Systemic SclerosisSCLERAMACNCT04244916
MPA AUC Monitoring in Patients Receiving MMF for Diffuse Cutaneous or Pulmonary Involvement in Systemic Sclerosis
An observational study of Systemic Sclerosis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Sclerosis
- What is being tested?
- AUC of MPA measure
- How many people
- 2 participants
- Where
- 1 siteFrance
- Study dates
- 25 May 2020to 4 Aug 2021
- Phase
- Observational
- 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
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
Who may be able to join
- 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.
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- 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.
Who cannot join
- -
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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.
- Conditions
- Systemic Sclerosis
- Being tested
- AUC of MPA measure
- Keywords
- Systemic sclerosis, diffuse, interstitial lung disease, therapeutic drug monitoring (TDM), mycophenolate mofetil
- Medical subject headings
- Scleroderma, Systemic, Lung Diseases, Interstitial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AUC of MPA measureBiological
Plasmatic AUC determination of MPA requires 3 blood punctures at H0, H30 and H2.
These punctures will be made at inclusion after 6 weeks, 12 weeks, 6 months and one year.
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 1 year
Secondary outcomes (2)
Pulmonary efficacy.1
Modification in FVC
Measured: 1 year
Pulmonary efficacy.2
Modification in DLCO
Measured: 6 months and 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2020
- Study start25 May 2020
- Primary completion4 Aug 2021
- Study completion4 Aug 2021
Why it stopped: Departure of the coordinator
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04244916
- Sponsor's protocol ID
- APHP190933
- Acronym
- SCLERAMAC
- Status last verified
- Sep 2025
- First submitted
- 6 Jan 2020
- Last updated
- 12 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 16628123Zahr 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.
PubMed 20506558Chaigne 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.
PubMed 24304103
Study at a glance
From the registry record, last updated by the sponsor on 12 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04244916
- Acronym / sponsor ID
- SCLERAMAC / APHP190933
- Conditions
- Systemic Sclerosis
- Keywords
- Systemic sclerosis, diffuse, interstitial lung disease, therapeutic drug monitoring (TDM), mycophenolate mofetil
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Departure of the coordinator
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- 25 May 2020 (actual)
- Primary completion
- 4 Aug 2021 (actual)
- Study completion
- 4 Aug 2021 (actual)
- First posted
- 28 Jan 2020 (actual)
- 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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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
Key inclusion criteria (11 listed)
- 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:
- In patients treated for lung damage:
Key exclusion criteria (1 listed)
- -
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 |
|---|---|---|
| AUC of MPA measurePlasmatic AUC determination of MPA requires 3 blood punctures at H0, H30 and H2. These punctures will be made at inclusion after 6 weeks, 12 weeks, 6 months and one year. | Biological | – |
Primary outcome
Skin efficacy
Time frame: 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).
2 secondary outcomes
Pulmonary efficacy.1
Time frame: 1 year
Pulmonary efficacy.2
Time frame: 6 months and 1 year
Results and safety
The study was stopped early (reason given: Departure of the coordinator). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Sep 2025.
- Background: PubMed 16628123van 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.
- Background: PubMed 20506558Zahr 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.
- Background: PubMed 24304103Chaigne 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.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Systemic Sclerosis, run by Assistance Publique - Hôpitaux de Paris at 1 site 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 11 inclusion and 1 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: stopped early, as of the sponsor's last update on 12 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
Are results available for this study?
The study was stopped early (reason given: Departure of the coordinator). Results are not available in the source record.
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?
Skin efficacy (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04244916, which also lists 3 linked publications.
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 12 Sep 2025.