Systemic Lupus ErythematosusPhase 1/2NCT00594932
Biomarker-Linked Outcomes of Cellcept in Lupus Arthritis
A Phase 1/2 study testing mycophenolate mofetil in people with Systemic Lupus Erythematosus and Arthritis, run by Oklahoma Medical Research Foundation at 1 site in United States.
Trial summary
We hypothesize that mycophenolate mofetil(Cellcept)is safe and effective for lupus arthritis. In this study, patients with lupus will be randomly assigned to receive mycophenolate mofetil or placebo (inert pills) for three months. Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- Systemic Lupus Erythematosus, Arthritis
- What is being tested?
- mycophenolate mofetil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2006to Apr 2009
- Phase
- Phase 1/2
- 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 14 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of SLE by the 1995 modification of revised ACR criteria (includes antiphospholipid antibodies)
- BILAG A arthritis or BILAG B arthritis with at least 6 tender and 4 swollen joints at screening and baseline
- Stable prednisone dose at 20 mg of less for one month at baseline.
- If on antimalarials must be stable for at least one month at baseline
- If on NSAIDS must be on a stable regimen for at least one month but can be prn dosing
- Must be willing to withdraw from azathioprine or MTX at the time of screening.
Show all 10Show fewer
- Between ages 14 and 70
- Women of childbearing potential must have a negative pregnancy test at screening and at each month during the study.
- All participants (male and female) must, if fertile, agree to practice contraception during the entire course of the study. This may include barrier, oral contraceptives, depo-provera, intrauterine device and/or abstinence.
- -
Who cannot join
- Inability to understand informed consent
- Drug or alcohol abuse within the past six months
- In the opinion of the investigator, it is not likely the patient can comply with the protocol for any reason, or participation in the protocol is not in the patient's best interest.
- Unstable medical condition that, in the opinion of the investigator would contraindicate study participation
- History of malignancy (except for basal cell carcinoma at any time and/or cervical cancer or squamous cell cancer at least five years previous to screening).
- Use of cyclosporine, leflunomide, cyclophosphamide or ay biologic agent within three months prior to screening.
- Participation in any clinical study of an investigational agent within three months of screening -
Trial locations and contacts
1 site in 1 country.
United States1 site
Oklahoma Medical Research Foundation
Oklahoma City, Oklahoma, 73104
About this study
We hypothesize that mycophenolate mofetil(Cellcept)is safe and effective for lupus arthritis. In this study, patients with lupus will be randomly assigned to receive mycophenolate mofetil or placebo (inert pills) for three months. At the end of three months all patients will receive mycophenolate mofetil for three additional months. The effectiveness on arthritis and other symptoms of lupus will be measured by joint counts and by the BILAG instrument (a measure of overall lupus disease activity. Additionally special blood tests aimed at understanding the biologic effects of mycophenolate mofetil will also be performed at some visits. The primary outcome measurement will be the safety and effectiveness of this treatment (as compared to placebo) at the three month point. The trial will continue in a blinded fashion (neither the investigator or the participants know who is getting mycophenolate and who is getting placebo) until 24 patients have completed the first three months of the protocol.
Read the detailed description
Patients and Methods:
27 patients with active BILAG B or A arthritis, with at least 6 swollen and 6 tender joints entered a six month study of MMF vs placebo for three months followed by open label MMF. 14 patients (12 women and 2 men) received placebo at baseline and 13 patients (11 women and 2 men) received MMF. Primary Outcome was Major Clinical Response at 3 months, then all patients received open label Cellcept for another 3 months. Blood was drawn for safety, lupus disease activity measures and exploratory Biomarkers, Joint counts were performed monthly. At baseline background DMARDs were stopped. Plaquenil was allowed. All patients received 160 mg depomedrol at baseline and were allowed 80 mg shots at subsequent months after blood draws and procedures had been completed.
DEFINITION of RESPONSE
Prespecified Primary Endpoint: Complete Clinical Response:
BILAG C in musculoskeletal by Week 12 and decrease to 0.25 or less of tender +swollen jt counts
Prespecified Secondary Endpoint: Partial response:
One letter drop in musculoskeletal by Week 12 OR decrease to 0.5 or less tender + swollen jt counts
Exploratory Measure (not prespecified): Major Clinical Response:
BILAG C in musculoskeletal by Week 12 and decrease to 0.5 or less of tender +swollen jt counts. (In the primary analysis the one patient who met this endpoint was designated as a partial responder since those prespecified criteria were also met.
Non response:
Does not meet above criteria for complete or partial response
Additional Measures: (prespecified secondary endpoints) included joint counts, changes in BILAG and SLEDAI and physician and patient global assessments.
- Conditions
- Systemic Lupus Erythematosus, Arthritis
- Being tested
- mycophenolate mofetil, placebo
- Keywords
- lupus, arthritis, mycophenolate, biomarkers
- Medical subject headings
- Lupus Erythematosus, Systemic, Arthritis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Also called mycophenolate, Cellcept
First treatment month: mycophenolate mofetil ascending doses orally Second treatment month to end of study: mycophenolate mofetil 3 gms/day (or less if tolerance issues arise)
placeboOther
Also called inert tablet, oral placebo pill
oral placebo will be given in ascending "doses" during the first month and at full "dose" during the second and third month (or at lower "dose" if tolerance issues warrant). During the fourth month mycophenolate mofetil will be given in ascending doses to 3 gms/day (or less if tolerance issues arise) and continues until the end of the study at 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Arm I: | Active comparator | Participants randomly assigned to Arm I will receive mycophenolate mofetil in ascending doses during Month 1, and 3 grams/day (or less if there are tolerance issues) for Months 2 through 6. During Month 1 these participants receive the same number of pills as every other month, but with ascending doses of mycophenolate mofetil and descending numbers of placebo pills. Week one for a total of 1.5 gm/day of mycophenolate mofetil, Week two 2.0 gm/day, Week three 2.5 gm/day and Week 4 3 gm/day. Dose can be held or decreased for tolerance issues at any time. Receives: Drug: mycophenolate mofetil |
| Arm 2 | Placebo comparator | Patients Randomly Assigned to Arm 2 will receive a placebo comparator. The placebo treatment will be structured so that they will undergo the same type of dosing in Month 1 that the ascending dose patient from Arm 1 undergo, but will have placebo in both bottles of pills. At the end of three months, after assessment of primary outcome, these patients enter open label treatment for three more months. During the fourth month this group continues to receive the same number of pills as they received before, with ascending doses of mycophenolate mofetil given vs descending placebo pills so that their induction is the same as those in Arm 1 at the first month. Receives: Other: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Arthritis Complete Response
Complete response at three months (This is defined as </= 0.25 of the total tender plus swollen joints observed at baseline and British Isles Lupus Assessment Group (BILAG) index C (mild) or D (no longer present) score in the Musculoskeletal system), comparing treatment to placebo group as complete responder or not complete responder. This was an intent to treat analysis so dropouts were counted as non-responders.
Measured: 3 months
Secondary outcomes (2)
Major Arthritis Response
This is defined as at least a 50% reduction in tender + swollen joint counts and severity rated as mild as defined by the British Isles Lupus Assessment Group Index
Measured: 3 months
Major and Partial Clinical Response
Those meeting the Criteria for Major or Complete Clinical Response as listed in the preceding endpoints or who meet criteria for partial response defined as at least a 25% decrease in tender and swollen joint count and improvement of at least one severity rating for arthritis as at least one level on the British Isles Lupus Assessment Group Index.
Measured: 3 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jan 2008 (estimated)
- Study startNov 2006
- Primary completionJul 2008
- Study completionApr 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 Systemic Lupus ErythematosusStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oklahoma Medical Research Foundation
- Sponsor type
- University, hospital or other
- Collaborators
- NYU Langone Health
- Responsible party
- Sponsor
- Investigators
- Joan T. Merrill, M.D., Principal investigator, Oklahoma Medical Research FoundationRobert Clancy, PhD, Study chair, NYU Langone Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00594932
- Sponsor's protocol ID
- OMRF 06-23
- Other IDs
- Aspreva Pharmaceuticals grant
- Status last verified
- Oct 2020
- First submitted
- 4 Jan 2008
- Last updated
- 9 Oct 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Biomarker-Linked Outcomes of Cellcept in Lupus Arthritis
- ClinicalTrials.gov ID
- NCT00594932
- Acronym / sponsor ID
- OMRF 06-23
- Conditions
- Systemic Lupus Erythematosus, Arthritis
- Keywords
- lupus, arthritis, mycophenolate, biomarkers
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Oklahoma Medical Research FoundationUniversity, hospital or other
- Collaborators
- NYU Langone Health
- Study start
- Nov 2006
- Primary completion
- Jul 2008 (actual)
- Study completion
- Apr 2009 (actual)
- First posted
- 16 Jan 2008 (estimated)
- Results
- Posted 9 Oct 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
We hypothesize that mycophenolate mofetil(Cellcept)is safe and effective for lupus arthritis. In this study, patients with lupus will be randomly assigned to receive mycophenolate mofetil or placebo (inert pills) for three months. At the end of three months all patients will receive mycophenolate mofetil for three additional months. The effectiveness on arthritis and other symptoms of lupus will be measured by joint counts and by the BILAG instrument (a measure of overall lupus disease activity. Additionally special blood tests aimed at understanding the biologic effects of mycophenolate mofetil will also be performed at some visits. The primary outcome measurement will be the safety and effectiveness of this treatment (as compared to placebo) at the three month point. The trial will continue in a blinded fashion (neither the investigator or the participants know who is getting mycophenolate and who is getting placebo) until 24 patients have completed the first three months of the protocol.
Detailed description
Patients and Methods:
27 patients with active BILAG B or A arthritis, with at least 6 swollen and 6 tender joints entered a six month study of MMF vs placebo for three months followed by open label MMF. 14 patients (12 women and 2 men) received placebo at baseline and 13 patients (11 women and 2 men) received MMF. Primary Outcome was Major Clinical Response at 3 months, then all patients received open label Cellcept for another 3 months. Blood was drawn for safety, lupus disease activity measures and exploratory Biomarkers, Joint counts were performed monthly. At baseline background DMARDs were stopped. Plaquenil was allowed. All patients received 160 mg depomedrol at baseline and were allowed 80 mg shots at subsequent months after blood draws and procedures had been completed.
DEFINITION of RESPONSE
Prespecified Primary Endpoint: Complete Clinical Response:
BILAG C in musculoskeletal by Week 12 and decrease to 0.25 or less of tender +swollen jt counts
Prespecified Secondary Endpoint: Partial response:
One letter drop in musculoskeletal by Week 12 OR decrease to 0.5 or less tender + swollen jt counts
Exploratory Measure (not prespecified): Major Clinical Response:
BILAG C in musculoskeletal by Week 12 and decrease to 0.5 or less of tender +swollen jt counts. (In the primary analysis the one patient who met this endpoint was designated as a partial responder since those prespecified criteria were also met.
Non response:
Does not meet above criteria for complete or partial response
Additional Measures: (prespecified secondary endpoints) included joint counts, changes in BILAG and SLEDAI and physician and patient global assessments.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I: | Active comparator | Drug: mycophenolate mofetilParticipants randomly assigned to Arm I will receive mycophenolate mofetil in ascending doses during Month 1, and 3 grams/day (or less if there are tolerance issues) for Months 2 through 6. During Month 1 these participants receive the same number of pills as every other month, but with ascending doses of mycophenolate mofetil and descending numbers of placebo pills. Week one for a total of 1.5 gm/day of mycophenolate mofetil, Week two 2.0 gm/day, Week three 2.5 gm/day and Week 4 3 gm/day. Dose can be held or decreased for tolerance issues at any time. |
| Arm 2 | Placebo comparator | Other: placeboPatients Randomly Assigned to Arm 2 will receive a placebo comparator. The placebo treatment will be structured so that they will undergo the same type of dosing in Month 1 that the ascending dose patient from Arm 1 undergo, but will have placebo in both bottles of pills. At the end of three months, after assessment of primary outcome, these patients enter open label treatment for three more months. During the fourth month this group continues to receive the same number of pills as they received before, with ascending doses of mycophenolate mofetil given vs descending placebo pills so that their induction is the same as those in Arm 1 at the first month. |
Who may qualify
- Ages
- 14 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Diagnosis of SLE by the 1995 modification of revised ACR criteria (includes antiphospholipid antibodies)
- BILAG A arthritis or BILAG B arthritis with at least 6 tender and 4 swollen joints at screening and baseline
- Stable prednisone dose at 20 mg of less for one month at baseline.
- If on antimalarials must be stable for at least one month at baseline
- If on NSAIDS must be on a stable regimen for at least one month but can be prn dosing
Key exclusion criteria (7 listed)
- Inability to understand informed consent
- Drug or alcohol abuse within the past six months
- In the opinion of the investigator, it is not likely the patient can comply with the protocol for any reason, or participation in the protocol is not in the patient's best interest.
- Unstable medical condition that, in the opinion of the investigator would contraindicate study participation
- History of malignancy (except for basal cell carcinoma at any time and/or cervical cancer or squamous cell cancer at least five years previous to screening).
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 mofetilAlso: mycophenolate, CellceptFirst treatment month: mycophenolate mofetil ascending doses orally Second treatment month to end of study: mycophenolate mofetil 3 gms/day (or less if tolerance issues arise) | Drug | Arm I: |
| placeboAlso: inert tablet, oral placebo pilloral placebo will be given in ascending "doses" during the first month and at full "dose" during the second and third month (or at lower "dose" if tolerance issues warrant). During the fourth month mycophenolate mofetil will be given in ascending doses to 3 gms/day (or less if tolerance issues arise) and continues until the end of the study at 6 months. | Other | Arm 2 |
Primary outcome
Arthritis Complete Response
Time frame: 3 months
Complete response at three months (This is defined as </= 0.25 of the total tender plus swollen joints observed at baseline and British Isles Lupus Assessment Group (BILAG) index C (mild) or D (no longer present) score in the Musculoskeletal system), comparing treatment to placebo group as complete responder or not complete responder. This was an intent to treat analysis so dropouts were counted as non-responders.
2 secondary outcomes
Major Arthritis Response
Time frame: 3 months
Major and Partial Clinical Response
Time frame: 3 Months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2020.
- 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.
Joan T. Merrill, M.D. principal investigator
Oklahoma Medical Research Foundation
Robert Clancy, PhD study chair
NYU Langone Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing mycophenolate mofetil in people with Systemic Lupus Erythematosus and Arthritis, run by Oklahoma Medical Research Foundation 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 10 inclusion and 7 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 9 Oct 2020. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Oct 2020). 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?
Arthritis Complete Response (time frame: 3 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 9 Oct 2020.