Hairy Cell LeukemiaPROXYNCT04125290
US Post-Marketing Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI)
An observational study of Hairy Cell Leukemia, run by AstraZeneca at 2 sites in United States.
Trial summary
This study is being conducted to satisfy a post-marketing requirement (PMR) to provide evidence characterizing 1) the safety of moxetumomab pasudotox-tdfk in patients who are 65 years of age and older and/or 2) the safety of moxetumomab pasudotox-tdfk in patients who have moderate renal impairment defined as an… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hairy Cell Leukemia
- How many people
- 2 participants
- Where
- 2 sitesUnited States
- Study dates
- 9 Dec 2019to 21 Jun 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study population will be patients who are prescribed moxetumomab-pasudotox-tdfk who are ≥65 years old at the time of starting initial treatment with moxetumomab pasudotox-tdfk or are ≥18 years old with moderate renal impairment defined as an estimated GFR of 30-59 ml/min, at the time of starting initial treatment with moxetumomab pasudotox-tdfk. These two populations may not be mutually exclusive.
Who may be able to join
- Provision of written informed consent, if required
- Patient received at least 1 dose of moxetumomab pasudotox-tdfk and has completed or discontinued the treatment
- Patient has a medical record available from the start of first dose of moxetumomab pasudotox tdfk
- AND at least 1 of the following:
- Patient is ≥65 years old at the time of starting initial treatment with moxetumomab pasudotox-tdfk OR
- Adult (≥18 years old) patient has moderate renal impairment, at the time of starting initial treatment with moxetumomab pasudotox-tdfk
Who cannot join
None listed.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Research Site
Bridgeton, Missouri, 63044
Rocky Mountain Cancer Centers
Pueblo, Colorado, 81008
About this study
Official title: US Post-Marketing Retrospective Observational Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI)(TM)
This study is being conducted to satisfy a post-marketing requirement (PMR) to provide evidence characterizing 1) the safety of moxetumomab pasudotox-tdfk in patients who are 65 years of age and older and/or 2) the safety of moxetumomab pasudotox-tdfk in patients who have moderate renal impairment defined as an estimated GFR of 30-59 ml/min
Read the detailed description
The pivotal Phase 3 study (Study 1053) supported full approval of moxetumomab pasudotox-tdfk from the US Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who received at least two prior systemic therapies, including treatment with a PNA, on 13 September 2018. Since HCL is a rare disease, clinical research has limited information concerning the safety of moxetumomab pasudotox-tdfk in elderly patient populations and patients with moderate renal impairment.This study is being conducted to satisfy a post-marketing requirement (PMR) to provide evidence characterizing the safety of moxetumomab in these patients.
- Conditions
- Hairy Cell Leukemia
- Keywords
- capillary leak syndrome, hemolytic uremic syndrome, renal toxicity, infusion-related reaction, electrolyte abnormalities, HCL and all relevant indications where LUMOXITI will be prescribed
- Medical subject headings
- Leukemia, Hairy Cell, Capillary Leak Syndrome, Hemolytic-Uremic Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
Incident proportion of capillary leak syndrome
Measured: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of hemolytic uremic syndrome
Measured: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of renal toxicity
Measured: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of infusion related reactions
Measured: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of electrolyte and biochemical abnormalities
Electrolyte and biochemical abnormalities are defined as laboratory measurements of interest that exceed local laboratory standards
Measured: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of other medical events related to moxetumomab pasudotox-tdfk interruption or discontinuation
Measured: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of other serious medical events that are life-threatening, resulting in hospitalizations and/or death
Measured: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2019
- Study start9 Dec 2019
- Primary completion21 Jun 2021
- Study completion21 Jun 2021
Why it stopped: Study stopped due to lack of recruitment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Hairy Cell LeukemiaStudies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- IQVIA Pty Ltd
- Responsible party
- Sponsor
- Investigators
- Archna Hale, Study director, AstraZenecaJuan Cuevas, Principal investigator, SSM Health DePaul HospitalTravis Arculeta, Principal investigator, Rocky Mountain Cancer CentersRoser Calvo, Study director, AstraZeneca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04125290
- Sponsor's protocol ID
- D3143R00004
- Acronym
- PROXY
- Status last verified
- Jul 2025
- First submitted
- 13 Sep 2019
- Last updated
- 18 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated device
- No
Publications
Bouroncle BA. Thirty-five years in the progress of hairy cell leukemia. Leuk Lymphoma. 1994;14 Suppl 1:1-12.
PubMed 7820038Jemal 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.
PubMed 17237035Kreitman 2018, Leukemia https://doi.org/10.1038/s41375-018-0210-1
Kreitman RJ, Cheson BD. Malignancy: Current Clinical Practice: Treatment of Hairy Cell Leukemia at the Close of the 20th Century. Hematology. 1999;4(4):283-303. doi: 10.1080/10245332.1999.11746452.
PubMed 11399570Kroft SH, Tallman MS, Shaw JM, Thangavelu M, Peterson LC. Myelodysplasia following treatment of chronic lymphocytic leukemia (CLL) with 2-chlorodeoxyadenosine (2-CdA). Leukemia. 1997 Jan;11(1):170. doi: 10.1038/sj.leu.2400523. No abstract available.
PubMed 9001435Leleu X, Soumerai J, Roccaro A, Hatjiharissi E, Hunter ZR, Manning R, Ciccarelli BT, Sacco A, Ioakimidis L, Adamia S, Moreau AS, Patterson CJ, Ghobrial IM, Treon SP. Increased incidence of transformation and myelodysplasia/acute leukemia in patients with Waldenstrom macroglobulinemia treated with nucleoside analogs. J Clin Oncol. 2009 Jan 10;27(2):250-5. doi: 10.1200/JCO.2007.15.1530. Epub 2008 Dec 8.
PubMed 19064987Seymour JF, Kurzrock R, Freireich EJ, Estey EH. 2-chlorodeoxyadenosine induces durable remissions and prolonged suppression of CD4+ lymphocyte counts in patients with hairy cell leukemia. Blood. 1994 May 15;83(10):2906-11.
PubMed 7910051Seymour JF, Talpaz M, Kurzrock R. Response duration and recovery of CD4+ lymphocytes following deoxycoformycin in interferon-alpha-resistant hairy cell leukemia: 7-year follow-up. Leukemia. 1997 Jan;11(1):42-7. doi: 10.1038/sj.leu.2400513.
PubMed 9001417
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- US Post-Marketing Retrospective Observational Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI)(TM)
- ClinicalTrials.gov ID
- NCT04125290
- Acronym / sponsor ID
- PROXY / D3143R00004
- Conditions
- Hairy Cell Leukemia
- Keywords
- capillary leak syndrome, hemolytic uremic syndrome, renal toxicity, infusion-related reaction, electrolyte abnormalities, HCL and all relevant indications where LUMOXITI will be prescribed
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Study stopped due to lack of recruitment.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Collaborators
- IQVIA Pty Ltd
- Study start
- 9 Dec 2019 (actual)
- Primary completion
- 21 Jun 2021 (actual)
- Study completion
- 21 Jun 2021 (actual)
- First posted
- 14 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is being conducted to satisfy a post-marketing requirement (PMR) to provide evidence characterizing 1) the safety of moxetumomab pasudotox-tdfk in patients who are 65 years of age and older and/or 2) the safety of moxetumomab pasudotox-tdfk in patients who have moderate renal impairment defined as an estimated GFR of 30-59 ml/min
Detailed description
The pivotal Phase 3 study (Study 1053) supported full approval of moxetumomab pasudotox-tdfk from the US Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who received at least two prior systemic therapies, including treatment with a PNA, on 13 September 2018. Since HCL is a rare disease, clinical research has limited information concerning the safety of moxetumomab pasudotox-tdfk in elderly patient populations and patients with moderate renal impairment.This study is being conducted to satisfy a post-marketing requirement (PMR) to provide evidence characterizing the safety of moxetumomab in these patients.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study population will be patients who are prescribed moxetumomab-pasudotox-tdfk who are ≥65 years old at the time of starting initial treatment with moxetumomab pasudotox-tdfk or are ≥18 years old with moderate renal impairment defined as an estimated GFR of 30-59 ml/min, at the time of starting initial treatment with moxetumomab pasudotox-tdfk. These two populations may not be mutually exclusive.
Key inclusion criteria (6 listed)
- Provision of written informed consent, if required
- Patient received at least 1 dose of moxetumomab pasudotox-tdfk and has completed or discontinued the treatment
- Patient has a medical record available from the start of first dose of moxetumomab pasudotox tdfk
- Patient is ≥65 years old at the time of starting initial treatment with moxetumomab pasudotox-tdfk OR
- Adult (≥18 years old) patient has moderate renal impairment, at the time of starting initial treatment with moxetumomab pasudotox-tdfk
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Incident proportion of capillary leak syndrome
Time frame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of hemolytic uremic syndrome
Time frame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of renal toxicity
Time frame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of infusion related reactions
Time frame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of electrolyte and biochemical abnormalities
Time frame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Electrolyte and biochemical abnormalities are defined as laboratory measurements of interest that exceed local laboratory standards
Incident proportion of other medical events related to moxetumomab pasudotox-tdfk interruption or discontinuation
Time frame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of other serious medical events that are life-threatening, resulting in hospitalizations and/or death
Time frame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Results and safety
The study was stopped early (reason given: Study stopped due to lack of recruitment.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2025.
- Background: PubMed 7820038Bouroncle BA. Thirty-five years in the progress of hairy cell leukemia. Leuk Lymphoma. 1994;14 Suppl 1:1-12.
- Background: PubMed 17237035Jemal 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.
- BackgroundKreitman 2018, Leukemia https://doi.org/10.1038/s41375-018-0210-1
- Background: PubMed 11399570Kreitman RJ, Cheson BD. Malignancy: Current Clinical Practice: Treatment of Hairy Cell Leukemia at the Close of the 20th Century. Hematology. 1999;4(4):283-303. doi: 10.1080/10245332.1999.11746452.
- Background: PubMed 9001435Kroft SH, Tallman MS, Shaw JM, Thangavelu M, Peterson LC. Myelodysplasia following treatment of chronic lymphocytic leukemia (CLL) with 2-chlorodeoxyadenosine (2-CdA). Leukemia. 1997 Jan;11(1):170. doi: 10.1038/sj.leu.2400523. No abstract available.
- Background: PubMed 19064987Leleu X, Soumerai J, Roccaro A, Hatjiharissi E, Hunter ZR, Manning R, Ciccarelli BT, Sacco A, Ioakimidis L, Adamia S, Moreau AS, Patterson CJ, Ghobrial IM, Treon SP. Increased incidence of transformation and myelodysplasia/acute leukemia in patients with Waldenstrom macroglobulinemia treated with nucleoside analogs. J Clin Oncol. 2009 Jan 10;27(2):250-5. doi: 10.1200/JCO.2007.15.1530. Epub 2008 Dec 8.
- Background: PubMed 7910051Seymour JF, Kurzrock R, Freireich EJ, Estey EH. 2-chlorodeoxyadenosine induces durable remissions and prolonged suppression of CD4+ lymphocyte counts in patients with hairy cell leukemia. Blood. 1994 May 15;83(10):2906-11.
- Background: PubMed 9001417Seymour JF, Talpaz M, Kurzrock R. Response duration and recovery of CD4+ lymphocytes following deoxycoformycin in interferon-alpha-resistant hairy cell leukemia: 7-year follow-up. Leukemia. 1997 Jan;11(1):42-7. doi: 10.1038/sj.leu.2400513.
- CSR Synopsis RedactedLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Archna Hale study director
AstraZeneca
Juan Cuevas principal investigator
SSM Health DePaul Hospital
Travis Arculeta principal investigator
Rocky Mountain Cancer Centers
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hairy Cell Leukemia, run by AstraZeneca at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 18 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Study stopped due to lack of recruitment.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incident proportion of capillary leak syndrome (time frame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04125290, which also lists 8 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jul 2025.