A trial to see the effects of ACP-196 (the test drug) in Patients who have Waldenstrom Macroglobulinemia
- Conditions
- Waldenström MacroglobulinemiaMedDRA version: 21.0Level: PTClassification code 10047804Term: Waldenstrom's macroglobulinaemia recurrentSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 21.0Level: PTClassification code 10047805Term: Waldenstrom's macroglobulinaemia refractorySystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)Therapeutic area: Diseases [C] - Cancer [C04]
- Registration Number
- EUCTR2014-003212-36-NL
- Lead Sponsor
- Acerta Pharma BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 88
Men and women = 18 years of age.
•Previously treated cohort only: A confirmed diagnosis of WM, which has relapsed after, or been refractory to = 1 prior therapy for WM and which requires treatment.
•Previously untreated cohort only: A confirmed diagnosis of previously untreated WM in subjects who require treatment and do not want to receive chemoimmunotherapy or have comorbidities that would preclude chemoimmunotherapy such as:
oSymptomatic hyperviscosity with an IgM = 5,000 mg/dL
oDisease-related neuropathy
• Serum concentration of IgM, as measured by serum protein
electrophoresis (SPEP) and immunofixation electrophoresis
(IFE), that exceeds the upper limits of normal or measurable
nodal WM (defined as the presence of =1 lymph node that
measures = 2.0 cm in the longest diameter and = 1.0 cm in the
longest perpendicular diameter).
• Eastern Cooperative Oncology Group (ECOG) performance
status of = 2.
• Agreement to use acceptable methods of contraception during
the study and for 30 days after the last dose of study drug if
sexually active and able to bear or beget children.
Agreement to refrain from sperm donation during the study and
for 30 days after the last dose of study drug.
• Willing and able to participate in all required evaluations and
procedures in this study protocol including swallowing capsules
without difficulty.
• Ability to understand the purpose and risks of the study and
provide signed and dated informed consent and authorization
to use protected health information (in accordanc
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 70
1. 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.
2. 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 ACP-196,
or put the study outcomes at undue risk.
3. 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 left ventricular ejection fraction (LVEF) = 40%, or QTc
> 480 msec.
4. Malabsorption syndrome, disease significantly affecting
gastrointestinal function, or resection of the stomach or small bowel or
ulcerative colitis, symptomatic inflammatory bowel disease, or partial or
complete bowel obstruction.
5. Any immunotherapy within 4 weeks of first dose of study drug.
6. 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).
7. Prior exposure to a BCR inhibitor (eg, Btk, phosphoinositide-3 kinase
[PI3K]]], or Syk inhibitors) or BCL-2 inhibitors (eg, ABT-199).
8. 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.
9. Grade = 2 toxicity (other than alopecia) continuing from prior
anticancer therapy including radiation.
10. Known history of human immunodeficiency virus (HIV) or active
infection with hepatitis C virus (HCV) or hepatitis B virus (HBV) or any
uncontrolled active systemic infection.
11. Major surgery within 4 weeks before first dose of study drug.
12. Uncontrolled autoimmune hemolytic anemia or idiopathic
thrombocytopenia purpura.
13. History of a bleeding diathesis (eg, hemophilia, von Willebrand
disease).
14. History of stroke or intracranial hemorrhage within 6 months before
the first dose of study drug.
15. Requires or receiving anticoagulation with warfarin or equivalent
vitamin K antagonists (eg, phenprocoumon) within 28 days of first dose
of study drug.
16. Requires treatment with long-acting proton pump inhibitors (eg,
omeprazole, esomeprazole, lansoprazole, dexlansoprazole, rabeprazole,
or pantoprazole).
17. Absolute neutrophil count (ANC) < 0.75 x 109/L or platelet count
<50 x 109/L. For subjects with disease involvement in the bone marrow,
ANC < 0.50 x 109/L or platelet count < 30 x 109/L.
18. Creatinine > 2.5 x institutional upper limit of normal (ULN); total
bilirubin > 2.5 x ULN; or aspartate aminotransferase (AST) or alanine
aminotransferase (ALT) > 3.0 x ULN
19. Lactating or pregnant.
20. Concurrent participation in another therapeutic clinical trial.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method