Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01313923
• Date of registration: 10/03/2011
• Prospective Registration: No
• Primary sponsor: University of California, Irvine
• Public title: Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus
• Scientific title: Evaluation of Sirolimus for the Treatment of the Autoimmune Blistering Dermatosis Pemphigus
• Date of first enrolment: February 2011
• Target sample size: 3
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01313923
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Early Phase 1

Countries of recruitment

United States

Contacts

• Name: Sergei Grando, MD, PhD
• Affiliation: University of California, Irvine

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Subject must be 18 years of age or older.

2. Subject must have an established diagnosis of pemphigus disorder via biopsy and/or
serologic titer, as determined appropriate by the lead researcher.

3. Subject must have active disease at the time of enrollment, as defined by a positive
Nikolsky sign.

4. Subject must not be taking any immunosuppressive medication or therapy other than
corticosteroids.

5. Subject must be able to understand and follow directions.

6. If female, subject is not currently breast feeding and/or pregnant as confirmed via
negative pregnancy test, no potential for pregnancy, or if of child-bearing age,
agrees to using birth control for entire duration of study and 12 weeks after end of
study.

Exclusion Criteria:

1. Subject may not be under 18 years old.

2. Subject cannot understand or follow directions.

3. Subject may not have any condition that could, in the opinion of the investigator,
compromise the subject's ability to give written consent and/or comply with the study
procedures, such as a history of substance abuse or a psychiatric condition.

4. If female and of child bearing age, is pregnant or unwilling to use birth control
during the study period.

5. Subject may not have any of the following laboratory abnormalities at baseline:

- total white blood cell count < 2,000/mm3 or platelet count < 100,000/mm3

- creatinine >1.5mg/dL

- urine analysis protein of 2+ or greater

- fasting triglycerides > 400 mg/dL, fasting total cholesterol > 300 mg/dL, or
fasting LDLcholesterol > 160 mg/dL

- transaminases > 2 times the upper limit of normal

6. Subjects may not be using any of the following medications: systemic antifungals,
antiepileptics, HIV protease inhibitors, cimetidine, cisapride, clarithromycin,
danazol, diltiazem, erythromycin, metoclopramide, rifabutin, rifampin, rifapentine,
troleandomycin, or verapamil

7. Subject may not consume grapefruit juice and/or St. John's Wort (hypericum perforatum)
throughout the duration of the study.

8. Subject may not have other significant concurrent medical conditions, including

- Active malignancy, including evidence of cutaneous basal or squamous cell
carcinoma or melanoma, or history of cancer (other than fully resected and
surgically cured cutaneous basal cell and squamous cell carcinoma) within 5 years
before the first 13 of 25 sirolimus dose. If malignancy occurred more than 5
years ago, documentation of disease-free state since treatment is required.

- Known immunodeficiency syndromes, including HIV

- Renal failure or insufficiency, as defined by laboratory parameters above

- Significant proteinuria, as defined by laboratory parameters above

- History of high cholesterol, lipids, or liver disease, as defined by laboratory
parameters above

- Uncontrolled hypertension, as defined by a blood pressure > 140/90 despite
optimal medical therapy, as prescribed by primary care doctor

- Any condition that, in the opinion of the investigator, might cause this study to
be detrimental to the subject

- Any active Common Terminology Criteria (CTC) grade 2 (localized infection;
requiring local intervention) or higher infection (including chronic or localized
infections) within 30 days prior to screening, at screening, or during screening
period prior to first dose of sirolimus



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pemphigus

Intervention(s)

Drug: Sirolimus (formerly known as Rapamycin)

Primary Outcome(s)

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

Secondary Outcome(s)

Statistical Measures [Time Frame: Study early termination by investigator - no participant completed any visits of the study - no measurements taken]

Secondary ID(s)

2010-7844

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 25/03/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01313923