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A Phase II Trial of Ofatumumab in Subjects With Waldenstrom's Macroglobulinemia

Phase 2
Completed
Conditions
Waldenstrom Macroglobulinaemia
Interventions
Biological: Ofatumumab
Registration Number
NCT00811733
Lead Sponsor
GlaxoSmithKline
Brief Summary

Given the tolerability and efficacy of ofatumumab in follicular lymphoma and Chronic Lymphocytic Leukemia, and the need to improve therapy for patients with WM utilizing a non-myelosuppressive agent this phase II trial of ofatumumab is being initiated in patients with Waldenstrom's Macroglobulinemia (WM).

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
37
Inclusion Criteria
  • Confirmed and active Waldenstrom's Macroglobulinemia requiring treatment.
  • Ambulatory and capable of all selfcare. Up and about more than 50% of waking hours.
  • Adequate organ function.
  • Detectable CD20 positive of the tumor cells.
  • Measurable disease as defined by a monoclonal IgM paraprotein level greater than 1000 mg/dL.
Exclusion Criteria
  • Treatment of WM within the past 28 days.
  • Treatment with rituximab or alemtuzamab within the past 3 months.
  • Certain heart problems, chronic or current active infection not controlled with oral antibiotics, other current cancer or within last 5 years.
  • Current participation in another interventional clinical study.
  • Lactating or pregnant women or female patients of child-bearing potential (or male patients with such partners) not willing to use adequate contraception.
  • Active cerebrovascular disease.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
OfatumumabOfatumumabOfatumumab is a fully human antibody, targeting a unique epitope on the CD20 molecule expressed on human B cells.
Primary Outcome Measures
NameTimeMethod
Number of Participants With Overall Response (OR) for Cycle 1 (Including the Redosing Cycle), as Assessed by the InvestigatorBaseline and up to 27 months from the first dose of Cycle 1 (Study Day 1), and before Cycle 2 treatment

OR (based on the Consensus Panel recommendations from the 2nd and 3rd International Workshop on WM) included Complete Response (CR), Partial Response (PR), or a Minor Response (MR). CR: Complete disappearance of serum monoclonal (SM) Immunoglobulin (Ig) E (IgE), measured centrally; resolution of adenopathy/organomegaly upon physical exam and computerized tomography (CT) scan; lymph nodes =\<1.5 centimeters; absence of malignant cell by bone marrow histologic examination. PR: a \>=50% reduction from baseline in the SM IgM concentration. MR: \>=25%, but a \<50% reduction of SM IgM from baseline.

Number of Participants With OR for Cycle 1 (Excluding the Redosing Cycle), as Assessed by the InvestigatorBaseline and up to Study Week 16

OR (based on the Consensus Panel recommendations from the 2nd and 3rd International Workshop on WM) included Complete Response (CR), Partial Response (PR), or a Minor Response (MR). CR: Complete disappearance of serum monoclonal (SM) Immunoglobulin (Ig) E (IgE), measured centrally; resolution of adenopathy/organomegaly upon physical exam and computerized tomography (CT) scan; lymph nodes =\<1.5 centimeters; absence of malignant cell by bone marrow histologic examination. PR: a \>=50% reduction from baseline in the SM IgM concentration. MR: \>=25%, but a \<50% reduction of SM IgM from baseline.

Secondary Outcome Measures
NameTimeMethod
Clearance of OfatumumabFrom the first dose (Cycle 1 Day 1) up to 6 months after the end of the last cycle of treatment; blood collected on each dosing day, weekly up to Week 8, and every 4 weeks up to Week 24/Month 7

Clearance (CL) is defined as the volume of plasma that is cleared of drug per unit of time. Blood samples for the quantification of ofatumumab were collected during each cycle and for up to 6 months after the end of each cycle.

Number of Participants With CR, PR, and MR for Cycle 1 (Excluding the Redosing Cycle), as Assessed by the InvestigatorBaseline and up to Study Week 16

Response criteria were based on the Consensus Panel recommendations from the 2nd and 3rd International Workshop on WM. CR: Complete disappearance of serum monoclonal (SM) Immunoglobulin (Ig) E (IgE), measured centrally; resolution of adenopathy/organomegaly upon physical exam and computerized tomography (CT) scan; lymph nodes =\<1.5 centimeters; absence of malignant cell by bone marrow histologic examination. PR: a \>=50% reduction from baseline in the SM IgM concentration. MR: \>=25%, but a \<50% reduction of SM IgM from baseline.

Number of Participants With IgM Flare for Cycle 1 Response (Including the Redosing Cycle)Baseline and up to 27 months from the first dose of Cycle 1 (Study Day 1), and before Cycle 2 treatment

IgM is a basic antibody that is produced by B cells. It is the first antibody to appear in response to initial exposure to antigen. IgM flare is defined as an IgM level that increases by \>25% from baseline (BL) and is associated with a response to treatment. Avoidance of IgM flare indicates the lack of an increase in IgM of \>25% from BL.

Progression-free SurvivalFrom baseline up to approximately 5 years

Time to disease progression is defined as the time from baseline to disease progression or death.

Time to Response for RespondersFrom baseline up to approximately 5 years

Time to response is defined as the time from baseline to the first response date.

Overall SurvivalFrom baseline up to approximately 5 years

Overall survival is defined as the time from baseline until death due to any cause.

Number of Participants With at Least One Confirmed Positive Post-ofatumumab HAHA ResultFrom baseline up to approximately 5 years

All human-antihuman antibody (HAHA) samples were first tested in a screening assay to identify potential HAHA positives. Next, samples that tested positive in the screening assay were further tested in the confirmation assay to determine the specificity of the signal to ofatumumab. Confirmed positive samples were reported as positive.

Number of Participants With the Indicated SAEs Related to Study DrugFrom baseline up to approximately 5 years

An SAE is any event occurring at any dose that results in any of the following: death, a life-threatening adverse drug experience (ADE; at immediate risk of death from the experience as it occurred), inpatient hospitalization/prolongation of existing hospitalization, a persistent/significant disability/incapacity, or a congenital anomaly/birth defect. Medical events that may not result in death, be life threatening, or require hospitalization may be considered to be a serious ADEs when based upon appropriate medical judgment. Relatedness was based on the Investigator's medical judgement.

Number of Participants With the Indicated AEs Leading to Permanent Discontinuation of Study Drug and Withdrawal From StudyFrom baseline up to approximately 5 years

Certain AEs led to permanent discontinuation of study drug and hence resulted in their withdrawal from the study.

Number of Participants With the Indicated >=Grade 3 AEsFrom baseline up to approximately 5 years

AEs were graded using the Common Toxicity Criteria for AEs from the Cancer Therapy Evaluation Program, Division of Cancer Therapy, National Cancer Institute. Grades: 0 = No AE or within normal limits; 1 = Mild AE; 2 = Moderate AE; 3 = Severe and undesirable AE; 4 = Life-threatening or disabling AE; 5 = Death related to AE.

Number of Participants With CR, PR, and MR for Cycle 1 (Including the Redosing Cycle), as Assessed by the InvestigatorBaseline and up to 27 months from the first dose of Cycle 1 (Study Day 1), and before Cycle 2 treatment

Response criteria were based on the Consensus Panel recommendations from the 2nd and 3rd International Workshop on WM. CR: Complete disappearance of serum monoclonal (SM) Immunoglobulin (Ig) E (IgE), measured centrally; resolution of adenopathy/organomegaly upon physical exam and computerized tomography (CT) scan; lymph nodes =\<1.5 centimeters; absence of malignant cell by bone marrow histologic examination. PR: a \>=50% reduction from baseline in the SM IgM concentration. MR: \>=25%, but a \<50% reduction of SM IgM from baseline.

Cmax and Ctrough of OfatumumabFrom the first dose (Cycle 1 Day 1) up to 6 months after the end of the last cycle of treatment; blood collected on each dosing day, weekly up to Week 8, and every 4 weeks up to Week 24/Month 7

Cmax is defined as the maximum observed drug concentration after administration, and Ctrough is defined as the drug concentration observed prior to the start of the next dose. Blood samples for the quantification of ofatumumab were collected during each cycle and for up to 6 months after the end of each cycle.

AUC(0-tau) and AUC(0-inf) of OfatumumabFrom the first dose (Cycle 1 Day 1) up to 6 months after the end of the last cycle of treatment; blood collected on each dosing day, weekly up to Week 8, and every 4 weeks up to Week 24/Month 7

AUC(0-tau) is the area under the drug concentration-time curve over the dosing interval (one week). AUC(0-inf) is the area under the drug concentration-time curve from time zero extrapolated to infinite time. Blood samples for the quantification of ofatumumab were collected during each cycle and for up to 6 months after the end of each cycle.

Duration of Response for All Responders (CR, PR, MR), as Assessed by the InvestigatorFrom baseline up to approximately 5 years

Duration of response is defined as the time from the initial response to relapse/disease progression (DP) or death. DP for CR is defined as the reappearance of the IgM protein, new signs/symptoms attributable to WM, evidence of active disease or recurrence of bone marrow involvement by lymphoplasmacytic cells, or the appearance of any new lymph node \>=1.5 centimeters on any axis. Progression for PR/MR is either a \>=25% increase in IgM from the lowest attained response value or progression of lymphadenopathy, organomegaly, cytopenias, or other clinically significant signs/symptoms caused by WM.

Volume of Distribution at Steady State of OfatumumabFrom the first dose (Cycle 1 Day 1) up to 6 months after the end of the last cycle of treatment; blood collected on each dosing day, weekly up to Week 8, and every 4 weeks up to Week 24/Month 7

Volume of distribution at steady state (Vss) is defined as the apparent volume of distribution of the drug in the body at steady state. Blood samples for the quantification of ofatumumab were collected during each cycle and for up to 6 months after the end of each cycle.

Half-life of OfatumumabFrom the first dose (Cycle 1 Day 1) up to 6 months after the end of the last cycle of treatment; blood collected on each dosing day, weekly up to Week 8, and every 4 weeks up to Week 24/Month 7

Half-life (t½) is defined as the time required for the concentration of the drug in plasma to decrease to one-half of its current value. Blood samples for the quantification of ofatumumab were collected during each cycle and for up to 6 months after the end of each cycle.

Change From Baseline in Blood Counts (CD4+, CD19+, CD50) at Month 3 After TreatmentBaseline and Month 3

CD4+ and CD19+ are two key flow cytometry parameters, and total hemolytic complement (CD50) is a complement parameter. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.

Number of Participants With the Indicated SAEs and Non-serious AEs Related to Study DrugFrom baseline up to approximately 5 years

An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. The Investigator assessed whether the AE was possibly or probably related to study drug.

Number of Participants With the Indicated Infusion-related >=Grade 3 AEFrom baseline up to approximately 5 years

Infusion-related AEs are the AEs that resulted from administration of study drug through infusion. AEs were graded using the Common Toxicity Criteria for AEs from the Cancer Therapy Evaluation Program, Division of Cancer Therapy, National Cancer Institute. Grades: 0 = No AE or within normal limits; 1 = Mild AE; 2 = Moderate AE; 3 = Severe and undesirable AE; 4 = Life-threatening or disabling AE; 5 = Death related to AE.

Trial Locations

Locations (1)

GSK Investigational Site

🇺🇸

San Antonio, Texas, United States

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