Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus

This study has been terminated.

(Lack of funding)

Sponsor:

Information provided by (Responsible Party):

Sergei Grando, University of California, Irvine

ClinicalTrials.gov Identifier:

NCT01313923

First received: March 10, 2011

Last updated: February 24, 2016

Last verified: January 2016

History of Changes

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.

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

	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

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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.
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Reporting Groups

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

Baseline Measures

	Sirolimus
Number of Participants [units: participants]	3
Age [units: participants]
<=18 years	0
Between 18 and 65 years	3
>=65 years	0
Age [units: years] Mean (Full Range)	59 (57 to 60)
Gender [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 ]

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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

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Other Adverse Events

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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
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
Study First Received:	March 10, 2011
Results First Received:	December 17, 2014
Last Updated:	February 24, 2016
Health Authority:	United States: Institutional Review Board

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