A Phase 2 interventional study of Rapamycin in Systemic Lupus Erythematosus (SLE), sponsored by State University of New York - Upstate Medical University. Completed at 1 site in United States. Open to participants aged 18 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2024-06-12.
Sponsored by State University of New York - Upstate Medical University · Phase 2, Interventional, and Treatment
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.
43 SLE subjects and 56 healthy controls are being recruited. The study will last 1 year with 9 study visits from day 0 to day 360. The healthy controls only need to donate blood once.
The study drug, sirolimus, is taken by mouth at a starting dose of 2mg/day. The dose is adjusted to achieve blood levels in the range of 6-15 ng/ml (the levels found to be effective for preventing organ rejections).
Blood samples are obtained before taking sirolimus, every two weeks for the first month, then every three months until 1 year, and then three months later to check the effect of discontinuing rapamycin. Each SLE subject will be asked to provide up to 100 ml (20 teaspoons) of blood at each visit. The first 6 visits will take place within 3 months and the remaining 3 visits every 3 months.
Routine laboratory work will be performed. Part of the blood drawn will be used for research and part will be used for routine lab work as part of standard of care.
The non-routine laboratory studies include:
The study drug levels will be checked at every visit. The non-routine laboratory studies will be performed at Visits 0 and 8 for SLE subjects and at Visit 0 for the healthy control subjects.
Healthy control subjects will be matched by age (a decade or less), gender, and ethnic origin. They will be recruited and analyzed only for immunobiological outcomes on the same day as lupus subjects.
All subjects will sign an informed consent at visit 0. There is a separate informed consent for the main study, one for the SLE subjects and one for the Healthy Controls. The same subjects can participate in the genetic sub-study. They must sign another informed consent for the genetic sub-study, one for the SLE subjects and one for the Healthy Controls. There is no need for additional blood drawing since part of the blood drawn for the main study can be used for the genetic sub-study.
1,202 studies on the registry are indexed under Lupus Erythematosus, Systemic; 399 are open to participants now.
This study's enrollment of 99 is above the median of 50 across 867 interventional studies indexed under Lupus Erythematosus, Systemic.
Browse Lupus Erythematosus, Systemic studies →State University of New York - Upstate Medical University is the lead sponsor of 154 studies on the registry; 17 are open to participants now.
Of its 11 completed or terminated interventional studies of FDA-regulated products, 4 (36%) have results posted.
Counted across the registry records on this site, refreshed daily.
For SLE Subjects:
For Healthy Control Subjects:
Exclusion Criteria:
For SLE Subjects:
For Healthy control Subjects:
SLE subjects receiving the study drug, Rapamune.
Drug: Rapamycin
Rapamycin, is given to this group at a starting dose of 2 mg/day.
Also known as: Rapamune, Sirolimus
Reduction of the Disease Activity as Measured by SLEDAI and BILAG Scores.
The Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) and The British Isles Lupus Assessment Group (BILAG) are clinical tools for assessing disease activity in lupus erythematosus patients. These indices play a central role as core determinants in SLE Responder Index. SLEDAI, comprising 24 items, quantifies disease activity on a scale of 0 to 105. Higher scores indicate more severe disease activity, reflecting cumulative impact of clinical and laboratory variables. BILAG, a comprehensive assessment, encompasses 97 items organized into 9 organ domains. The scoring ranges from A to E: A: No activity B: Mild activity C: Moderate activity D: Severe activity E: Very severe activity Total BILAG score is sum of individual item scores across all domains, with a potential range from 0 (if all items are graded as A, denoting no activity) to 97 (if all items are graded as E, signifying very severe activity). A lower total BILAG score indicates less severe disease activity.
Time frame: 1 year
Decrease of the Amount of Prednisone Needed to Control Disease Activity in SLE Patients.
Secondary endpoints were prednisone dose required to control disease activity. The average daily dosage of prednisone that was employed to control disease activity was diminished from 24.3±4.7 mg/day upon enrollment at visit 1 to 7.2±2.3 mg/day upon termination of the 12-month treatment period at visit 6.
Time frame: 1 year
| Milestone | SLE Subjects Receiving the Study Drug | Controls |
|---|---|---|
| Started | 43 | 56 |
| Completed | 29 | 56 |
| Not completed | 14 | 0 |
The Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) and The British Isles Lupus Assessment Group (BILAG) are clinical tools for assessing disease activity in lupus erythematosus patients. These indices play a central role as core determinants in SLE Responder Index. SLEDAI, comprising 24 items, quantifies disease activity on a scale of 0 to 105. Higher scores indicate more severe disease activity, reflecting cumulative impact of clinical and laboratory variables. BILAG, a comprehensive assessment, encompasses 97 items organized into 9 organ domains. The scoring ranges from A to E: A: No activity B: Mild activity C: Moderate activity D: Severe activity E: Very severe activity Total BILAG score is sum of individual item scores across all domains, with a potential range from 0 (if all items are graded as A, denoting no activity) to 97 (if all items are graded as E, signifying very severe activity). A lower total BILAG score indicates less severe disease activity.
| score on a scale | SLE Subjects Receiving the Study Drug |
|---|---|
| SLEDAI | 10.2 ± 5.6 |
| BILAG | 28.4 ± 12.4 |
Secondary endpoints were prednisone dose required to control disease activity. The average daily dosage of prednisone that was employed to control disease activity was diminished from 24.3±4.7 mg/day upon enrollment at visit 1 to 7.2±2.3 mg/day upon termination of the 12-month treatment period at visit 6.
| mg | SLE Subjects Receiving the Study Drug |
|---|---|
| Prednisone dose upon enrollment at visit 1 | 24.3 ± 4.7 |
| Prednisone dose upon termination of the 12-month treatment period at visit 6 | 7.2 ± 2.3 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| SLE Subjects Receiving the Study Drug | 0/40 (0%) | 0/40 (0%) | 1/40 (2.5%) |
| Controls | 0/56 (0%) | 0/56 (0%) | 0/56 (0%) |
| Event | SLE Subjects Receiving the Study Drug | Controls |
|---|---|---|
| Oral ulcerGastrointestinal disorders | 1/40 | — |
SLE patients and healthy controls (Blood samples of healthy individuals were obtained as controls only for immunobiological outcomes)
| Age, Categorical(Participants) | SLE Subjects Receiving the Study Drug | Controls | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 37 | 55 | 92 |
| >=65 years | 3 | 1 | 4 |
| Age, Continuous(years) | SLE Subjects Receiving the Study Drug | Controls | Total |
|---|---|---|---|
| Mean | 45 ± 14 | 45 ± 12 | 45 ± 13 |
| Sex: Female, Male(Participants) | SLE Subjects Receiving the Study Drug | Controls | Total |
|---|---|---|---|
| Female | 38 | 51 | 89 |
| Male | 2 | 5 | 7 |
| Region of Enrollment(participants) | SLE Subjects Receiving the Study Drug | Controls | Total |
|---|---|---|---|
| United States | 40 | 56 | 96 |
This study is completed, as verified in May 2024. You cannot join it, but the record below documents what was studied.
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Lupus Erythematosus, Systemic→
State University of New York - Upstate Medical University