Safety and Pharmacokinetics of Intravenous and Oral APX001 in Patients With Acute Myeloid Leukemia (AML) and Neutropenia

Phase 1

Completed

• Conditions: Acute Myeloid Leukemia
• Interventions: Drug: APX001 with Standard of Care anti-fungal agent

• Registration Number: NCT03333005
• Lead Sponsor: Basilea Pharmaceutica

Brief Summary

An open-label, multi-center, Phase Ib study to determine the safety and pharmacokinetics of intravenous and oral APX001 in patients undergoing chemotherapy for Acute Myeloid Leukemia with neutropenia.

A total of 20 patients will be enrolled in this study. 10 patients in Cohort I, intravenous drug dosing and 10 patients will be enrolled in Cohort II, oral drug dosing.

All patients will receive chemotherapy for their AML according to local clinical standard of care as well as antifungal prophylaxis.

APX001 will be administered for 14 consecutive days, beginning on Study Day 3 after onset of chemotherapy

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-10-23
• Study First Submitted QC: 2017-11-02
• Study First Posted: 2017-11-06
• Study Start: 2017-11-27
• Primary Completion: 2018-10-26
• Study Completion: 2018-10-26
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-16
• Last Update Posted: 2024-05-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• Provision of written consent
• Ages 18-75 inclusive, male or female
• Diagnosis of Acute Myeloid Leukemia
• Patients entering first induction treatment chemotherapy
• Expected to be neutropenic (<500 ANC/ul) for >/= 10 days

Key

Exclusion Criteria

• Patients who received systemic antifungal therapy for proven or probable fungal infections within the last 12 months
• Current fever (> 38 degrees Celsius)
• Concomitant use of rifampin, rifabutin, ergot alkaloids, terfenadine, astemizole, cisapride, pimozide, quinidine, long acting barbiturates, neostigmine and carbamazepine

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
APX001 with Standard of Care Anti-fungal agent	APX001 with Standard of Care anti-fungal agent	-

Primary Outcome Measures

Name	Time	Method
Number of patients with treatment-related adverse events as assessed and reported by CTCAE v4.0	One to forty-four days

Secondary Outcome Measures

Name	Time	Method
Area under the plasma concentration versus time curve (AUC)	One to forty-four days
Peak Plasma Concentration (Cmax)	One to forty-four days

Trial Locations

Locations (3)

University of Munich, Grosshadern Campus; 🇩🇪 Munich, Germany

Johannes Gutenberg, University of Mainz; 🇩🇪 Mainz, Germany

University of Cologne, Center for Integrated Oncology (CIO); 🇩🇪 Cologne, Germany