Phase 3
Completed N=80
Moxetumomab Pasudotox for Advanced Hairy Cell Leukemia
Source: ClinicalTrials.gov NCT01829711 ↗Enrolled (actual)
80
Serious AEs
35.0%
Results posted
Aug 2018
Primary outcomePrimary: Percentage of Participants With Durable Complete Response (CR) Assessed by Blinded Independent Central Review — 36.3 Percentage of participants
◆ Published Evidence
Established
87citations · ~17 / year
Moxetumomab pasudotox in heavily pre-treated patients with relapsed/refractory hairy cell leukemia (HCL): long-term follow-up from the pivotal trial.
Summary
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.
Linked Publications (2)
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Moxetumomab pasudotox in heavily pre-treated patients with relapsed/refractory hairy cell leukemia (HCL): long-term follow-up from the pivotal trial.
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Moxetumomab pasudotox for the treatment of relapsed and/or refractory hairy cell leukemia.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Durable Complete Response (CR) Assessed by Blinded Independent Central Review |
36.3 | — |
| PRIMARY Percentage of Participants With Durable CR by Investigator's Assessment |
48.8 | — |
| SECONDARY Percentage of Participants With Minimal Residual Disease (MRD) Positive or MRD Negative CR Assessed by Blinded Independent Central Review |
33.8; 7.5 | — |
| SECONDARY Percentage of Participants With MRD Positive or MRD Negative CR by Investigator's Assessment |
32.5; 7.5 | — |
| SECONDARY Time to CR Assessed by Blinded Independent Central Review |
5.9 | — |
| SECONDARY Duration of CR Assessed by Blinded Independent Central Review |
62.8 | — |
| SECONDARY Duration of Hematologic Remission |
45.8 | — |
| SECONDARY Time to Hematologic Remission |
1.1 | — |
| SECONDARY Percentage of Participants With Objective Response (OR) Assessed by Blinded Independent Central Review |
75.0 | — |
| SECONDARY Percentage of Participants With Objective Response by Investigator's Assessment |
78.8 | — |
| SECONDARY Time to Objective Response Assessed by Blinded Independent Central Review |
5.7 | — |
| SECONDARY Duration of Objective Response Assessed by Blinded Independent Central Review |
66.7 | — |
| SECONDARY Progression-free Survival (PFS) Assessed by Blinded Independent Central Review |
41.5 | — |
| SECONDARY Time to Treatment Failure (TTF) Assessed by Blinded Independent Central Review |
41.5 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) |
79; 28 | — |
| SECONDARY Number of Participants With Abnormal Clinical Laboratory Results Reported as TEAEs |
17; 1; 5; 1; 2; 1 | — |
| SECONDARY Number of Participants With Abnormal Vital Signs Reported as TEAEs |
9; 3; 12; 6; 25 | — |
| SECONDARY Number of Participants With Abnormal Electrocardiogram (ECG) Reported as TEAEs |
2; 1; 3; 1; 1; 1 | — |
| SECONDARY Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox |
0.567; 0.550; 0.583 | — |
| SECONDARY Maximum Observed Plasma Concentration (Cmax) of Moxetumomab Pasudotox |
192; 435; 379 | — |
| SECONDARY Time of Last (Tlast) Measurable Concentration of Moxetumomab Pasudotox |
0.841; 3.37; 2.16 | — |
| SECONDARY Area Under the Plasma Concentration-time Curve From Time Zero to Time of the Last Quantifiable Concentration (AUC0-last) of Moxetumomab Pasudotox |
120; 820; 626 | — |
| SECONDARY Area Under the Plasma Concentration-time Curve From Time Zero to 3 Hours (AUC0-3hr) Post End of Moxetumomab Pasudotox |
869; 856; 1030 | — |
| SECONDARY Area Under the Plasma Concentration-time Curve From Time Zero to Infinity (AUC0-inf) of Moxetumomab Pasudotox |
1300; 1470 | — |
| SECONDARY Area Under the Plasma Concentration-time Curve Extrapolated (AUCExt) of Moxetumomab Pasudotox |
13.2; 14.3; 20.2 | — |
| SECONDARY Systemic Clearance (CL) of Moxetumomab Pasudotox |
44.6; 31.8 | — |
| SECONDARY Terminal Half Life (t1/2) of Moxetumomab Pasudotox |
1.38; 1.39 | — |
| SECONDARY Percentage of Participants With Positive Anti-drug Antibodies (ADA), Neutralizing Anti-drug Antibodies (nAb) and Specificity (CD22 and PE38) Positive to Moxetumomab Pasudotox |
87.5; 83.8; 55.2; 98.5 | — |
Eligibility Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT01829711) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.