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Ibrutinib (PCI-32765) in Waldenstrom's Macroglobulinemia

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ClinicalTrials.gov Identifier: NCT01614821
Recruitment Status : Completed
First Posted : June 8, 2012
Results First Posted : January 21, 2020
Last Update Posted : January 21, 2020
Sponsor:
Information provided by (Responsible Party):
Steven P. Treon, MD, PhD, Dana-Farber Cancer Institute

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Waldenstrom's Macroglobulinemia
Intervention Drug: PCI-32765
Enrollment 63
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Treatment Arm
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PCI-32765; ibrutinib

PCI-32765: Taken orally, once daily in the morning

Period Title: Overall Study
Started 63
Completed 63
Not Completed 0
Arm/Group Title Treatment Arm
Hide Arm/Group Description

PCI-32765; ibrutinib

PCI-32765: Taken orally, once daily in the morning

Overall Number of Baseline Participants 63
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 63 participants
<=18 years
0
   0.0%
Between 18 and 65 years
32
  50.8%
>=65 years
31
  49.2%
Age, Continuous  
Median (Standard Deviation)
Unit of measure:  Years
Number Analyzed 63 participants
63  (10.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 63 participants
Female
15
  23.8%
Male
48
  76.2%
Race and Ethnicity Not Collected   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants
[1]
Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 63 participants
63
1.Primary Outcome
Title Overall Response Rate
Hide Description To assess the overall response rate (>25% reduction in serum IgM from baseline).
Time Frame 4 years
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[Not Specified]
Arm/Group Title Treatment Arm
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PCI-32765; ibrutinib

PCI-32765: Taken orally, once daily in the morning

Overall Number of Participants Analyzed 63
Measure Type: Count of Participants
Unit of Measure: Participants
57
  90.5%
2.Secondary Outcome
Title Safety and Tolerability of PCI-32765
Hide Description To assess the safety and tolerability of PCI-32765 in symptomatic WM patients with relapsed/refractory disease. Grade > or = 2 Adverse Events determined to be associated with PCI-32765 and subsequent outcomes will constitute the safety profile of PCI-32765 in WM. Percent of participants who experienced at least 1 grade 2 or higher treatment emergent adverse event.
Time Frame 4 years
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment Arm
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PCI-32765; ibrutinib

PCI-32765: Taken orally, once daily in the morning

Overall Number of Participants Analyzed 63
Measure Type: Count of Participants
Unit of Measure: Participants
62
  98.4%
3.Secondary Outcome
Title Determine Progression Free Survival
Hide Description To determine Progression Free Survival (PFS in symptomatic WM patients with relapsed/refractory disease. Participants were treated for 40 cycles and then followed for 2 years or until next therapy or death. 40 participants were censored prior to disease progression.
Time Frame 6 years
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment Arm
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PCI-32765; ibrutinib

PCI-32765: Taken orally, once daily in the morning

Overall Number of Participants Analyzed 63
Median (Full Range)
Unit of Measure: months
39
(0 to 67)
4.Secondary Outcome
Title To Determine Time to Next Therapy (TTNT) of PCI-32765 in Symptomatic WM Patients With Relapsed/Refractory Disease
Hide Description Time to Next Therapy is the duration of time from of starting ibrutinib until next therapy. Participants were treated for 40 cycles and then followed for 2 years or until next therapy or death. Participants had the option to continue ibrutinib commercially. 40 participants were censored while still on commercial ibrutinib therapy.
Time Frame 6 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment Arm
Hide Arm/Group Description:

PCI-32765; ibrutinib

PCI-32765: Taken orally, once daily in the morning

Overall Number of Participants Analyzed 63
Median (Full Range)
Unit of Measure: months
49
(1 to 67)
5.Secondary Outcome
Title Major Response Rates
Hide Description To assess the major response rate (>50% reduction in serum IgM from baseline)
Time Frame 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment Arm
Hide Arm/Group Description:

PCI-32765; ibrutinib

PCI-32765: Taken orally, once daily in the morning

Overall Number of Participants Analyzed 63
Measure Type: Count of Participants
Unit of Measure: Participants
49
  77.8%
6.Secondary Outcome
Title Very Good Partial Response Rate
Hide Description To assess the very good partial response rate (>90% reduction in serum IgM from baseline)
Time Frame 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment Arm
Hide Arm/Group Description:

PCI-32765; ibrutinib

PCI-32765: Taken orally, once daily in the morning

Overall Number of Participants Analyzed 63
Measure Type: Count of Participants
Unit of Measure: Participants
17
  27.0%
Time Frame Adverse events were collected from Cycle 1 Day 1 through 30 days after discontinuing protocol ibrutinib therapy, an average of 3 years.
Adverse Event Reporting Description Grade 2 and above treatment emergent adverse events were collected.
 
Arm/Group Title Treatment Arm
Hide Arm/Group Description

PCI-32765; ibrutinib

PCI-32765: Taken orally, once daily in the morning

All-Cause Mortality
Treatment Arm
Affected / at Risk (%)
Total   1/63 (1.59%)    
Hide Serious Adverse Events
Treatment Arm
Affected / at Risk (%) # Events
Total   30/63 (47.62%)    
Blood and lymphatic system disorders   
Neutropenic Fever and Chills   1/63 (1.59%)  1
Anemia   2/63 (3.17%)  2
Cardiac disorders   
Atrial Fibrillation   2/63 (3.17%)  2
Pericarditis   1/63 (1.59%)  1
General disorders   
Fever and Tachycardia   1/63 (1.59%)  1
Malaise   1/63 (1.59%)  1
Fever   3/63 (4.76%)  3
Hepatobiliary disorders   
Cholecystitis   1/63 (1.59%)  1
Infections and infestations   
Flu   1/63 (1.59%)  1
Cellulitis   1/63 (1.59%)  1
Pneumonia   11/63 (17.46%)  11
Endocarditis   1/63 (1.59%)  1
Herpes Zoster   1/63 (1.59%)  1
Upper Respiratory Infection   1/63 (1.59%)  1
Group A Streptococcus   1/63 (1.59%)  1
Foot Infection   1/63 (1.59%)  1
Kidney Infection   1/63 (1.59%)  1
Injury, poisoning and procedural complications   
Femur Fracture   1/63 (1.59%)  1
Investigations   
Platelet Count Decreased   2/63 (3.17%)  2
Neutrophil count Decreased   1/63 (1.59%)  1
Metabolism and nutrition disorders   
Dehydration   1/63 (1.59%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Disease Transformation DLBCL   2/63 (3.17%)  2
Myelodysplastic Syndrome   1/63 (1.59%)  1
Adenocarcinoma of the rectum   1/63 (1.59%)  1
Squamous cell carcinoma   2/63 (3.17%)  2
Prostate Cancer   1/63 (1.59%)  1
Nervous system disorders   
Syncope   1/63 (1.59%)  1
Psychiatric disorders   
Depression and Suicidal Ideation   1/63 (1.59%)  1
Respiratory, thoracic and mediastinal disorders   
Pleural effusion   2/63 (3.17%)  2
Vascular disorders   
Hematoma   1/63 (1.59%)  1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Treatment Arm
Affected / at Risk (%) # Events
Total   62/63 (98.41%)    
Blood and lymphatic system disorders   
Anemia   40/63 (63.49%)  40
Febrile Neutropenia   2/63 (3.17%)  2
Leukocytosis   1/63 (1.59%)  1
Cardiac disorders   
Atrial Fibrillation   9/63 (14.29%)  9
Palpiatations   3/63 (4.76%)  3
Sick sinus syndrome   1/63 (1.59%)  1
Sinus bradycardia   2/63 (3.17%)  2
Sinus tachycardia   2/63 (3.17%)  2
Ventricular arrhythmia   1/63 (1.59%)  1
Ear and labyrinth disorders   
Hearing impaired   1/63 (1.59%)  1
Tinnitis   2/63 (3.17%)  2
Endocrine disorders   
Hypothyroidism   1/63 (1.59%)  1
Eye disorders   
Blurred vision   7/63 (11.11%)  7
Cataract   1/63 (1.59%)  1
Conjunctivitis   1/63 (1.59%)  1
Eye pain   2/63 (3.17%)  2
Retinal detachment   2/63 (3.17%)  2
Retinal tear   1/63 (1.59%)  1
Watering eyes   1/63 (1.59%)  1
Gastrointestinal disorders   
Abdominal Pain   7/63 (11.11%)  7
Bloating   3/63 (4.76%)  3
Constipation   8/63 (12.70%)  8
Diarrhea   20/63 (31.75%)  20
Duodenal ulcer   1/63 (1.59%)  1
Dyspepsia   1/63 (1.59%)  1
Dysphagia   3/63 (4.76%)  3
Gastric ulcer   1/63 (1.59%)  1
Gastroesophageal reflux disease   12/63 (19.05%)  12
Hemorrhoidal hemmorhage   1/63 (1.59%)  1
Hemorrhoids   1/63 (1.59%)  1
Oral Mucositis   8/63 (12.70%)  8
Nausea   9/63 (14.29%)  9
Oral hemorrhage   4/63 (6.35%)  4
Proctitis   1/63 (1.59%)  1
Rectal perforation   1/63 (1.59%)  1
Small intestinal obstruction   1/63 (1.59%)  1
Toothache   1/63 (1.59%)  1
Vomiting   7/63 (11.11%)  7
General disorders   
Chills   3/63 (4.76%)  3
Facial edema   1/63 (1.59%)  1
Edema limbs   9/63 (14.29%)  9
Fatigue   33/63 (52.38%)  33
Fever   13/63 (20.63%)  13
Fly-like symptoms   4/63 (6.35%)  4
Malaise   6/63 (9.52%)  6
Non-cardiac chest pain   2/63 (3.17%)  2
Pain   7/63 (11.11%)  7
Infections and infestations   
Bladder Infection   3/63 (4.76%)  3
Bronchial infection   7/63 (11.11%) 
Eye infection   1/63 (1.59%)  1
Gum infection   1/63 (1.59%)  1
Lip infection   1/63 (1.59%)  1
Lung infection   14/63 (22.22%)  14
Mucosal infection   3/63 (4.76%)  3
Nail infection   3/63 (4.76%)  3
Otitis media   3/63 (4.76%)  3
Penile Infection   1/63 (1.59%)  1
Pharyngitis   1/63 (1.59%)  1
Sinusitis   21/63 (33.33%)  21
Skin infection   11/63 (17.46%)  11
Soft tissue infection   1/63 (1.59%)  1
Tooth infection   1/63 (1.59%)  1
Upper respiratory infection   19/63 (30.16%)  19
Urinary tract infection   9/63 (14.29%)  9
Vaginal infection   1/63 (1.59%)  1
Wound infection   1/63 (1.59%)  1
Injury, poisoning and procedural complications   
Bruising   8/63 (12.70%)  8
Fall   2/63 (3.17%)  2
Fracture   4/63 (6.35%)  4
Wound complication   2/63 (3.17%)  2
Investigations   
Alanine aminotransferase increased   2/63 (3.17%)  2
Alkaline phosphatase increased   2/63 (3.17%)  2
Aspartate aminotransferase increased   2/63 (3.17%)  2
Blood bilirubin increased   4/63 (6.35%)  4
Creatinine increased   1/63 (1.59%)  1
Lipase increased   2/63 (3.17%)  2
Neutrophil count decreased   26/63 (41.27%)  26
Platelet count decreased   29/63 (46.03%)  29
Weight gain   2/63 (3.17%)  2
Weight loss   4/63 (6.35%)  4
White blood cell decreased   2/63 (3.17%)  2
Metabolism and nutrition disorders   
Anorexia   2/63 (3.17%)  2
Dehydration   4/63 (6.35%)  4
Hypercalcemia   1/63 (1.59%)  1
Hyperglycemia   1/63 (1.59%)  1
Hypoalbuminemia   5/63 (7.94%)  5
Hypoglycemia   1/63 (1.59%)  1
Hypokalemia   4/63 (6.35%)  4
Hypomagnesemia   1/63 (1.59%)  1
Hyponatremia   2/63 (3.17%)  2
Musculoskeletal and connective tissue disorders   
Arthralgia   17/63 (26.98%)  17
Back pain   5/63 (7.94%)  5
Bone pain   3/63 (4.76%)  3
Chest wall pain   1/63 (1.59%)  1
Muscle weakness lower limb   1/63 (1.59%)  1
Myalgia   14/63 (22.22%)  14
Neck Pain   1/63 (1.59%)  1
Pain in extremity   2/63 (3.17%)  2
Nervous system disorders   
Dizziness   11/63 (17.46%)  11
Dysgeusia   1/63 (1.59%)  1
Headache   11/63 (17.46%)  11
Paresthesia   7/63 (11.11%)  7
Peripheral motor neuropathy   9/63 (14.29%)  9
Peripheral sensory neuropathy   12/63 (19.05%)  12
Stroke   1/63 (1.59%)  1
Syncope   1/63 (1.59%)  1
Psychiatric disorders   
Anxiety   6/63 (9.52%)  6
Depression   4/63 (6.35%)  4
Insomnia   5/63 (7.94%)  5
Renal and urinary disorders   
Acute kidney injury   1/63 (1.59%)  1
Hematuria   4/63 (6.35%)  4
Renal calculi   2/63 (3.17%)  2
urinary tract obstruction   2/63 (3.17%)  2
Urinary tract pain   1/63 (1.59%)  1
Urinary urgency   2/63 (3.17%)  2
Reproductive system and breast disorders   
Irregular menstruation   1/63 (1.59%)  1
Respiratory, thoracic and mediastinal disorders   
Allergic rhinitis   1/63 (1.59%)  1
Cough   11/63 (17.46%)  11
Dyspnea   6/63 (9.52%)  6
Epistaxis   8/63 (12.70%)  8
Hypoxia   1/63 (1.59%)  1
Nasal congestion   3/63 (4.76%)  3
Pleural effusion   10/63 (15.87%)  10
Pneumonitis   1/63 (1.59%)  1
Postnasal drip   1/63 (1.59%)  1
Productive cough   1/63 (1.59%)  1
Respiratory failure   1/63 (1.59%)  1
Sore throat   2/63 (3.17%)  2
Wheezing   2/63 (3.17%)  2
Skin and subcutaneous tissue disorders   
Alopecia   1/63 (1.59%)  1
Dry eye   1/63 (1.59%)  1
Nail ridging   7/63 (11.11%)  7
Pruritis   5/63 (7.94%)  5
Purpura   1/63 (1.59%)  1
Rash acneiform   3/63 (4.76%)  3
Rash maculo-papular   7/63 (11.11%)  7
scalp pain   1/63 (1.59%)  1
Skin hyperpigmentation   1/63 (1.59%)  1
Urticaria   1/63 (1.59%)  1
Vascular disorders   
Hematoma   5/63 (7.94%)  5
Hot flashes   1/63 (1.59%)  1
Hypertension   9/63 (14.29%)  9
Hypotension   2/63 (3.17%)  2
Lymphedema   1/63 (1.59%)  1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Steven Treon
Organization: Dana-Farber Cancer Institute
Phone: 617-632-2681
EMail: steven_treon@dfci.harvard.edu
Layout table for additonal information
Responsible Party: Steven P. Treon, MD, PhD, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT01614821    
Other Study ID Numbers: 12-015
First Submitted: May 17, 2012
First Posted: June 8, 2012
Results First Submitted: November 13, 2019
Results First Posted: January 21, 2020
Last Update Posted: January 21, 2020