Clinical Trial: Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: A Multicenter, Open Label, Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) (Taliglucerase Alfa) In Pediatric Subjects With Type 1 Gaucher Disease

Brief Summary:

In August of 2014, the FDA approved ELELYSO for long-term enzyme replacement therapy (ERT) for pediatric subjects with a confirmed diagnosis of Type 1 Gaucher disease. The recommended dosage for treatment-naïve adult and pediatric subjects 4 years of age and older is 60 units per kg of body weight administered every other week as a 60 to 120 minute intravenous infusion. As a postmarketing commitment, the Sponsor agreed to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of Elelyso (taliglucerase alfa) in pediatric subjects with Type 1 Gaucher Disease. in at least 5 subjects with body weight less than 15 kg; at least 5 subjects with body weight 15 to less than 20 kg; and at least 5 subjects with body weight of 20-25 kg with Type 1 Gaucher disease dosed at 60 units/kg every other week.

When applicable, PD measurements for children enrolled in the PK study may be obtained through the taliglucerase alfa registry (PMR 1895-5) and will include organ volumes (spleen and liver), hematological values (hemoglobin and platelets) as well as growth (height and weight) data. Safety data, including any serious hypersensitivity reactions, such as anaphylaxis, as well as changes in antibody status (ie, detection and titers of binding and neutralizing antibodies, and detection of IgE antibodies), will also be collected through the taliglucerase alfa registry.


Detailed Summary:

This study (B3031003) is an open-label study in pediatric subjects with Type 1 Gaucher Disease to characterize PK, PD and safety following an infusion of taliglucerase alfa in at least 5 subjects with body weight less than 15 kg; at least 5 subjects with body weight 15 to less than 20 kg; and at least 5 subjects with body weight of 20-25 kg. The PK sample collection will take approximately 4 hours to complete and will be performed one month or up to 6 months after the subject's first taliglucerase infusion in the registry study. The subject will be contacted the day after the PK samples are collected to assess any continuing or new adverse events and to review the subject's concomitant medications. Body weight at the time of the PK blood draw will determine the weight category for each subject. Subjects enrolled into the study will be assigned the same unique subject number assigned to them in the registry study (B3031002). This will allow linkage to relevant data from the registry study for analysis in this study.

For the purposes of this study, baseline evaluations will be obtained from the registry study and must be performed prior to the subject's first dose of taliglucerase alfa. The Month 6 and Month 12 evaluations will be performed 6 months and 12 months after the start of taliglucerase alfa treatment, respectively.

Subjects will be eligible for the PK study (B3031003) only if the PD assessments (spleen volume/size, hemoglobin/platelet counts and height/weight measurements) immunogenicity data and Gaucher disease diagnostic history are available from the registry study baseline visit and prior to the start of taliglucerase alfa treatment. If liver volume/size is available, it will also be analyzed but is not necessary for eligibility for the PK study.

Pediatric subje
Sponsor: Pfizer

Current Primary Outcome:

  • Pharmacokinetics - AUCinf [ Time Frame: Up to 6 Months ]
    AUCinf of taliglucerase alfa assessed within 6 months from the subject's first dose of taliglucerase alfa.
  • Pharmacokinetics - AUClast [ Time Frame: Up to 6 Months ]
    AUClast of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
  • Pharmockinetics - Cmax [ Time Frame: Up to 6 Months ]
    Cmax of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
  • Pharmacokinetics - t½ [ Time Frame: Up to 6 Months ]
    t½ of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
  • Pharmacokinetics - CL [ Time Frame: Up to 6 Months ]
    CL of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
  • Pharmacokinetics - Vss [ Time Frame: Up to 6 Months ]
    Vss of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Pharmacodynamics - Spleen volume/size [ Time Frame: 12 Months ]
    Spleen volume/size measured at Baseline and at Month 12.
  • Pharmacodynamics - Liver volume/size [ Time Frame: 12 Months ]
    Liver volume/size measured at Baseline and at Month 12.
  • Pharmacodynamics - Growth measurements [ Time Frame: 12 Months ]
    Growth measurements taken at Baseline and at Month 12.
  • Pharmacodynamics - Hemoglobin and Platelet Counts [ Time Frame: 12 Months ]
    Hemoglobin and platelet counts measured at Baseline and at Month 12.
  • Safety - Antibody Assessment [ Time Frame: 12 Months ]
    Antibody assessment measured at Month 6 and Month 12.
  • Safety - Adverse Events related to IV Infusion [ Time Frame: 12 Months ]
    Adverse events related to intravenous infusions of taliglucerase alfa.
  • Safety - Vital Signs [ Time Frame: 1 Day ]
    Vital signs prospectively collected during the taligluceras alfa infusion associated with PK measurements.


Original Secondary Outcome: Same as current

Information By: Pfizer

Dates:
Date Received: September 23, 2016
Date Started: May 30, 2017
Date Completion: June 26, 2020
Last Updated: May 18, 2017
Last Verified: May 2017