Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus

This study has been terminated.
(Lack of funding)
Information provided by (Responsible Party):
Sergei Grando, University of California, Irvine Identifier:
First received: March 10, 2011
Last updated: February 24, 2016
Last verified: January 2016
Results First Received: December 17, 2014  
Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Pemphigus
Intervention: Drug: Sirolimus (formerly known as Rapamycin)

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
three patients were screened and enrolled in the study.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment

Only major consideration for the pre-assignment is to exclude any patient with underlying immunodeficiency or hematological disorder. Investigational product is itself can suppress hematopoiesis and is a immuno-modulator.

All three patients screened were enrolled in the study.

Reporting Groups
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.

Participant Flow:   Overall Study
Physician Decision                3 

  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
Sirolimus All patient will be open-label; Sirolimus. Dosage is variable based on FDA guidelines.

Baseline Measures
Overall Participants 
[units: participants]
[units: participants]
<=18 years   0 
Between 18 and 65 years   3 
>=65 years   0 
[units: years]
Mean (Full Range)
 (57 to 60) 
[units: participants]
Female   3 
Male   0 
Region of Enrollment 
[units: participants]
United States   3 

  Outcome Measures

1.  Primary:   Improvement of ABSIS Score While Reducing Steroid Dosage   [ Time Frame: Expected time line 24 months ]

2.  Secondary:   Statistical Measures   [ Time Frame: Intended assessment at 24 months ]
Results not yet reported.   Anticipated Reporting Date:   12/2016   Safety Issue:   No

  Serious Adverse Events

  Other Adverse Events

  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No results as study has been terminated early by the investigator.

  More Information
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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 Identifier: NCT01313923     History of Changes
Other Study ID Numbers: 2010-7844
Study First Received: March 10, 2011
Results First Received: December 17, 2014
Last Updated: February 24, 2016
Health Authority: United States: Institutional Review Board