Clinical trial · Interventional
Moxetumomab Pasudotox for Advanced Hairy Cell Leukemia
A Pivotal Multicenter Trial of Moxetumomab Pasudotox in Relapsed/ Refractory Hairy Cell Leukemia
NCT01829711CI-TRIAL-00044556completedPhase 3Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Background: \- 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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Leukemia, Hairy Cell | Hairy Cell Leukemia | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| IV Bag Protectant for Moxetumomab pasudotox | Drug | — | UNRESOLVED |
| Moxetumomab pasudotox | Drug | Moxetumomab Pasudotox | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Moxetumomab pasudotox 40 µg/kg
- description
- Patients will receive Moxetumomab Pasudotox intravenously (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.
- interventionNames
- Drug: Moxetumomab pasudotox
- Drug: IV Bag Protectant for Moxetumomab pasudotox
Primary outcomes (2)
- measure
- Percentage of Participants With Durable Complete Response (CR) Assessed by Blinded Independent Central Review
- timeFrame
- Full disease assessment (CBC, bone marrow and imaging) at end of treatment (EOT; up to 24 weeks) and post EOT Day 181; CBC monthly for 6 months post EOT, every 3 months post Day 181 for first 2 years and every 6 months thereafter (approximately 6 years)
- description
- Durable CR was defined as overall response that meets blood, bone marrow and imaging criteria for CR, followed by a \>180 day duration of hematologic remission (HR). CR requires all of the following to be present: No evidence of leukemic cells in peripheral blood and/or by routine H/E staining of bone marrow; Resolution of any hepatomegaly, splenomegaly, and abnormal (\>= 2 cm minimum length) lymphadenopathy by CT or MRI (maximum diameter of spleen should be either \< 17 cm or have decreased by \>25% from its baseline.); HR requires normal complete blood count (CBC) as exhibited by: Neutrophils \>= 1.5 x 10\^9/L, Platelets \>= 100 x 10\^9/L, and hemoglobin \>= 11.0 g/dL without transfusions or growth factors for at least 4 weeks.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 100 Years
Show eligibility criteria text
* INCLUSION CRITERIA: * Patients must have histologically confirmed hairy cell leukemia or hairy cell leukemia variant .with a need for therapy * Patients must be Pseudomonas-immunotoxin naive * Patients must have had at least 2 prior purine analogs, or at least 1 course of purine analog and 1 of either rituximab or BRAF inhibitor. * Men or women age greater than or equal to 18 years. * ECOG performance status less than or equal to 2. * Patients must have adequate organ function EXCLUSION CRITERIA * Patients who have had chemotherapy, immunotherapy or radiotherapy within 4 weeks prior to entering the study. * Patients who are receiving any other investigational agents. * Patients with known brain metastases should be excluded from this clinical trial * Patients with clinically significant ophthalmologic findings during screening * Pregnant or breastfeeding females. * Positive for Hepatitis B core antibody or surface antigen unless the patient is on Lamivudine or Entecavir and Hepatitis B Viral DNA load is less than 2000 IU/mL. * Active second malignancy requiring treatment other than minor resection of indolent cancers like basal cell and squamous skin cancers * HIV-positive patients unless taking appropriate anti-HIV medications with a CD4 count of greater than 200. * History of allogeneic bone marrow transplant. * Patients with history of both thromboembolism and known congenital hypercoagulable conditions. * Uncontrolled pulmonary infection, pulmonary edema. * Adequate oxygen saturation * Radioimmunotherapy within 2 years prior to enrollment in study. * Adequate hematologic function * Adequate lung function * Patients with history of thrombotic microangiopathy or thrombotic microangiopathy / hemolytic uremic syndrome * Patients with QTc interval (Friderica) elevation \> 500 msec based on at least 2 separate 12-lead ECGs * Patient on high dose estrogen * Patients with clinical evidence of disseminated intravascular coagulation
References
Publications (7)
- BACKGROUNDBouroncle BA. Thirty-five years in the progress of hairy cell leukemia. Leuk Lymphoma. 1994;14 Suppl 1:1-12. PMID 7820038
- BACKGROUNDJemal A, Siegel R, Ward E, Murray T, Xu J, Thun MJ. Cancer statistics, 2007. CA Cancer J Clin. 2007 Jan-Feb;57(1):43-66. doi: 10.3322/canjclin.57.1.43. PMID 17237035
- BACKGROUNDSharpe RW, Bethel KJ. Hairy cell leukemia: diagnostic pathology. Hematol Oncol Clin North Am. 2006 Oct;20(5):1023-49. doi: 10.1016/j.hoc.2006.06.010. PMID 16990105
- BACKGROUNDKreitman RJ, Tallman MS, Robak T, Coutre S, Wilson WH, Stetler-Stevenson M, Fitzgerald DJ, Lechleider R, Pastan I. Phase I trial of anti-CD22 recombinant immunotoxin moxetumomab pasudotox (CAT-8015 or HA22) in patients with hairy cell leukemia. J Clin Oncol. 2012 May 20;30(15):1822-8. doi: 10.1200/JCO.2011.38.1756. Epub 2012 Feb 21. PMID 22355053
- BACKGROUNDKreitman RJ, Dearden C, Zinzani PL, Delgado J, Karlin L, Robak T, Gladstone DE, le Coutre P, Dietrich S, Gotic M, Larratt L, Offner F, Schiller G, Swords R, Bacon L, Bocchia M, Bouabdallah K, Breems DA, Cortelezzi A, Dinner S, Doubek M, Gjertsen BT, Gobbi M, Hellmann A, Lepretre S, Maloisel F, Ravandi F, Rousselot P, Rummel M, Siddiqi T, Tadmor T, Troussard X, Yi CA, Saglio G, Roboz GJ, Balic K, Standifer N, He P, Marshall S, Wilson W, Pastan I, Yao NS, Giles F. Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia. Leukemia. 2018 Aug;32(8):1768-1777. doi: 10.1038/s41375-018-0210-1. Epub 2018 Jul 20. PMID 30030507
- DERIVEDKreitman RJ, Dearden C, Zinzani PL, Delgado J, Robak T, le Coutre PD, Gjertsen BT, Troussard X, Roboz GJ, Karlin L, Gladstone DE, Kuptsova-Clarkson N, Liu S, Patel P, Rotolo F, Mitry E, Pastan I, Giles F; Study 1053 investigators. Moxetumomab pasudotox in heavily pre-treated patients with relapsed/refractory hairy cell leukemia (HCL): long-term follow-up from the pivotal trial. J Hematol Oncol. 2021 Feb 24;14(1):35. doi: 10.1186/s13045-020-01004-y.