SCLERAMAC: MPA AUC Monitoring in Patients Receiving MMF for Diffuse Cutaneous or Pulmonary Involvement in Systemic Sclerosis

Sponsor

Assistance Publique - Hôpitaux de Paris (Other)

Overall Status

Recruiting

CT.gov ID

NCT04244916

Collaborator

(none)

Enrollment

Location

Anticipated Duration (Months)

1.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

To define a target value of AUC MPA to improve the modified Rodnan score and / or respiratory impairment (DLCO or FVC) at one year in patients receiving MMF for the treatment of diffuse cutaneous or interstitial lung damage of systemic sclerosis.

Condition or Disease	Intervention/Treatment	Phase
Systemic Sclerosis	Biological: AUC of MPA measure

Detailed Description

In the treatment of autoimmune diseases, MMF is almost always prescribed at a fixed dose, regardless of AUC, or based on the target of AUC determined for organ transplantation. One study looked at determining an "effective" AUC threshold in systemic lupus erythematosus, which appears to be 35mg / h / l. This was also done for ANCA vasculitis.

We therefore conducted this study to determine a correlation between AUC MPA and the effectiveness of MMF in systemic sclerosis.

Prospective, observational, open study.

Main objective: define a target value of AUC MPA to improve the modified Rodnan score and / or respiratory impairment (DLCO or FVC) at one year in patients receiving MMF for the treatment of diffuse skin involvement or pulmonary function in systemic sclerosis.

The main endpoint will be evaluated on the evolution of the modified Rodnan score at 1 year after the initiation of MMF and / or the evolution of FVC and DLCO at 1 year after the initiation of MMF.

Study Design

Study Type:

Observational

Anticipated Enrollment :

50 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Prospective Study to Investigate the Relevance of Monitoring Area Under the Curve of Mycophenolic Acid in Patients Receiving Mycophenolate Mofetil to Treat a Diffuse Cutaneous or a Pulmonary Involvement of Systemic Sclerosis

Actual Study Start Date :

May 25, 2020

Anticipated Primary Completion Date :

May 25, 2023

Anticipated Study Completion Date :

Sep 25, 2023

Outcome Measures

Primary Outcome Measures

Skin efficacy [1 year]
Modified Rodnan skin score (mRSS) : min 0 max 51. A diminution in the mRSS of more than 25% from the initial value was considered as improved. On the contrary, an increase in the mRSS over 25% was considered as deteriorated. All other variations of mRSS were classified as stable. Minimal clinically important difference was also tested (worsening of mRSS ≥ 4.7).

Secondary Outcome Measures

Pulmonary efficacy.1 [1 year]
Modification in FVC
Pulmonary efficacy.2 [6 months and 1 year]
Modification in DLCO

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Systemic sclerosis meeting the ACR / EULAR criteria of 2013
Equal or more than 18 years old, able to freely consent to study
In patients treated for skin damage:
Diffuse skin sclerosis (rising above the elbows and / or knees)
First clinical sign of systemic sclerosis outside of Raynaud's phenomenon going back less than three years
Failure to take other concomitant immunosuppressive treatments or in the last 3 months except corticosteroids.
In patients treated for lung damage:
Interstitial lung damage identified on chest CT, chest x-ray
Any duration of progression of systemic scleroderma
Prescription of MMF in first line or in relay of a treatment with Cyclophosphamide.
Absence of biotherapy in the last 6 months.