NCT00095329

Brief Summary

The purpose of this study is to determine the safety and tolerability of the drug sirolimus in patients with multiple sclerosis (MS) who have failed other treatments.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started May 2003

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2003

Completed
1.5 years until next milestone

First Submitted

Initial submission to the registry

November 2, 2004

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 3, 2004

Completed
4 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2005

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2005

Completed
Last Updated

September 22, 2016

Status Verified

September 1, 2016

Enrollment Period

1.8 years

First QC Date

November 2, 2004

Last Update Submit

September 20, 2016

Conditions

Multiple Sclerosis (MS) - Relapsing-remitting

Keywords

relapsing-remitting MSnonresponsive to standard of care MSsirolimus

Outcome Measures

Primary Outcomes (3)

  • Safety of sirolimus, including number of lesions detected by brain MRI

  • Tolerability of sirolimus

  • Mean number of new and overall total number of gadolinium-enhancing lesions reported on sequential brain MRIs

Secondary Outcomes (2)

  • Efficacy, as measured by change in the mean number of new and overall total number of gadolinium-enhancing lesions on pre-treatment brain MRIs, compared to post-treatment

  • Effect of sirolimus therapy on the immune function of patients with relapsing-remitting multiple sclerosis (RRMS)

Study Arms (1)

sirolimus

EXPERIMENTAL
Biological: sirolimus

Interventions

sirolimusBIOLOGICAL
sirolimus

Eligibility Criteria

Age18 Years - 58 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • Relapsing-remitting MS
  • Evidence of demyelination on magnetic resonance imaging (MRI) scan
  • Expanded Disability Status Scale (EDSS) score between 0 and 6
  • Nonresponsive to beta-interferon or Glatiramer acetate therapy
  • Discontinuation of beta-interferon or Glatiramer acetate therapy within 1 month prior to study entry
  • Willing to use acceptable methods of contraception

You may not qualify if:

  • Primary progressive MS
  • Prior treatment with immunosuppressants
  • Steroid therapy within 1 month prior to study entry
  • Evidence of active infection or cancer
  • Heart or hematologic dysfunction
  • High levels of lipids in the blood
  • Use of lipid-lowering agents
  • History of cirrhosis or liver disease requiring treatment
  • History of hepatitis B or C
  • Active cytomegalovirus infection
  • Kidney disease requiring treatment
  • Active lung disease
  • Diabetes
  • Hyperthyroidism
  • HIV infection
  • +4 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Center for Neurological Diseases, Brigham and Women's Hospital, Harvard Medical School

Boston, Massachusetts, 02115, United States

Location

Related Publications (4)

  • Meier DS, Weiner HL, Khoury SJ, Guttmann CR. Magnetic resonance imaging surrogates of multiple sclerosis pathology and their relationship to central nervous system atrophy. J Neuroimaging. 2004 Jul;14(3 Suppl):46S-53S. doi: 10.1177/1051228404266268.

    PMID: 15228759BACKGROUND

  • Gonsette RE. New immunosuppressants with potential implication in multiple sclerosis. J Neurol Sci. 2004 Aug 15;223(1):87-93. doi: 10.1016/j.jns.2004.04.025.

    PMID: 15261567BACKGROUND

  • Lucchinetti C, Bruck W. The pathology of primary progressive multiple sclerosis. Mult Scler. 2004 Jun;10 Suppl 1:S23-30. doi: 10.1191/1352458504ms1027oa.

    PMID: 15218806BACKGROUND

  • Kovarik JM, Burtin P. Immunosuppressants in advanced clinical development for organ transplantation and selected autoimmune diseases. Expert Opin Emerg Drugs. 2003 May;8(1):47-62. doi: 10.1517/14728214.8.1.47.

    PMID: 14610911BACKGROUND

Related Links

MeSH Terms

Conditions

Multiple Sclerosis, Relapsing-Remitting

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Multiple SclerosisDemyelinating Autoimmune Diseases, CNSAutoimmune Diseases of the Nervous SystemNervous System DiseasesDemyelinating DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Officials

  • Samia J. Khoury, MD

    Center for Neurological Diseases, Brigham and Women's Hospital, Harvard Medical School

    PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

First Submitted

November 2, 2004

First Posted

November 3, 2004

Study Start

May 1, 2003

Primary Completion

March 1, 2005

Study Completion

March 1, 2005

Last Updated

September 22, 2016

Record last verified: 2016-09

Locations

US(1)