Dose Escalation and Dose Expansion Study of Tirabrutinib in Combination With Other Targeted Anti-cancer Therapies in Adults With B-cell Malignancies

NCT ID: NCT02457598

Last Updated: 2025-06-17

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

203 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-06-16

Study Completion Date

2024-09-25

Brief Summary

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The primary objectives of this study are to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary efficacy of tirabrutinib (ONO/GS-4059) in combination with other targeted anti-cancer therapies and to evaluate the long-term safety of tirabrutinib as a monotherapy and in combination with other targeted anti-cancer therapies in adults with relapsed or refractory B-cell lymphoproliferative malignancies.

This study consists of three parts: Dose Escalation, Dose Expansion, and Long-term Safety Monitoring. During the Dose Escalation phase, participants will be sequentially enrolled in a standard 3 + 3 dose escalation study design, to receive oral tirabrutinib combined with idelalisib entospletinib +/- obinutuzumab. The Dose Expansion Phase will enroll additional participants with a single B-cell lymphoproliferative malignancy disease type to further evaluate efficacy, safety, tolerability, PK, and pharmacodynamics. The Long-term Safety Monitoring phase will evaluate the long-term safety of tirabrutinib both as a monotherapy and in combination with other anti-cancer therapies. As of Amendment 9, all participants currently on the study who have no clinical evidence of disease progression will transition into long-term safety monitoring. Participants from the ongoing Study GS-US-401-1787 and participants who came off Study GS-US-401-1757 and Study GS-US-401-1787 but continued to receive treatment via named patient use (or individual expanded use) will be enrolled into the long-term safety monitoring group (Group VI). Participants enrolled in Group VI will continue the same treatment regimen in Study GS-US-401-1787 or named patient use (or individual expanded use). As of Protocol Amendment 8, the maximum treatment duration for any participant is an additional 6 years from the date of this amendment (ie. until November 2025). As of Amendment 9, entospletinib will be provided until 31 December 2020 to participants who are currently receiving entospletinib. Participants treated with entospletinib as part of a combination regimen with tirabrutinib will stop receiving entospletinib by 31 December 2020 but may continue to be treated with tirabrutinib monotherapy. Idelalisib will be provided as 50 mg tablets until 31 December 2020 and 100 mg tablets until study completion. Participants assigned to the 50 mg tablet will be given the option, at the investigator's discretion, to switch to 100 mg once daily idelalisib dose.

Detailed Description

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Conditions

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B-cell Malignancies

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Enrollment and treatment were parallel in different combinations used in the study.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Combination I (Tirabrutinib 20 mg QD+ Idelalisib 50 mg BID)

Participants will receive tirabrutinib 20 mg tablet once daily (QD) for up to 209 weeks and idelalisib 50 mg tablet twice daily (BID) orally for up to 120 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Idelalisib

Intervention Type DRUG

Tablets administered orally twice daily

Combination I (Tirabrutinib 40 mg QD+ Idelalisib 50 mg BID)

Participants will receive tirabrutinib 40 mg tablet QD for up to 251 weeks and idelalisib 50 mg tablet BID orally, for up to 245 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Idelalisib

Intervention Type DRUG

Tablets administered orally twice daily

Combination I (Tirabrutinib 80 mg QD+ Idelalisib 50 mg BID)

Participants will receive tirabrutinib 80 mg tablet QD for up to 212 weeks and idelalisib 50 mg tablet BID orally, for up to 26 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Idelalisib

Intervention Type DRUG

Tablets administered orally twice daily

Combination I (Tirabrutinib 80 mg QD+ Idelalisib 100 mg QD)

Participants will receive tirabrutinib 80 mg tablet QD for up to 227 weeks and idelalisib 100 mg tablet QD orally, for up to 227 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Idelalisib

Intervention Type DRUG

Tablets administered orally twice daily

Combination I (Tirabrutinib 160 mg QD+ Idelalisib 100 mg QD)

Participants will receive tirabrutinib 160 mg tablet QD for up to 151 weeks and idelalisib 100 mg tablet QD orally, for up to 151 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Idelalisib

Intervention Type DRUG

Tablets administered orally twice daily

Combination I (Tirabrutinib 20 mg BID+ Idelalisib 50 mg BID)

Participants will receive tirabrutinib 20 mg tablet BID for up to 207 weeks and idelalisib 50 mg tablet BID orally, for up to 207 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Idelalisib

Intervention Type DRUG

Tablets administered orally twice daily

Combination II (Tirabrutinib 40 mg QD+ Entospletinib 200 mg QD)

Participants will receive tirabrutinib 40 mg tablet QD for up to 250 weeks and entospletinib 200 mg tablet QD orally, for up to 250 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Entospletinib

Intervention Type DRUG

Tablets administered orally

Combination II (Tirabrutinib 80 mg QD+ Entospletinib 200 mg QD)

Participants will receive tirabrutinib 80 mg tablet QD for up to 234 weeks and entospletinib 200 mg tablet QD orally, for up to 220 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Entospletinib

Intervention Type DRUG

Tablets administered orally

Combination II (Tirabrutinib 150 mg QD+ Entospletinib 200 mg QD)

Participants will receive tirabrutinib 150 mg tablet QD for up to 103 weeks and entospletinib 200 mg tablet QD orally, for up to 88 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Entospletinib

Intervention Type DRUG

Tablets administered orally

Combination II (Tirabrutinib 40 mg QD+ Entospletinib 400 mg QD)

Participants will receive tirabrutinib 40 mg tablet QD for up to 119 weeks and entospletinib 400 mg tablet QD orally, for up to 119 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Entospletinib

Intervention Type DRUG

Tablets administered orally

Combination II (Tirabrutinib 80 mg QD+ Entospletinib 400 mg QD)

Participants will receive tirabrutinib 80 mg tablet QD for up to 232 weeks and entospletinib 400 mg tablet QD orally, for up to 229 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Entospletinib

Intervention Type DRUG

Tablets administered orally

Combination II (Tirabrutinib 160 mg QD+ Entospletinib 400 mg QD)

Participants will receive tirabrutinib 160 mg tablet QD for up to 44 weeks and entospletinib 400 mg tablet QD orally, for up to 44 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Entospletinib

Intervention Type DRUG

Tablets administered orally

Combination III (Tirabrutinib 80 mg QD+ Idelalisib 100 mg QD+ obinutuzumab 1000 mg)

Participants will receive tirabrutinib 80 mg tablet QD for up to 199 weeks; Idelalisib 100 mg tablet QD orally for up to 199 weeks, and single dose of obinutuzumab 1000 mg intravenously (IV) for up to 22 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Idelalisib

Intervention Type DRUG

Tablets administered orally twice daily

Obinutuzumab

Intervention Type DRUG

Administered intravenously

Combination IV (Tirabrutinib 80 mg QD+ entospletinib 400 mg QD+ obinutuzumab 1000 mg)

Participants will receive tirabrutinib 80 mg tablet QD for up to 196 weeks; entospletinib 400 mg tablet QD orally for up to 196 weeks and single dose of obinutuzumab 1000 mg IV for up to 22 weeks. Both drugs will be administered for 28-day cycles.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Entospletinib

Intervention Type DRUG

Tablets administered orally

Obinutuzumab

Intervention Type DRUG

Administered intravenously

Group V Single Agent Tirabrutinib

Participants will receive tirabrutinib 80 mg tablet QD orally, for each 28-day cycle up to 194 weeks.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Group VI Single Agent Tirabrutinib: CLL

Participants with CLL who received tirabrutinib in this or a previous tirabrutinib study will continue to receive tirabrutinib at the original dose (between the range of 40 - 400 mg), tablet QD, for each 28-day cycle up to 441 weeks. Participants will be monitored for the long-term safety of tirabrutinib in this group.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Group VI Single Agent Tirabrutinib: NHL

Participants with NHL who received tirabrutinib in this or a previous tirabrutinib study will continue to receive tirabrutinib at the original dose (between the range of 60 - 480 mg), tablet QD, for each 28-day cycle up to 441 weeks. Participants will be monitored for the long-term safety of tirabrutinib in this group.

Group Type EXPERIMENTAL

Tirabrutinib

Intervention Type DRUG

Capsules or tablets administered orally

Interventions

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Tirabrutinib

Capsules or tablets administered orally

Intervention Type DRUG

Idelalisib

Tablets administered orally twice daily

Intervention Type DRUG

Entospletinib

Tablets administered orally

Intervention Type DRUG

Obinutuzumab

Administered intravenously

Intervention Type DRUG

Other Intervention Names

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ONO/GS-4059 Zydelig® GS-1101 CAL-101 GS-9973

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of follicular lymphoma (FL), marginal zone lymphoma (MZL), small lymphocytic lymphoma (SLL), chronic lymphocytic leukemia (CLL) (meeting International Workshop on Chronic Lymphocytic Leukemia (IWCLL) Criteria 2008), mantle cell lymphoma (MCL), Waldenstrom's macroglobulinemia (WM), or non-germinal center B-cell lymphoma (GCB) diffuse large B-cell lymphoma (DLBCL) as documented by medical records on World Health Organization (WHO) criteria
* Prior treatment for FL, MZL, SLL, MCL, WM with ≥ 2 or for CLL or non-GCB DLBCL ≥ 1 chemotherapy-based or immunotherapy-based regimen, and not transplant eligible and have had either progressive disease (PD) or no response to previous treatment
* For diseases other than Waldenstrom's macroglobulinemia (WM), presence of radiographically measurable presence of ≥ 1 lesion that measures ≥ 2.0 cm in the longest dimension (LD) and ≥ 1.0 cm in the longest perpendicular dimension (LPD)
* Eastern Cooperative Oncology Group (ECOG) ≤ 2
* Platelets ≥ 50 x 10\^9/L; Hb ≥ 8.0 g/dL; absolute neutrophil count (ANC) ≥ 1.0 x 10\^9/L
* Without transfusion and growth factors within 7 days
* Aspartate transaminase/alanine transaminase (AST/ALT) ≤ 2.5 x upper limit of normal (ULN)
* Total bilirubin ≤ 1.5 x ULN
* Creatinine ≤ 1.5 x ULN or creatinine clearance (CrCl) ≥ 60 mL/min
* Not pregnant
* Willingness and ability to comply with protocol-specified Pneumocystis jirovecii pneumonia (PJP) prophylaxis
* Long-term Safety Monitoring group only (Group VI):

* Currently enrolled in Study GS-US-401-1787 or previously enrolled in Study GS-US-401-1757 or Study GS-US-401-1787 and currently receiving continued treatment via named patient use
* Continuing to benefit from the current treatment regimen in the opinion of the investigator/treating physician

Exclusion Criteria

* Hepatitis B surface antigen (HBsAG) positive or hepatitis B core antibody positive
* Hepatitis C virus (HCV) antibody positive
* History of long QT syndrome or whose corrected QT(QTc) interval measured (Fridericia method) at screening is prolonged (\>450 ms)
* Long-term Safety Monitoring group only (Group VI):

* Evidence of clinical or radiological disease progression
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Gilead Sciences

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Gilead Study Director

Role: STUDY_DIRECTOR

Gilead Sciences

Locations

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Georgetown University Medical Center

Washington D.C., District of Columbia, United States

Site Status

Indiana University Health Goshen Center for Cancer Care

Goshen, Indiana, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

CHRU de Lille, Hopital Claude Huriez

Lille, , France

Site Status

Hopital Saint Eloi

Montpellier, , France

Site Status

Chu Haut Leveque

Pessac, , France

Site Status

Centre Hospitalier de Lyon Sud

Pierre-Bénite, , France

Site Status

Institut Universitaire du Cancer-Oncopole I.U.C.T-O

Toulouse, , France

Site Status

Cambridge University Hospitals NHS Foundation Trust

Cambridge, , United Kingdom

Site Status

Cardiff and Vale Health Board, Clinical Research Facility

Cardiff, , United Kingdom

Site Status

Leeds Teaching Hosptials NHS Trust, Dept of Haematology

Leeds, , United Kingdom

Site Status

University Hospitals of Leicester NHS Trust

Leicester, , United Kingdom

Site Status

The Christie NHS Foundation Trust

Manchester, , United Kingdom

Site Status

Plymouth Hospitals NHS Trust

Plymouth, , United Kingdom

Site Status

University Hospital Southampton NHS Foundation Trust

Southampton, , United Kingdom

Site Status

Countries

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United States France United Kingdom

References

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Danilov AV, Herbaux C, Walter HS, Hillmen P, Rule SA, Kio EA, Karlin L, Dyer MJS, Mitra SS, Yi PC, Humeniuk R, Huang X, Zhou Z, Bhargava P, Jurgensmeier JM, Fegan CD. Phase Ib Study of Tirabrutinib in Combination with Idelalisib or Entospletinib in Previously Treated Chronic Lymphocytic Leukemia. Clin Cancer Res. 2020 Jun 15;26(12):2810-2818. doi: 10.1158/1078-0432.CCR-19-3504. Epub 2020 Mar 10.

Reference Type BACKGROUND
PMID: 32156743 (View on PubMed)

Morschhauser F, Dyer MJS, Walter HS, Danilov AV, Ysebaert L, Hodson DJ, Fegan C, Rule SA, Radford J, Cartron G, Bouabdallah K, Davies AJ, Spurgeon S, Rajakumaraswamy N, Li B, Humeniuk R, Huang X, Bhargava P, Jurgensmeier JM, Salles G. Phase 1b study of tirabrutinib in combination with idelalisib or entospletinib in previously treated B-cell lymphoma. Leukemia. 2021 Jul;35(7):2108-2113. doi: 10.1038/s41375-020-01108-x. Epub 2020 Dec 17. No abstract available.

Reference Type BACKGROUND
PMID: 33328591 (View on PubMed)

Salles, G., Dyer, MJS,. Hodson, DJ., et-al. Updated Preliminary Results of a Phase 1b Dose Escalation and Dose Expansion Study of Tirabrutinib in Combination with Entospletinib in Patients with B-Cell Lymphoma. Blood 2018; 132 (Supplement 1): 5344.

Reference Type BACKGROUND

Walter HS, Rule SA, Dyer MJ, Karlin L, Jones C, Cazin B, Quittet P, Shah N, Hutchinson CV, Honda H, Duffy K, Birkett J, Jamieson V, Courtenay-Luck N, Yoshizawa T, Sharpe J, Ohno T, Abe S, Nishimura A, Cartron G, Morschhauser F, Fegan C, Salles G. A phase 1 clinical trial of the selective BTK inhibitor ONO/GS-4059 in relapsed and refractory mature B-cell malignancies. Blood. 2016 Jan 28;127(4):411-9. doi: 10.1182/blood-2015-08-664086. Epub 2015 Nov 5.

Reference Type BACKGROUND
PMID: 26542378 (View on PubMed)

Walter HS, Salles GA, Dyer MJ. New Agents to Treat Chronic Lymphocytic Leukemia. N Engl J Med. 2016 Jun 2;374(22):2185-6. doi: 10.1056/NEJMc1602674. No abstract available.

Reference Type BACKGROUND
PMID: 27248633 (View on PubMed)

Morschhauser, F., Radford, J., Ysebaert, L., et-al. Updated Preliminary Results of a Phase 1b Dose Escalation and Dose Expansion Study of Tirabrutinib in Combination with Idelalisib in Patients with B-Cell Lymphoma. Blood 2018; 132 (Supplement 1): 5345.

Reference Type BACKGROUND

Walter HS, Jayne S, Rule SA, Cartron G, Morschhauser F, Macip S, Karlin L, Jones C, Herbaux C, Quittet P, Shah N, Hutchinson CV, Fegan C, Yang Y, Mitra S, Salles G, Dyer MJS. Long-term follow-up of patients with CLL treated with the selective Bruton's tyrosine kinase inhibitor ONO/GS-4059. Blood. 2017 May 18;129(20):2808-2810. doi: 10.1182/blood-2017-02-765115. Epub 2017 Apr 4. No abstract available.

Reference Type BACKGROUND
PMID: 28377400 (View on PubMed)

Yahiaoui A, Meadows SA, Sorensen RA, Cui ZH, Keegan KS, Brockett R, Chen G, Queva C, Li L, Tannheimer SL. PI3Kdelta inhibitor idelalisib in combination with BTK inhibitor ONO/GS-4059 in diffuse large B cell lymphoma with acquired resistance to PI3Kdelta and BTK inhibitors. PLoS One. 2017 Feb 8;12(2):e0171221. doi: 10.1371/journal.pone.0171221. eCollection 2017.

Reference Type DERIVED
PMID: 28178345 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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Other Identifiers

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2015-000834-30

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GS-US-401-1757

Identifier Type: -

Identifier Source: org_study_id

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