Autologous Mesenchymal Stem Cell (MSC) Transplantation in MS

NCT00813969 | Completed Phase 1 DMC

• 1 other identifier: MS-MSC-001
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Relapsing-Remitting Multiple Sclerosis; Secondary Progressive Multiple Sclerosis; Progressive Relapsing Multiple Sclerosis

Keywords

Mesenchymal Stem Cell; MSC; Autologous; Multiple Sclerosis

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (2)

• To evaluate effects on MS disease activity measured by the number of Gd-enhancing brain MRI lesions

  1 month

• To evaluate safety and tolerability of autologous MSC transplantation over 6 months

  6 months

Study Arms (1)

Autologous MSC transplantation

EXPERIMENTAL

Biological: Autologous mesenchymal stem cell transplantation

Interventions

Autologous mesenchymal stem cell transplantation BIOLOGICAL

A single IV infusion of up to 2 million cells per kg based on the MSC numbers achieved after culture expansion

Autologous MSC transplantation

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• The Cleveland Clinic: lead
• University Hospitals Cleveland Medical Center: collaborator

Study Sites (1)

Cleveland Clinic Mellen Center

Cleveland, Ohio, 44195, United States

Location

Related Publications (1)

• Cohen JA, Imrey PB, Planchon SM, Bermel RA, Fisher E, Fox RJ, Bar-Or A, Sharp SL, Skaramagas TT, Jagodnik P, Karafa M, Morrison S, Reese Koc J, Gerson SL, Lazarus HM. Pilot trial of intravenous autologous culture-expanded mesenchymal stem cell transplantation in multiple sclerosis. Mult Scler. 2018 Apr;24(4):501-511. doi: 10.1177/1352458517703802. Epub 2017 Apr 6.

  PMID: 28381130 DERIVED

MeSH Terms

Conditions

Multiple Sclerosis, Relapsing-Remitting; Multiple Sclerosis, Chronic Progressive; Multiple Sclerosis

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Jeffrey A Cohen, M.D.

  The Cleveland Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2008
• First Posted: December 23, 2008
• Study Start: March 1, 2011
• Primary Completion: January 1, 2014
• Study Completion: May 1, 2014
• Last Updated: March 30, 2016

Record last verified: 2016-03

Locations

US (1)