A Study Investigating the Safety, Tolerability and Efficacy of ASP7517 in Subjects With Relapsed/Refractory Acute Myeloid Leukemia (AML) and Relapsed/Refractory Higher Risk Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndrome Clinical Trial

Official title: A Phase 1/2 Open-label Study Investigating the Safety, Tolerability and Efficacy of ASP7517 in Subjects With Relapsed/Refractory Acute Myeloid Leukemia (AML) and Relapsed/Refractory Higher Risk Myelodysplastic Syndrome (MDS)

Status Completed

Clinical Trial Summary

The purpose of this study is to evaluate the safety and tolerability and to determine the recommended phase 2 dose (RP2D) and/or the maximum tolerated dose (MTD) of ASP7517.

This study will also evaluate the clinical response of ASP7517 as well as other measures of anticancer activity of ASP7517.

Clinical Trial Description

This study consists of 2 parts: phase 1 dose escalation and phase 2 dose expansion.

Phase 1 Dose Escalation:

Approximately 18 subjects with either relapsed/refractory (R/R) AML or R/R higher risk MDS will be enrolled. Participants will receive 2 single doses of ASP7517 via intravenous infusion. Dosing will occur on day 1 of each cycle. Each cycle is defined as 28 days with a total of 2 treatment cycles.

Participants must be managed under hospitalization for at least 7 days during the first cycle of the dose escalation phase. In addition, prior to hospital discharge, participant safety must be ensured by performing medical tests and procedures listed on day 7 of cycle 1 and tests considered clinically necessary to evaluate the participant's general condition and adverse event (AE) resolution. The participant should also be followed on an outpatient basis on planned visits during cycles 1 and 2 after hospital discharge during the dose limiting toxicity (DLT) assessment period to closely monitor any AEs. Participants may be hospitalized days 1 to 7 during cycle 2.

Phase 2 Dose Expansion:

Approximately 104 participants per dose level will be enrolled. Each dose level may enroll up to 52 R/R AML participants and up to 52 R/R higher risk MDS participants. Both groups of participants will enroll in parallel and independently. The number of dose levels investigated during phase 2 will be based upon the data from phase 1. When escalation and expansion cohorts are both open for enrollment, enrollment into escalation cohorts takes priority such that participants who are eligible for both will be preferentially enrolled in the escalation cohorts. ;

Related Conditions & MeSH terms

• Acute Myeloid Leukemia (AML)
• Leukemia
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute
• Myelodysplastic Syndrome
• Myelodysplastic Syndromes
• Preleukemia
• Syndrome

• NCT number: NCT04079296
• Study type: Interventional
• Source: Astellas Pharma Inc
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: September 19, 2019
• Completion date: April 21, 2023