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

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

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.

Study Overview

• Status: Terminated
• Conditions: Systemic Sclerosis
• Intervention / Treatment: 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 Type: Observational
• Enrollment (Actual): 2

Contacts and Locations

Study Locations

• France
  • France
    • Paris, France, France, 75014
      • Cochin Hospital, AP-HP

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients receiving MMF for another indication than skin or lung damage to systemic sclerosis (after lung transplantation for example).

• Contraindication to MMF: Hypersensitivity to the product. Pregnancy.

• In patients treated for skin damage:

  • Failure to take other concomitant immunosuppressive treatments or in the last 3 months except corticosteroid therapy.
  • No treatment with biotherapy in the last 6 months

• In patients treated for lung damage:

  • No treatment with biotherapy in the last 6 months

Description

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 Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Skin efficacy	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).	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pulmonary efficacy.1	Modification in FVC	1 year
Pulmonary efficacy.2	Modification in DLCO	6 months and 1 year

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborators: URC Necker Cochin, France

Publications and helpful links

General Publications

• van Gelder T, Le Meur Y, Shaw LM, Oellerich M, DeNofrio D, Holt C, Holt DW, Kaplan B, Kuypers D, Meiser B, Toenshoff B, Mamelok RD. Therapeutic drug monitoring of mycophenolate mofetil in transplantation. Ther Drug Monit. 2006 Apr;28(2):145-54. doi: 10.1097/01.ftd.0000199358.80013.bd.
• Zahr N, Arnaud L, Marquet P, Haroche J, Costedoat-Chalumeau N, Hulot JS, Funck-Brentano C, Piette JC, Amoura Z. Mycophenolic acid area under the curve correlates with disease activity in lupus patients treated with mycophenolate mofetil. Arthritis Rheum. 2010 Jul;62(7):2047-54. doi: 10.1002/art.27495.
• Chaigne B, Gatault P, Darrouzain F, Barbet C, Degenne D, Francois M, Szymanski P, Rabot N, Golea G, Diot E, Maillot F, Lebranchu Y, Nivet H, Paintaud G, Halimi JM, Guillevin L, Buchler M. Mycophenolate mofetil in patients with anti-neutrophil cytoplasmic antibody-associated vasculitis: a prospective pharmacokinetics and clinical study. Clin Exp Immunol. 2014 May;176(2):172-9. doi: 10.1111/cei.12246.

Study record dates

Study Major Dates

• Study Start (Actual): May 25, 2020
• Primary Completion (Actual): August 4, 2021
• Study Completion (Actual): August 4, 2021

Study Registration Dates

• First Submitted: January 6, 2020
• First Submitted That Met QC Criteria: January 27, 2020
• First Posted (Actual): January 28, 2020

Study Record Updates

• Last Update Posted (Estimated): September 12, 2025
• Last Update Submitted That Met QC Criteria: September 5, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: interstitial lung disease; mycophenolate mofetil; therapeutic drug monitoring (TDM); Systemic sclerosis; diffuse
• Additional Relevant MeSH Terms: Connective Tissue Diseases; Respiratory Tract Diseases; Lung Diseases; Skin Diseases; Skin and Connective Tissue Diseases; Lung Diseases, Interstitial; Scleroderma, Systemic
• Other Study ID Numbers: APHP190933

Plan for Individual participant data (IPD)

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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No