Trial Outcomes & Findings for Prospective Study of Rapamycin for the Treatment of SLE (NCT NCT00779194)

NCT ID: NCT00779194

Last Updated: 2024-06-12

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 99 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2024-06-12

Participant Flow

Participant milestones

Measure	SLE Subjects Receiving the Study Drug SLE subjects receiving the study drug, Rapamune. Rapamycin: Rapamycin, is given to this group at a starting dose of 2 mg/day.	Controls Healthy control group donating blood only for immunobiological outcomes.
Overall Study STARTED	43	56
Overall Study COMPLETED	29	56
Overall Study NOT COMPLETED	14	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Prospective Study of Rapamycin for the Treatment of SLE

Baseline characteristics by cohort

Measure	SLE Subjects Receiving the Study Drug n=40 Participants SLE Subjects Receiving the Study Drug, Rapamune. Rapamycin: Rapamycin, is given to this group at a starting dose of 2mg/day.	Controls n=56 Participants Blood samples of healthy individuals were obtained as controls only for immunobiological outcomes.	Total n=96 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	37 Participants n=5 Participants	55 Participants n=7 Participants	92 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants
Age, Continuous	45 years STANDARD_DEVIATION 14 • n=5 Participants	45 years STANDARD_DEVIATION 12 • n=7 Participants	45 years STANDARD_DEVIATION 13 • n=5 Participants
Sex: Female, Male Female	38 Participants n=5 Participants	51 Participants n=7 Participants	89 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	5 Participants n=7 Participants	7 Participants n=5 Participants
Region of Enrollment United States	40 participants n=5 Participants	56 participants n=7 Participants	96 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Population: SLE patients

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.

Outcome measures

Measure	SLE Subjects Receiving the Study Drug n=40 Participants SLE subjects receiving the study drug Rapamune. Rapamycin: Rapamycin, is given to this group at a starting dose of 2 mg/day.
Reduction of the Disease Activity as Measured by SLEDAI and BILAG Scores. SLEDAI	10.2 score on a scale Standard Deviation 5.6
Reduction of the Disease Activity as Measured by SLEDAI and BILAG Scores. BILAG	28.4 score on a scale Standard Deviation 12.4

SECONDARY outcome

Timeframe: 1 year

Population: 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.

Outcome measures

Measure	SLE Subjects Receiving the Study Drug n=40 Participants SLE subjects receiving the study drug Rapamune. Rapamycin: Rapamycin, is given to this group at a starting dose of 2 mg/day.
Decrease of the Amount of Prednisone Needed to Control Disease Activity in SLE Patients. Prednisone dose upon enrollment at visit 1	24.3 mg Standard Deviation 4.7
Decrease of the Amount of Prednisone Needed to Control Disease Activity in SLE Patients. Prednisone dose upon termination of the 12-month treatment period at visit 6	7.2 mg Standard Deviation 2.3

Adverse Events

SLE Subjects Receiving the Study Drug

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Controls

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	SLE Subjects Receiving the Study Drug n=40 participants at risk SLE Subjects receiving the study drug, Rapamune. Rapamycin: Rapamycin, is given to this group at a starting dose of 2 g/day	Controls n=56 participants at risk Healthy control group donating blood only for immunobiological outcomes.
Gastrointestinal disorders Oral ulcer	2.5% 1/40	0.00% 0/56

Additional Information

Andras Perl

STATE UNIVERSITY OF NEW YORK

Phone: 3154644194

Email: perla@upstate.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place