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Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus

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

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Pemphigus
Intervention Drug: Sirolimus (formerly known as Rapamycin)
Enrollment 3
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
Hide 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
Arm/Group Title Sirolimus
Hide Arm/Group Description All patient will be open-label; Sirolimus. Dosage is variable based on FDA guidelines.
Overall Number of Baseline Participants 3
Hide 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
1.Primary Outcome
Title Improvement of ABSIS Score While Reducing Steroid Dosage
Hide 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
Show Outcome Measure DataHide Outcome Measure Data
Hide 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)
Hide 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
Hide 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
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)
Hide 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   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Sirolimus (Formerly Known as Rapamycin)
Affected / at Risk (%)
Total   0/0 
Show Other (Not Including Serious) Adverse Events Hide 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 
No results as study has been terminated early by the investigator.
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
Responsible Party: Sergei Grando, University of California, Irvine
ClinicalTrials.gov Identifier: NCT01313923     History of Changes
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