Moxetumomab Pasudotox for Advanced Hairy Cell Leukemia
- Moxetumomab pasudotox is an experimental non-chemotherapy cancer treatment drug. It targets CD22, a molecule on the surface of essentially all hairy cell leukemia cells. Moxetumomab pasudotox binds to CD22, goes into the cell, and releases a toxin which kills the cell. In a phase I trial it had activity in relapsed/refractory hairy cell leukemia with safety profile supporting further clinical study (http://ncbi.nlm.nih.gov/pubmed/22355053). This is a phase III multicenter trial designed to confirm these results.
Leukemia, Hairy Cell
Drug: moxetumomab pasudotox
Drug: IV Bag Protectant for Moxetumomab pasudotox
|Study Design:||Endpoint Classification: Efficacy Study
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Pivotal Multicenter Trial of Moxetumomab Pasudotox in Relapsed/ Refractory Hairy Cell Leukemia|
- Rate of CR in patients treated with study drug [ Time Frame: Every 4 weeks ] [ Designated as safety issue: No ]
- Overall response rate [ Time Frame: every 4 weeks. ] [ Designated as safety issue: No ]
- Relapse free survival [ Time Frame: Once patients have a Complete Response (CR), they will be followed with monthly blood work for 6 months then every 3 months for 2 years, then yearly thereafter. Bone marrow examinations at 6 months. ] [ Designated as safety issue: No ]
- Progression free survival [ Time Frame: Once patients have a Complete Response (CR), they will be followed with monthly blood work for 6 months then every 3 months for 2 years, then yearly thereafter. Bone marrow examinations at 6 months. ] [ Designated as safety issue: No ]
- Time to Response [ Time Frame: Duration of Study ] [ Designated as safety issue: No ]TTR will be measured from start of administartion to the first documentation of response (CR or PR)
- Safety [ Time Frame: 1st Administration through 30 Days after Last Dose ] [ Designated as safety issue: Yes ]Occurence of AEs, abnormal laboratory values, and SAEs reported from 1st administration of moxetumomab pasudotox through 30 days after last dose
- Pharmacokinetic [ Time Frame: Duration of Treatment ] [ Designated as safety issue: Yes ]
- Immunogenic Potential [ Time Frame: Duration of Treatment ] [ Designated as safety issue: Yes ]
|Study Start Date:||April 2013|
|Estimated Study Completion Date:||May 2017|
|Estimated Primary Completion Date:||May 2017 (Final data collection date for primary outcome measure)|
Experimental: Arm A
Patients will receive Moxetumomab Pasudotox IV over 30 minutes on days 1, 3, 5 of each 28 day cycle for a maximum of 6 cycles or until disease progression, unacceptable toxivity, initiation of alternate therapy or documented CR.
|Drug: moxetumomab pasudotox Drug: IV Bag Protectant for Moxetumomab pasudotox|
- Hairy cell leukemia is an indolent B-cell leukemia comprising 2% of all leukemias, or approximately 900 of the 44,000 new cases of leukemia/year in the US
- Over the last two decades, immunotoxin research has accumulated to support a role for CD22-targeted therapy in the treatment of HCL.
- Moxetumomab pasudotox is a recombinant immunotoxin containing an Fv fragment of an anti-CD22 monoclonal antibody and truncated Pseudomonas exotoxin.
- Moxetumomab pasudotox has demonstrated a high complete response (CR) rate in patients with chemoresistant hairy cell leukemia (HCL) and has shown activity in pediatric acute lymphoblastic leukemia as well.
- Modification of the structure of moxetumomab pasudotox has greatly improved binding and cytotoxicity toward CD22 expressing malignant cells compared to the precursor molecule. Preclinical and clinical studies have demonstrated that this increase in binding affinity results in improved antitumor activity and tolerability
- Currently there are no approved agents with significant efficacy for HCL patients after failure of standard therapy
- This is a multicenter, single-arm study of moxetumomab pasudotox in patients with relapsed/refractory hairy cell leukemia.
- 77 patients will be enrolled to receive moxetumomab pasudotox IV on days 1, 3 and 5 of each 28 day cycle for a maximum of 6 cycles or until disease progression, unacceptable toxicity occurs, the subject begins alternate therapy, or documented CR (for subjects who have no assessable minimal residual disease and not to exceed 6 cycles). If less than or equal to 2 of the first 25 patients do not achieve durable CR, no additional patients will be accrued.
- The overall IRB accrual ceiling is currently set at 80 to allow for a small number of patients that cannot be assessed for response.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01829711
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|Study Director:||MedImmune LLC||MedImmune LLC|