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A Study of Intravenous MabThera/Rituxan in Pediatric Patients With Severe Granulomatosis With Polyangiitis (Wegener's) or Microscopic Polyangiitis

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2016 by Hoffmann-La Roche
Information provided by (Responsible Party):
Hoffmann-La Roche Identifier:
First received: December 13, 2012
Last updated: July 1, 2016
Last verified: July 2016
This multicenter, open-label, uncontrolled study will evaluate the safety and pharmacokinetics of MabThera/Rituxan (rituximab) in pediatric patients with severe granulomatosis with polyangiitis (Wegener's) or microscopic polyangiitis. Patients will receive MabThera/Rituxan 375 mg/m2 intravenously on Days 1, 8, 15 and 22.

Condition Intervention Phase
Wegener's Granulomatosis, Microscopic Polyangiitis
Drug: rituximab [MabThera/Rituxan]
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • Safety: Incidence of adverse events [ Time Frame: approximately 3.5 years ] [ Designated as safety issue: No ]
  • Safety: Nature and severity of adverse events [ Time Frame: approximately 3.5 years ] [ Designated as safety issue: No ]
  • Pharmacokinetics: Clearance (CL) [ Time Frame: up to Day 22 and at Month 1, 2, 4 and 6 ] [ Designated as safety issue: No ]
  • Pharmacokinetics: Volume of distribution [ Time Frame: up to Day 22 and at Month 1, 2, 4 and 6 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Pharmacokinetics: Area under the concentration-time curve (AUC) [ Time Frame: up to Day 22 and at Month 1, 2, 4 and 6 ] [ Designated as safety issue: No ]
  • Pharmacokinetics: Maximum concentration (Cmax) [ Time Frame: up to Day 22 and at Month 1, 2, 4 and 6 ] [ Designated as safety issue: No ]

Estimated Enrollment: 25
Study Start Date: May 2013
Estimated Study Completion Date: March 2018
Estimated Primary Completion Date: March 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: MabThera/Rituxan Drug: rituximab [MabThera/Rituxan]
375 mg/m2 iv on Days 1, 8, 15 and 22


Ages Eligible for Study:   2 Years to 17 Years   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age at screening between >/= 2 and < 18 years
  • Diagnosis of granulomatosis with polyangiitis (EULAR/PRINTO/PRES 2008, Ankara criteria for childhood Wegener's granulomatosis) or diagnosis of microscopic polyangiitis (according to the Chapel Hill Consensus Conference)
  • Newly diagnosed patients or patients with relapsing disease according to the following definition:

The recurrence or new onset of potentially organ- or life-threatening disease (i.e. one or more major BVAS/WG items as listed in the protocol or disease severe enough to require treatment with cyclophosphamide)

  • For patients of reproductive potential (males and females), use of reliable means of contraception throughout the study participation
  • For all eligible patients, mandatory prophylactic treatment for Pneumocystis jirovecii infection

Exclusion Criteria:

  • Diagnosis of Churg-Strauss syndrome, as defined by the Chapel Hill consensus Conference (Jennette 1994)
  • Limited disease that would not normally be treated with cyclophosphamide
  • Severe disease requiring mechanical ventilation due to alveolar hemorrhage
  • Requirement for plasmapheresis or dialysis at screening
  • Pregnancy or breastfeeding
  • Evidence of other significant uncontrolled concomitant disease, or of disorder or condition that, in the investigator's opinion, would preclude or interfere with patient participation
  • Primary or secondary immunodeficiency (history of or currently active), including known history of human immunodeficiency virus (HIV) infection
  • Known active infection of any kind (excluding fungal infections of nail beds), or any major episode of infection requiring hospitalization with IV anti-infective agents within 4 weeks of baseline or completion of oral anti-infective agents within 2 weeks prior to baseline
  • History of deep space/tissue infection within 24 weeks prior to baseline
  • History of serious recurrent or chronic infection
  • History of cancer (except for basal cell and squamous cell carcinoma of the skin that have been excised and cured)
  • Currently active alcohol or drug abuse or history of alcohol or drug abuse
  • History of severe allergic or anaphylactic reaction to a biologic agent or known hypersensitivity to any component of rituximab or to murine proteins
  • Previous treatment with rituximab
  • Previous treatment with any cell-depleting therapies
  • Receipt of oral or IV cyclophosphamide within the previous 4 months prior to the baseline visit
  • Receipt of infliximab within 3 months, adalimumab within 2 months or etanercept within 1 month prior to the baseline visit
  • Treatment with any investigational agent within 28 days of baseline or 5 half-lives of the investigational drug (whichever is longer)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01750697

Contact: Reference Study ID Number: WA25615 888-662-6728 (U.S. and Canada)

  Show 23 Study Locations
Sponsors and Collaborators
Hoffmann-La Roche
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

Responsible Party: Hoffmann-La Roche Identifier: NCT01750697     History of Changes
Other Study ID Numbers: WA25615  2012-002062-13 
Study First Received: December 13, 2012
Last Updated: July 1, 2016
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Granulomatosis with Polyangiitis
Systemic Vasculitis
Microscopic Polyangiitis
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
Vascular Diseases
Cardiovascular Diseases
Autoimmune Diseases
Immune System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Antineoplastic Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents processed this record on September 23, 2016