Adults 18 to 99, any sex, with Hairy Cell Leukemia. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Dose Limiting Toxicities (DLTs)Primary· Cycle 1 Day 1 to Cycle 2 Day 10 (each cycle duration was 28 days)
Adverse events are suspected of causal relationship to drug and are \>=Grade (G) 3 in severity considered DLTs: G 3 or 4 hematologic abnormalities at baseline due to disease were not evaluable for hematologic DLT, G 2 allergic reactions of bronchospasm or urticaria, or any \>= G3 allergic reaction, in presence of pre-medication were considered DLTs. Following \>= G 3 non-hematological treatment-related toxicities not considered DLTs: Tumor lysis syndrome, G 3 low electrolyte levels with pre-existing low levels of same electrolytes, anticoagulant therapy, G 3 or 4 infection or neutropenic fever
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
0
40 mcg/kg
0
50 mcg/kg
0
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)Primary· From start of study drug administration until 30 days after the last dose of study drug (approximately 8 years)
The TEAEs are defined as adverse events (AEs) present at baseline that worsened in intensity after administration of investigational product or events absent at baseline that emerged after administration of study drug, for the period extending to 30 days after the last dose of study drug. A serious adverse event (SAE) is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening situation (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly/birth d
TEAEs
Group
Value
95% CI
5 mcg/kg
3
10 mcg/kg
3
20 mcg/kg
3
30 mcg/kg
3
40 mcg/kg
4
50 mcg/kg
33
TESAEs
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
2
40 mcg/kg
0
50 mcg/kg
4
Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)Primary· From start of study drug administration until 30 days after the last dose of study drug (approximately 8 years)
Vital signs abnormalities reported as TEAEs included pyrexia, weight increased, dyspnoea, hypoxia, hypertension, hypotension.
Pyrexia
Group
Value
95% CI
5 mcg/kg
1
10 mcg/kg
1
20 mcg/kg
2
30 mcg/kg
3
40 mcg/kg
1
50 mcg/kg
16
Weight increased
Group
Value
95% CI
5 mcg/kg
1
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
0
40 mcg/kg
0
50 mcg/kg
6
Dyspnoea
Group
Value
95% CI
5 mcg/kg
1
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
1
40 mcg/kg
0
50 mcg/kg
6
Hypoxia
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
1
40 mcg/kg
0
50 mcg/kg
1
Hypertension
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
1
20 mcg/kg
0
30 mcg/kg
1
40 mcg/kg
0
50 mcg/kg
0
Hypotension
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
2
40 mcg/kg
0
50 mcg/kg
10
Number of Participants With Clinically Relevant Electrocardiogram (ECG) Abnormalities Recorded as Adverse Events (AEs)Primary· From start of study drug administration until 30 days after the last dose of study drug (approximately 8 years)
Cardiac AEs observed in participants with clinically significant ECG abnormalities included; ECG QT prolonged, Sinus tachycardia and Atrioventricular block first degree.
ECG QT prolonged
Group
Value
95% CI
5 mcg/kg
1
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
2
40 mcg/kg
0
50 mcg/kg
2
Sinus tachycardia
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
1
40 mcg/kg
0
50 mcg/kg
2
Atrioventricular block first degree
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
0
40 mcg/kg
1
50 mcg/kg
0
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)Primary· From start of study drug administration until 30 days after the last dose of study drug (approximately 8 years)
An abnormal laboratory finding which required an action or intervention by the investigator, or a finding judged by the investigator to represent a change beyond the range of normal physiologic fluctuation were reported as an adverse event.
Alanine aminotransferase increased
Group
Value
95% CI
5 mcg/kg
1
10 mcg/kg
1
20 mcg/kg
1
30 mcg/kg
3
40 mcg/kg
2
50 mcg/kg
25
Aspartate aminotransferase increased
Group
Value
95% CI
5 mcg/kg
2
10 mcg/kg
1
20 mcg/kg
1
30 mcg/kg
2
40 mcg/kg
1
50 mcg/kg
24
Blood alkaline phosphatase increased
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
1
40 mcg/kg
0
50 mcg/kg
5
Blood bicarbonate decreased
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
0
40 mcg/kg
0
50 mcg/kg
5
Blood bilirubin increased
Group
Value
95% CI
5 mcg/kg
2
10 mcg/kg
1
20 mcg/kg
1
30 mcg/kg
0
40 mcg/kg
1
50 mcg/kg
2
Blood creatine phosphokinase increased
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
1
20 mcg/kg
0
30 mcg/kg
1
40 mcg/kg
0
50 mcg/kg
4
Blood creatinine increased
Group
Value
95% CI
5 mcg/kg
1
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
1
40 mcg/kg
0
50 mcg/kg
11
Blood triglycerides increased
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
1
20 mcg/kg
0
30 mcg/kg
1
40 mcg/kg
1
50 mcg/kg
2
Number of Participants With Objective Response Rate (ORR): Complete Response (CR) or Partial Response (PR)Primary· From Baseline (predose of Cycle 1 Day 1) through end of study (until CR, PD, initiation of alternative therapy, unacceptable toxicity, development of neutralizing antibodies, or other study discontinuation reasons) (approximately 8 years)
Objective response rate defined as proportion of participants with confirmed CR or confirmed PR according to Response Evaluation Criteria for hairy cell leukemia (HCL). A CR is defined as: No evidence of leukemic cells by routine H/E stains of peripheral blood and bone marrow. No hepatomegaly, splenomegaly, or lymphadenopathy by physical examination and/or appropriate radiographic techniques. Normal complete blood count as: Neutrophils \>= 1,500/mcL, Platelets \>= 100,000/mcL, and Hemoglobin \>= 11.0 gm/dL without transfusions or growth factors for at least 4 weeks. Partial response requires a
Group
Value
95% CI
5 mcg/kg
3
10 mcg/kg
3
20 mcg/kg
2
30 mcg/kg
2
40 mcg/kg
3
50 mcg/kg
29
Number of Participants With Complete Response (CR)Primary· From Baseline (predose of Cycle 1 Day 1) through end of study (until CR, PD, initiation of alternative therapy, unacceptable toxicity, development of neutralizing antibodies, or other study discontinuation reasons) (approximately 8 years)
The CR is defined by: No evidence of leukemic cells by routine H/E stains of the peripheral blood and bone marrow. No hepatomegaly, splenomegaly, or lymphadenopathy by physical examination and/or appropriate radiographic techniques. Normal complete blood count as exhibited by: Neutrophils \>= 1,500/microliter (mcL), Platelets \>= 100,000/mcL, and Hemoglobin \>= 11.0 gram per deciliter (gm/dL) without transfusions or growth factors for at least 4 weeks.
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
2
20 mcg/kg
2
30 mcg/kg
1
40 mcg/kg
2
50 mcg/kg
21
Number of Participants With Partial Response (PR)Primary· From Baseline (predose of Cycle 1 Day 1) through end of study (until CR, PD, initiation of alternative therapy, unacceptable toxicity, development of neutralizing antibodies, or other study discontinuation reasons) (approximately 8 years)
Partial response requires all of the following: \>=50% decrease in peripheral blood lymphocyte count from the pretreatment baseline value, \>=50% reduction in lymphadenopathy, \>=50% reduction in abnormal hepatosplenomegaly by computed tomography or physical exam, Neutrophils \>= 1,500/mcL or 50% improvement over baseline without growth factors for at least 4 weeks, Platelets \>=100,000/mcL or 50% improvement over baseline, and Hemoglobin \>= 11.0 g/dL or 50% improvement over baseline without transfusions or growth factors for at least 4 weeks. For participants who are transfusion-dependent at
Group
Value
95% CI
5 mcg/kg
3
10 mcg/kg
1
20 mcg/kg
0
30 mcg/kg
1
40 mcg/kg
1
50 mcg/kg
8
Number of Participants With Stable Disease (SD)Primary· From Baseline (predose of Cycle 1 Day 1) through end of study (until CR, PD, initiation of alternative therapy, unacceptable toxicity, development of neutralizing antibodies, or other study discontinuation reasons) (approximately 8 years)
Stable disease was characterized by not meeting the criteria for CR, PR or PD.
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
0
20 mcg/kg
1
30 mcg/kg
1
40 mcg/kg
0
50 mcg/kg
4
Number of Participants With Progressive Disease (PD)Primary· From Baseline (predose of Cycle 1 Day 1) through end of study (until CR, PD, initiation of alternative therapy, unacceptable toxicity, development of neutralizing antibodies, or other study discontinuation reasons) (approximately 8 years)
Progressive disease is defined by at least one of the following compared to pretreatment:\>= 25% increase in the sum of the products of the greatest perpendicular dimensions of at least two lymph nodes on two consecutive examinations at least 2 weeks apart (at least 1 node must be \>= 2 cm) or appearance of new palpable lymph nodes, \>=25% increase in the size of the liver and/or spleen as determined by measurement below the respective costal margin, or appearance of new palpable hepatomegaly or splenomegaly that was not previously present, \>=50% increase in the absolute number of circulating
Group
Value
95% CI
5 mcg/kg
0
10 mcg/kg
0
20 mcg/kg
0
30 mcg/kg
0
40 mcg/kg
1
50 mcg/kg
0
Time to Complete ResponsePrimary· From Baseline (predose of Cycle 1 Day 1) through end of study (until CR, PD, initiation of alternative therapy, unacceptable toxicity, development of neutralizing antibodies, or other study discontinuation reasons) (approximately 8 years)
Time to complete response (CR) was measured from the start of moxetumomab pasudotox treatment to the first documentation of CR and was evaluated only in participants who received any treatment of moxetumomab pasudotox and had achieved a CR. Time to complete response was summarized using Kaplan-Meier estimates (median time, 95% confidence interval \[CI\] for median time).
Group
Value
95% CI
10 mcg/kg
2.53
2.30 – 2.76
20 mcg/kg
9.56
4.11 – 15.01
30 mcg/kg
3.71
3.71 – 3.71
40 mcg/kg
2.53
2.27 – 2.79
50 mcg/kg
3.94
2.30 – 6.31
Time to Objective ResponsePrimary· From Baseline (predose of Cycle 1 Day 1) through end of study (until CR, PD, initiation of alternative therapy, unacceptable toxicity, development of neutralizing antibodies, or other study discontinuation reasons) (approximately 8 years)
Time to OR was measured from the start of moxetumomab pasudotox treatment to the first documentation of OR and was evaluated only in participants who received any treatment of moxetumomab pasudotox and had achieved an OR (CR or PR). Objective response (OR) was defined as the participants with confirmed CR or confirmed PR according to Response Evaluation Criteria.
Group
Value
95% CI
5 mcg/kg
3.02
2.04 – 4.80
10 mcg/kg
1.12
0.92 – 2.76
20 mcg/kg
8.2
1.38 – 15.01
30 mcg/kg
8.18
1.87 – 14.49
40 mcg/kg
1.08
0.95 – 2.79
50 mcg/kg
1.05
0.92 – 1.58
Adverse events — posted to ClinicalTrials.gov
Time frame: From start of study drug administration until 30 days after the last dose of study drug (approximately 8 years).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
A dose-escalation study to identify the dose-limiting toxicity (DLT) and maximum tolerated dose (MTD), defined as the highest dose that can safely be given to a participant and establish the safest dose based on the highest tolerated dose for clinical testing.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT06965114 — Testing the Combination of Anti-cancer Drugs, Tovorafenib Plus Rituximab, in Patients With Hairy Cell Leukemia
· Phase 1, PHASE2
· recruiting
NCT06561360 — A Study of Vemurafenib and Obinutuzumab Compared to Cladribine and Rituximab in People With Hairy Cell Leukemia (HCL)
· Phase 2
· recruiting
NCT04815356 — Phase I Study of Anti-CD22 Chimeric Receptor T Cells in Patients With Relapsed/Refractory Hairy Cell Leukemia and Varian
· Phase 1
· recruiting
NCT05388123 — Low Dose Vemurafenib and Rituximab in Hairy Cell Leukemia
· Phase 2
· active not recruiting
NCT04775745 — Study of Oral Administration of LP-168 in Patients With Relapsed or Refractory B-cell Malignancies.
· Phase 1
· recruiting
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by MedImmune LLC
Last refreshed: 2 April 2019
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00586924.