Multiple SclerosisPhase 1NCT01933802
Intrathecal Administration of Autologous Mesenchymal Stem Cell-derived Neural Progenitors (MSC-NP) in Patients With Multiple Sclerosis
A Phase 1 study testing intrathecal administration of autologous MSC-NP in people with Multiple Sclerosis, run by Tisch Multiple Sclerosis Research Center of New York at 1 site in United States.
Trial summary
The study is an open-label, phase I clinical trial designed to evaluate the safety and tolerability of intrathecal administration of autologous mesenchymal stem cell-derived neural progenitor cells (MSC-NP) in patients with progressive multiple sclerosis. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Multiple Sclerosis
- What is being tested?
- intrathecal administration of autologous MSC-NP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2014to Mar 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 70 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 18-70 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.
Who cannot join
- 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
Show all 18Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Tisch MS Research Center of New York
New York, New York, 10019
About this study
Official 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
The study is an open-label, phase I clinical trial designed to evaluate the safety and tolerability of intrathecal administration of autologous mesenchymal stem cell-derived neural progenitor cells (MSC-NP) in patients with progressive multiple sclerosis. Study participants will receive three intrathecal injections of culture-expanded autologous MSC-NPs at three month intervals.
- Conditions
- Multiple Sclerosis
- Being tested
- intrathecal administration of autologous MSC-NP
- 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 administration of autologous MSC-NPBiological
Autologous MSC-NPs administered intrathecally at a dose between 2 and 10 million cells, depending on ex vivo expansion characteristics. Three doses will be administered at 3 month intervals.
| Study group | Role | What they receive |
|---|---|---|
| autologous MSC-NP | Experimental | intrathecal administration of autologous MSC-NP in three doses at three month intervals Receives: Biological: intrathecal administration of autologous MSC-NP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with adverse events
The primary objective of the study is to assess the safety and tolerability of intrathecal therapy with autologous MSCNPs in MS. Number of participants with adverse events will be documented 1 day, 1 week, 1 month and 2 months post treatment for three treatments (followup for third treatment is 3 months instead of 2 months).
Measured: 9 months
Number of participants with adverse events .
The co-primary objective will be to observe long term safety of the treatment 6 months and 30 months following the last treatment.
Measured: 30 months
Secondary outcomes (1)
Preliminary evaluation of efficacy
The secondary objective is to observe trends in efficacy over the course of the experimental treatment. Outcome measures include evoked potentials (baseline and 3 months post 3rd dose) ,quality of life questionnaire, EDSS, and MSFC (baseline, at the time of each dose, and 3 and 6 months after third dose), and MRI (baseline, 2 months after first dose, and 3 and 24 months after third dose).
Measured: 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2013 (estimated)
- Study startApr 2014
- Primary completionDec 2016
- Study completionMar 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Multiple SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tisch Multiple Sclerosis Research Center of New York
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Saud A Sadiq, MD, Principal investigator, Tisch MS Research Center of New York
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01933802
- Sponsor's protocol ID
- TISCHMS-MSCNP-001
- Other IDs
- IND 13889
- Status last verified
- Mar 2018
- First submitted
- 23 Aug 2013
- Last updated
- 19 Mar 2018
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Harris VK, Faroqui R, Vyshkina T, Sadiq SA. Characterization of autologous mesenchymal stem cell-derived neural progenitors as a feasible source of stem cells for central nervous system applications in multiple sclerosis. Stem Cells Transl Med. 2012 Jul;1(7):536-47. doi: 10.5966/sctm.2012-0015. Epub 2012 Jun 28.
PubMed 23197858Harris VK, Yan QJ, Vyshkina T, Sahabi S, Liu X, Sadiq SA. Clinical and pathological effects of intrathecal injection of mesenchymal stem cell-derived neural progenitors in an experimental model of multiple sclerosis. J Neurol Sci. 2012 Feb 15;313(1-2):167-77. doi: 10.1016/j.jns.2011.08.036. Epub 2011 Oct 1.
PubMed 21962795Harris 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 29449193Harris VK, Stark JW, Yang S, Zanker S, Tuddenham J, Sadiq SA. Mesenchymal stem cell-derived neural progenitors in progressive MS: Two-year follow-up of a phase I study. Neurol Neuroimmunol Neuroinflamm. 2020 Dec 4;8(1):e928. doi: 10.1212/NXI.0000000000000928. Print 2021 Jan.
PubMed 33277427
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT01933802
- Acronym / sponsor ID
- TISCHMS-MSCNP-001
- Conditions
- Multiple Sclerosis
- Keywords
- mesenchymal stem cells, neural progenitors, autologous, bone marrow, multiple sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Tisch Multiple Sclerosis Research Center of New YorkUniversity, hospital or other
- Study start
- Apr 2014
- Primary completion
- Dec 2016 (actual)
- Study completion
- Mar 2017 (actual)
- First posted
- 2 Sep 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is an open-label, phase I clinical trial designed to evaluate the safety and tolerability of intrathecal administration of autologous mesenchymal stem cell-derived neural progenitor cells (MSC-NP) in patients with progressive multiple sclerosis. Study participants will receive three intrathecal injections of culture-expanded autologous MSC-NPs at three month intervals.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| autologous MSC-NP | Experimental | Biological: intrathecal administration of autologous MSC-NPintrathecal administration of autologous MSC-NP in three doses at three month intervals |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (18 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| intrathecal administration of autologous MSC-NPAutologous MSC-NPs administered intrathecally at a dose between 2 and 10 million cells, depending on ex vivo expansion characteristics. Three doses will be administered at 3 month intervals. | Biological | autologous MSC-NP |
Primary outcome
Number of participants with adverse events
Time frame: 9 months
The primary objective of the study is to assess the safety and tolerability of intrathecal therapy with autologous MSCNPs in MS. Number of participants with adverse events will be documented 1 day, 1 week, 1 month and 2 months post treatment for three treatments (followup for third treatment is 3 months instead of 2 months).
Number of participants with adverse events .
Time frame: 30 months
The co-primary objective will be to observe long term safety of the treatment 6 months and 30 months following the last treatment.
1 secondary outcome
Preliminary evaluation of efficacy
Time frame: 9 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2018.
- Background: PubMed 23197858Harris VK, Faroqui R, Vyshkina T, Sadiq SA. Characterization of autologous mesenchymal stem cell-derived neural progenitors as a feasible source of stem cells for central nervous system applications in multiple sclerosis. Stem Cells Transl Med. 2012 Jul;1(7):536-47. doi: 10.5966/sctm.2012-0015. Epub 2012 Jun 28.
- Background: PubMed 21962795Harris VK, Yan QJ, Vyshkina T, Sahabi S, Liu X, Sadiq SA. Clinical and pathological effects of intrathecal injection of mesenchymal stem cell-derived neural progenitors in an experimental model of multiple sclerosis. J Neurol Sci. 2012 Feb 15;313(1-2):167-77. doi: 10.1016/j.jns.2011.08.036. Epub 2011 Oct 1.
- Results publication: PubMed 29449193Harris 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.
- Derived from this study: PubMed 33277427Harris VK, Stark JW, Yang S, Zanker S, Tuddenham J, Sadiq SA. Mesenchymal stem cell-derived neural progenitors in progressive MS: Two-year follow-up of a phase I study. Neurol Neuroimmunol Neuroinflamm. 2020 Dec 4;8(1):e928. doi: 10.1212/NXI.0000000000000928. Print 2021 Jan.
- Tisch MS Research Center of New YorkLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Saud A Sadiq, MD principal investigator
Tisch MS Research Center of New York
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing intrathecal administration of autologous MSC-NP 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 18 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 19 Mar 2018. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of participants with adverse events (time frame: 9 months). The study lists 2 primary outcomes in all. 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 NCT01933802, which also lists 4 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 19 Mar 2018.