Scleroderma, SystemicNCT00333437
Pulmonary Involvement in Scleroderma: A Clinical Study of the Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients With Lung Involvement
A study testing Mycophenolate mofetil in people with Scleroderma, Systemic, run by University of California, San Francisco at 1 site in United States.
Trial summary
Researchers from the Division of Pulmonary and Critical Care Medicine at University of California, San Francisco (UCSF) are conducting a study to evaluate whether mycophenolate mofetil (an immunosuppressive medication, trade named CellCept) is safe and effective for preventing the lung damage from scleroderma from… Read more about this study
- Who is it for?
- Ages 21 to 70 years
- Conditions the study is for
- Scleroderma, Systemic
- What is being tested?
- Mycophenolate mofetil
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- May 2006to Jan 2009
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 21 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- To participate in this study, patients must first undergo a BAL and HRCT. To be eligible to undergo HRCT and BAL (under the purview of this trial), prospective patients must meet the following criteria:
- Aged 21-70.
- Negative pregnancy test (with a sensitivity of at least 50 mIU/mL) for females of child-bearing potential
- All patients must fulfill the criteria for SSc by American College of Rheumatology (ACR) criteria (Subcommittee for Scleroderma Criteria 1980).
- FVC < 85% of predicted.
- SSc for no more than 7 years with onset defined as the date of the first non-Raynaud manifestation.
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- Patients may have limited (cutaneous thickening distal but not proximal to elbows and knees, with or without facial involvement) or diffuse (cutaneous thickening proximal to elbows and knees, often involving the chest or abdomen) cutaneous SSc (Medsger 1995).
- Abnormal DLCO and abnormalities on the plain chest radiograph are not required, although a normal DLCO would be unusual in the face of significant ventilatory restriction due to SSc lung disease.
- To be eligible to take study medication, the patient must meet not only the criteria above, but also must have ≥ 3.0% neutrophils or ≥ 2.0% eosinophils in screening BAL fluid and/or ground glass opacification on HRCT.
- Women of childbearing potential should have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL within 1 week before beginning therapy. CellCept therapy will not be initiated until a report of a negative pregnancy test has been obtained.
- Effective contraception must be used before beginning CellCept therapy, during therapy, and for 6 weeks following discontinuation of therapy, even where there has been a history of infertility, unless due to hysterectomy. Two reliable forms of contraception must be used simultaneously unless abstinence is the chosen method. If pregnancy does occur during treatment, the physician and patient should discuss the desirability of continuing the pregnancy.
Who cannot join
- FVC < 45% of predicted or DLCO (corrected for hemoglobin [Hgb] but not for alveolar volume) < 35% of predicted (suggestive of severe, probably irreparable, disease).
- Leukopenia (white blood cell count < 4000) or thrombocytopenia (platelet count < 100,000).
- Serum creatinine ≥ 2.0 mg/dl.
- Pregnancy, breast feeding, unreliability, drug abuse, or chronic debilitating disease.
- Uncontrolled congestive heart failure.
- Active infection of the lung, or elsewhere, whose management would be compromised by mycophenolate mofetil.
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- Prior treatment for alveolitis with mycophenolate mofetil or prior or current treatment for alveolitis with: D-penicillamine, methotrexate, colchicine, Potaba, or azathioprine.
- Other serious concomitant medical illness (e.g., cancer).
- Forced expiratory volume in 1 second (FEV1)/FVC ratio < 65%.
- If of childbearing potential, failure regularly to be employing two reliable means of contraception (i.e., condom, abstinence, intrauterine device (IUD), tubal ligation, vasectomy)
- Pulmonary hypertension (defined as an estimated systolic blood pressure (SBP) ≥ 35 mmHg measured by echocardiogram).
- Smoking of cigars, pipes, or cigarettes during the past 6 months.
- Clinically significant abnormalities on chest x-ray or HRCT scan other than interstitial lung disease (e.g., lung mass, evidence of active pulmonary infection).
- Use of prednisone (or equivalent) in doses > 10 mg per day.
- Does not have ≥ 3.0% neutrophils or ≥ 2.0% eosinophils on screening BAL fluid and does not have ground glass opacification on HRCT.
- Unable to take oral medication.
- Not able to comply with study procedures in the opinion of the investigator.
Trial locations and contacts
1 site in 1 country.
United States1 site
UCSF, 400 Parnassus Ave
San Francisco, California, 94143
About this study
Official title: Pulmonary Involvement in Scleroderma: Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients
Researchers from the Division of Pulmonary and Critical Care Medicine at University of California, San Francisco (UCSF) are conducting a study to evaluate whether mycophenolate mofetil (an immunosuppressive medication, trade named CellCept) is safe and effective for preventing the lung damage from scleroderma from getting worse.
Read the detailed description
The proposed study is designed to evaluate the safety and efficacy of mycophenolate mofetil (CellCept) for the treatment of symptomatic pulmonary alveolitis due to systemic sclerosis (SSc). This study utilizes a prospective, open-label, experimental design.
Primary Hypothesis: The alveolitis in patients with SSc, as defined by decreased forced vital capacity (FVC), bronchoalveolar lavage (BAL), and High Resolution Chest Tomography (HRCT) is responsive to 1 year of daily mycophenolate mofetil therapy.
Secondary Hypothesis: Quality of life, six-minute walk and single-breath diffusing capacity for carbon monoxide (DLCO) improve in patients with SSc mediated alveolitis after therapy with mycophenolate mofetil. This response to therapy is associated with a change in the inflammatory cytokine profile present in BAL fluid.
- Conditions
- Scleroderma, Systemic
- Being tested
- Mycophenolate mofetil
- Keywords
- Scleroderma, Systemic
- Medical subject headings
- Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Mycophenolate Mofetil Receives: Drug: Mycophenolate mofetil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Change From Baseline in Forced Vital Capacity (FVC)
compare pre- and post-therapy FVC (post- minus pre-). Forced vital capacity (FVC) is the volume of air (liters) that can forcibly be blown out after full inspiration.
Measured: Baseline, 12 months
Secondary outcomes (4)
Mean Change in Bronchoalveolar Lavage (BAL) Components (Neutrophils, Eosinophils)
BAL samples were colleected from the affected lobe (as determined by lung CT scans) before beginning and after completing study therapy.
Measured: Baseline, 12 months
Change in Shortness of Breath (Self-reported)
Participants reported frequency of shortness of breath experienced with exertion
Measured: Baseline, 12 months
Mean Change in Six Minute Walk Distance
Comparison of 6-minute walk distance before beginning and after completing study therapy
Measured: 12 months
Mean Change in Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)
DLCO was measured before beginning and after completion of study therapy
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2006 (estimated)
- Study startMay 2006
- Primary completionJan 2009
- Study completionJan 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Scleroderma, SystemicStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Collaborators
- Roche Pharma AG
- Responsible party
- Sponsor
- Investigators
- Jeffrey A Golden, MD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00333437
- Sponsor's protocol ID
- CEL371
- Status last verified
- Sep 2013
- First submitted
- 2 Jun 2006
- Last updated
- 13 Nov 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pulmonary Involvement in Scleroderma: Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients
- ClinicalTrials.gov ID
- NCT00333437
- Acronym / sponsor ID
- CEL371
- Conditions
- Scleroderma, Systemic
- Keywords
- Scleroderma, Systemic
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- Roche Pharma AG
- Study start
- May 2006
- Primary completion
- Jan 2009 (actual)
- Study completion
- Jan 2009 (actual)
- First posted
- 5 Jun 2006 (estimated)
- Results
- Posted 17 May 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Researchers from the Division of Pulmonary and Critical Care Medicine at University of California, San Francisco (UCSF) are conducting a study to evaluate whether mycophenolate mofetil (an immunosuppressive medication, trade named CellCept) is safe and effective for preventing the lung damage from scleroderma from getting worse.
Detailed description
The proposed study is designed to evaluate the safety and efficacy of mycophenolate mofetil (CellCept) for the treatment of symptomatic pulmonary alveolitis due to systemic sclerosis (SSc). This study utilizes a prospective, open-label, experimental design.
Primary Hypothesis: The alveolitis in patients with SSc, as defined by decreased forced vital capacity (FVC), bronchoalveolar lavage (BAL), and High Resolution Chest Tomography (HRCT) is responsive to 1 year of daily mycophenolate mofetil therapy.
Secondary Hypothesis: Quality of life, six-minute walk and single-breath diffusing capacity for carbon monoxide (DLCO) improve in patients with SSc mediated alveolitis after therapy with mycophenolate mofetil. This response to therapy is associated with a change in the inflammatory cytokine profile present in BAL fluid.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: Mycophenolate mofetilMycophenolate Mofetil |
Who may qualify
- Ages
- 21 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- To participate in this study, patients must first undergo a BAL and HRCT. To be eligible to undergo HRCT and BAL (under the purview of this trial), prospective patients must meet the following criteria:
- To be eligible to take study medication, the patient must meet not only the criteria above, but also must have ≥ 3.0% neutrophils or ≥ 2.0% eosinophils in screening BAL fluid and/or ground glass opacification on HRCT.
- Women of childbearing potential should have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL within 1 week before beginning therapy. CellCept therapy will not be initiated until a report of a negative pregnancy test has been obtained.
- Effective contraception must be used before beginning CellCept therapy, during therapy, and for 6 weeks following discontinuation of therapy, even where there has been a history of infertility, unless due to hysterectomy. Two reliable forms of contraception must be used simultaneously unless abstinence is the chosen method. If pregnancy does occur during treatment, the physician and patient should discuss the desirability of continuing the pregnancy.
Key exclusion criteria (17 listed)
- FVC < 45% of predicted or DLCO (corrected for hemoglobin [Hgb] but not for alveolar volume) < 35% of predicted (suggestive of severe, probably irreparable, disease).
- Leukopenia (white blood cell count < 4000) or thrombocytopenia (platelet count < 100,000).
- Serum creatinine ≥ 2.0 mg/dl.
- Pregnancy, breast feeding, unreliability, drug abuse, or chronic debilitating disease.
- Uncontrolled congestive heart failure.
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 |
|---|---|---|
| Mycophenolate mofetil | Drug | Treatment |
Primary outcome
Mean Change From Baseline in Forced Vital Capacity (FVC)
Time frame: Baseline, 12 months
compare pre- and post-therapy FVC (post- minus pre-). Forced vital capacity (FVC) is the volume of air (liters) that can forcibly be blown out after full inspiration.
4 secondary outcomes
Mean Change in Bronchoalveolar Lavage (BAL) Components (Neutrophils, Eosinophils)
Time frame: Baseline, 12 months
Change in Shortness of Breath (Self-reported)
Time frame: Baseline, 12 months
Mean Change in Six Minute Walk Distance
Time frame: 12 months
Mean Change in Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Nov 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Jeffrey A Golden, MD principal investigator
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Mycophenolate mofetil in people with Scleroderma, Systemic, run by University of California, San Francisco at 1 site in United States.
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 17 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 13 Nov 2013. 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 United States. 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?
Yes. Results are posted in the registry (first posted 17 May 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Mean Change From Baseline in Forced Vital Capacity (FVC) (time frame: Baseline, 12 months). 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 13 Nov 2013.