Treating Multiple Sclerosis With Sirolimus, an Immune System Suppressor
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ClinicalTrials.gov Identifier: NCT00095329 |
Recruitment Status :
Terminated
(Due to slow recruitment.)
First Posted : November 3, 2004
Last Update Posted : September 22, 2016
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Multiple Sclerosis (MS) - Relapsing-remitting | Biological: sirolimus | Phase 1 Phase 2 |
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.
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 |

Arm | Intervention/treatment |
---|---|
Experimental: sirolimus |
Biological: sirolimus |
- 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
- 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)

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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

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 ClinicalTrials.gov identifier (NCT number): NCT00095329
United States, Massachusetts | |
Center for Neurological Diseases, Brigham and Women's Hospital, Harvard Medical School | |
Boston, Massachusetts, United States, 02115 |
Principal Investigator: | Samia J. Khoury, MD | Center for Neurological Diseases, Brigham and Women's Hospital, Harvard Medical School |
Publications:
Responsible Party: | National Institute of Allergy and Infectious Diseases (NIAID) |
ClinicalTrials.gov 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 |
relapsing-remitting MS nonresponsive to standard of care MS sirolimus |
Multiple Sclerosis Sclerosis Pathologic Processes Demyelinating Autoimmune Diseases, CNS Autoimmune Diseases of the Nervous System Nervous System Diseases Demyelinating Diseases Autoimmune Diseases Immune System Diseases |
Sirolimus Anti-Bacterial Agents Anti-Infective Agents Antibiotics, Antineoplastic Antineoplastic Agents Antifungal Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |