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

• Conditions: Systemic Sclerosis

• Registration Number: NCT04244916
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

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.

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 Timeline

• Study First Submitted: 2020-01-06
• Study First Submitted QC: 2020-01-27
• Study First Posted: 2020-01-28
• Study Start: 2020-05-25
• Status Verified: 2021-11
• Last Update Submitted: 2021-11-19
• Last Update Posted: 2021-11-22
• Primary Completion: 2023-05-25
• Study Completion: 2023-09-25

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 50

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.

Exclusion Criteria

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Pulmonary efficacy.1	1 year	Modification in FVC
Pulmonary efficacy.2	6 months and 1 year	Modification in DLCO

Trial Locations

Locations (1)

Cochin hospital, AP-HP; 🇫🇷 Paris, France