Trial Outcomes & Findings for Moxetumomab Pasudotox-tdfk (Lumoxiti(TM)) and Either Rituximab (Rituxan(R)) or Ruxience for Relapsed Hairy Cell Leukemia (NCT NCT03805932)

Last Updated: 2025-07-24

Results Overview

Recommended safe dose of moxetumomab pasudotox-tdfk is defined as the treatment of 10 participants with no more than 2 experiencing a dose-limiting toxicity (DLT). A DLT is defined as all treatment related Grade 3-5 adverse events (AEs) occurring from the initiation of moxetumomab pasudotox-tdfk therapy to within 30 days after the last dose of moxetumomab pasudotox-tdfk treatment assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). Grade 3 is severe, Grade 4 is life-threatening, and Grade 5 is death related to adverse event.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE1

Target enrollment

18 participants

Primary outcome timeframe

4 weeks

Results posted on

2025-07-24

Participant Flow

Participant milestones

Participant milestones
Measure	Moxetumomab - Dose Escalation 30 mcg/kg Participants with Relapsed/Refractory Hairy Cell Leukemia (HCL) or Hairy Cell Leukemia (HCLv) Arm 1 Moxetumomab Pasudotox-tdfk + Rituximab Dose escalation treatment with moxetumomab -pasudotox-tdfk and rituximab Moxetumomab pasudotox-tdfk is administered at 30 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Rituximab will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Rituximab is given on day -2, on subsequent cycles, Rituximab is given on day 1.	Moxetumomab - Dose Expansion 40 mcg/kg Dose expansion; treatment with moxetumomab -pasudotox-tdfk and Ruxience Arm 1 and Arm 2 Moxetumomab Pasudotox-tdfk + Ruxience Moxetumomab pasudotox-tdfk is administered at 40 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Ruxience will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Ruxience is given on day -2, on subsequent cycles, Ruxience is given on day 1 (Delta)
Overall Study STARTED	3	15
Overall Study COMPLETED	3	15
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Moxetumomab Pasudotox-tdfk (Lumoxiti(TM)) and Either Rituximab (Rituxan(R)) or Ruxience for Relapsed Hairy Cell Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Moxetumomab - Dose Escalation 30 mcg/kg n=3 Participants Participants with Relapsed/Refractory Hairy Cell Leukemia (HCL) or Hairy Cell Leukemia (HCLv) Arm 1 Moxetumomab Pasudotox-tdfk + Rituximab Dose escalation treatment with moxetumomab -pasudotox-tdfk and rituximab Moxetumomab pasudotox-tdfk is administered at 30-40 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Rituximab will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Rituximab is given on day -2, on subsequent cycles, Rituximab is given on day 1.	Moxetumomab - Dose Expansion 40 mcg/kg n=15 Participants Dose expansion; treatment with moxetumomab -pasudotox-tdfk and Ruxience Arm 1 and Arm 2 Moxetumomab Pasudotox-tdfk + Ruxience Moxetumomab pasudotox-tdfk is administered at 30-40 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Ruxience will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Ruxience is given on day -2, on subsequent cycles, Ruxience is given on day 1 (Delta)	Total n=18 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants	10 Participants n=7 Participants	11 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	5 Participants n=7 Participants	7 Participants n=5 Participants
Age, Continuous	61.17 years STANDARD_DEVIATION 8.98 • n=5 Participants	60.07 years STANDARD_DEVIATION 8.64 • n=7 Participants	60.25 years STANDARD_DEVIATION 8.44 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	14 Participants n=7 Participants	17 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants	15 Participants n=7 Participants	18 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	14 Participants n=7 Participants	17 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants	15 participants n=7 Participants	18 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 weeks

Outcome measures

Outcome measures
Measure	All Participants n=18 Participants All participants in Group 1 and Group 2 who received at least one dose of Moxetumomab 30-40 mcg/kg.	Moxetumomab - Dose Expansion 40 mcg/kg Dose expansion; treatment with moxetumomab -pasudotox-tdfk and Ruxience Arm 1 and Arm 2 Moxetumomab Pasudotox-tdfk + Ruxience Moxetumomab pasudotox-tdfk is administered at 30-40 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Ruxience will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Ruxience is given on day -2, on subsequent cycles, Ruxience is given on day 1 (Delta)
Recommended Safe Dose of Moxetumomab Pasudotox-tdfk	40 mcg/kg	—

PRIMARY outcome

Timeframe: 4 weeks

Recommended safe dose of rituximab/Ruxience is defined as the treatment of 10 participants with no more than 2 experiencing a dose-limiting toxicity (DLT). A DLT is defined as all treatment related Grade 3-5 adverse events (AEs) occurring from the initiation of moxetumomab pasudotox-tdfk therapy to within 30 days after the last dose of moxetumomab pasudotox-tdfk treatment assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). Grade 3 is severe, Grade 4 is life-threatening, and Grade 5 is death related to adverse event.

Outcome measures

Outcome measures
Measure	All Participants n=18 Participants All participants in Group 1 and Group 2 who received at least one dose of Moxetumomab 30-40 mcg/kg.	Moxetumomab - Dose Expansion 40 mcg/kg Dose expansion; treatment with moxetumomab -pasudotox-tdfk and Ruxience Arm 1 and Arm 2 Moxetumomab Pasudotox-tdfk + Ruxience Moxetumomab pasudotox-tdfk is administered at 30-40 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Ruxience will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Ruxience is given on day -2, on subsequent cycles, Ruxience is given on day 1 (Delta)
Recommended Safe Dose of Rituximab/Ruxience	40 mcg/kg	—

SECONDARY outcome

Timeframe: 28-42 days after day 1 of the last treatment.

Number of participants whose cancer shrinks or disappears after treatment defined as minimal residual disease. MRD is no hairy cell leukemia (HCL) in the blood and bone marrow aspirate flow determined by immunohistochemistry (IHC) and flow cytometry of blood and bone marrow aspirate.

Outcome measures

Outcome measures
Measure	All Participants n=3 Participants All participants in Group 1 and Group 2 who received at least one dose of Moxetumomab 30-40 mcg/kg.	Moxetumomab - Dose Expansion 40 mcg/kg n=15 Participants Dose expansion; treatment with moxetumomab -pasudotox-tdfk and Ruxience Arm 1 and Arm 2 Moxetumomab Pasudotox-tdfk + Ruxience Moxetumomab pasudotox-tdfk is administered at 30-40 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Ruxience will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Ruxience is given on day -2, on subsequent cycles, Ruxience is given on day 1 (Delta)
Number of Participants Whose Cancer Shrinks or Disappears After Treatment	2 Participants	11 Participants

SECONDARY outcome

Timeframe: 28-42 days after day 1 of the last treatment.

MRD-free is defined as participants with no hairy cell leukemia (HCL) in the blood and bone marrow aspirate flow determined by immunohistochemistry (IHC) and flow cytometry of blood and bone marrow aspirate.

Outcome measures

Outcome measures
Measure	All Participants n=3 Participants All participants in Group 1 and Group 2 who received at least one dose of Moxetumomab 30-40 mcg/kg.	Moxetumomab - Dose Expansion 40 mcg/kg n=15 Participants Dose expansion; treatment with moxetumomab -pasudotox-tdfk and Ruxience Arm 1 and Arm 2 Moxetumomab Pasudotox-tdfk + Ruxience Moxetumomab pasudotox-tdfk is administered at 30-40 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Ruxience will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Ruxience is given on day -2, on subsequent cycles, Ruxience is given on day 1 (Delta)
Number of Participants Who Are Minimal Residual Disease (MRD)-Free	2 Participants	11 Participants

SECONDARY outcome

Timeframe: 28-42 days after day 1 of the last treatment.

DOR is defined as the time of initial response until documented tumor progression. Response was assessed by the and is defined as an increase in symptoms or \>25% decline in hematologic parameters related to disease, based on the judgement of the principal investigator. ≥50% increase in sum of products of perpendicular diameters of evaluable (\> 2cm) lymphadenopathy or appearance of new evaluable lymph nodes \> 2 cm short axis.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: From the initiation of moxetumomab pasudotox-tdfk therapy to within 30 days after the last dose

Dose limiting toxicity (DLT) is defined as all treatment related Grade 3-5 adverse events (AEs) occurring from the initiation of moxetumomab pasudotox-tdfk therapy to within 30 days after the last dose of moxetumomab pasudotox-tdfk treatment. Adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). Grade 3 is severe, Grade 4 is life-threatening, and Grade 5 is death related to adverse event.

Outcome measures

Outcome measures
Measure	All Participants n=3 Participants All participants in Group 1 and Group 2 who received at least one dose of Moxetumomab 30-40 mcg/kg.	Moxetumomab - Dose Expansion 40 mcg/kg n=15 Participants Dose expansion; treatment with moxetumomab -pasudotox-tdfk and Ruxience Arm 1 and Arm 2 Moxetumomab Pasudotox-tdfk + Ruxience Moxetumomab pasudotox-tdfk is administered at 30-40 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Ruxience will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Ruxience is given on day -2, on subsequent cycles, Ruxience is given on day 1 (Delta)
Number of Participants With a Dose-limiting Toxicity (DLT)	0 Participants	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group.

Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Outcome measures
Measure	All Participants n=3 Participants All participants in Group 1 and Group 2 who received at least one dose of Moxetumomab 30-40 mcg/kg.	Moxetumomab - Dose Expansion 40 mcg/kg n=15 Participants Dose expansion; treatment with moxetumomab -pasudotox-tdfk and Ruxience Arm 1 and Arm 2 Moxetumomab Pasudotox-tdfk + Ruxience Moxetumomab pasudotox-tdfk is administered at 30-40 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Ruxience will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Ruxience is given on day -2, on subsequent cycles, Ruxience is given on day 1 (Delta)
Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)	3 Participants	15 Participants

Adverse Events

Moxetumomab - Dose Escalation 30 mcg/kg

Serious events: 0 serious events

Other events: 3 other events

Deaths: 1 deaths

Moxetumomab - Dose Expansion 40 mcg/kg

Serious events: 2 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Moxetumomab - Dose Escalation 30 mcg/kg n=3 participants at risk Participants with Relapsed/Refractory Hairy Cell Leukemia (HCL) or Hairy Cell Leukemia (HCLv) Arm 1 Moxetumomab Pasudotox-tdfk + Rituximab Dose escalation treatment with moxetumomab -pasudotox-tdfk and rituximab Moxetumomab pasudotox-tdfk is administered at 30 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Rituximab will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Rituximab is given on day -2, on subsequent cycles, Rituximab is given on day 1.	Moxetumomab - Dose Expansion 40 mcg/kg n=15 participants at risk Dose expansion; treatment with moxetumomab -pasudotox-tdfk and Ruxience Arm 1 and Arm 2 Moxetumomab Pasudotox-tdfk + Ruxience Moxetumomab pasudotox-tdfk is administered at 40 ug/Kg intravenous (iv) over 30 min given on days 1, 3, 5 of each cycle. Ruxience will be administered at 375mg/m\^2 iv, 50-400 mg/hour (hr). On cycle 1, Ruxience is given on day -2, on subsequent cycles, Ruxience is given on day 1 (Delta)
Infections and infestations Catheter related infection	0.00% 0/3 • Date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group.	6.7% 1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group.
General disorders Fever	0.00% 0/3 • Date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group.	6.7% 1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group.
Blood and lymphatic system disorders Hemolytic uremic syndrome	0.00% 0/3 • Date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group.	6.7% 1/15 • Number of events 1 • Date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group.

Other adverse events

Additional Information

Dr. Robert J. Kreitman

National Cancer Institute

Phone: 301-480-6187

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place