Autologous Mesenchymal Stem Cell (MSC) Transplantation in MS

Relapsing-Remitting Multiple Sclerosis Clinical Trial

Official title:

A Phase I Study to Assess the Feasibility, Safety, and Tolerability of Autologous Mesenchymal Stem Cell Transplantation in Patients With Relapsing Forms of Multiple Sclerosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00813969
• Other study ID #: MS-MSC-001
• Status: Completed
• Phase: Phase 1
• First received: December 22, 2008
• Last updated: March 28, 2016
• Start date: March 2011
• Est. completion date: May 2014

Study information

• Verified date: March 2016
• Source: The Cleveland Clinic
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

The study is an investigator-run, open-label Phase 1 safety study of autologous mesenchymal stem cell transplantation, involving approximately 24 ambulatory participants with relapsing forms of MS (approximately equal numbers with relapsing-remitting and secondary progressive/ progressive relapsing MS) and evidence of involvement of the anterior afferent visual system.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: May 2014
• Est. primary completion date: January 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 55 Years

Eligibility

Inclusion Criteria:

• Age 18 to 55, inclusive.

• Diagnosis of MS

• Relapsing form of MS (relapsing-remitting, secondary progressive, or progressive-relapsing course).

• EDSS score 3.0-6.5, inclusive. (Must be able to walk)

• Active disease during prior 24 months.

• Documented evidence of involvement of the anterior afferent visual system: previous optic neuritis, optic atrophy or an afferent pupillary defect on exam, RNFL thickness on OCT <LLN in at least one eye OR documented VEP latency in at least 1 eye.

• Cranial MRI scan demonstrating T2-hyperintense lesions satisfying diagnostic criteria for MS

• Ability to perform the component tests of the MSFC (T25FW, 9HPT, PASAT3).

• Ability to perform SLCLA.

• Has given written informed consent to participate in the study.

Exclusion Criteria:

• A clinically significant infectious illness (e.g., cellulitis, abscess, pneumonia, septicemia) within 30 days of the Screening Visit.

• History of cancer other than basal cell carcinoma of the skin.

• History or laboratory results indicative of any significant cardiac, endocrine, hematologic, hepatic, immunologic, infectious, metabolic, urologic, pulmonary, gastrointestinal, dermatologic, psychiatric, renal, neoplastic, or other disorder that in the opinion of the Principal Investigator would preclude the safe performance of BM aspiration, infusion of autologous MSCs, or performance of any of the planned study assessments.

• Abnormal blood tests which exceed designated limits.

• Positive screening tests for hepatitis B, hepatitis C, HIV 1&2, HTLV I/II, CMV, West Nile virus, syphilis, blood parasite infection.

• Clinically significant abnormality on chest X-ray.

• Clinically significant abnormality on EKG.

• Oxygen-saturation <90% on room air.

• History of alcohol or drug abuse within one year.

• Any metallic material or electronic device in the body, or condition that precludes the participant from undergoing MRI with Gd administration.

• Uncontrolled glaucoma or other ocular condition that precludes performing OCT or interpreting the results.

• MS relapse with onset within 30 days prior to the Screening Visit or the participant has not stabilized from a previous relapse at the time of the Screening Visit.

• Current treatment with an investigational MS disease therapy.

• Prior treatment with:

Total lymphoid irradiation. Cladribine. T-cell or T-cell receptor vaccination. Campath-1h (alemtuzumab). Rituxan (rituximab).

• Prior treatment within three months with:

Tysabri (natalizumab). Gilenya (Fingolimod/FTY720). Zenapax (daclizumab). Cytoxan (cyclophosphamide). Novantrone (mitoxantrone). Cyclosporine. CellCept (mycophenolate mofetil). Imuran (azathioprine). Rheumatrex (methotrexate). IV gamma globulin. Plasma exchange.

• Prior treatment within one month:

Systemic corticosteroids with daily dose equivalent to Prednisone 60 mg or greater.

• Female participants who are not post-menopausal for at least one year, not surgically sterile, or not willing to practice effective contraception.

• Nursing mothers, pregnant women, or women planning to become pregnant during the study.

• Male participants who are not willing to practice effective contraception.

• Unwillingness or inability to comply with the requirements of this protocol including the presence of any condition (physical, mental, or social) that, in the opinion of the Principal Investigator, is likely to affect the participant's ability to comply with the study protocol.

• Any other reason that, in the opinion of the Principal Investigator, makes the participant unsuitable for participation in the study.

Study Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Multiple Sclerosis
• Multiple Sclerosis, Chronic Progressive
• Multiple Sclerosis, Relapsing-Remitting
• Progressive Relapsing Multiple Sclerosis
• Relapsing-Remitting Multiple Sclerosis
• Sclerosis
• Secondary Progressive Multiple Sclerosis

Intervention

Biological:
• Autologous mesenchymal stem cell transplantation
A single IV infusion of up to 2 million cells per kg based on the MSC numbers achieved after culture expansion

Locations

Country	Name	City	State
United States	Cleveland Clinic Mellen Center	Cleveland	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
The Cleveland Clinic	University Hospital Case Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the feasibility of culturing MSCs, and infusion-related safety and tolerability of autologous MSC transplantation over one month in patients with relapsing forms of MS		1 month	Yes
Secondary	To evaluate effects on MS disease activity measured by the number of Gd-enhancing brain MRI lesions		1 month	No
Secondary	To evaluate safety and tolerability of autologous MSC transplantation over 6 months		6 months	Yes