A Phase IIa Study of Intravenous Rituximab in Pediatric Participants With Severe Granulomatosis W… (NCT01750697) | Clinical Trial Compass
CompletedPhase 2
A Phase IIa Study of Intravenous Rituximab in Pediatric Participants With Severe Granulomatosis With Polyangiitis (Wegener's) or Microscopic Polyangiitis
United States, Canada, France25 participantsStarted 2013-05-23
Plain-language summary
This Phase IIa international multicenter, open-label, uncontrolled study will evaluate the safety and pharmacokinetics of rituximab (MabThera/Rituxan) in pediatric participants with severe granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). Participants will receive rituximab 375 milligrams per square meter (mg/m\^2) intravenously (IV) on Days 1, 8, 15 and 22.
Who can participate
Age range
2 Years – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Diagnosis of GPA (EULAR/PRINTO/PRES 2008, Ankara criteria for childhood Wegener's granulomatosis) or diagnosis of MPA (according to the Chapel Hill Consensus Conference)
* Newly diagnosed participants or participants 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 Birmingham Vasculitis Activity Score for Wegener's Granulomatosis \[BVAS/WG\] items or disease severe enough to require treatment with cyclophosphamide)
* For participants of reproductive potential (males and females), use of reliable means of contraception throughout the study participation
* For all eligible participants mandatory prophylactic treatment for Pneumocystis jirovecii infection
Exclusion Criteria:
* Diagnosis of Churg-Strauss syndrome, as defined by the Chapel Hill Consensus Conference
* 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
* Incomplete recovery from recent surgery or less than (\<) 12 weeks since surgery prior to baseline or planned within 24 weeks of baseline
* Lack of peripheral venous access
* Pregnancy or breast-feeding
* Evidence of other significant uncontrolled concomitant disease, or of disorder or condition that, in the investigator's opinion, would preclude or interfere with particip…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percentage of Participants With Adverse Events (AEs), Including Serious AEs
Timeframe: Baseline (Day 1) up to last visit (1.5-5 years)
2
Pharmacokinetics: Rituximab Clearance (CL)
Timeframe: From Day 1 to Day 180
3
Pharmacokinetics: Volume of Distribution (Vd) of Rituximab