Phase 2
N=99
Prospective Study of Rapamycin for the Treatment of SLE
Systemic Lupus Erythematosus (SLE)
Bottom Line
View on ClinicalTrials.gov: NCT00779194 ↗Enrolled (actual)
99
Serious AEs
0.0%
Results posted
Jun 2024
Primary outcome: Primary: Reduction of the Disease Activity as Measured by SLEDAI and BILAG Scores. — 10.2; 28.4 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Rapamycin (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- State University of New York - Upstate Medical University
- Primary completion
- Dec 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Reduction of the Disease Activity as Measured by SLEDAI and BILAG Scores. |
10.2; 28.4 | — |
| SECONDARY Decrease of the Amount of Prednisone Needed to Control Disease Activity in SLE Patients. |
24.3; 7.2 | — |
Summary
Systemic lupus erythematosus (SLE) is an autoimmune disease of unknown origin. It involves multiple organs including the joints, skin, kidneys and central nervous system. The disease process is caused by a dysfunction of the immune system. The drugs currently used for the treatment of SLE are only partially effective and carry significant risks for side-effects. Patients that were resistant or intolerant to conventional medication have been effectively treated with Rapamycin and were able to decrease the amount of prednisone they needed.
The purpose of this study is to prospectively determine the therapeutic efficacy and mechanism of action of Rapamune in patients with SLE. Healthy subjects not receiving Rapamune will be asked to donate blood to serve as controls only for immunobiological outcomes.
As part of the research effort to understand the reason for the variations in the effects of treatment drugs by different individuals, a sub-study of the DNA makeup of subjects enrolled in the trial will also be done. The purpose of the sub-study is to possibly determine whether different responses to the drugs used to treat SLE have a correlation with the differences in the genetic makeup of the subjects.
Eligibility Criteria
Inclusion Criteria
For SLE Subjects:
- SLE patients who exhibit ongoing disease activity by SLEDAI greater or equal to 4.
- SLE patients whose disease activity is controlled by administration of corticosteroids, most commonly, at least 10 mg/day of prednisone.
- 18 years of age or older.
- Updated vaccinations prior to study entry.
- Use of effective contraception for male patients before, during and up to 12 weeks after sirolimus therapy.
For Healthy Control Subjects:
- 18 years of age or older
- Must be matched with one of the SLE patients enrolled in the study by age, gender and ethnic origin
- Must not have any acute or chronic illness.
Exclusion Criteria
For SLE Subjects:
- Patients who are pregnant.
- Patients with allergy or intolerance to sirolimus.
- Patients with life-threatening manifestations of SLE.
- Patients with proteinuria exceeding 500 mg/24 h or urine protein/creatine ratio >0.5.
- Patients with total cholesterol > 300 mg/dl or triglyceride > 400 mg.dl will be excluded.
- Patients with acute infection requiring antibiotics.
- Patients on sirolimus who develop infections and require intravenous antibiotics and fail to show clinical improvement in 5 days.
- Patients concurrently undergoing B cell-depleting therapy, cyclophosphamide, cyclosporine, and tacrolimus.
- Patients who have received investigational biologic B-cell depleting products within one year of study initiation.
- Patients with a history of chronic viral infections (e.g., HIV, hepatitis B, hepatitis C) or with a history of a malignancy (except non-melanoma skin cancer).
- Due to interference with sirolimus metabolism, subjects will not be allowed to receive concomitant rifampin, ketoconazole,voriconazole, itraconazole, erythromycin, or clarithromycin during the study.
- Patients with any type of interstitial lung disease.
For Healthy control Subjects:
- Subjects who are pregnant.
- Subjects with any acute or chronic illness.
Data sourced from ClinicalTrials.gov (NCT00779194). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.