Intrathecal Administration of Autologous Mesenchymal Stem Cell-derived Neural Progenitors (MSC-NP) in Progressive Multiple Sclerosis: in progressive MS, phase 2

Autologous, Bone Marrow-Derived Mesenchymal Stem Cell-Derived Neural Progenitor Cells (MSC-NP), Expanded Ex Vivo; Administered Intrathecally

CompletedPhase 2Progressive MSNCT03355365

What it found

Missed its main goal

The main measure did not show a significant benefit.

Measure	Estimate	95% CI	p
Expanded Disability Status Scale (EDSS) Plus			1.0

Statistical analysis as posted on ClinicalTrials.gov.

What was tested

Treatment tested

Intrathecal MSC-NP injection

Placebo

Intrathecal saline injection

Results by group

Expanded Disability Status Scale (EDSS) Plus

Baseline and 13 months

Group	Value	Interval	Number analysed
Treatment (IT MSC-NP)	17 Participants		49
Placebo (IT Saline)	17 Participants		49

Treatment (IT MSC-NP)

Placebo (IT Saline)

p = 1.0

Values as posted on ClinicalTrials.gov. Outcomes measured differently across trials are not directly comparable.

Safety

Group	Participants at risk	Serious adverse events
Intrathecal MSC-NP Injection (Year 1)	27	2 (7.4%)
Intrathecal Saline Injection (Year 1)	27	0 (0.0%)
Intrathecal MSC-NP Injection (Year 2)	27	3 (11.1%)
Intrathecal Saline Injection (Year 2)	24	3 (12.5%)

As posted on ClinicalTrials.gov. Serious adverse events include any serious health event during the trial, whether or not the treatment caused it.

What it measured

Main measures

Expanded Disability Status Scale (EDSS) Plus
Baseline and 13 months
Disability progressionWalking and mobility

Other measures (8)

Change in Multiple Sclerosis Functional Composite (MSFC) Z-Score From Baseline
Baseline and 13 months
Disability progressionThinking and memoryWalking and mobilityArm and hand function
Change in EDSS From Baseline
Baseline and 13 months
Disability progressionWalking and mobility
Percent Change in T25FW (Timed 25 Foot Walk) From Baseline
Baseline and 13 months
Disability progressionWalking and mobility
Percent Change in 6MWT (6 Minute Walk Test) From Baseline
Baseline and 13 months
Walking and mobility
Percent Change in 9HPT-D (9 Hole Peg Test in Dominant Hand) From Baseline
Baseline and 13 months
Disability progressionArm and hand function
Percent Change in 9HPT-ND (9 Hole Peg Test in Non-Dominant Hand) From Baseline
Baseline and 13 months
Disability progressionArm and hand function
Change in MSWS-12 (12 Item MS Walking Scale) Score From Baseline
Baseline and 13 months
Walking and mobilityQuality of life (reported by participants)
Change in PASAT (Paced Auditory Serial Addition Test) Score From Baseline
Baseline and 13 months
Thinking and memory

Who could take part

Progressive MS · 18 to 65

Show the full 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 >20 years at time of screening * Change of disease modifying agent \< 12 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 implant 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.

As registered. Criteria can change, so ask the trial team or your neurologist.

About this trial

In the sponsor's words

This is a phase II, double-blinded, placebo-controlled, randomized, cross-over Study designed to determine the 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 treatments in a second year.

Papers

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History

Started
Sept 2018
Main results due
Feb 2023
Finished
Apr 2023
Results posted
Jun 2025

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