NCT00377637

Brief Summary

This 2 arm study assessed the efficacy of Mycophenolate Mofetil (MMF; CellCept) compared to cyclophosphamide in inducing a response in patients with lupus nephritis, and the long term efficacy of MMF compared to azathioprine in maintaining remission and renal function. Patients were randomized to receive either MMF (1.5 g twice daily \[bid\]) or cyclophosphamide (0.5-1.0 g/m\^2 in monthly pulses) in the induction phase. Those patients meeting criteria for response were re-randomized for entry into the maintenance phase, to receive either MMF (1 g bid) or azathioprine (2 mg/kg/day).

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
370

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Jul 2005

Longer than P75 for phase_3

Geographic Reach
17 countries

108 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2005

Completed
1.2 years until next milestone

First Submitted

Initial submission to the registry

September 15, 2006

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 18, 2006

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2007

Completed
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2010

Completed
1.8 years until next milestone

Results Posted

Study results publicly available

December 6, 2011

Completed
Last Updated

December 6, 2011

Status Verified

October 1, 2011

Enrollment Period

1.7 years

First QC Date

September 15, 2006

Results QC Date

August 11, 2011

Last Update Submit

October 31, 2011

Conditions

Outcome Measures

Primary Outcomes (2)

  • Induction Phase: Number of Patients Showing Treatment Response

    Treatment response was adjudicated by a blinded clinical endpoints committee (CEC) and defined as: a) Decrease in proteinuria, defined as a decrease in the urine protein to creatinine ratio (UPCr) to \<3 in subjects with baseline proteinuria ≥3 UPCr or a decrease in the UPCr by ≥50% in subjects with proteinuria \<3 UPCr at Baseline, and b) Stabilization of serum creatinine or improvement. UPCr were derived from the 24 hour urine collection. Patients who did not show a treatment response at Week 24 or who withdrew earlier than Week 24 were considered non-responders.

    24 weeks

  • Maintenance Phase: Kaplan-Meier Estimates of Percentage of Participants Treatment Failure Free, by Time Interval

    Treatment Failure was adjudicated by a clinical endpoints committee and was defined as the time to the earliest occurrence of any one of the following: death, end stage renal disease, sustained doubling of serum creatinine, renal flare, or a requirement for rescue therapy for exacerbation or deterioration of Lupus nephritis. Kaplan-Meier survival curves were estimated from the observed time to treatment failure for each patient. The data presented are the percentage of participants who were treatment-failure free at each time interval as estimated by Kaplan-Meier.

    From the start of the Maintenance Phase to Month 36

Secondary Outcomes (12)

  • Induction Phase: Number of Participants Achieving Complete Remission

    24 weeks

  • Induction Phase: Change From Baseline to Week 24 in Serum Creatinine

    Baseline, Week 24

  • Induction Phase: Change From Baseline to Week 24 in 24-hour Urine Protein

    Baseline, Week 24

  • Induction Phase: Change From Baseline to Week 24 in Serum Albumin

    Baseline, Week 24

  • Induction Phase: Change in Renal British Isles Lupus Assessment Group (BILAG) Score

    Baseline, 24 weeks

  • +7 more secondary outcomes

Study Arms (4)

Induction Phase: Mycophenolate mofetil

EXPERIMENTAL

Participants received oral mycophenolate mofetil (MMF) 1.5 g twice a day and concomitant corticosteroids for the 24 weeks of the Induction Phase.

Drug: Mycophenolate mofetil (MMF)Drug: Corticosteroid

Induction Phase: Cyclophosphamide

ACTIVE COMPARATOR

Participants received monthly infusions of cyclophosphamide, 0.5 to 1.0 g per square meter of body surface area and concomitant treatment with corticosteroids for the 24 week Induction Phase.

Drug: CyclophosphamideDrug: Corticosteroid

Maintenance Phase: Mycophenolate mofetil

EXPERIMENTAL

Participants received mycophenolate mofetil (MMF) 1.0 g orally twice a day, placebo to azathioprine orally once a day and corticosteroid for the 36 weeks Maintenance Phase.

Drug: Mycophenolate mofetil (MMF)Drug: Placebo to AzathioprineDrug: Corticosteroid

Maintenance Phase: Azathioprine

ACTIVE COMPARATOR

Participants received azathioprine (AZA) 2 mg/kg/day orally once a day, placebo to mycophenolate mofetil orally twice a day and corticosteroid for the 36 weeks Maintenance Phase.

Drug: AzathioprineDrug: Placebo to Mycophenolate mofetilDrug: Corticosteroid

Interventions

Supplied as 500 mg tablets taken orally twice a day (BID). Dose specific for each arm. Dosing started at 500 mg BID for the first week, increasing by 500 mg in subsequent weeks until the final target dose was reached.

Also known as: CellCept
Induction Phase: Mycophenolate mofetilMaintenance Phase: Mycophenolate mofetil

Intravenous cyclophosphamide (IVC) was administered every four weeks (monthly) to a total of six infusions. Dosing was started at 0.75 g/m\^2 of body surface area for the first month, with subsequent doses at 0.5-1.0 g/m\^2. The target dose was 1.0 g/m\^2, but doses were titrated by 0.25 g/m\^2 increments to maintain nadir leukocyte count between 2500-4000/mm\^3.

Also known as: Endoxan®
Induction Phase: Cyclophosphamide

2 mg/kg/day orally, provided as 50 mg capsules to be taken after meals.

Also known as: Imuran®
Maintenance Phase: Azathioprine

Placebo capsules matching Azathioprine taken orally once a day.

Maintenance Phase: Mycophenolate mofetil

Placebo tablets matching Mycophenolate mofetil taken orally twice daily.

Maintenance Phase: Azathioprine

Oral prednisolone (or equivalent) starting at a dose of 0.75-1.0 mg/kg/day (maximum 60 mg/day) tapered to 10 mg/day.

Induction Phase: CyclophosphamideInduction Phase: Mycophenolate mofetilMaintenance Phase: AzathioprineMaintenance Phase: Mycophenolate mofetil

Eligibility Criteria

Age12 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • male or female patients, 12-75 years of age;
  • diagnosis of systemic lupus erythematosus;
  • kidney biopsy within 6 months of study, with histological diagnosis of lupus nephritis;
  • laboratory evidence of active nephritis.

You may not qualify if:

  • continuous dialysis starting \>2 weeks before randomization into induction phase, and/or with an anticipated duration of \>8 weeks;
  • previous or planned kidney transplant;
  • other clinically significant active medical conditions.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (108)

Unknown Facility

Huntsville, Alabama, 35801, United States

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La Jolla, California, 92037, United States

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Los Angeles, California, 90095, United States

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San Francisco, California, 94143, United States

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San Leandro, California, 94578, United States

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Torrance, California, 90502, United States

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Miami, Florida, 33136, United States

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Atlanta, Georgia, 30303, United States

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Chicago, Illinois, 60611, United States

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Chicago, Illinois, 60637, United States

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Baltimore, Maryland, 21205, United States

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Boston, Massachusetts, 02115, United States

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Ann Arbor, Michigan, 48109-0358, United States

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Columbia, Missouri, 65212, United States

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Brooklyn, New York, 11203, United States

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Lake Success, New York, 11042, United States

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New York, New York, 10003, United States

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New York, New York, 10021, United States

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New York, New York, 10032, United States

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Chapel Hill, North Carolina, 27599-7280, United States

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Durham, North Carolina, 27710, United States

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Cleveland, Ohio, 44136, United States

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Columbus, Ohio, 43210, United States

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Oklahoma City, Oklahoma, 73104, United States

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Philadelphia, Pennsylvania, 19104, United States

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Pittsburgh, Pennsylvania, 15213, United States

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Charleston, South Carolina, 29425, United States

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Dallas, Texas, 75390, United States

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Buenos Aires, C1015ABO, Argentina

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Buenos Aires, C1425DQK, Argentina

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Córdoba, 5016, Argentina

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San Isidro, B1602BPPD, Argentina

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San Miguel de Tucumán, T4000AXL, Argentina

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Adelaide, SA 5000, Australia

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Camperdown, 2050, Australia

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Melbourne, 3168, Australia

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Parkville, 3052, Australia

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Woodville, 5011, Australia

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Brussels, 1200, Belgium

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Leuven, 3000, Belgium

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Liège, 4000, Belgium

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Rio de Janeiro, 20551-030, Brazil

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São Paulo, 04039-020, Brazil

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Sorocaba, 18030-210, Brazil

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Vancouver, British Columbia, V5Z 1L7, Canada

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Victoria, British Columbia, V8Z 7X8, Canada

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Halifax, Nova Scotia, B3H 2Y9, Canada

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London, Ontario, N6A 4V2, Canada

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Toronto, Ontario, M5T 2S8, Canada

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Montreal, Quebec, H1T 2M4, Canada

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Beijing, 100034, China

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Guangdong, 510008, China

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Guangzhou, 510630, China

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Jiangsu, 210002, China

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Shanghai, 200001, China

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Brno, 656 91, Czechia

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Prague, 128 08, Czechia

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Lille, 59037, France

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Lyon, 69437, France

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Nantes, 44035, France

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Paris, 75679, France

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Paris, 75877, France

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Toulouse, 31059, France

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Aachen, 52074, Germany

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Bad Bramstedt, 24576, Germany

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Berlin, 10117, Germany

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Berlin, 14059, Germany

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Dresden, 01307, Germany

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Düsseldorf, 40225, Germany

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Erlangen, 91054, Germany

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Hanover, 30625, Germany

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Leipzig, 04103, Germany

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München, 80336, Germany

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München, 81675, Germany

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Münster, 48149, Germany

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Athens, 11521, Greece

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Athens, 11527, Greece

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Heraklion, 71500, Greece

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Debrecen, 4032, Hungary

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Pécs, 7632, Hungary

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Szeged, 2724, Hungary

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Brescia, 25125, Italy

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Milan, 20149, Italy

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Padua, 35128, Italy

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Pisa, 56100, Italy

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Udine, 33100, Italy

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Mexico City, 14000, Mexico

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Mérida, 97000, Mexico

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San Luis Potosí City, 78240, Mexico

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Lisbon, Portugal

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Porto, 4200-319, Portugal

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Alicante, 03010, Spain

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Barcelona, 08035, Spain

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Barcelona, 08036, Spain

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Madrid, 28041, Spain

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Málaga, 29010, Spain

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Santander, 39008, Spain

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Seville, 41013, Spain

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Birmingham, B15 2TT, United Kingdom

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Cambridge, CB2 2QQ, United Kingdom

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Leeds, LS1 3EX, United Kingdom

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London, SE1 7EH, United Kingdom

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London, W12 OHS, United Kingdom

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London, WIT 4NJ, United Kingdom

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Manchester, M13 9WL, United Kingdom

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Newcastle upon Tyne, NE7 7DN, United Kingdom

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Sheffield, S10 2JF, United Kingdom

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Southampton, SO16 6YD, United Kingdom

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Related Publications (5)

  • Sundel R, Solomons N, Lisk L; Aspreva Lupus Management Study (ALMS) Group. Efficacy of mycophenolate mofetil in adolescent patients with lupus nephritis: evidence from a two-phase, prospective randomized trial. Lupus. 2012 Nov;21(13):1433-43. doi: 10.1177/0961203312458466. Epub 2012 Aug 24.

  • Dooley MA, Jayne D, Ginzler EM, Isenberg D, Olsen NJ, Wofsy D, Eitner F, Appel GB, Contreras G, Lisk L, Solomons N; ALMS Group. Mycophenolate versus azathioprine as maintenance therapy for lupus nephritis. N Engl J Med. 2011 Nov 17;365(20):1886-95. doi: 10.1056/NEJMoa1014460.

  • Dall'Era M, Stone D, Levesque V, Cisternas M, Wofsy D. Identification of biomarkers that predict response to treatment of lupus nephritis with mycophenolate mofetil or pulse cyclophosphamide. Arthritis Care Res (Hoboken). 2011 Mar;63(3):351-7. doi: 10.1002/acr.20397. Epub 2010 Nov 15.

  • Ginzler EM, Wofsy D, Isenberg D, Gordon C, Lisk L, Dooley MA; ALMS Group. Nonrenal disease activity following mycophenolate mofetil or intravenous cyclophosphamide as induction treatment for lupus nephritis: findings in a multicenter, prospective, randomized, open-label, parallel-group clinical trial. Arthritis Rheum. 2010 Jan;62(1):211-21. doi: 10.1002/art.25052.

  • Isenberg D, Appel GB, Contreras G, Dooley MA, Ginzler EM, Jayne D, Sanchez-Guerrero J, Wofsy D, Yu X, Solomons N. Influence of race/ethnicity on response to lupus nephritis treatment: the ALMS study. Rheumatology (Oxford). 2010 Jan;49(1):128-40. doi: 10.1093/rheumatology/kep346. Epub 2009 Nov 20.

MeSH Terms

Conditions

Lupus Nephritis

Interventions

Mycophenolic AcidCyclophosphamideAzathioprineAdrenal Cortex Hormones

Condition Hierarchy (Ancestors)

GlomerulonephritisNephritisKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesLupus Erythematosus, SystemicConnective Tissue DiseasesSkin and Connective Tissue DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

CaproatesAcids, AcyclicCarboxylic AcidsOrganic ChemicalsFatty AcidsLipidsPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsPhosphoramidesOrganophosphorus CompoundsThionucleosidesSulfur CompoundsMercaptopurinePurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesHormonesHormones, Hormone Substitutes, and Hormone Antagonists

Limitations and Caveats

For many of the Maintenance Phase Outcomes, the total number of events precluded a meaningful time to event statistical analysis, therefore, only events numbers are reported.

Results Point of Contact

Title
Medical Communications
Organization
Hoffman-LaRoche

Study Officials

  • Clinical Trials

    Hoffmann-La Roche

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 15, 2006

First Posted

September 18, 2006

Study Start

July 1, 2005

Primary Completion

March 1, 2007

Study Completion

March 1, 2010

Last Updated

December 6, 2011

Results First Posted

December 6, 2011

Record last verified: 2011-10

Locations