Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus

• ClinicalTrials.gov Identifier: NCT01313923
• Recruitment Status: Terminated
• First Posted: March 14, 2011
• Results First Posted: March 25, 2016
• Last Update Posted: June 9, 2017
• Sponsor: University of California, Irvine
• Information provided by (Responsible Party): Sergei Grando, University of California, Irvine

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Pemphigus
• Intervention: Drug: Sirolimus (formerly known as Rapamycin)
• Enrollment: 3

Participant Flow

Recruitment Details	three patients were screened and enrolled in the study.
Pre-assignment Details	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.

Arm/Group Title	Sirolimus
Arm/Group Description	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.
Period Title: Overall Study
Started	3
Completed	0
Not Completed	3
Reason Not Completed
Physician Decision	3

Baseline Characteristics

Arm/Group Title		Sirolimus
Arm/Group Description		All patient will be open-label; Sirolimus. Dosage is variable based on FDA guidelines.
Overall Number of Baseline Participants		3
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	3 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	3 100.0%
	>=65 years	0 0.0%
Age, Continuous; Mean (Full Range); Unit of measure: Years
	Number Analyzed	3 participants
		59; (57 to 60)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	3 participants
	Female	3 100.0%
	Male	0 0.0%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
United States	Number Analyzed	3 participants
		3

Outcome Measures

1. Primary Outcome

Title	Improvement of ABSIS Score While Reducing Steroid Dosage
Description	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.
Time Frame	Expected time line 24 months

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Sirolimus (Formerly Known as Rapamycin)
Arm/Group Description:	No results. Study has been terminated by investigator.
Overall Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

2. Secondary Outcome

Title	Statistical Measures
Description	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.
Time Frame	Study early termination by investigator - no participant completed any visits of the study - no measurements taken

Outcome Measure Data Not Reported

Adverse Events

Time Frame	Serious and Other (Not Including Serious) Adverse Events were not collected/assessed
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Sirolimus (Formerly Known as Rapamycin)
Arm/Group Description	No results as study has been terminated early by the investigator.
All-Cause Mortality
	Sirolimus (Formerly Known as Rapamycin)
	Affected / at Risk (%)
Total	--/--
Serious Adverse Events
	Sirolimus (Formerly Known as Rapamycin)
	Affected / at Risk (%)
Total	0/0
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Sirolimus (Formerly Known as Rapamycin)
	Affected / at Risk (%)
Total	0/0

Limitations and Caveats

No results as study has been terminated early by the investigator.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Sergei A. Grando, MD, PhD, DSc
• Organization: UC Irvine Dermatology Research Center
• Phone: 9498242713
• EMail: sgrando@uci.edu

• Responsible Party: Sergei Grando, University of California, Irvine
• ClinicalTrials.gov Identifier: NCT01313923
• Other Study ID Numbers: 2010-7844
• First Submitted: March 10, 2011
• First Posted: March 14, 2011
• Results First Submitted: December 17, 2014
• Results First Posted: March 25, 2016
• Last Update Posted: June 9, 2017