Ibrutinib With Rituximab in Adults With Waldenström's Macroglobulinemia

NCT ID: NCT02165397

Last Updated: 2021-03-03

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

181 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-07-07

Study Completion Date

2019-11-07

Brief Summary

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The purpose of this study is to evaluate the safety and efficacy of ibrutinib in combination with rituximab in participants with Waldenström's macroglobulinemia (WM).

Detailed Description

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Conditions

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Waldenström's Macroglobulinemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Randomized Study (Ibrutinib + Rituximab)

Ibrutinib: 420 mg (3 capsules x 140 mg) orally administered daily beginning from Day 1. Rituximab: 375 mg/m\^2 intravenous (IV) per package insert weekly for four consecutive weeks, followed by a second four-weekly rituximab course after a three-month interval.

Group Type EXPERIMENTAL

Ibrutinib

Intervention Type DRUG

Participants will receive 420 mg of Ibrutinib orally.

Rituximab

Intervention Type DRUG

Participants will receive rituximab 375 mg/m\^2 IV.

Randomized Study (Placebo + Rituximab)

Placebo: 3 capsules of placebo orally administered daily beginning from Day 1. Rituximab: 375 mg/m\^2 IV per package insert weekly for four consecutive weeks, followed by a second four-weekly rituximab course after a three-month interval.

Group Type EXPERIMENTAL

Placebo

Intervention Type DRUG

Participants will receive placebo capsules orally.

Rituximab

Intervention Type DRUG

Participants will receive rituximab 375 mg/m\^2 IV.

Open-Label Substudy (Ibrutinib)

Ibrutinib: 420 mg (3 capsules) orally administered daily beginning from Day 1.

Group Type EXPERIMENTAL

Ibrutinib

Intervention Type DRUG

Participants will receive 420 mg of Ibrutinib orally.

Interventions

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Ibrutinib

Participants will receive 420 mg of Ibrutinib orally.

Intervention Type DRUG

Placebo

Participants will receive placebo capsules orally.

Intervention Type DRUG

Rituximab

Participants will receive rituximab 375 mg/m\^2 IV.

Intervention Type DRUG

Other Intervention Names

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PCI-32765 Rituxan

Eligibility Criteria

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Inclusion Criteria

* Untreated or previously treated for WM. Previously treated subjects must have either documented disease progression or had no response (stable disease) to the most recent treatment regimen
* Centrally confirmed clinicopathological diagnosis of WM
* Measurable disease defined as serum monoclonal immunoglobulin M (IgM) \>0.5 g/dL
* Symptomatic disease meeting at least 1 of the recommendations from the Second International Workshop on Waldenström Macroglobulinemia for requiring treatment
* Hematology and biochemical values within protocol-defined limits
* Men and women ≥ 18 years of age
* Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2

Exclusion Criteria

* Known involvement of the central nervous system by WM
* Disease that is refractory to the last prior rituximab-containing therapy defined as either

* Relapse after the last rituximab-containing therapy \< 12 months since last dose of rituximab, OR
* Failure to achieve at least a minor response (MR) after the last rituximab-containing therapy If the subject meets this exclusion criterion and therefore is excluded from the main randomized study, participation in the non randomized substudy (Arm C) may be considered
* Rituximab treatment within the last 12 months before the first dose of study drug
* Known anaphylaxis or (immunoglobulin E) IgE-mediated hypersensitivity to murine proteins or to any component of rituximab
* Prior exposure to ibrutinib or other Bruton's tyrosine kinase (BTK) inhibitors
* Known bleeding disorders (eg, von Willebrand's disease) or hemophilia
* History of stroke or intracranial hemorrhage within 12 months prior to enrollment.
* Any uncontrolled active systemic infection.
* Any life-threatening illness, medical condition, or organ system dysfunction that, in the investigator's opinion, could compromise the subject's safety or put the study outcomes at undue risk.
* Currently active, clinically significant cardiovascular disease
* Requires treatment with a strong cytochrome P450 (CYP) 3A inhibitor

Eligibility Criteria for Open-label Substudy Treatment Arm C


* Relapse after the last rituximab-containing therapy \<12 months since last dose of rituximab, OR
* Failure to achieve at least a MR after the last rituximab-containing therapy.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Janssen Research & Development, LLC

INDUSTRY

Sponsor Role collaborator

Pharmacyclics LLC.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Bernhard Hauns, MD

Role: STUDY_DIRECTOR

Pharmacyclics LLC.

Locations

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University of California Los Angeles

Los Angeles, California, United States

Site Status

Stanford Cancer Center

Palo Alto, California, United States

Site Status

Colorado Blood Cancer Institute

Denver, Colorado, United States

Site Status

Emory University Hospital

Atlanta, Georgia, United States

Site Status

Northwestern Memorial Hospital

Chicago, Illinois, United States

Site Status

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status

Weill Cornell Medical Center

New York, New York, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

The Canberra Hospital

Garran, Australian Capital Territory, Australia

Site Status

Concord Repartriation General Hospital

Concord, New South Wales, Australia

Site Status

Flinders Medical Center

Bedford Park, South Australia, Australia

Site Status

Peter MacCallum Cancer Center

Melbourne, Victoria, Australia

Site Status

Cross Cancer Institute

Edmonton, Alberta, Canada

Site Status

Queen Elizabeth II Health Sciences Center

Halifax, Nova Scotia, Canada

Site Status

Princess Margaret Hospital

Toronto, Ontario, Canada

Site Status

McGill University Health Center

Montreal, Quebec, Canada

Site Status

Institut Paoli-Calmettes

Marseille, Bouches-du-Rhône, France

Site Status

Centre Hospitalier de Saint Brieuc Hopital Yves le Foll

Saint-Brieuc, Finistère, France

Site Status

Hôtel Dieu

Nantes, Loire-Atlantique, France

Site Status

CHU de Nancy-Hopital Brabois Adulte

Vandœuvre-lès-Nancy, Meurthe-et-Moselle, France

Site Status

Hôpital Claude Huriez

Lille, Nord, France

Site Status

CHU Estaing

Clermont-Ferrand, Puy-de-Dôme, France

Site Status

Centre Hospitalier Lyon Sud

Pierre-Bénite, Rhône, France

Site Status

Hopital Henri Mondor

Créteil, , France

Site Status

Hôpital Saint Louis

Paris, , France

Site Status

Groupe Hospitalier Pitié Salpétrière

Paris, , France

Site Status

Stauferklinikum Schwäbisch Gmünd

Mutlangen, Baden-Wurttemberg, Germany

Site Status

Universitätsmedizin der Johannes Gutenberg-Universität Mainz

Mainz, Rhineland-Palatinate, Germany

Site Status

Universität Des Saarlandes

Homburg, Saarland, Germany

Site Status

DIAKO Evangelische Diakonie Krankenhaus gGmbH

Bremen, , Germany

Site Status

LMU Klinikum der Universität München

München, , Germany

Site Status

University General Hospital of Patras

Pátrai, Achaia, Greece

Site Status

Alexandra Hospital

Athens, Attica, Greece

Site Status

University General Hospital of Thessaloniki "AHEPA"

Thessaloniki, Macedonia, Greece

Site Status

Laiko General Hospital of Athens

Athens, , Greece

Site Status

Azienda Ospedaliera Città della Salute e della Scienza di Torino

Turin, Piedmont, Italy

Site Status

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

ASST Grande Ospedale Metropolitano Niguarda

Milan, , Italy

Site Status

ASST di Pavia - Fondazione IRCCS Policlinico San Matteo di Pavia

Pavia, , Italy

Site Status

Azienda Ospedaliero Universitaria Santa Maria della Misericordia di Udine

Udine, , Italy

Site Status

Hospital Universitari Germans Trias i Pujol

Badalona, Barcelona, Spain

Site Status

Hospital Universitario de Salamanca

Salamanca, Castille and León, Spain

Site Status

Hospital Clinic de Barcelona

Barcelona, , Spain

Site Status

Hospital de La Santa Creu i Sant Pau

Barcelona, , Spain

Site Status

Hospital Universitario Infanta Leonor

Madrid, , Spain

Site Status

Royal Bournemouth Hospital

Bournemouth, Dorset, United Kingdom

Site Status

Countries

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United States Australia Canada France Germany Greece Italy Spain United Kingdom

References

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Dimopoulos MA, Tedeschi A, Trotman J, Garcia-Sanz R, Macdonald D, Leblond V, Mahe B, Herbaux C, Tam C, Orsucci L, Palomba ML, Matous JV, Shustik C, Kastritis E, Treon SP, Li J, Salman Z, Graef T, Buske C; iNNOVATE Study Group and the European Consortium for Waldenstrom's Macroglobulinemia. Phase 3 Trial of Ibrutinib plus Rituximab in Waldenstrom's Macroglobulinemia. N Engl J Med. 2018 Jun 21;378(25):2399-2410. doi: 10.1056/NEJMoa1802917. Epub 2018 Jun 1.

Reference Type BACKGROUND
PMID: 29856685 (View on PubMed)

Dimopoulos MA, Trotman J, Tedeschi A, Matous JV, Macdonald D, Tam C, Tournilhac O, Ma S, Oriol A, Heffner LT, Shustik C, Garcia-Sanz R, Cornell RF, de Larrea CF, Castillo JJ, Granell M, Kyrtsonis MC, Leblond V, Symeonidis A, Kastritis E, Singh P, Li J, Graef T, Bilotti E, Treon S, Buske C; iNNOVATE Study Group and the European Consortium for Waldenstrom's Macroglobulinemia. Ibrutinib for patients with rituximab-refractory Waldenstrom's macroglobulinaemia (iNNOVATE): an open-label substudy of an international, multicentre, phase 3 trial. Lancet Oncol. 2017 Feb;18(2):241-250. doi: 10.1016/S1470-2045(16)30632-5. Epub 2016 Dec 10.

Reference Type BACKGROUND
PMID: 27956157 (View on PubMed)

Guijosa A, Ramirez-Gamero A, Sarosiek S, Branagan AR, von Keudell G, Treon SP, Castillo JJ. Ibrutinib plus rituximab vs ibrutinib monotherapy in patients with Waldenstrom macroglobulinemia: a pooled analysis. Blood Adv. 2025 Sep 23;9(18):4705-4715. doi: 10.1182/bloodadvances.2025016536.

Reference Type DERIVED
PMID: 40674744 (View on PubMed)

Buske C, Tedeschi A, Trotman J, Garcia-Sanz R, MacDonald D, Leblond V, Mahe B, Herbaux C, Matous JV, Tam CS, Heffner LT, Varettoni M, Palomba ML, Shustik C, Kastritis E, Treon SP, Ping J, Hauns B, Arango-Hisijara I, Dimopoulos MA. Plain Language Summary of the iNNOVATE study: ibrutinib plus rituximab is well-tolerated and effective in people with Waldenstrom's macroglobulinemia. Future Oncol. 2023 Feb;19(5):345-353. doi: 10.2217/fon-2022-1015. Epub 2023 Feb 23.

Reference Type DERIVED
PMID: 36815271 (View on PubMed)

Buske C, Tedeschi A, Trotman J, Garcia-Sanz R, MacDonald D, Leblond V, Mahe B, Herbaux C, Matous JV, Tam CS, Heffner LT, Varettoni M, Palomba ML, Shustik C, Kastritis E, Treon SP, Ping J, Hauns B, Arango-Hisijara I, Dimopoulos MA. Ibrutinib Plus Rituximab Versus Placebo Plus Rituximab for Waldenstrom's Macroglobulinemia: Final Analysis From the Randomized Phase III iNNOVATE Study. J Clin Oncol. 2022 Jan 1;40(1):52-62. doi: 10.1200/JCO.21.00838. Epub 2021 Oct 4.

Reference Type DERIVED
PMID: 34606378 (View on PubMed)

Trotman J, Buske C, Tedeschi A, Matous JV, MacDonald D, Tam CS, Tournilhac O, Ma S, Treon SP, Oriol A, Ping J, Briso EM, Arango-Hisijara I, Dimopoulos MA. Single-Agent Ibrutinib for Rituximab-Refractory Waldenstrom Macroglobulinemia: Final Analysis of the Substudy of the Phase III InnovateTM Trial. Clin Cancer Res. 2021 Nov 1;27(21):5793-5800. doi: 10.1158/1078-0432.CCR-21-1497. Epub 2021 Aug 11.

Reference Type DERIVED
PMID: 34380643 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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PCYC-1127-CA

Identifier Type: -

Identifier Source: org_study_id

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