Trial Outcomes & Findings for Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus (NCT NCT01313923)

Results Overview

Measurement of disease severity will be quantified using ABSIS (Autoimmune Bullous Skin Disorder Intensity Score). Improvement in disease control is quantified by the maintenance or improvement of ABSIS score while reducing steroid dosage. No results as study has been terminated early by the investigator.

Recruitment status

TERMINATED

Study phase

EARLY_PHASE1

Target enrollment

3 participants

Primary outcome timeframe

Expected time line 24 months

Results posted on

2017-06-09

Participant Flow

three patients were screened and enrolled in the study.

Only major consideration for the pre-assignment is to exclude any patient with underlying immunodeficiency or hematological disorder. Investigational product itself can suppress hematopoiesis and is an immuno-modulator. All three patients screened were enrolled in the study.

Participant milestones

Participant milestones
Measure	Sirolimus There is one arm to the study. All patients will be open-label, Sirolimus. There is no set dosage: medication dose will be based on FDA approved guidelines.
Overall Study STARTED	3
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Sirolimus There is one arm to the study. All patients will be open-label, Sirolimus. There is no set dosage: medication dose will be based on FDA approved guidelines.
Overall Study Physician Decision	3

Baseline Characteristics

Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sirolimus n=3 Participants All patient will be open-label; Sirolimus. Dosage is variable based on FDA guidelines.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	59 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Expected time line 24 months

Population: This outcome was not assessed because no participant completed any visits of the study

Measurement of disease severity will be quantified using ABSIS (Autoimmune Bullous Skin Disorder Intensity Score). Improvement in disease control is quantified by the maintenance or improvement of ABSIS score while reducing steroid dosage. No results as study has been terminated early by the investigator.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Study early termination by investigator - no participant completed any visits of the study - no measurements taken

The statistical goal is to observe "success," an improvement in disease control while up-titrating sirolimus dosage. As there will be no control group, the subject or progress at the end of the study will be compared to their baseline at the beginning of the study. The subject and disease severity at the beginning of the study will be compared to the disease severity at each visit and be correlated with the dosage of sirolimus and corticosteroid. However, since no patient completed the study, the outcome and any data collected was not assessed.

Outcome measures

Outcome data not reported

Adverse Events

Sirolimus (Formerly Known as Rapamycin)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Sergei A. Grando, MD, PhD, DSc

UC Irvine Dermatology Research Center

Phone: 9498242713

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place