Biomarker-Linked Outcomes of Cellcept in Lupus Arthritis

NCT00594932 | Completed Phase 1 Results

• 2 other identifiers: OMRF 06-23Aspreva Pharmaceuticals grant
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Systemic Lupus Erythematosus; Arthritis

Keywords

lupus, arthritis, mycophenolate, biomarkers

Outcome Measures

Primary Outcomes (1)

• 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.

  3 months

Secondary Outcomes (2)

• Major Arthritis Response

  3 months

• Major and Partial Clinical Response

  3 Months

Study Arms (2)

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.

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.

Other: placebo

Interventions

mycophenolate mofetil DRUG

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)

Also known as: mycophenolate, Cellcept

Arm I:

placebo OTHER

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.

Also known as: inert tablet, oral placebo pill

Arm 2

Eligibility Criteria

• Age: 14 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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 -

Sponsors & Collaborators

• Oklahoma Medical Research Foundation: lead
• NYU Langone Health: collaborator

Study Sites (1)

Oklahoma Medical Research Foundation

Oklahoma City, Oklahoma, 73104, United States

Location

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Arthritis

Interventions

Mycophenolic Acid

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Joint Diseases; Musculoskeletal Diseases

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids

Results Point of Contact

• Title: Joan T Merrill M.D.
• Organization: Oklahoma Medical Research Foundation

joan-merrill@omrf.org

405 271 7805

Study Officials

• Joan T. Merrill, M.D.

  Oklahoma Medical Research Foundation

  PRINCIPAL INVESTIGATOR

• Robert Clancy, PhD

  NYU Langone Health

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 4, 2008
• First Posted: January 16, 2008
• Study Start: November 1, 2006
• Primary Completion: July 1, 2008
• Study Completion: April 1, 2009
• Last Updated: October 9, 2020
• Results First Posted: October 9, 2020

Record last verified: 2020-10

Locations

US (1)