Rituximab for the Otolaryngologic Manifestations of Granulomatosis With Polyangiitis (RENTGPA)
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|ClinicalTrials.gov Identifier: NCT02626845|
Recruitment Status : Terminated (Slow recruitment)
First Posted : December 10, 2015
Last Update Posted : March 22, 2018
|Condition or disease||Intervention/treatment||Phase|
|Granulomatosis With Polyangiitis (Wegener's Granulomatosis)||Drug: Rituximab Other: Placebo||Phase 4|
Patients with GPA and active ENT disease in at least two ENT domains, as defined after endoscopic visualization of the upper airway and audiometric evaluation by a single otolaryngologist using a validated GPA ENT disease activity score, will be eligible for inclusion. ENT disease may be new, grumbling or relapsing.
All patients entering the trial will receive standard induction therapy with rituximab (375mg/m2 per week x 4). At week 16, patients will be randomized to receive maintenance rituximab (1000mg) every 4 months or placebo infusions. The primary outcome will be assessed at week 52. Patients will be treated with a standardized prednisone taper according to whether they had severe or limited disease at study entry, prednisone taper will be completed at week 16.
The investigators plan to enroll 28 patients who will be randomized in a 1:1 fashion to rituximab or placebo. The investigators estimate accrual of these subjects will take 18 months from study initiation. Once enrolled, subjects are followed for 52 weeks until the primary endpoint is assessed.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Investigator, Outcomes Assessor)|
|Official Title:||Rituximab for the Otolaryngologic Manifestations of Granulomatosis With Polyangiitis|
|Study Start Date :||December 2015|
|Actual Primary Completion Date :||July 2017|
|Actual Study Completion Date :||July 2017|
Active Comparator: Rituximab Arm
All subjects in this arm will receive standard of care induction therapy, and then will receive two additional rituximab infusions at week 16 and week 32.
Standard of care induction with Rituximab: 375mg/m2 weekly x 4 weeks. Once randomized, the rituximab dose will be 1000mg IV every 4 months x 2.
Other Name: Rituxan
Placebo Comparator: Placebo Arm
All subjects in this arm will receive standard of care induction therapy, and then will receive two additional placebo infusions at week 16 and week 32.
Will be given at two time-points (week 16 and week 32) to subjects in the Placebo Arm.
- Proportion of patients in ENT remission without relapse at week 52 in each treatment group. [ Time Frame: Assessed at week 52 ]ENT remission is defined as a GPA ENT disease activity score of 0.
- Comparison of mean ENT disease activity scores between treatment arms [ Time Frame: Assessed at week 52 ]
- Cumulative steroid dose [ Time Frame: Assessed at week 52 ]
- Duration of steroid free remission [ Time Frame: Assessed at week 52 ]
- Proportion of subject in remission without relapse and completed steroid taper [ Time Frame: Assessed at week 52 ]
- Quality of Life as measured by the SNOT-22 Questionnaire [ Time Frame: Assessed at week 0, 16, and 52 ]
- Change in VDI in the ENT domain [ Time Frame: Assessed at week 52 ]
- Number of surgical procedures in the ENT domain required during the study period [ Time Frame: Assessed at week 52 ]
- Number of ENT flares as measured by the ENT GPA DAS [ Time Frame: Assessed at week 52 ]
- Number of GPA flares as measured by BVAS-WG [ Time Frame: Assessed at week 52 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02626845
|United States, New York|
|Hospital for Special Surgery|
|New York, New York, United States, 100214898|
|Principal Investigator:||Robert F Spiera, MD||Hospital for Special Surgery, New York|