatment to patients with progressive MS who do not meet the inclusion/exclusion criteria of our Phase II stem cell trial.

Conditions

Multiple Sclerosis

Interventions

Intrathecal MSC-NP injection

Between 5 to 10 million MSC-NPs will be administered intrathecally in each dose.

10 million cells is the maximum dose. Treatment will consist of 3-5 doses spaced 3 months apart.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Diagnosis of MS as defined by the McDonald criteria
• Diagnosis of primary progressive or secondary progressive MS
• Patients will be 18 years or older
• Significant disability shown by an Expanded Disability Status Score (EDSS, [5]) ≥4.5 that was not acquired within the last 12 months *(Patients with moderate to severe cerebellar dysfunction who have an EDSS of <6.5 who are not eligible for our Ph II Version Date: 11/21/18 Version #4 9 study will be included. This is because EDSS scores are heavily related to muscle strength and not balance or coordination and most accurately reflect paraparesis. Also there are patients with MS who have disabling upper limb coordination dysfunction but EDSS scores of better than 6.5 who are not suitable for the Phase II study but may benefit from this therapy).
• 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

Exclusion Criteria

• 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
• 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
• History of central nervous system infection or immunodeficiency syndromes due to increased risk of CNS infection
• Patients who are anticipated to have difficultly accessing the intrathecal space related to scoliosis, obesity, or any other relevant factors determined by the PI.
• Patients who have not tried available therapies for their progressive MS

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Tisch Multiple Sclerosis Research Center of New York

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Saud A Sadiq, MD, FAAN

Role: PRINCIPAL_INVESTIGATOR

Tisch MS Research Center of New York

Locations

Tisch MS Research Center of New York

New York, New York, United States

Site Status: AVAILABLE

Countries

United States

Central Contacts

Saud A Sadiq, MD, FAAN

Role: CONTACT

ssadiq@tischms.org

212 265 8070

Violaine Harris, PhD

Role: CONTACT

vharris@tischms.org

212 265 8070

Facility Contacts

Saud Sadiq, MD

Role: primary

ssadiq@tischms.org

212 265 8070

References

Harris VK, Stark J, Vyshkina T, Blackshear L, Joo G, Stefanova V, Sara G, Sadiq SA. Phase I Trial of Intrathecal Mesenchymal Stem Cell-derived Neural Progenitors in Progressive Multiple Sclerosis. EBioMedicine. 2018 Mar;29:23-30. doi: 10.1016/j.ebiom.2018.02.002. Epub 2018 Feb 3.

Reference Type: BACKGROUND

PMID: 29449193 (View on PubMed)

Other Identifiers

TISCHMS-MSCNP-003

Identifier Type: -

Identifier Source: org_study_id