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

NCT03333005 | Completed Phase 1

• 1 other identifier: APX001-103
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 3

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

Conditions

Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Number of patients with treatment-related adverse events as assessed and reported by CTCAE v4.0

  One to forty-four days

Secondary Outcomes (2)

• Area under the plasma concentration versus time curve (AUC)

  One to forty-four days

• Peak Plasma Concentration (Cmax)

  One to forty-four days

Study Arms (1)

APX001 with Standard of Care Anti-fungal agent

EXPERIMENTAL

Drug: APX001 with Standard of Care anti-fungal agent

Interventions

APX001 with Standard of Care anti-fungal agent DRUG

safety assessment

APX001 with Standard of Care Anti-fungal agent

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Basilea Pharmaceutica: lead
• The Clinical Trials Centre Cologne: collaborator

Study Sites (3)

University of Cologne, Center for Integrated Oncology (CIO)

Cologne, 50931, Germany

Location

Johannes Gutenberg, University of Mainz

Mainz, 55131, Germany

Location

University of Munich, Grosshadern Campus

Munich, 81377, Germany

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Marc Engelhardt

  Basilea Pharmaceutica International Ltd, Allschwil

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The first 10 patients enrolled will be administered IV APX001. The second 10 patients enrolled will be administered oral APX001

Study Record Dates

• First Submitted: October 23, 2017
• First Posted: November 6, 2017
• Study Start: November 27, 2017
• Primary Completion: October 26, 2018
• Study Completion: October 26, 2018
• Last Updated: May 17, 2024

Record last verified: 2024-05

Locations

DE (3)