CLINICAL TRIALS PROFILE FOR IBRUTINIB

505(b)(2) Clinical Trials for IBRUTINIB

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT02436707 ↗	Novel Combination Therapy in the Treatment of Relapsed and Refractory Aggressive B-Cell Lymphoma	Recruiting	Janssen, LP	Phase 2	2015-05-05	The purpose of this study is to find out what effects new combinations of treatment will have this disease. New promising treatment strategies will be added to this study as they are available to be compared against the standard treatment.
New Combination	NCT02436707 ↗	Novel Combination Therapy in the Treatment of Relapsed and Refractory Aggressive B-Cell Lymphoma	Recruiting	Karyopharm Therapeutics Inc	Phase 2	2015-05-05	The purpose of this study is to find out what effects new combinations of treatment will have this disease. New promising treatment strategies will be added to this study as they are available to be compared against the standard treatment.
New Combination	NCT02436707 ↗	Novel Combination Therapy in the Treatment of Relapsed and Refractory Aggressive B-Cell Lymphoma	Recruiting	Roche Pharma AG	Phase 2	2015-05-05	The purpose of this study is to find out what effects new combinations of treatment will have this disease. New promising treatment strategies will be added to this study as they are available to be compared against the standard treatment.
New Combination	NCT02436707 ↗	Novel Combination Therapy in the Treatment of Relapsed and Refractory Aggressive B-Cell Lymphoma	Recruiting	Canadian Cancer Trials Group	Phase 2	2015-05-05	The purpose of this study is to find out what effects new combinations of treatment will have this disease. New promising treatment strategies will be added to this study as they are available to be compared against the standard treatment.
New Formulation	NCT02841150 ↗	Study to Assess the Bioequivalence of Ibrutinib 560- Milligram (mg) Tablet to Four 140 -mg IMBRUVICA Capsules	Completed	Janssen Research & Development, LLC	Phase 1	2016-06-01	The purpose of this study is to demonstrate the bioequivalence (BE) of a new formulation of ibrutinib to the marketed Imbruvica formulation in healthy adults under fasted conditions.
New Formulation	NCT02877225 ↗	Study to Assess the Bioequivalence of Ibrutinib 140 Milligram (mg) Tablet to 140 mg IMBRUVICA Capsule	Completed	Janssen Research & Development, LLC	Phase 1	2016-08-29	The purpose of this study is to demonstrate the bioequivalence (BE) of a new formulation of ibrutinib to the marketed Imbruvica formulation in healthy adults under fasted conditions.

All Clinical Trials for IBRUTINIB

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01217749 ↗	Efficacy and Safety Study of PCI-32765 Combine With Ofatumumab in CLL	Completed	Ohio State University	Phase 1/Phase 2	2010-12-01	The purpose of this study is to determine the efficacy and safety of a fixed-dose, daily regimen of orally administered PCI-32765 combined with ofatumumab in subjects with relapsed/refractory CLL/SLL and related diseases
NCT01217749 ↗	Efficacy and Safety Study of PCI-32765 Combine With Ofatumumab in CLL	Completed	Pharmacyclics LLC.	Phase 1/Phase 2	2010-12-01	The purpose of this study is to determine the efficacy and safety of a fixed-dose, daily regimen of orally administered PCI-32765 combined with ofatumumab in subjects with relapsed/refractory CLL/SLL and related diseases
NCT01236391 ↗	Safety and Efficacy of PCI-32765 in Participants With Relapsed/Refractory Mantle Cell Lymphoma (MCL)	Completed	Janssen Pharmaceuticals	Phase 2	2011-02-01	The primary objective of this study was to evaluate the efficacy of ibrutinib in participants with relapsed or refractory MCL. The secondary objective was to evaluate the safety of a fixed daily dosing regimen (560 mg daily) of PCI-32765 in this population.
NCT01236391 ↗	Safety and Efficacy of PCI-32765 in Participants With Relapsed/Refractory Mantle Cell Lymphoma (MCL)	Completed	Pharmacyclics LLC.	Phase 2	2011-02-01	The primary objective of this study was to evaluate the efficacy of ibrutinib in participants with relapsed or refractory MCL. The secondary objective was to evaluate the safety of a fixed daily dosing regimen (560 mg daily) of PCI-32765 in this population.
NCT01325701 ↗	Safety and Efficacy Study of a BTK Inhibitor in Subjects With Relapsed or Refractory Diffuse Large B-cell Lymphoma	Completed	Pharmacyclics LLC.	Phase 2	2011-05-01	The purpose of this study is to evaluate the efficacy of ibrutinib (PCI-32765) in relapsed/refractory de novo activated B-cell (ABC) and germinal-cell B-Cell (GCB) Diffuse Large B-cell Lymphoma (DLBCL).
NCT01478581 ↗	Study of the Bruton's Tyrosine Kinase Inhibitor in Subjects With Relapsed or Relapsed and Refractory Multiple Myeloma	Completed	Pharmacyclics LLC.	Phase 2	2012-03-01	The primary objective of this study is to determine the efficacy of PCI-32765, both as a single agent and in combination with dexamethasone, in subjects with relapsed or relapsed and refractory Multiple Myeloma (MM)
NCT01500733 ↗	PCI-32765 for Special Cases of Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma	Active, not recruiting	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2011-11-28	Background: - Chronic lymphocytic leukemia and small lymphocytic lymphoma (CLL/SLL) are types of blood or lymph node cancers that mostly affect the elderly. CLL/SLL both create abnormal white blood cells that hurt the immune system and make it more difficult to fight infections. These cancers are usually diagnosed after age 50; more than half of the people with CLL/SLL are over age 70. Elderly people often do not respond well to standard chemotherapy for CLL/SLL. They may have other health problems that make chemotherapy difficult. In addition, individuals who have a genetic abnormality called 17p deletion also do not respond well to standard treatments for CLL/SLL. Researchers want to test a new cancer treatment drug, PCI-32765, to see if it can treat CLL/SLL in these hard-to-treat groups. Objectives: - To see if PCI-32765 is a safe and effective treatment for CLL/SLL in older people and people with 17p deletion. Eligibility: - Individuals over 65 years of age who have CLL/SLL. - Individuals at least 18 years of age who have CLL/SLL and 17p deletion. Design: - Participants will be screened with a medical history, physical exam, and imaging studies. Blood and urine samples will be taken. Optional bone marrow and lymph node biopsies may also be taken. - Participants will take PCI-32765 capsules every day for 28 days (one cycle of treatment). Treatment will be monitored with frequent blood tests and clinic visits. - PCI-32765 will be given for six cycles of treatment. Those who benefit from the drug will continue to take it as long as there are no side effects and the disease does not progress. Those who do not benefit will stop treatment and have regular followup exams.

Clinical Trial Conditions for IBRUTINIB

Condition Name

Condition Name for IBRUTINIB
Intervention	Trials
Chronic Lymphocytic Leukemia	73
Mantle Cell Lymphoma	41
Small Lymphocytic Lymphoma	36
Lymphoma	20

Condition MeSH

Condition MeSH for IBRUTINIB
Intervention	Trials
Lymphoma	189
Leukemia, Lymphocytic, Chronic, B-Cell	137
Leukemia, Lymphoid	131
Leukemia	123

Clinical Trial Locations for IBRUTINIB

Trials by Country

Trials by Country for IBRUTINIB
Location	Trials
Spain	92
France	90
United Kingdom	88
Italy	88
China	82

Trials by US State

Trials by US State for IBRUTINIB
Location	Trials
California	101
New York	94
Texas	91
Ohio	85
New Jersey	66

Clinical Trial Progress for IBRUTINIB

Clinical Trial Phase

Clinical Trial Phase for IBRUTINIB
Clinical Trial Phase	Trials
PHASE4	1
PHASE2	12
PHASE1	8

Clinical Trial Status

Clinical Trial Status for IBRUTINIB
Clinical Trial Phase	Trials
Recruiting	115
Active, not recruiting	101
Completed	75

Clinical Trial Sponsors for IBRUTINIB

Sponsor Name

Sponsor Name for IBRUTINIB
Sponsor	Trials
Pharmacyclics LLC.	85
National Cancer Institute (NCI)	77
Janssen Research & Development, LLC	43

Sponsor Type

Sponsor Type for IBRUTINIB
Sponsor	Trials
Other	332
Industry	325
NIH	83

IBRUTINIB Market Analysis and Financial Projection

Last updated: April 23, 2026

Ibrutinib (Imbruvica) Clinical Trials Update, Market Analysis, and Projection

What is the current clinical-trials footprint for ibrutinib?

Ibrutinib is the first-in-class Bruton’s tyrosine kinase (BTK) inhibitor for multiple B-cell malignancies. The clinical program remains active through label expansion, sequencing/combination studies, and next-generation BTK inhibitor positioning.

Core indications with established clinical evidence (global):

• Mantle cell lymphoma (MCL) (after at least one prior therapy): accelerated-to-approval pathway historically; current practice increasingly uses BTK-based and BCL2-based combinations.
• Chronic lymphocytic leukemia (CLL) / Small lymphocytic lymphoma (SLL): frontline and relapsed settings; major use in chemo-free regimens.
• Waldenström macroglobulinemia (WM): combination and relapse sequencing.
• Marginal zone lymphoma (MZL): after anti-CD20 therapy and in relapsed settings.
• Graft-versus-host disease (cGVHD): ibrutinib has been investigated and used in specific settings where BTK signaling is implicated.
• Other lymphoid malignancies: ongoing studies have assessed combinations with anti-CD20 antibodies, BCL2 inhibitors, and other signaling agents.

Trials landscape (what is changing):

• The highest commercial impact studies are those that refine frontline sequencing (monotherapy vs fixed-duration combinations) and define depth-of-response and duration off therapy endpoints.
• Clinical development increasingly focuses on combination tolerability (cardiac safety signals, bleeding risk, infections) and long-term outcomes versus chemo-free standards.
• The competitive set has shifted from “BTK vs chemotherapy” toward “BTK plus fixed-duration regimens vs next-generation BTK inhibition strategies,” including covalent BTK alternatives and non-covalent BTK inhibitors.

Regulatory anchor for current use (US):

• Ibrutinib is marketed by AbbVie and Janssen under Imbruvica for multiple hematologic indications (FDA label basis across CLL/SLL, MCL, WM, MZL, and others). FDA label details are the binding reference for trial-to-market translation. Source: FDA prescribing information. [1]

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Which competitive dynamics drive market share risk for ibrutinib?

Ibrutinib faces a maturing BTK-inhibitor market where differentiators are safety profile, time-to-therapy discontinuation, and tolerability in older, comorbid populations.

Key competitive vectors:

1. Next-generation covalent BTK inhibitors
   • Aligned to improved tolerability and simplified dosing.
2. Non-covalent BTK inhibitors
   • Built for resistance circumvention, especially where C481 mutations and intolerance drive discontinuation.
3. Chemo-free combination regimens with fixed-duration intent
   • Shifts value from indefinite BTK monotherapy to combination protocols with defined stop rules and deeper molecular responses.

What this means for ibrutinib commercial trajectory:

• Ibrutinib’s share tends to hold best where clinicians prioritize proven long-term efficacy and where payer coverage and formulary status remain entrenched.
• Uptake pressure rises in geographies and centers adopting newer BTK agents and fixed-duration strategies that lower toxicity and reduce total time on therapy.

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Market analysis: where ibrutinib sits in current payer and prescriber reality

How large is the commercial opportunity today and how does it evolve?

Ibrutinib’s market is supported by multi-indication use in CLL/SLL, MCL, WM, and MZL. The addressable population is concentrated in older cohorts with frequent comorbidities, which is also where tolerability and drug discontinuation outcomes become high-leverage differentiators.

Market sizing approach used for projection (directional framework):

• Start with treated prevalence for each label indication.
• Apply BTK penetration (share of patients receiving a BTK inhibitor).
• Apply substitution rate from ibrutinib to newer BTK inhibitors and combinations.
• Apply dose intensity and duration-off-therapy reductions where combination protocols shorten treatment length.

Directional drivers:

• Frontline CLL/SLL remains the largest volume pool; value depends on whether regimens use continuous BTK therapy or transition to fixed-duration protocols.
• Relapsed/refractory MCL and WM sustain steady use but are exposed to increasing competition from newer BTK inhibitors and non-BTK strategies.
• MZL has smaller volume but can be sensitive to payer formulary changes when newer BTK options show improved tolerability.

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What are the pricing and reimbursement pressures likely to do to net sales?

Net sales are shaped by:

• Indication mix (frontline volume vs relapsed volume).
• Competition-driven formulary moves (tier placement changes).
• Patient out-of-pocket exposure under co-pay and PAP structures.
• Regional oncology procurement cycles.

Because ibrutinib is a widely used branded therapy, market value erosion typically follows a pattern:

• Initial stability in entrenched formularies.
• Gradual share dilution as newer BTK inhibitors gain guideline-based preference in frontline and fit/older-but-tolerant cohorts.
• Additional dilution if next-generation agents show clear tolerability advantages leading to lower discontinuation.

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Projections: base-case, downside, and upside

What is the likely ibrutinib revenue path over the next 3 to 5 years?

A full numerical forecast requires live paid-access financial models and latest consensus updates. The task here is to provide a projection structure tied to observable market mechanisms, not to invent figures.

Projection framework (how the revenue curve changes):

• Base-case: modest net sales decline driven by:
  • Share shift to next-generation BTK inhibitors in CLL/SLL frontline,
  • Offset from continued relapsed use in MCL/WM/MZL,
  • Continued clinical retention due to long track record and guideline inertia.
• Downside: faster loss of share if:
  • Payers expand formularies for newer BTK inhibitors,
  • Clinicians move toward fixed-duration combinations that reduce BTK monotherapy persistence,
  • Higher discontinuation pressure in real-world due to known ibrutinib tolerability issues.
• Upside: slower erosion if:
  • Combination protocols include ibrutinib with durable responses and manageable toxicity,
  • Real-world persistence stays higher than expected versus competitive alternatives,
  • Stronger-than-expected uptake in additional sub-sets from ongoing studies.

Operational milestones that determine which curve dominates:

• Guideline updates and payer medical policy changes in major markets.
• Evidence maturity from late-phase combination trials that can move practice toward or away from continuous BTK.
• Conversion rates of relapsed patients from older BTK exposures to other agents.

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Clinical value: what endpoints matter commercially

Which clinical outcomes most affect adoption of ibrutinib?

Ibrutinib adoption and retention depend on:

• Overall response rate (ORR) and duration of response (DOR)
• Progression-free survival (PFS) and time to progression
• Overall survival (OS) where available
• Safety-driven discontinuation: atrial fibrillation/flutter, bleeding events, infection rates
• Treatment duration and “time to next therapy” in real-world practice

Why this drives market dynamics:

• Faster-moving frontline standards compress treatment length and shift the value proposition from indefinite therapy to fixed-duration or response-guided strategies.
• Safety profile affects persistence, which directly impacts volume and net sales.

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Key Takeaways

• Ibrutinib retains a broad, entrenched multi-indication footprint anchored by FDA labeling. [1]
• Market value is increasingly driven by substitution risk to next-generation BTK inhibitors and practice shift to fixed-duration or combination strategies that reduce continuous BTK exposure.
• Clinical trial activity remains commercially relevant mainly when it changes frontline sequencing, improves durability of response, or shifts treatment duration and discontinuation rates.
• Near-term revenue trajectory is best modeled by share dilution versus persistence across CLL/SLL frontline and relapsed MCL/WM/MZL volumes rather than by new molecular breakthroughs.

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FAQs

1) What are the main cancers where ibrutinib is used today?

CLL/SLL, MCL, WM, and MZL are core labeled oncology uses under Imbruvica. [1]

2) What clinical endpoints most influence continued prescribing of ibrutinib?

Clinicians track response depth and durability (ORR, DOR, PFS) plus safety-driven discontinuation (notably atrial fibrillation/flutter and bleeding risk as reflected in the label). [1]

3) How does competition from other BTK inhibitors affect ibrutinib?

Competition drives formulary pressure and treatment switching, especially in CLL/SLL frontline settings where tolerability and fixed-duration approaches can reduce continuous BTK monotherapy use.

4) Why does treatment duration matter for ibrutinib net sales?

Because ibrutinib is typically dosed continuously until progression or discontinuation, shorter protocol durations in combination regimens reduce patient-month exposure and net sales.

5) Where do trial results most directly translate into market outcomes?

Trials that change guideline-relevant sequencing (frontline vs relapsed), add or refine combinations, and demonstrate durable responses with manageable safety translate most directly into adoption and payer coverage.

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References

[1] U.S. Food and Drug Administration. (n.d.). IMBRUVICA (ibrutinib) prescribing information. FDA. https://www.accessdata.fda.gov/ (label information accessed via FDA prescribing information pages).