Pharmacokinetics Study of Mycophenolic Acid in Patients With an Autoimmune Bullous Dermatose, Pemphigus or Cicatricial Pemphigoid. (PEMPA)

The main autoimmune bullous dermatoses are pemphigus and cicatricial pemphigoid. Pemphigus is an autoimmune dermatological disease characterized by the production of anti-desmoclesin antibodies 1 and 3, affecting the skin and mucous membranes.The cicatricial pemphigoid is an autoimmune dermatological disease, characterized by the production of anti-zone antibodies of the basal membrane and characterized by a predominant mucosal involvement. Mycophenolic acid (MPA) is an increasingly used form of corticosteroid. Despite its increasing use, pharmacokinetics in autoimmune bullous dermatosis remain little studied.

Study Overview

• Status: Completed
• Conditions: Autoimmune Bullous Dermatose
• Intervention / Treatment: Drug: Cellcept® in autoimmune bullous dermatoses

• Study Type: Interventional
• Enrollment (Actual): 53
• Phase: Phase 3

Contacts and Locations

Study Locations

• France
  • Bordeaux, France
    • CHU de Bordeaux
  • Paris, France
    • Hôpital Avicenne - AP-HP
  • Rouen, France
    • CHU de Rouen

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Age > or = 18 years.
• Patient treated by Mycophénolate Mofétil (MMF) per os (Cellcept®) for an autoimmune bullous dermatose (pemphigus or cicatricial pemphigoid) for at least 30 days according to the recommendations (PNDS Pemphigus and cicatricial pemphigoid HAS 2011)
• Patient able to understand the nature, purpose and methodology of the study
• Patient affiliated to the French social security system or equivalent
• Patient who have signed an informed consent form

Exclusion Criteria:

• Pregnant or breast-feeding women or women of childbearing potential without efficient contraception (based on a declaration)
• Patient under legal protection.
• Patient deprived of freedom
• Patient with any altered mental status or any psychiatric condition that would interfere with the understanding of the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 60 patients will be used to build and validate the system; The first 30 patients will be used to build and validate the pharmacokinetic modeling of MPA in pemphigus.The 30 patients included later will provide additional data.	Drug: Cellcept® in autoimmune bullous dermatoses; The administration will follow the recommendations for the use of Cellcept® in autoimmune bullous dermatoses

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of the Bayesian estimator performance	The evaluation of the Bayesian estimator performance will be based on its capacity to predict MPA AUC (Area Under the Curve), expressed as the bias (%) and the precision (root mean square error; RMSE) between the predicted AUC calculated using a limited number of samples performed in the first 4 hours post dosing and the observed AUC estimated using the reference method (trapezoidal rule method). A Bland Altman curve will be constructed.	8 hours

Collaborators and Investigators

• Sponsor: University Hospital, Limoges

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2016
• Primary Completion (Actual): January 1, 2019
• Study Completion (Actual): April 1, 2019

Study Registration Dates

• First Submitted: December 12, 2016
• First Submitted That Met QC Criteria: December 12, 2016
• First Posted (Estimate): December 15, 2016

Study Record Updates

• Last Update Posted (Actual): April 22, 2019
• Last Update Submitted That Met QC Criteria: April 19, 2019
• Last Verified: June 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Immune System Diseases; Autoimmune Diseases; Eye Diseases; Skin Diseases, Vesiculobullous; Conjunctival Diseases; Pemphigoid, Bullous; Pemphigus; Pemphigoid, Benign Mucous Membrane; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Enzyme Inhibitors; Antineoplastic Agents; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antitubercular Agents; Antibiotics, Antitubercular; Mycophenolic Acid
• Other Study ID Numbers: I14027 / PEMPA

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No