Study of Mycamine® in Children With Fungal Infections to Evaluate Safety and Blood Levels of the Drug

A Phase 1, Open-Label Study Of The Safety And Pharmacokinetics Of Repeated-Dose Micafungin (FK463) In Children (2-5 Years AND 6-11 Years) And Adolescents (12-16 Years) With Esophageal Candidiasis Or Other Invasive Candidiasis

Children with fungal infections will be divided into two groups by weight. Children weighing < 25 kg will receive a higher dose of study medication (per kg body weight) for 10 to 14 days than children weighing > 25 kg.

Study Overview

• Status: Completed
• Conditions: Candidemia; Invasive Candidiasis; Esophageal Candidiasis; Oropharyngeal Candidiasis
• Intervention / Treatment: Drug: Micafungin

Detailed Description

This is a prospective, multicenter, open-label, repeat-dose study conducted in two groups. Subjects in Group 1 and Group 2 will receive treatment for 10 to 14 days (per investigator clinical judgment); dose level (3.0 mg/kg or 4.5 mg/kg) will be determined by the subject's weight at baseline. Study procedures in both groups will be similar except that serial blood samples for assessment of pharmacokinetics will be collected in Group 1. Subjects may only participate in one group of the study.

At least 24 subjects, with at least 8 in each of three age ranges, will be enrolled in Group 1 (PK + safety). At least 36 subjects will be enrolled in Group 2 (safety), with at least 12 in each of the three age ranges.

• Study Type: Interventional
• Enrollment (Actual): 84
• Phase: Phase 1

Contacts and Locations

Study Locations

• South Africa
  • Benoni, South Africa, 1500
    • Benmed Park Clinic
  • Cape Town, South Africa, 7924
    • Grootte Schuur Hospital
  • Lynn East, South Africa, 0039
    • GCT -Sunnyside Medi-clinic
  • Mariannhill, South Africa, 3610
    • St. Mary's Hospital
  • Temba, South Africa, 0400
    • Jubilee Hospital
  • Wynberg, South Africa, 7824
    • 2 Military Hospital
• United States
  • California
    • Los Angeles, California, United States, 90095
      • University of California Los Angeles Medical Center
    • Orange, California, United States, 92868
      • Children's Hospital of Orange County
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Children's Mercy Hospital
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science University
  • Texas
    • Dallas, Texas, United States, 75390
      • University of Texas Southwestern
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 16 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Subject is 2 to 16 years inclusive
2. Subject has suspected, proven or probable candidiasis, candidemia or other invasive candidiasis
3. Subject has sufficient venous access to permit administration of study medication, collection of pharmacokinetic samples, and monitoring of laboratory safety variables

Exclusion Criteria:

1. Subject has evidence of significant liver disease, as defined by aspartate transaminase (AST), alanine transaminase (ALT), bilirubin or alkaline phosphatase > 5 times the upper limit of normal (ULN)
2. Subject has a concomitant medical condition that in the opinion of the investigator and/or medical monitor precludes enrollment into the study
3. Subject has a history of anaphylaxis, hypersensitivity, or any serious reaction to the echinocandin class of antifungals
4. Subject has received treatment with an echinocandin within one week prior to first dosing
5. Subject status is unstable and subject is unlikely to complete all study required procedures

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 1. Micafungin 3.0 mg; IV	Drug: Micafungin; IV; Other Names: FK463
Experimental: 2. Micafungin 4.5 mg; IV	Drug: Micafungin; IV; Other Names: FK463

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
PK parameters (AUCtau, Tmax, and Cmax)	10 - 14 Days

Secondary Outcome Measures

Outcome Measure	Time Frame
Adverse events	Day 1 to End of Study
Vital signs	Day 1 to End of Study
Hematology and chemistry laboratory tests	Day 1 to End of Study
12-lead ECGs and Physical examination	Day 1 to End of Study

Collaborators and Investigators

• Sponsor: Astellas Pharma Inc

Publications and helpful links

Helpful Links

• Link to results on the Astellas Clinical Study Results website

Study record dates

Study Major Dates

• Study Start (Actual): October 14, 2007
• Primary Completion (Actual): September 8, 2011
• Study Completion (Actual): September 8, 2011

Study Registration Dates

• First Submitted: January 23, 2008
• First Submitted That Met QC Criteria: January 23, 2008
• First Posted (Estimate): February 6, 2008

Study Record Updates

• Last Update Posted (Actual): July 31, 2018
• Last Update Submitted That Met QC Criteria: July 28, 2018
• Last Verified: July 1, 2018

More Information

Terms related to this study

• Keywords: Micafungin; Candidiasis; Echinocandin; Antifungal
• Additional Relevant MeSH Terms: Pathologic Processes; Infections; Systemic Inflammatory Response Syndrome; Inflammation; Bacterial Infections and Mycoses; Sepsis; Mycoses; Invasive Fungal Infections; Fungemia; Candidiasis; Candidemia; Candidiasis, Invasive; Anti-Infective Agents; Antifungal Agents; Micafungin
• Other Study ID Numbers: 9463-CL-2101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Access to anonymized individual participant level data collected during the trial, in addition to study-related supporting documentation, is planned for trials conducted with approved product indications and formulations, as well as compounds terminated during development. Conditions and exceptions are described under the Sponsor Specific Details for Astellas on www.clinicalstudydatarequest.com.
• IPD Sharing Time Frame: Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data.
• IPD Sharing Access Criteria: Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR