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An Open-label, Phase 2 Study of Acalabrutinib in Subjects With Waldenström Macroglobulinemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02180724
Recruitment Status : Active, not recruiting
First Posted : July 3, 2014
Results First Posted : April 24, 2020
Last Update Posted : April 24, 2020
Sponsor:
Information provided by (Responsible Party):
Acerta Pharma BV

Study Description
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Brief Summary:
This study is evaluating the safety and efficacy of acalabrutinib for the treatment of subjects with Waldenström Macroglobulinemia (WM)

Condition or disease Intervention/treatment Phase
Waldenström Macroglobulinemia (WM) Drug: Acalabrutinib Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 106 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Phase 2 Study of Acalabrutinib in Subjects With Waldenström Macroglobulinemia
Actual Study Start Date : July 2014
Actual Primary Completion Date : February 2018
Estimated Study Completion Date : August 2021

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Previously Treated
Previously treated, N=92
 Drug: Acalabrutinib
Experimental: Treatment Naïve
Treatment Naïve, N=14
 Drug: Acalabrutinib


Outcome Measures
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Primary Outcome Measures :
  1. Overall Response Rate (ORR) of Acalabrutinib in Subjects as Assessed by Investigator [ Time Frame: Up to approximately 3.8 years. Data cut at when last patient have completed Cycle 27 (28 days per Cycle). ]
    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 Outcome Measures :
  1. Progression-free Survival (PFS) of Acalabrutinib by Investigator [ Time Frame: Up to approximately 3.8 years. Data cut at last subject have completed Cycle 27 (28 days per Cycle). ]
    Kaplan-Meier (K-M) estimates of the PFS assessments and its 95% confidence interval are provided using both modified 3rd and 6th IWWM criteria by investigator. K-M estimates at a certain time (ex. all subjects complete Cycle 27) provides the estimated percentage of the subjects who are still alive or have not progressed by the given time over all patients at risk. Per 6th IWWM criteria, the progressive disease is defined as >= 25% increase in serum IgM level with an absolute increase of at least 500 mg/dL from lowest nadir (requires confirmation on at least 2 consecutive measurements at least 4 weeks apart) and/or progression of clinical features attributable to the disease. Per modified 3rd IWWM criteria, besides IgM requirement as 6th criteria, it could also includes progression of clinically significant disease related symptoms and/or death from any cause or initiation of a new anti-neoplastic therapy.

  2. Overall Survival (OS) of Acalabrutinib by Investigator [ Time Frame: Primary analysis occur when all subjects have completed Cycle 27 or have discontinued before Cycle 27. ]
    Kaplan-Meier (K-M) estimates of the OS assessments and its 95% confidence interval are provided using both 3rd and 6th IWWM criteria by investigator. K-M estimates at a certain time (ex. all subjects complete Cycle 27) provides the estimated percentage of the subjects who are still alive by the given time over all patients at risk.

  3. Summary of Duration of Response (DOR) [ Time Frame: Primary analysis occur when all subjects have completed Cycle 27 or have excit the study ]
    DOR is defined as the interval from the first documentation of Complete Response (CR), Very Good Partial Response (VGPR), Partial Response (PR) or Minor Response (MR) to the earlier of the first documentation of definitive PD or death from any cause. The summary statistics are provided for DOR.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Men and women ≥ 18 years of age
  • A confirmed diagnosis of WM, which requires treatment.
  • Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2.
  • Agreement to use contraception during the study and for 30 days after the last dose of study drugs if sexually active and able to bear or beget children.

Exclusion Criteria:

  • 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 LVEF < 40%
  • 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.
  • Breast feeding or pregnant.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02180724


Locations
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United States, District of Columbia
Washington, District of Columbia, United States
United States, New York
New York, New York, United States
United States, Texas
Houston, Texas, United States
Netherlands
Amsterdam, Netherlands
United Kingdom
Leicester, United Kingdom
Sponsors and Collaborators
Acerta Pharma BV
Investigators
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Study Director: Acerta Clinical Trials 1-888-292-9613; acertamc@dlss.com
  Study Documents (Full-Text)

Documents provided by Acerta Pharma BV:
Study Protocol  [PDF] April 16, 2019
Statistical Analysis Plan  [PDF] January 31, 2018

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Acerta Pharma BV
ClinicalTrials.gov Identifier: NCT02180724    
Other Study ID Numbers: ACE-WM-001
First Posted: July 3, 2014    Key Record Dates
Results First Posted: April 24, 2020
Last Update Posted: April 24, 2020
Last Verified: April 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Acerta Pharma BV:
Bruton tyrosine kinase inhibitor
Btk
Waldenström Macroglobulinemia
 WM
Waldenström
Macroglobulinemia
Additional relevant MeSH terms:
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Waldenstrom Macroglobulinemia
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Neoplasms
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
 Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases