Autoimmune Bullous DermatosePhase 3PEMPANCT02993133
Pharmacokinetics Study of Mycophenolic Acid in Patients With an Autoimmune Bullous Dermatose, Pemphigus or Cicatricial Pemphigoid.
A Phase 3 study testing Cellcept® in autoimmune bullous dermatoses in people with Autoimmune Bullous Dermatose, run by University Hospital, Limoges at 3 sites in France.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Bullous Dermatose
- What is being tested?
- Cellcept® in autoimmune bullous dermatoses
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 53 participants
- Where
- 3 sitesFrance
- Study dates
- Dec 2016to Apr 2019
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country.
France3 sites
CHU de Bordeaux
Bordeaux
Hôpital Avicenne - AP-HP
Paris
CHU de Rouen
Rouen
About this study
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.
- Conditions
- Autoimmune Bullous Dermatose
- Being tested
- Cellcept® in autoimmune bullous dermatoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cellcept® in autoimmune bullous dermatosesDrug
The administration will follow the recommendations for the use of Cellcept® in autoimmune bullous dermatoses
| Study group | Role | What they receive |
|---|---|---|
| 60 patients will be used to build and validate the system | Experimental | 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. Receives: Drug: Cellcept® in autoimmune bullous dermatoses |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 8 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2016 (estimated)
- Study startDec 2016
- Primary completionJan 2019
- Study completionApr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune Bullous DermatoseStudies of the same condition, open and under way.
- More trials run by University Hospital, LimogesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 53 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Limoges
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02993133
- Sponsor's protocol ID
- I14027 / PEMPA
- Acronym
- PEMPA
- Status last verified
- Jun 2018
- First submitted
- 12 Dec 2016
- Last updated
- 22 Apr 2019
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacokinetics Study of Mycophenolic Acid in Patients With an Autoimmune Bullous Dermatose, Pemphigus or Cicatricial Pemphigoid.
- ClinicalTrials.gov ID
- NCT02993133
- Acronym / sponsor ID
- PEMPA / I14027 / PEMPA
- Conditions
- Autoimmune Bullous Dermatose
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 53 participants (actual)
- Lead sponsor
- University Hospital, LimogesUniversity, hospital or other
- Study start
- Dec 2016 (actual)
- Primary completion
- Jan 2019 (actual)
- Study completion
- Apr 2019 (actual)
- First posted
- 15 Dec 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 60 patients will be used to build and validate the system | Experimental | Drug: Cellcept® in autoimmune bullous dermatosesThe 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cellcept® in autoimmune bullous dermatosesThe administration will follow the recommendations for the use of Cellcept® in autoimmune bullous dermatoses | Drug | 60 patients will be used to build and validate the system |
Primary outcome
Evaluation of the Bayesian estimator performance
Time frame: 8 hours
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2019.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Cellcept® in autoimmune bullous dermatoses in people with Autoimmune Bullous Dermatose, run by University Hospital, Limoges at 3 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of the Bayesian estimator performance (time frame: 8 hours). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2019.