Clinical trial NCT03822858: Expanded Access to Intrathecal Administration of Autologous Mesenchymal Stem Cell-derived Neural Progenitors (MSC-NP) in Progressive Multiple Sclerosis
Official title: Autologous, Bone Marrow-Derived Mesenchymal Stem Cell-Derived Neural Progenitor Cells (MSC-NP), Expanded Ex Vivo; Administered Intrathecally
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Multiple Sclerosis
Keywords: Mesenchymal Stem Cells
Neural Progenitors
Autologous
Bone Marrow
Multiple Sclerosis
Lead sponsor: Tisch Multiple Sclerosis Research Center of New York
Sponsor class: OTHER
First posted: 2019-01-30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT

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.


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
* Does not qualify for the inclusion/exclusion criteria of our Phase II stem cell trial. For example, patients with EDSS \>6.5 or MS symptom onset and/or duration of disease \>15 years

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

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: New York
country: United States
facility: Tisch MS Research Center of New York
state: New York
Study officials:
affiliation: Tisch MS Research Center of New York
name: Saud A Sadiq, MD, FAAN
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.