Trial · NCT01829711
Moxetumomab Pasudotox for Advanced Hairy Cell Leukemia
NCT01829711, Phase 3, Completed, MedImmune LLC; results first posted 2018-08-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Moxetumomab Pasudotox for Advanced Hairy Cell Leukemia. Trials, record NCT01829711. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01829711
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Moxetumomab Pasudotox for Advanced Hairy Cell Leukemia
- NCT number
- NCT01829711
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 80 participants, actual
- Lead sponsor
- MedImmune LLC
- Conditions
- Leukemia, Hairy Cell
- Interventions
- Moxetumomab pasudotox, IV Bag Protectant for Moxetumomab pasudotox
- First posted
- 2013-04-11
- Last updated
- 2020-04-08
- Results first posted
- 2018-08-27
Source ClinicalTrials.gov
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Durable Complete Response (CR) Assessed by Blinded Independent Central Review | 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) | 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. |
| Primary | Percentage of Participants With Durable CR by Investigator's Assessment | 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) | Durable CR was defined as overall response that meets blood, bone marrow and imaging criteria for CR, followed by a \>180 day duration of 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. |
| Secondary | Percentage of Participants With Minimal Residual Disease (MRD) Positive or MRD Negative CR Assessed by Blinded Independent Central Review | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | The MRD status by blinded independent review refers specifically to results of central pathologist read of bone marrow biopsy by immunohistochemistry. The CR with Positive or Negative MRD requires all of the following to be present: * No evidence of leukemic cells in the peripheral blood and/or by routine H/E staining of bone marrow. Minimal Residual Disease: CR with HCL evident in blood or in bone marrow biopsy by immunohistochemistry. * Resolution of any hepatomegaly, splenomegaly, and abnormal (\>= 2 cm minimum length) lymphadenopathy by CT or MRI. Although a normal spleen size is not defined, the maximum diameter of the spleen should be either \< 17 cm or have decreased by \>25% from its baseline. * Normal 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. |
| Secondary | Percentage of Participants With MRD Positive or MRD Negative CR by Investigator's Assessment | Prior to each treatment cycle, end of treatment, and at follow-up visits every 3 months for the next 24 months and every 6 months thereafter (Approximately 6 years) | The MRD status by investigator refers to results of investigator assessment of bone marrow biopsy or bone marrow aspirate by immunohistochemistry and/or flow cytometry. The CR with Positive or Negative MRD requires all of the following to be present: * No evidence of leukemic cells in the peripheral blood and/or by routine H/E staining of bone marrow. Minimal Residual Disease: CR with HCL evident in blood or marrow by flow cytometry. * Resolution of any hepatomegaly, splenomegaly, and abnormal (\>= 2 cm minimum length) lymphadenopathy by CT or MRI. Although a normal spleen size is not defined, the maximum diameter of the spleen should be either \< 17 cm or have decreased by \>25% from its baseline. * Normal 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. |
| Secondary | Time to CR Assessed by Blinded Independent Central Review | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | Time to CR was defined as the time from the start of moxetumomab pasudotox administration to the first documentation of CR. |
| Secondary | Duration of CR Assessed by Blinded Independent Central Review | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | Duration of CR was defined as the duration from documentation of CR to the time of relapse from CR. Relapse from CR was defined as any CR criteria (blood counts, imaging or bone marrow) no longer consistent with CR. 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); normal 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. |
| Secondary | Duration of Hematologic Remission | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | Duration of HR was defined as the duration from documentation of HR to the time of relapse. HR was defined as the blood counts required for CR as normal 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. Duration of HR was censored on the date of the last hematologic assessment for participants who have no documented relapse based on blood count prior to data cutoff, dropout, or initiation of alternative anticancer therapy. |
| Secondary | Time to Hematologic Remission | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | Time to HR was defined as the time from the start of moxetumomab pasudotox administration to the first documentation of HR. HR was defined as the blood counts required for CR as normal 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. |
| Secondary | Percentage of Participants With Objective Response (OR) Assessed by Blinded Independent Central Review | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | The OR was defined as number of participants with a best response of CR or PR. CR requires all of 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); normal CBC (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. PR requires all of following for a period of at least 4 weeks: \>=50% decrease or normalization (\<5.0 x 10\^9/L) in peripheral blood lymphocyte count and \>=50% reduction in lymphadenopathy and in abnormal haepatosplenomegaly by CT or MRI from pre-treatment baseline value; normal CBC as mentioned above or 50% improvement in CBC values over baseline without transfusions or growth factors for at least 4 weeks. |
| Secondary | Percentage of Participants With Objective Response by Investigator's Assessment | Prior to each treatment cycle, end of treatment, and at follow-up visits every 3 months for the next 24 months and every 6 months thereafter (Approximately 6 years) | The OR was defined as number of participants with a best response of CR or PR. CR requires all of 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); normal CBC (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. PR requires all of following for a period of at least 4 weeks: \>=50% decrease or normalization (\<5.0 x 10\^9/L) in peripheral blood lymphocyte count and \>=50% reduction in lymphadenopathy and in abnormal haepatosplenomegaly by CT or MRI from pre-treatment baseline value; normal CBC as mentioned above or 50% improvement in CBC values over baseline without transfusions or growth factors for at least 4 weeks. |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 registered outcomesOutcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Durable Complete Response (CR) Assessed by Blinded Independent Central Review | 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) | The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. | Percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Durable CR by Investigator's Assessment | 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) | The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Minimal Residual Disease (MRD) Positive or MRD Negative CR Assessed by Blinded Independent Central Review | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With MRD Positive or MRD Negative CR by Investigator's Assessment | Prior to each treatment cycle, end of treatment, and at follow-up visits every 3 months for the next 24 months and every 6 months thereafter (Approximately 6 years) | The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. | Percentage of participants | NUMBER |
| SECONDARY | Time to CR Assessed by Blinded Independent Central Review | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. Time to CR was evaluated for participants who achieved CR per independent central review. | Months | MEDIAN |
| SECONDARY | Duration of CR Assessed by Blinded Independent Central Review | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. Duration of CR was evaluated for participants who achieved CR per independent central review. | Months | MEDIAN |
| SECONDARY | Duration of Hematologic Remission | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. Duration of HR was evaluated for participants who achieved HR. | Months | MEDIAN |
| SECONDARY | Time to Hematologic Remission | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. Time to HR was evaluated for participants in the ITT population who achieved HR. | Months | MEDIAN |
| SECONDARY | Percentage of Participants With Objective Response (OR) Assessed by Blinded Independent Central Review | Prior to each treatment cycle, EOT (up to 24 weeks), monthly from the EOT assessment until the Day 181 assessment (only for CBC), at follow-up visits every 3 months for the next 24 months, and every 6 months thereafter (approximately 6 years) | The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Objective Response by Investigator's Assessment | Prior to each treatment cycle, end of treatment, and at follow-up visits every 3 months for the next 24 months and every 6 months thereafter (Approximately 6 years) | The ITT population included participants who entered into the study and treated with moxetumomab pasudotox. | Percentage of participants | NUMBER |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group103
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Durable Complete Response (CR) Assessed by Blinded Independent Central Review | Moxetumomab Pasudotox 40 µg/kg | 36.3 | 95% Confidence Interval 25.8 to 47.8 | Percentage of participants | |
| Percentage of Participants With Durable CR by Investigator's Assessment | Moxetumomab Pasudotox 40 µg/kg | 48.8 | 95% Confidence Interval 37.4 to 60.2 | Percentage of participants | |
| Percentage of Participants With Minimal Residual Disease (MRD) Positive or MRD Negative CR Assessed by Blinded Independent Central Review | Moxetumomab Pasudotox 40 µg/kg | MRD negative CR | 33.8 | 95% Confidence Interval 23.6 to 45.2 | Percentage of participants |
| Percentage of Participants With Minimal Residual Disease (MRD) Positive or MRD Negative CR Assessed by Blinded Independent Central Review | Moxetumomab Pasudotox 40 µg/kg | MRD positive CR | 7.5 | 95% Confidence Interval 2.8 to 15.6 | Percentage of participants |
| Percentage of Participants With MRD Positive or MRD Negative CR by Investigator's Assessment | Moxetumomab Pasudotox 40 µg/kg | MRD negative CR | 32.5 | 95% Confidence Interval 22.4 to 43.9 | Percentage of participants |
| Percentage of Participants With MRD Positive or MRD Negative CR by Investigator's Assessment | Moxetumomab Pasudotox 40 µg/kg | MRD positive CR | 7.5 | 95% Confidence Interval 2.8 to 15.6 | Percentage of participants |
| Time to CR Assessed by Blinded Independent Central Review | Moxetumomab Pasudotox 40 µg/kg | 5.9 | Full Range 1.8 to 13.2 | Months | |
| Duration of CR Assessed by Blinded Independent Central Review | Moxetumomab Pasudotox 40 µg/kg | 62.8 | 95% Confidence Interval 35.7 to 62.8 | Months | |
| Duration of Hematologic Remission | Moxetumomab Pasudotox 40 µg/kg | 45.8 | 95% Confidence Interval 25.9 to 71.5 | Months | |
| Time to Hematologic Remission | Moxetumomab Pasudotox 40 µg/kg | 1.1 | Full Range 0.2 to 12.9 | Months |
1 to 10 of 103Source ClinicalTrials.gov
See all 103 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Moxetumomab Pasudotox 40 µg/kg | 80 | |
| Overall Study | COMPLETED | Moxetumomab Pasudotox 40 µg/kg | 37 | |
| Overall Study | NOT COMPLETED | Moxetumomab Pasudotox 40 µg/kg | 43 |
Source ClinicalTrials.gov
Adverse events114
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Moxetumomab Pasudotox 40 µg/kg | 17 | 80 | 41 |
| other | Neutropenia | Blood and lymphatic system disorders | Moxetumomab Pasudotox 40 µg/kg | 4 | 80 | 5 |
| other | Sinus tachycardia | Cardiac disorders | Moxetumomab Pasudotox 40 µg/kg | 6 | 80 | 6 |
| other | Cataract | Eye disorders | Moxetumomab Pasudotox 40 µg/kg | 4 | 80 | 4 |
| other | Dry eye | Eye disorders | Moxetumomab Pasudotox 40 µg/kg | 6 | 80 | 6 |
| other | Vision blurred | Eye disorders | Moxetumomab Pasudotox 40 µg/kg | 7 | 80 | 8 |
| other | Abdominal distension | Gastrointestinal disorders | Moxetumomab Pasudotox 40 µg/kg | 10 | 80 | 12 |
| other | Abdominal pain | Gastrointestinal disorders | Moxetumomab Pasudotox 40 µg/kg | 7 | 80 | 7 |
| other | Abdominal pain upper | Gastrointestinal disorders | Moxetumomab Pasudotox 40 µg/kg | 4 | 80 | 5 |
| other | Constipation | Gastrointestinal disorders | Moxetumomab Pasudotox 40 µg/kg | 18 | 80 | 21 |
1 to 10 of 114Source ClinicalTrials.gov
See all 114 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Moxetumomab Pasudotox 40 µg/kg | 4 | 80 |
| Total, other adverse events | Moxetumomab Pasudotox 40 µg/kg | 77 | 80 |
| Total, serious adverse events | Moxetumomab Pasudotox 40 µg/kg | 28 | 80 |
Source ClinicalTrials.gov
Papers7
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01829711
GET https://conductscience.org/api/trials/v1/NCT01829711/registered?page=2