US Post-Marketing Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI) (PROXY)

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

Study Overview

• Status: Terminated
• Conditions: Hairy Cell Leukemia

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 Type: Observational
• Enrollment (Actual): 2

Contacts and Locations

Study Locations

• United States
  • Colorado
    • Pueblo, Colorado, United States, 81008
      • Rocky Mountain Cancer Centers
  • Missouri
    • Bridgeton, Missouri, United States, 63044
      • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incident proportion of capillary leak syndrome		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		From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.
Incident proportion of renal toxicity		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		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	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		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		From day 1 of moxetumomab pasudotox-tdfk initiation up to 30 days after last dose of moxetumomab pasudotox-tdfk.

Collaborators and Investigators

• Sponsor: AstraZeneca
• Collaborators: Iqvia Pty Ltd
• Investigators: Study Director: Archna Hale, AstraZeneca; Principal Investigator: Juan Cuevas, SSM Health DePaul Hospital; Principal Investigator: Travis Arculeta, Rocky Mountain Cancer Centers; Study Director: Roser Calvo, AstraZeneca

Publications and helpful links

General Publications

• Jemal 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.
• Bouroncle BA. Thirty-five years in the progress of hairy cell leukemia. Leuk Lymphoma. 1994;14 Suppl 1:1-12.
• Kreitman 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.
• Kroft 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.
• Leleu 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.
• Seymour 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.
• Seymour 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.

Study record dates

Study Major Dates

• Study Start (Actual): December 9, 2019
• Primary Completion (Actual): June 21, 2021
• Study Completion (Actual): June 21, 2021

Study Registration Dates

• First Submitted: September 13, 2019
• First Submitted That Met QC Criteria: October 11, 2019
• First Posted (Actual): October 14, 2019

Study Record Updates

• Last Update Posted (Actual): April 21, 2023
• Last Update Submitted That Met QC Criteria: April 20, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Keywords: renal toxicity; capillary leak syndrome; hemolytic uremic syndrome; infusion-related reaction; electrolyte abnormalities; HCL and all relevant indications where LUMOXITI will be prescribed
• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Leukemia; Leukemia, Hairy Cell
• Other Study ID Numbers: D3143R00004

Drug and device information, study documents

• Studies a U.S. FDA-regulated device product: No