Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Pharmacokinetics of Botulism Antitoxin Heptavalent in Pediatric Patients

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Cangene Corporation
ClinicalTrials.gov Identifier:
NCT02051062
First received: January 29, 2014
Last updated: December 23, 2014
Last verified: December 2014
  Purpose

The purpose of this study is to verify the pediatric dosing recommendations for BAT® in pediatric patients that are treated with BAT® due to a confirmed or suspected case of botulism. One 5 mL blood sample will be obtained within 24 hours post BAT® administration. Study BT-011 will be run concurrently with the BAT patient registry (BT-010).


Condition Intervention
Botulism
Procedure: One 5 mL of blood will be collected.

Study Type: Interventional
Study Design: Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Basic Science
Official Title: Pharmacokinetics of Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) - (Equine) (BAT™) in Pediatric Patients With a Confirmed or Suspected Exposure to Botulinum Toxin.

Resource links provided by NLM:


Further study details as provided by Cangene Corporation:

Primary Outcome Measures:
  • Area Under the Concentration-Time Curve (AUC 0-24h) [ Time Frame: 24 hours ] [ Designated as safety issue: No ]

    One 5 mL of blood collected to yield 2 mL serum sample will be collected post-BAT® administration.

    The blood sample should be collected no later than 24 hours post BAT® administration. To ensure sufficient detectable circulating levels of BAT® for Pharmacokinetic analysis the target window of time for collection should be between 6 and 24 hours post-BAT® administration.

    The serum concentration(s) obtained will be modeled using a population Pharmacokinetics approach based on a previously developed model for BAT™ serotypes A-G in healthy adult human subjects.

    Individual Bayesian Pharmacokinetic parameters (i.e. clearance (CL), volume of distribution (Vc) , volume of distribution of the central and the peripheral compartments (Vp)) and corresponding exposure metrics will be derived from the population analysis.



Estimated Enrollment: 10
Study Start Date: October 2014
Estimated Study Completion Date: April 2018
Estimated Primary Completion Date: October 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: One 5 mL blood sample will be collected
The blood sample should be collected no later than 24 hours post BAT® administration. To ensure sufficient detectable circulating levels of BAT® for pharmacokinetic analysis the target window of time for collection should be between 6 and 24 hours post-BAT® administration.
Procedure: One 5 mL of blood will be collected.
The blood sample should be collected no later than 24 hours post BAT® administration. To ensure sufficient detectable circulating levels of BAT® for pharmacokinetic analysis the target window of time for collection should be between 6 and 24 hours post-BAT® administration.

Detailed Description:

Objectives: The purpose of this study is to collect one serum sample from pediatric patients in order to analyze the pharmacokinetics of BAT® to verify the currently approved pediatric dosing recommendations.

Protocol Design: This is a single arm, multi-site Pharmacokinetics study in pediatric patients treated with BAT®. The study begins once participation in the study is confirmed by the physician and informed consent/assent is obtained from the patient and/or guardian. Concurrent participation in the BAT patient registry (BT-010) is encouraged to collect safety and clinical outcome data.

Assessments: One 5 mL of blood collected to yield 2 mL serum sample will be collected post-BAT administration. The blood sample should be collected no later than 24 hours post BAT® administration. To ensure sufficient detectable circulating levels of BAT® for pharmacokinetic analysis the target window of time for collection should be between 6 and 24 hours post-BAT administration.

Pharmacokinetic or Efficacy Parameters: The serum concentration(s) obtained will be modeled using a population pharmacokinetic approach based on a previously developed model for BAT serotypes A-G in healthy adult human subjects.

Individual Bayesian Pharmacokinetics parameters (i.e. clearance (CL), volume of distribution (Vc), volume of distribution of the central and the peripheral compartments (Vp)) and corresponding exposure metrics will be derived from the population analysis.

Primary Endpoints: The primary endpoint is the dosage level at which pharmacokinetic equivalence is reached i.e. the dosage at which BAT® is most effective against the symptoms of botulism. Following the estimation of exposure in pediatric patients, and similar to what was done for adult subjects, the margin of efficacy for 90% survival will be estimated in order to verify the appropriateness of the administered pediatric dose.

  Eligibility

Ages Eligible for Study:   up to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Informed consent/assent (as applicable) is required for provision of a serum sample to Cangene.
  • Pediatric patients [age category: pediatric—preterm and term newborn infants (0 to 27 days), infants and toddlers (28 days to 23 months), children (2 to 11-years), and adolescents (12 to 16-years)]
  • Confirmed or suspected exposure to botulinum toxin.
  • Treatment with BAT® deployed from the Strategic National Stockpile or state stockpiles.

Exclusion Criteria:

  • Blood sample cannot be collected prior to 24 hours post-BAT® administration.
  • The 5 mL blood sample volume is deemed to be unsafe based on patient weight.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02051062

Sponsors and Collaborators
Cangene Corporation
Investigators
Study Chair: Jason S Richardson, PhD Clinical Research Scientist for Cangene Corporation (doing business as Emergent Biosolutions)
Study Chair: Matt Cromie, MSc Clinical Research Senior Manager for Cangene Corporation (doing business as Emergent Biosolutions)
Study Chair: Christine Hall, PhD Director Clinical Research for Cangene Corporation (doing business as Emergent Biosolutions)
  More Information

Additional Information:
Publications:
Responsible Party: Cangene Corporation
ClinicalTrials.gov Identifier: NCT02051062     History of Changes
Other Study ID Numbers: BT-011
Study First Received: January 29, 2014
Last Updated: December 23, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Cangene Corporation:
Pharmacokinetics
BAT
Pediatric

Additional relevant MeSH terms:
Botulism
Bacterial Infections
Chemically-Induced Disorders
Clostridium Infections
Foodborne Diseases
Gram-Positive Bacterial Infections
Nervous System Diseases
Neuromuscular Diseases
Neuromuscular Junction Diseases
Neurotoxicity Syndromes
Poisoning
Botulinum Antitoxin
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on February 25, 2015