Trials Sponsors Conditions Drugs Pricing

Conditions

Waldenström Macroglobulinemia (WM)Waldenström Macroglobulinemia

Keywords

Bruton tyrosine kinase inhibitorBtkWaldenström MacroglobulinemiaWMWaldenströmMacroglobulinemia

Outcome Measures

Primary Outcomes (2)

Overall Response Rate (ORR) of Acalabrutinib in Subjects as Assessed by Investigator Per IWWM 6th Criteria
ORR defined as the rate of subjects achieving a Miner Response (MR) or better including complete response (CR), very good partial response (VGPR), partial response (PR) and MR; The definition of responses are evaluated by investigators using both Modified 6th criteria (refer to protocol table 4-2 , 4-3) and Modified 3rd International Workshop of Waldenström Macroglobulinemia (IWWM) criteria (refer to protocol table 4-4 and table 4-5 for definition). For Modified 6th criteria, MR is defined as (1) monoclonal IgM protein is detectable, (2) no new signs or symptoms of active disease, (3) and patient has \>=25% but \< 50% reduction in serum monoclonal IgM level from baseline. PR and VGPR both require (1) and (2) as MR, but PR also requires the \>=50% and \<90% reduction in serum monoclonal IgM level as well as reduction in extramedullary disease. VGPR requires \>= 90% reduction in serum IgM and complete resolution of extramedullary disease.
Up to approximately 3.8 years. Data cut at when last patient has completed Cycle 27 (28 days per Cycle).
Overall Response Rate (ORR) of Acalabrutinib in Subjects as Assessed by Investigator Per IWWM 3rd Criteria
ORR defined as the rate of subjects achieving a Miner Response (MR) or better including complete response (CR), very good partial response (VGPR), partial response (PR) and MR; The definition of responses are evaluated by investigators using both Modified 6th criteria (refer to protocol table 4-2 , 4-3) and Modified 3rd International Workshop of Waldenström Macroglobulinemia (IWWM) criteria (refer to protocol table 4-4 and table 4-5 for definition). For Modified 6th criteria, MR is defined as (1) monoclonal IgM protein is detectable, (2) no new signs or symptoms of active disease, (3) and patient has \>=25% but \< 50% reduction in serum monoclonal IgM level from baseline. PR and VGPR both require (1) and (2) as MR, but PR also requires the \>=50% and \<90% reduction in serum monoclonal IgM level as well as reduction in extramedullary disease. VGPR requires \>= 90% reduction in serum IgM and complete resolution of extramedullary disease.

Secondary Outcomes (3)

Progression-free Survival (PFS) of Acalabrutinib by Investigator
Up to approximately 3.8 years. Data cut at last subject have completed Cycle 27 (28 days per Cycle).
Overall Survival (OS) of Acalabrutinib by Investigator
Primary analysis occur when all subjects have completed Cycle 27 or have discontinued before Cycle 27.
Summary of Duration of Response (DOR)
Primary analysis occur when all subjects have completed Cycle 27 or have exit the study

Study Arms (2)

Previously Treated

EXPERIMENTAL

Subjects previously treated with Waldenström Macroglobulinemia N=92

Drug: Acalabrutinib (ACP-196)

Treatment Naïve

EXPERIMENTAL

Subjects with treatment-naïve Waldenström Macroglobulinemia. N=14

Drug: Acalabrutinib (ACP-196)

Interventions

Acalabrutinib (ACP-196)Drug

Also known as: Acalabrutinib

Previously Treated

Acalabrutinib (ACP-196)Drug

Also known as: Acalabrutinib

Treatment Naïve

Registry intervention entries

These entries preserve how the registry describes the intervention. Their treatment roles are shown separately from molecular identities.

Formulated product
Calquence 100 mg hard capsules
The registry describes a specific formulation. Its exact chemical ingredients and approval status require separate evidence.
CAPSULE, HARD
View registry record
Formulated product
Calquence 100 mg film-coated tablets
The registry describes a specific formulation. Its exact chemical ingredients and approval status require separate evidence.
FILM-COATED TABLET
View registry record

Ingredients named in source formulations

The registry names these ingredients. The formulation’s exact chemical form has not been confirmed.

Calquence 100 mg hard capsules
Named ingredient: acalabrutinib
View registry record
Calquence 100 mg film-coated tablets
Named ingredient: acalabrutinib
View registry record

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

Men and women ≥18 years of age.
Previously treated cohort only: A confirmed diagnosis of WM, which has relapsed after, or been refractory to ≥1prior therapy for WM and which requires treatment.

Sponsors & Collaborators

Acerta Pharma BV
Industrylead
Acerta Pharma B.V.
Industrylead

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Study Sites (38)

Research Site

Santa Barbara, California, 93105, United States

Research Site

Aurora, Colorado, 80012, United States

Research Site

Washington D.C., District of Columbia, 20007, United States

Research Site

Minneapolis, Minnesota, 55404, United States

Research Site

New York, New York, 10021, United States

Research Site

Nashville, Tennessee, 37203, United States

Research Site

Austin, Texas, 78705, United States

Related Publications (3)

Owen RG, McCarthy H, D'Sa S, Thomas SK, Tournilhac O, Forconi F, Kersten MJ, Zinzani PL, Iyengar S, Kothari J, Minnema MC, Kastritis E, Cheson BD, Walter HS, Greenwald D, Calvo R, Li Y, Rule S, Furman RR. Acalabrutinib in Waldenstrom macroglobulinemia yields durable responses with 5 years of follow-up. Blood Adv. 2026 Jul 28;10(14):5058-5069. doi: 10.1182/bloodadvances.2025017713.
PMID: 41985004DERIVED
Alfaifi A, Bahashwan S, Alsaadi M, Ageel AH, Ahmed HH, Fatima K, Malhan H, Qadri I, Almehdar H. Advancements in B-Cell Non-Hodgkin's Lymphoma: From Signaling Pathways to Targeted Therapies. Adv Hematol. 2024 Nov 12;2024:5948170. doi: 10.1155/2024/5948170. eCollection 2024.
PMID: 39563886DERIVED

MeSH Terms

Conditions

Waldenstrom Macroglobulinemia

Interventions

acalabrutinib

Condition Hierarchy (Ancestors)

Neoplasms, Plasma CellNeoplasms by Histologic TypeNeoplasmsHemostatic DisordersVascular DiseasesCardiovascular DiseasesParaproteinemiasBlood Protein DisordersHematologic DiseasesHemic and Lymphatic DiseasesHemorrhagic DisordersLymphoproliferative Disorders

Results Point of Contact

Title: Global Clinical Lead
Organization: Acerta Pharma

Study Officials

AstraZeneca Clinical study Information Center
1-877-240-9479 information.center@astrazeneca.com
STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: OTHER
Restrictive Agreement: Yes

Enrollment

107 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 2
Allocation: NON RANDOMIZED
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

June 30, 2014

First Posted

July 3, 2014

Study Start

September 11, 2014

Actual (reported)

You may not qualify if:

Prior malignancy, except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer from which the subject has been disease free for ≥2 years or which will not limit survival to \<2 years. Note: These cases must be discussed with the medical monitor.
A life-threatening illness, medical condition, or organ system dysfunction which, in the investigator's opinion, could compromise the subject's safety, interfere with the absorption or metabolism of acalabrutinib, or put the study outcomes at undue risk.
Significant cardiovascular disease such as uncontrolled or symptomatic arrhythmias, congestive heart failure, or myocardial infarction within 6 months of screening, or any Class 3 or 4 cardiac disease as defined by the New York Heart Association Functional Classification, or QTc \>480 msec.
Malabsorption syndrome, disease significantly affecting gastrointestinal function, or resection of the stomach or small bowel or gastric bypass, symptomatic inflammatory bowel disease, or partial or complete bowel obstruction.
Any immunotherapy within 4 weeks of first dose of study drug.
For subjects with recent chemotherapy or experimental therapy, the first dose of study drug must occur after 5 times the half-life of the agent(s).
Prior exposure to a BCR inhibitor (e.g., BTK,PI3K, or SYK inhibitors) or BCL-2 inhibitors (e.g., ABT-199).
Ongoing immunosuppressive therapy, including systemic or enteric corticosteroids for treatment of WM or other conditions. Note: Subjects may use topical or inhaled corticosteroids or low-dose steroids (≤10 mg of prednisone or equivalent per day) as therapy for comorbid conditions. During study participation, subjects may also receive systemic or enteric corticosteroids as needed for treatment-emergent comorbid conditions.
Grade ≥2 toxicity (other than alopecia) continuing from prior anticancer therapy including radiation.
Known history of HIV or active infection with HCV or hepatitis B virus (HBV) or any uncontrolled active systemic infection.
Major surgery within 4 weeks before first dose of study drug.
Uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenia purpura.
History of a bleeding diathesis (e.g., hemophilia, von Willebrand disease).
History of stroke or intracranial hemorrhage within 6 months before the first dose of acalabrutinib.
Requires or receiving anticoagulation with warfarin or equivalent vitamin K antagonists (e.g., phenprocoumon) within 28 days of first dose of study drug.
+5 more criteria

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Results Posted

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (3)

Study Arms (2)

Previously Treated

Treatment Naïve

Interventions

Registry intervention entries

Ingredients named in source formulations

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (38)

Related Publications (3)

Related Links

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source records

Documents

Locations