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Treating Multiple Sclerosis With Sirolimus, an Immune System Suppressor

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00095329
Recruitment Status : Terminated (Due to slow recruitment.)
First Posted : November 3, 2004
Last Update Posted : September 22, 2016
Autoimmunity Centers of Excellence
Information provided by (Responsible Party):
National Institute of Allergy and Infectious Diseases (NIAID)

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.

Condition or disease Intervention/treatment Phase
Multiple Sclerosis (MS) - Relapsing-remitting Biological: sirolimus Phase 1 Phase 2

Detailed Description:

MS is a chronic autoimmune disease of the central nervous system in which myelin, the protein sheath that protects nerve cells, is degraded by T cells and macrophages, leading to an eventual loss of neurologic function. MS can be classified as either relapsing-remitting, in which patients experience worsening in symptoms followed by partial or complete recovery of function; or progressive, in which patients have a gradual increase in symptoms, with or without relapses. Standard treatments used to treat relapsing-remitting MS are only modestly effective and may be associated with significant toxicity. There is a need to develop therapies with lower toxicities that can be administered early during the course of disease and have the potential to stop disease progression altogether. Sirolimus has been demonstrated to provide potent immunosuppression in recent clinical trials involving kidney transplantation, and may help people with autoimmune diseases like MS. This study will determine the benefit of sirolimus in MS patients.

Blood and urine collection will occur at screening. Participants will take daily doses of sirolimus for 6 months. There will be nine study visits; they will occur at Days 14, 28, 42, 56, 90, 120, 150, 180, and 225. Medication adverse events, concomitant medications, and vital signs will be recorded at Visits 1 through 8. At all visits, patient compliance to the sirolimus regimen will be measured, and blood and urine collection will occur. Physical and neurological exams, magnetic resonance imaging (MRI) brain scans, MS status tests, and a chest x-ray will be conducted at selected times throughout the study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II, Open-Label Pilot Trial to Evaluate the Safety of Rapamune (Sirolimus) in Patients With Multiple Sclerosis
Study Start Date : May 2003
Actual Primary Completion Date : March 2005
Actual Study Completion Date : March 2005

Resource links provided by the National Library of Medicine

Drug Information available for: Sirolimus

Arm Intervention/treatment
Experimental: sirolimus Biological: sirolimus

Primary Outcome Measures :
  1. Safety of sirolimus, including number of lesions detected by brain MRI
  2. Tolerability of sirolimus
  3. Mean number of new and overall total number of gadolinium-enhancing lesions reported on sequential brain MRIs

Secondary Outcome Measures :
  1. 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
  2. Effect of sirolimus therapy on the immune function of patients with relapsing-remitting multiple sclerosis (RRMS)

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 58 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  • Tuberculosis
  • History of alcohol or drug abuse within 6 months prior to study entry
  • Claustrophobia or inability to undergo MRI
  • Pregnancy or breast-feeding

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00095329

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United States, Massachusetts
Center for Neurological Diseases, Brigham and Women's Hospital, Harvard Medical School
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
National Institute of Allergy and Infectious Diseases (NIAID)
Autoimmunity Centers of Excellence
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Principal Investigator: Samia J. Khoury, MD Center for Neurological Diseases, Brigham and Women's Hospital, Harvard Medical School
Additional Information:
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Responsible Party: National Institute of Allergy and Infectious Diseases (NIAID) Identifier: NCT00095329    
Other Study ID Numbers: DAIT AMS02
First Posted: November 3, 2004    Key Record Dates
Last Update Posted: September 22, 2016
Last Verified: September 2016
Keywords provided by National Institute of Allergy and Infectious Diseases (NIAID):
relapsing-remitting MS
nonresponsive to standard of care MS
Additional relevant MeSH terms:
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Multiple Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs