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Multiple SclerosisPhase 2NCT03355365

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

A Phase 2 study testing Intrathecal MSC-NP injection and Intrathecal saline injection in people with Multiple Sclerosis, run by Tisch Multiple Sclerosis Research Center of New York at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Multiple Sclerosis
What is being tested?
Intrathecal MSC-NP injection and Intrathecal saline injection
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
54 participants
Where
1 siteUnited States
Study dates
21 Sep 2018to 17 Apr 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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)
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  • 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.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Tisch MS Research Center of New York

    New York, New York, 10019

Other trials in United States

About this study

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

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.

Read the detailed description

The IT-MSC-NP treatments and all clinical assessments will take place at a single center (Tisch MSRCNY). Study subjects will be assigned to blocks stratified by baseline EDSS score (3.0-4.0, 4.5-5.5, 6.0, and 6.5) and disease subtype (SPMS or PPMS). Study subjects are randomized in an equal fashion (1:1) to study treatment and placebo at initial randomization. Subjects in each block will be randomized into placebo or treatment group. In the second year, treated subjects will cross over to the placebo group and placebo subjects will cross over to the treated group.

The total study duration will be 3 years upon enrollment. Each study subject will be required to attend up to 18 study visits, to include 1 screening visit, 1 bone marrow visit, 1 baseline visit, followed by study visits every 2 months during the treatment period of two years (12 treatment/LP procedure visits and 2 outcome visits), and an additional follow-up visit at the end of year 3.

Being tested
Intrathecal MSC-NP injection, Intrathecal saline injection
Keywords
Mesenchymal Stem Cells, Neural Progenitors, Autologous, Bone Marrow, Multiple Sclerosis
Medical subject headings
Multiple Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Intrathecal MSC-NP injectionBiological

    MSC-NPs represent a neural subpopulation of MSCs from bone marrow with reduced pluripotency and minimized risk of ectopic differentiation, thus are likely to be more suitable for CNS delivery. Importantly, characterization of MSC-NPs demonstrated their immunoregulatory and trophic properties, and MSC-NPs derived from MS and non-MS patients alike were therapeutically viable.

  • Intrathecal saline injectionOther

    Placebo

Study groups in NCT03355365
Study groupRoleWhat they receive
Intrathecal MSC-NP injectionExperimentalPatients will receive six autologous stem cell injections through spinal taps every 2 months over a year.
Receives: Biological: Intrathecal MSC-NP injection
Intrathecal saline injectionPlacebo comparatorPatients will receive six placebo injections through spinal taps every 2 months over a year.
Receives: Other: Intrathecal saline injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Expanded Disability Status Scale (EDSS) Plus

    Changes in disability assessed based on composite score of EDSS, timed 25-foot walk (T25FW), and nine hole peg test (9HPT) (EDSS-Plus). Improvement is defined by at least one of the following three measures: ≥0.5 decrease in EDSS (if EDSS at entry is ≥ 6.0) or ≥ 1.0 decrease in EDSS (if EDSS at entry is ≤5.5), ≥20% increase in T25FW, or ≥20% increase in 9HPT in either dominant or non-dominant hand. Assessments were made at baseline and month 13. The number of patients who improved in any of the 3 composite measures in month 13 compared to baseline is reported.

    Measured: Baseline and 13 months

Secondary outcomes (8)
  1. Change in Multiple Sclerosis Functional Composite (MSFC) Z-Score From Baseline

    The Multiple Sclerosis Functional Composite (MSFC) is a standardized tool used to quantify disability in people with multiple sclerosis (MS) by measuring leg function/ambulation (timed 25 foot walk), arm/hand function (9-hole peg test), and cognitive function (Paced Auditory Serial Addition Test). Each assessment is converted into a Z score. A Z-score of 0 represents the population mean. The composite MSFC Z-score represents an average of the 3 Z-scores. A positive change in composite Z-score in month 13 compared to baseline represents better performance and a negative change in composite Z score indicates worse performance.

    Measured: Baseline and 13 months

  2. Change in EDSS From Baseline

    The Expanded Disability Status Scale (EDSS) is a standardized measure used to assess and track the level of disability in people with multiple sclerosis (MS), ranging from 0 (normal neurological status) to 10 (death due to MS) in 0.5 increments. Only ambulatory subjects were enrolled in this study with an EDSS between 3.0 and 6.5. Subgroups of participants were analyzed based on the degree of walking disability. EDSS 3.0-5.5 represents moderate to severe disability but able to walk without assistance, and EDSS 6.0-6.5 represents a need for unilateral or bilateral assistance to ambulate. A decrease in EDSS at month 13 compared to baseline is considered improvement.

    Measured: Baseline and 13 months

  3. Percent Change in T25FW (Timed 25 Foot Walk) From Baseline

    The timed 25-foot walk (T25FW) is a test used in multiple sclerosis (MS) to assess a person's mobility and leg function, where individuals walk 25 feet as quickly and safely as possible, and the time taken is recorded. A positive percentage change indicates improved walking in month 13 compared to baseline, whereas a negative percentage change indicates worsening.

    Measured: Baseline and 13 months

  4. Percent Change in 6MWT (6 Minute Walk Test) From Baseline

    The 6-minute walk test (6MWT) in multiple sclerosis (MS) measures functional walking capacity by assessing the distance a person can walk in 6 minutes. A positive percentage change indicates improved walking capacity at month 13 compared to baseline, whereas a negative percentage change indicates worsening.

    Measured: Baseline and 13 months

  5. Percent Change in 9HPT-D (9 Hole Peg Test in Dominant Hand) From Baseline

    The Nine-Hole Peg Test (9HPT) is a standardized, quantitative assessment used to measure manual dexterity and upper extremity function in individuals with multiple sclerosis (MS). Each hand, dominant (D) and non-dominant (ND) is assessed seperately. A positive percentage change indicates improved function at month 13 compared to baseline, whereas a negative percentage change indicates worsening.

    Measured: Baseline and 13 months

  6. Percent Change in 9HPT-ND (9 Hole Peg Test in Non-Dominant Hand) From Baseline

    The Nine-Hole Peg Test (9HPT) is a standardized, quantitative assessment used to measure manual dexterity and upper extremity function in individuals with multiple sclerosis (MS). Each hand, dominant (D) and non-dominant (ND) is assessed seperately. A positive percentage change indicates improved function at month 13 compared to baseline, whereas a negative percentage change indicates worsening.

    Measured: Baseline and 13 months

  7. Change in MSWS-12 (12 Item MS Walking Scale) Score From Baseline

    The 12-item Multiple Sclerosis Walking Scale (MSWS-12) is a patient-reported outcome measure that assesses the impact of multiple sclerosis (MS) on walking ability. The self-administered questionnaire consists of 12 items addressing how MS has affected various aspects of walking over the past two weeks. Each of the 12 items is scored from 1 to 5 from: 1 = Not at all to 5 = Extremely. Raw total score ranges from 12 (no impairment) to 60 (maximum impairment). Raw score is converted to a standardized score (0-100 scale) producing a final score from 0 = No impact of MS on walking to 100 = Maximum impact. The change in score from baseline to post-intervention was calculated. A decrease in score at month 13 compared to baseline was considered improvement.

    Measured: Baseline and 13 months

  8. Change in PASAT (Paced Auditory Serial Addition Test) Score From Baseline

    The Paced Auditory Serial Addition Test (PASAT) is a neuropsychological test used to assess cognitive impairments, attention, information processing speed, and working memory. The test involves listening to a series of single-digit numbers and the individual must add each new number to the one immediately before it. The score is the number of correct responses out of 60 items. The change in score from baseline to post-intervention is calculated. A positive change in score means an increase in the number of correct answers at month 13 compared to baseline, generally indicating improvement in information processing speed and sustained attention.

    Measured: Baseline and 13 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Nov 2017
  2. Study start21 Sep 2018
  3. Primary completion9 Feb 2023
  4. Study completion17 Apr 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Model description
Compassionate crossover design
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
54 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03355365
Sponsor's protocol ID
TISCHMS-MSCNP-002
Status last verified
May 2025
First submitted
14 Nov 2017
Last updated
11 Jun 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 29449193
  • Harris VK, Stark J, Williams A, Roche M, Malin M, Kumar A, Carlson AL, Kizilbash C, Wollowitz J, Andy C, Gerber LM, Sadiq SA. Efficacy of intrathecal mesenchymal stem cell-neural progenitor therapy in progressive MS: results from a phase II, randomized, placebo-controlled clinical trial. Stem Cell Res Ther. 2024 May 23;15(1):151. doi: 10.1186/s13287-024-03765-6.

    PubMed 38783390

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Intrathecal MSC-NP injection and Intrathecal saline injection in people with Multiple Sclerosis, run by Tisch Multiple Sclerosis Research Center of New York at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Expanded Disability Status Scale (EDSS) Plus (time frame: Baseline and 13 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03355365, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2025.

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