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

• ClinicalTrials.gov Identifier: NCT04125290
• Recruitment Status: Not yet recruiting
• First Posted: October 14, 2019
• Last Update Posted: October 14, 2019
• Sponsor: AstraZeneca
• Collaborator: Iqvia Pty Ltd
• Information provided by (Responsible Party): AstraZeneca

Tracking Information

• First Submitted Date: September 13, 2019
• First Posted Date: October 14, 2019
• Last Update Posted Date: October 14, 2019
• Estimated Study Start Date: December 1, 2019
• Estimated Primary Completion Date: September 11, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 11, 2019)

• 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. ]

• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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)
• 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 .
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Retrospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• 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.
• Condition: Hairy Cell Leukemia
• Intervention: Not Provided
• Study Groups/Cohorts: Not Provided

Publications *

• Bouroncle BA. Thirty-five years in the progress of hairy cell leukemia. Leuk Lymphoma. 1994;14 Suppl 1:1-12. Review.
• 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.
• 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.
• 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.
• 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 Waldenström 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.

Recruitment Information

• Recruitment Status: Not yet recruiting
• Estimated Enrollment (submitted: October 11, 2019): 80
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: September 11, 2023
• Estimated Primary Completion Date: September 11, 2023 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No

Contacts

Contact: AstraZeneca Clinical Study Information Center; 1-877-240-9479; information.center@astrazeneca.com

• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT04125290
• Other Study ID Numbers: D3143R00004
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Responsible Party: AstraZeneca
• Study Sponsor: AstraZeneca
• Collaborators: Iqvia Pty Ltd

Investigators

• Study Director: Katherine Ruffner, MD; Acerta Pharma, LLC
• Study Director: Katherine Coyle, PharmD; AstraZeneca

• PRS Account: AstraZeneca
• Verification Date: October 2019