Clinical trial NCT04125290: US Post-Marketing Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI)
Official title: US Post-Marketing Retrospective Observational Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI)(TM)
Study type: OBSERVATIONAL
Overall status: TERMINATED
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
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
Collaborators: name: Iqvia Pty Ltd
First posted: 2019-10-14
Enrollment: 2
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

Brief 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 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.

Eligibility criteria: Inclusion Criteria:

* 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

Primary outcomes:
measure: Incident proportion of capillary leak syndrome
timeFrame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
measure: Incident proportion of hemolytic uremic syndrome
timeFrame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
measure: Incident proportion of renal toxicity
timeFrame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
measure: Incident proportion of infusion related reactions
timeFrame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
measure: Incident proportion of electrolyte and biochemical abnormalities
timeFrame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
measure: Incident proportion of other medical events related to moxetumomab pasudotox-tdfk interruption or discontinuation
timeFrame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
measure: Incident proportion of other serious medical events that are life-threatening, resulting in hospitalizations and/or death
timeFrame: From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Secondary outcomes:
Other outcomes:
Locations:
city: Pueblo
country: United States
facility: Rocky Mountain Cancer Centers
state: Colorado
city: Bridgeton
country: United States
facility: Research Site
state: Missouri
Study officials:
affiliation: AstraZeneca
name: Archna Hale
role: STUDY_DIRECTOR
affiliation: SSM Health DePaul Hospital
name: Juan Cuevas
role: PRINCIPAL_INVESTIGATOR
affiliation: Rocky Mountain Cancer Centers
name: Travis Arculeta
role: PRINCIPAL_INVESTIGATOR
affiliation: AstraZeneca
name: Roser Calvo
role: STUDY_DIRECTOR
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