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

May 16, 2024 updated by: Basilea Pharmaceutica

An Open Label, Multi-Center Study to Determine the Safety and Pharmacokinetics of Intravenous and Oral APX001 in Patients Undergoing Chemotherapy for Acute Myeloid Leukemia With Neutropenia

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

20

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Cologne, Germany, 50931
        • University of Cologne, Center for Integrated Oncology (CIO)
      • Mainz, Germany, 55131
        • Johannes Gutenberg, University of Mainz
      • Munich, Germany, 81377
        • University of Munich, Grosshadern Campus

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key 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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: APX001 with Standard of Care Anti-fungal agent
Drug: APX001 with Standard of Care anti-fungal agent
safety assessment

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
Area under the plasma concentration versus time curve (AUC)
Time Frame: One to forty-four days
One to forty-four days
Peak Plasma Concentration (Cmax)
Time Frame: One to forty-four days
One to forty-four days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Basilea Pharmaceutica

Collaborators

The Clinical Trials Centre Cologne

Investigators

  • Study Director: Marc Engelhardt, Basilea Pharmaceutica International Ltd, Allschwil

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 27, 2017

Primary Completion (Actual)

October 26, 2018

Study Completion (Actual)

October 26, 2018

Study Registration Dates

First Submitted

October 23, 2017

First Submitted That Met QC Criteria

November 2, 2017

First Posted (Actual)

November 6, 2017

Study Record Updates

Last Update Posted (Actual)

May 17, 2024

Last Update Submitted That Met QC Criteria

May 16, 2024

Last Verified

May 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • APX001-103

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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