CA-4948-101: Open-Label, Dose Escalation and Expansion Trial of Emavusertib (CA-4948) in Relapsed or Refractory Primary Central Nervous System Lymphoma (R/R PCNSL)

NCT ID: NCT03328078

Last Updated: 2025-06-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

152 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-28

Study Completion Date

2026-10-31

Brief Summary

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This is a multi-center, open-label study to evaluate the safety, pharmacokinetics (PK), and anti-cancer activity of oral administration of emavusertib alone or in combination with ibrutinib in adult participants with relapsed or refractory (R/R) hematologic malignancies.

This trial will be completed in four parts. In Part A1, emavusertib will be evaluated first in a dose escalating monotherapy setting to establish the safety and tolerability (complete). In Part A2, emavusertib will be evaluated in combination with ibrutinib at 560 milligrams (mg) once daily (QD) or 420 mg QD as indicated by disease (Part A2 complete).

Part B will comprise 2 cohorts to assess safety and efficacy of emavusertib in combination with ibrutinib in participants with R/R primary central nervous system lymphoma (PCNSL) who have directly progressed on a bruton tyrosine kinase inhibitor (BTKi). In this part of the study, emavusertib will be dosed at 100 mg or 200 mg twice daily (BID) in combination with ibrutinib in 28-day treatment cycles.

Part C will comprise 3 treatment arms in the second-line setting to assess the efficacy and safety of emavusertib monotherapy, ibrutinib monotherapy, and emavusertib in combination with ibrutinib in participants with R/R PCNSL who are naïve to BTKi treatment. In this part of the study, eligible second-line participants with R/R PCNSL who are naïve to BTKi treatment will be randomized 1:1:1 to 1 of 3 treatment arms: (1) emavusertib 200 mg BID, (2) ibrutinib 560 mg QD, or (3) emavusertib 200 mg BID in combination with ibrutinib 560 mg QD.

Detailed Description

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Conditions

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Relapsed Hematologic Malignancy Refractory Hematologic Malignancy Relapsed Primary Central Nervous System Lymphoma Refractory Primary Central Nervous System Lymphoma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Part A will evaluate escalating doses of emavusertib (CA-4948) either as monotherapy (Part A1) or in combination with ibrutinib (Part A2). Enrollment is closed for Part A1 and A2. Part B will comprise two expansion cohorts to assess efficacy and safety of emavusertib in combination with ibrutinib in R/R PCNSL. Part C will comprise three cohorts to assess the efficacy and safety of emavusertib monotherapy, ibrutinib monotherapy, and emavusertib in combination with ibrutinib in participants with R/R PCNSL who are naïve to BTKi treatment.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Part B is non-randomized and Part C is randomized.

Study Groups

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Emavusertib (CA-4948) dose escalation

Part A1: Dose-level cohorts with up to approximately 6 participants each will be used to define the Maximum Tolerated Dose (MTD) for emavusertib.

Group Type EXPERIMENTAL

Emavusertib

Intervention Type DRUG

Emavusertib will be provided as a tablet dosage form to be taken BID.

Emavusertib (CA-4948) and ibrutinib dose escalation

Part A2: Evaluate escalating dose levels of oral emavusertib in combination with 560 mg QD or 420 mg QD of oral ibrutinib. The starting dose of emavusertib to be used in combination will be 200 mg BID. It is anticipated that 12 to 20 participants at a potential dose level will be required to establish optimal combination dosing.

Group Type EXPERIMENTAL

Emavusertib

Intervention Type DRUG

Emavusertib will be provided as a tablet dosage form to be taken BID.

Ibrutinib

Intervention Type DRUG

Ibrutinib will be provided as a tablet or capsule dosage form to be taken QD.

Emavusertib (CA-4948) and ibrutinib dose expansion

In two Expansion Cohorts (Part B), emavusertib in combination with ibrutinib will be administered in participants with R/R PCNSL who have progressed on a BTKi. In Cohort 1, emavusertib 100 mg BID will be administered with ibrutinib 560 mg QD consecutively in 28-day treatment cycles. In Cohort 2, emavusertib 200 mg BID will be administered with ibrutinib 560 mg QD consecutively in 28-day treatment cycles.

Group Type EXPERIMENTAL

Emavusertib

Intervention Type DRUG

Emavusertib will be provided as a tablet dosage form to be taken BID.

Ibrutinib

Intervention Type DRUG

Ibrutinib will be provided as a tablet or capsule dosage form to be taken QD.

Emavusertib (CA-4948) and ibrutinib

In this part of the study (Part C), eligible second-line participants with R/R PCNSL who are naïve to BTKi treatment will receive 1 of 3 treatment arms: (1) emavusertib 200 mg BID, (2) ibrutinib 560 mg QD, or (3) emavusertib 200 mg BID in combination with ibrutinib 560 mg QD. Treatments will be administered continuously in 28-day treatment cycles.

Group Type EXPERIMENTAL

Emavusertib

Intervention Type DRUG

Emavusertib will be provided as a tablet dosage form to be taken BID.

Ibrutinib

Intervention Type DRUG

Ibrutinib will be provided as a tablet or capsule dosage form to be taken QD.

Interventions

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Emavusertib

Emavusertib will be provided as a tablet dosage form to be taken BID.

Intervention Type DRUG

Ibrutinib

Ibrutinib will be provided as a tablet or capsule dosage form to be taken QD.

Intervention Type DRUG

Other Intervention Names

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CA-4948

Eligibility Criteria

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Inclusion Criteria

1. Males and females greater than or equal to 18 years of age
2. Life expectancy of at least 3 months
3. Eastern Cooperative Oncology Group (ECOG) Performance Status of 0, 1 or 2
4. Histopathologically confirmed diagnosis of PCNSL (medical record is acceptable). Cerebral biopsies are not required if imaging reveals typical images of PCNSL.

1. Participants with parenchymal lesions must have unequivocal evidence of disease progression (e.g., presence of at least 1 measurable target lesion \[≥ 10 millimeters (mm) and ≤ 40 mm in the longest diameter on brain magnetic resonance imaging \[MRI\] or head computed tomography \[CT\] on imaging within 28 days prior to Cycle 1 Day 1\]). In cases where the tumor size is smaller but still measurable and located at a critical central nervous system (CNS) location, disabling the participant and/or causing symptoms, this participant may be eligible following a discussion with the Sponsor Medical Monitor.
2. For participants limited to leptomeningeal involvement, cerebrospinal fluid (CSF) analysis (cytology and/or flow cytometry) with or without additional imaging (MRI) of the spine as clinically indicated is required to document abnormal cells within 28 days prior to Cycle 1 Day 1.

Exclusion Criteria

1. Participants with only intraocular PCNSL without brain lesion or CSF involvement, T-cell lymphoma, systemic presence of lymphoma, or non-CNS lymphoma metastatic to the CNS
2. Evidence of systemic lymphoma. This must be demonstrated by a positron emission tomography (PET) scan (or CT scan with contrast if applicable) of the chest, abdomen, and pelvis at Screening (testicular ultrasound may be considered to exclude a testicular lymphoma disseminated to the brain).
3. Prior history of malignancies other than lymphoma (except for basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix or breast) or prior history of systemic lymphoma, unless the participant has been free of the disease for ≥ 3 years.
4. Active malignancy other than PCNSL requiring systemic therapy
5. Previous BTKi treatment (Part C only).
6. History of Grade ≥ 3 rhabdomyolysis without complete recovery
7. Requirement for urgent therapy due to uncontrolled tumor mass/edema effects.
8. Received external beam radiation therapy to the CNS within 28 days prior to Cycle 1 Day 1.
9. Received prior investigational drugs (including treatment in clinical research, unapproved combination products, and new dosage forms) within 28 days or 5 half-lives, whichever is shorter, prior to Cycle 1 Day 1; allogeneic hematopoietic stem cell transplant (HSCT) within 60 days prior to Cycle 1 Day 1; or had clinically significant graft-versus-host disease (GVHD) requiring ongoing up-titration of immunosuppressive medications prior to Screening (with the exception of a BTKi for Part B only).

Note: The use of a stable or tapering dose of immunosuppressive therapy post-HSCT and/or topical steroids for ongoing skin GVHD is permitted with Sponsor Medical Monitor approval
10. Any prior systemic anti-cancer treatment such as chemotherapy, immunomodulatory drug therapy, etc., received within 14 days or 5 half-lives, whichever is shorter, prior to Cycle 1 Day 1 (with the exception of ibrutinib or other BTKi for Part B only, which may be continued until the day before Cycle 1 Day 1)
11. Prior history of hypersensitivity or anaphylaxis to emavusertib, ibrutinib or any of their excipients.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Curis, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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St. Joseph's Hospital and Medical Center

Phoenix, Arizona, United States

Site Status RECRUITING

Mayo Clinic

Phoenix, Arizona, United States

Site Status RECRUITING

City of Hope

Duarte, California, United States

Site Status RECRUITING

Providence St. John's Health Center

Santa Monica, California, United States

Site Status RECRUITING

UCLA Department of Medicine - Hematology/Oncology

Santa Monica, California, United States

Site Status WITHDRAWN

Smilow Cancer Hospital at Yale-New Haven

New Haven, Connecticut, United States

Site Status RECRUITING

Mayo Clinic

Jacksonville, Florida, United States

Site Status RECRUITING

Northwestern Memorial Hospital

Chicago, Illinois, United States

Site Status RECRUITING

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status RECRUITING

Mayo Clinic

Rochester, Minnesota, United States

Site Status RECRUITING

Fred and Pamela Buffett Cancer Center

Omaha, Nebraska, United States

Site Status RECRUITING

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status ACTIVE_NOT_RECRUITING

Roswell Park Comprehensive Cancer Center

Buffalo, New York, United States

Site Status RECRUITING

Mt Sinai

New York, New York, United States

Site Status RECRUITING

Columbia University Irving Medical Center

New York, New York, United States

Site Status RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status RECRUITING

Duke University Medical Center, Duke Cancer Center

Durham, North Carolina, United States

Site Status RECRUITING

Cleveland Clinic

Cleveland, Ohio, United States

Site Status RECRUITING

Providence Neurological Specialties West

Portland, Oregon, United States

Site Status RECRUITING

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status COMPLETED

UPMC Hilman Cancer Center

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

University of Tennessee Medical Center

Knoxville, Tennessee, United States

Site Status ACTIVE_NOT_RECRUITING

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

The University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status RECRUITING

Huntsman Cancer Institute, University of Utah

Salt Lake City, Utah, United States

Site Status RECRUITING

Swedish Cancer Institute

Seattle, Washington, United States

Site Status COMPLETED

University of Washington Medical Center

Seattle, Washington, United States

Site Status RECRUITING

Všeobecná fakultní nemocnice v Praze

Prague, , Czechia

Site Status RECRUITING

Institut Bergonie

Bordeaux, , France

Site Status RECRUITING

Hopital de la Timone

Marseille, , France

Site Status RECRUITING

Hospital Pitie Salpetriere

Paris, , France

Site Status RECRUITING

Institut Curie Hospital

Paris, , France

Site Status RECRUITING

Hematology Department Soroka UMC / Heanatology Department

Beersheba, , Israel

Site Status RECRUITING

Hadassah Medical Center / Ein-Carem

Jerusalem, , Israel

Site Status RECRUITING

Università di Torino Croce e Carle

Cuneo, , Italy

Site Status RECRUITING

SODc Ematologia Azienda Ospedaliera Universitaria Careggi

Florence, , Italy

Site Status RECRUITING

IRST - Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori

Meldola, , Italy

Site Status RECRUITING

IRCCS San Raffaele Scientific Institute

Milan, , Italy

Site Status RECRUITING

Uniwersyteckie Centrum Kliniczne Osrodek Badan Klinicznych Wczesnych Faz

Gdansk, , Poland

Site Status RECRUITING

Oddzial Kliniczny Hematologii

Krakow, , Poland

Site Status WITHDRAWN

NarodowyInstytutu Onkologii im. Marii Sklodowskiej-Curie-Panstwowy Instytutu Badawczy

Warsaw, , Poland

Site Status RECRUITING

MD Anderson Cancer Center Madrid

Madrid, , Spain

Site Status RECRUITING

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status RECRUITING

Countries

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United States Czechia France Israel Italy Poland Spain

Central Contacts

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Ahmed Hamdy, MD

Role: CONTACT

617-503-6500

Other Identifiers

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2024-513312-95-00

Identifier Type: CTIS

Identifier Source: secondary_id

2022-000891-20

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CA-4948-101

Identifier Type: -

Identifier Source: org_study_id

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