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Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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):

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)

  Participant Flow

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 itself can suppress hematopoiesis and is an immuno-modulator.

All three patients screened were enrolled in the study.


Reporting Groups
  Description
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
    Sirolimus
STARTED   3 
COMPLETED   0 
NOT COMPLETED   3 
Physician Decision                3 



  Baseline Characteristics

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

Baseline Measures
   Sirolimus 
Overall Participants Analyzed 
[Units: Participants]
 3 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      0   0.0% 
Between 18 and 65 years      3 100.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Full Range)
 59 
 (57 to 60) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      3 100.0% 
Male      0   0.0% 
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: Study early termination by investigator - no participant completed any visits of the study - no measurements taken ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

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

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
e-mail: sgrando@uci.edu



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