<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03021941</url>
  </required_header>
  <id_info>
    <org_study_id>B3031003</org_study_id>
    <nct_id>NCT03021941</nct_id>
  </id_info>
  <brief_title>Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease</brief_title>
  <official_title>A MULTICENTER, OPEN LABEL, PHARMACOKINETICS, PHARMACODYNAMICS AND SAFETY STUDY OF ELELYSO(TM) (TALIGLUCERASE ALFA) IN PEDIATRIC SUBJECTS WITH TYPE 1 GAUCHER DISEASE</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Pfizer</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Pfizer</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      In August of 2014, the FDA approved ELELYSO for long-term enzyme replacement therapy (ERT)&#xD;
      for pediatric subjects with a confirmed diagnosis of Type 1 Gaucher disease. The recommended&#xD;
      dosage for treatment-naïve adult and pediatric subjects 4 years of age and older is 60 units&#xD;
      per kg of body weight administered every other week as a 60 to 120 minute intravenous&#xD;
      infusion. As a postmarketing commitment, the Sponsor agreed to evaluate the pharmacokinetics&#xD;
      (PK), pharmacodynamics (PD), and safety of Elelyso (taliglucerase alfa) in pediatric subjects&#xD;
      with Type 1 Gaucher Disease. in at least 5 subjects with body weight less than 15 kg; at&#xD;
      least 5 subjects with body weight 15 to less than 20 kg; and at least 5 subjects with body&#xD;
      weight of 20-25 kg with Type 1 Gaucher disease dosed at 60 units/kg every other week.&#xD;
&#xD;
      When applicable, PD measurements for children enrolled in the PK study may be obtained&#xD;
      through the taliglucerase alfa registry (PMR 1895-5) and will include organ volumes (spleen&#xD;
      and liver), hematological values (hemoglobin and platelets) as well as growth (height and&#xD;
      weight) data. Safety data, including any serious hypersensitivity reactions, such as&#xD;
      anaphylaxis, as well as changes in antibody status (ie, detection and titers of binding and&#xD;
      neutralizing antibodies, and detection of IgE antibodies), will also be collected through the&#xD;
      taliglucerase alfa registry.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This study (B3031003) is an open-label study in pediatric subjects with Type 1 Gaucher&#xD;
      Disease to characterize PK, PD and safety following an infusion of taliglucerase alfa in at&#xD;
      least 5 subjects with body weight less than 15 kg; at least 5 subjects with body weight 15 to&#xD;
      less than 20 kg; and at least 5 subjects with body weight of 20-25 kg. The PK sample&#xD;
      collection will take approximately 4 hours to complete and will be performed one month or up&#xD;
      to 6 months after the subject's first taliglucerase infusion in the registry study. The&#xD;
      subject will be contacted the day after the PK samples are collected to assess any continuing&#xD;
      or new adverse events and to review the subject's concomitant medications. Body weight at the&#xD;
      time of the PK blood draw will determine the weight category for each subject. Subjects&#xD;
      enrolled into the study will be assigned the same unique subject number assigned to them in&#xD;
      the registry study (B3031002). This will allow linkage to relevant data from the registry&#xD;
      study for analysis in this study.&#xD;
&#xD;
      For the purposes of this study, baseline evaluations will be obtained from the registry study&#xD;
      and must be performed prior to the subject's first dose of taliglucerase alfa. The Month 6&#xD;
      and Month 12 evaluations will be performed 6 months and 12 months after the start of&#xD;
      taliglucerase alfa treatment, respectively.&#xD;
&#xD;
      Subjects will be eligible for the PK study (B3031003) only if the PD assessments (spleen&#xD;
      volume/size, hemoglobin/platelet counts and height/weight measurements) immunogenicity data&#xD;
      and Gaucher disease diagnostic history are available from the registry study baseline visit&#xD;
      and prior to the start of taliglucerase alfa treatment. If liver volume/size is available, it&#xD;
      will also be analyzed but is not necessary for eligibility for the PK study.&#xD;
&#xD;
      Pediatric subjects prescribed 60 units/kg of taliglucerase alfa every other week by their&#xD;
      physician will be recruited from the registry study. Subjects can be screened for the PK&#xD;
      study at the same time as they are enrolled into the registry study (ERT treatment-naïve&#xD;
      subjects) or up to 6 months after they are enrolled into the registry study (previously ERT&#xD;
      naïve subjects) if PD assessments (spleen volume/size, hemoglobin/platelet counts and&#xD;
      height/weight measurements), immunogenicity data and Gaucher disease diagnostic history&#xD;
      (residual enzyme activity and genotype data) were performed at baseline of the registry study&#xD;
      and prior to the start of taliglucerase alfa treatment.&#xD;
&#xD;
      Baseline data from the registry study for PD and immunogenicity testing are defined as PD&#xD;
      measurements (spleen volume/size, liver volume/size (if available), hemoglobin and platelet&#xD;
      counts and growth measures), Gaucher disease diagnostic history and immunogenicity samples&#xD;
      that were collected at entry into the registry study and prior to the start of taliglucerase&#xD;
      alfa treatment.&#xD;
&#xD;
      For the secondary PD endpoints, spleen volume/size and liver volume/size (if available) will&#xD;
      be measured using MRI, CT or ultrasound, whichever is the standard of care according to the&#xD;
      investigator. The method used to measure spleen and liver volume/size at Baseline will be the&#xD;
      same one used at Month 12. Change from Baseline and percent change from Baseline at Month 12&#xD;
      of registry study will be calculated for spleen volume, liver volume (if available),&#xD;
      hemoglobin and platelet counts, and growth measures (height, weight and Z-scores).&#xD;
&#xD;
      For each subject enrolled in this study, safety data while on taliglucerase alfa, starting&#xD;
      from the baseline visit (entry into the registry study) and continuing until 28 days after&#xD;
      the Month 12 PD data collection visit, will be obtained for reporting.&#xD;
&#xD;
      Safety assessments for the study will include collection of all adverse event and serious&#xD;
      adverse event data, including serious hypersensitivity reactions, procedures for&#xD;
      immunogenicity testing (ie, detection and titers of binding and neutralizing antibodies and&#xD;
      detection of IgE antibodies) as well as vital signs taken as standard of care during&#xD;
      infusions. A pre-dose blood sample for testing of antidrug antibodies (ADA) on the day of PK&#xD;
      Sample Visit will be collected in an effort to assess the impact of immunogenicity on PK.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    In a communication dated June 12, 2019 the FDA has released Pfizer from the Postmarketing&#xD;
    Commitment (PMC 2767-2) that was associated with study B3031003.&#xD;
  </why_stopped>
  <start_date type="Anticipated">July 31, 2019</start_date>
  <completion_date type="Anticipated">June 30, 2020</completion_date>
  <primary_completion_date type="Anticipated">June 30, 2020</primary_completion_date>
  <phase>Phase 4</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Other</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Pharmacokinetics - AUCinf</measure>
    <time_frame>Up to 6 Months</time_frame>
    <description>AUCinf of taliglucerase alfa assessed within 6 months from the subject's first dose of taliglucerase alfa.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetics - AUClast</measure>
    <time_frame>Up to 6 Months</time_frame>
    <description>AUClast of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmockinetics - Cmax</measure>
    <time_frame>Up to 6 Months</time_frame>
    <description>Cmax of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetics - t½</measure>
    <time_frame>Up to 6 Months</time_frame>
    <description>t½ of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetics - CL</measure>
    <time_frame>Up to 6 Months</time_frame>
    <description>CL of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetics - Vss</measure>
    <time_frame>Up to 6 Months</time_frame>
    <description>Vss of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Pharmacodynamics - Spleen volume/size</measure>
    <time_frame>12 Months</time_frame>
    <description>Spleen volume/size measured at Baseline and at Month 12.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacodynamics - Liver volume/size</measure>
    <time_frame>12 Months</time_frame>
    <description>Liver volume/size measured at Baseline and at Month 12.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacodynamics - Growth measurements</measure>
    <time_frame>12 Months</time_frame>
    <description>Growth measurements taken at Baseline and at Month 12.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacodynamics - Hemoglobin and Platelet Counts</measure>
    <time_frame>12 Months</time_frame>
    <description>Hemoglobin and platelet counts measured at Baseline and at Month 12.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Safety - Antibody Assessment</measure>
    <time_frame>12 Months</time_frame>
    <description>Antibody assessment measured at Month 6 and Month 12.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Safety - Adverse Events related to IV Infusion</measure>
    <time_frame>12 Months</time_frame>
    <description>Adverse events related to intravenous infusions of taliglucerase alfa.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Safety - Vital Signs</measure>
    <time_frame>1 Day</time_frame>
    <description>Vital signs prospectively collected during the taligluceras alfa infusion associated with PK measurements.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">0</enrollment>
  <condition>Type 1 Gaucher Disease</condition>
  <arm_group>
    <arm_group_label>Elelyso 60 units/kg</arm_group_label>
    <arm_group_type>Other</arm_group_type>
    <description>All patients receive 60 units/kg of Elelyso.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Elelyso 60 units/kg</intervention_name>
    <description>All patients receive Elelyso 60 units/kg.</description>
    <arm_group_label>Elelyso 60 units/kg</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Male or female pediatric subjects with a diagnosis of Type 1 Gaucher disease (evidence&#xD;
             of leukocyte acid-glucosidase activity 30% of the mean of the reference range for&#xD;
             healthy persons), and meeting one of three weight categories: less than15 kg; 15 to&#xD;
             less than 20 kg or 20-25 kg.&#xD;
&#xD;
          2. ERT treatment-naïve, or able to perform the PK assessment within the initial 6 months&#xD;
             of initial Elelyso treatment (provided the subject was ERT naïve prior to the start of&#xD;
             Elelyso).&#xD;
&#xD;
          3. Have had baseline (ie, prior to the first dose of study medication) PD measurements&#xD;
             (spleen volume/size measured by MRI, CT or ultrasound, hemoglobin/platelet counts and&#xD;
             growth measures including height and weight), immunogenicity sample collection and&#xD;
             Gaucher disease diagnosis history documented in advance of treatment start.&#xD;
&#xD;
          4. Presence of splenomegaly at baseline defined as spleen volume/size measurement of 5&#xD;
             MN.&#xD;
&#xD;
          5. Subjects prescribed the nominal dose of 60 units/kg every two weeks and can tolerate&#xD;
             an infusion rate of 1 mL/min.&#xD;
&#xD;
          6. Evidence of a personally signed and dated informed consent document from parent/legal&#xD;
             guardian (or adult caregiver) capable of providing informed consent indicating that&#xD;
             the subject's parent(s)/legal guardian has been informed of all pertinent aspects of&#xD;
             the study before any screening procedures are performed. When age appropriate, written&#xD;
             assent must also be obtained.&#xD;
&#xD;
          7. Have parent/legal guardian (or adult caregiver) capable and willing to comply with&#xD;
             scheduled visits, treatment plan, laboratory tests and other study procedures.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Evidence or history of clinically significant issue or the presence of a medical,&#xD;
             emotional, behavioral or psychological condition that, in the judgment of the&#xD;
             Investigator, would interfere with the subject's participation in the study, cause&#xD;
             harm to the subject or decrease compliance with the study requirements.&#xD;
&#xD;
          2. Treatment with an investigational drug within 30 days (or as determined by the local&#xD;
             requirement) or 5 half-lives preceding the PK sample collection visit, whichever is&#xD;
             longer.&#xD;
&#xD;
          3. A diagnosis of Type 2 or 3 Gaucher disease, or the presence of neurological signs and&#xD;
             symptoms characteristic of Type 2 or 3 Gaucher disease.&#xD;
&#xD;
          4. Any change during the registry study to the subjects dose of taliglucerase alfa&#xD;
             infusion (ie, change from 60 units/kg every two weeks to a different dose of&#xD;
             taliglucerase alfa) or a change in infusion duration or rate or a change of ERT&#xD;
             medication (ie, switch from taliglucerase alfa to a different ERT).&#xD;
&#xD;
          5. In the judgment of the Investigator, the subject's vital signs (eg, blood pressure,&#xD;
             pulse) prior to infusion on the day of the PK visit indicate that participation in the&#xD;
             study would not be in the study candidate's best interest.&#xD;
&#xD;
          6. A hemoglobin level of &lt;10 g/dL within 30 days of the PK sample visit or on the day of&#xD;
             the PK sample visit.&#xD;
&#xD;
          7. History of sensitivity to heparin or heparin-induced thrombocytopenia. (Note: applies&#xD;
             only if heparin lock or flush is to be used on the day of the PK sample visit).&#xD;
&#xD;
          8. Parents or legal guardians who are investigational site staff members directly&#xD;
             involved in the conduct of the study and their family members, children of site staff&#xD;
             members otherwise supervised by the Investigator, or subjects who are children of&#xD;
             Pfizer employees directly involved in the conduct of the study.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>12 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Pfizer CT.gov Call Center</last_name>
    <role>Study Director</role>
    <affiliation>Pfizer</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>O&amp;O Alpan LLC</name>
      <address>
        <city>Fairfax</city>
        <state>Virginia</state>
        <zip>22030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=B3031003&amp;StudyName=A+Multicenter%2C+Open+Label%2C+Pharmacokinetics%2C+Pharmacodynamics+And+Safety+Study+Of+Elelyso%28tm%29+%28taliglucerase+Alfa%29+In+Pediatric+Subjects+With+Type+1+Gaucher+Disease</url>
    <description>To obtain contact information for a study center near you, click here.</description>
  </link>
  <verification_date>August 2019</verification_date>
  <study_first_submitted>September 23, 2016</study_first_submitted>
  <study_first_submitted_qc>January 11, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 16, 2017</study_first_posted>
  <last_update_submitted>September 20, 2019</last_update_submitted>
  <last_update_submitted_qc>September 20, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">September 24, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Gaucher Disease</keyword>
  <keyword>Enzyme Replacement Therapy</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Gaucher Disease</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.</ipd_description>
    <ipd_url>https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests</ipd_url>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03021941
org study id: B3031003
nct id: NCT03021941
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
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.
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 subjects prescribed 60 units/kg of taliglucerase alfa every other week by their
      physician will be recruited from the registry study. Subjects can be screened for the PK
      study at the same time as they are enrolled into the registry study (ERT treatment-naïve
      subjects) or up to 6 months after they are enrolled into the registry study (previously ERT
      naïve subjects) if PD assessments (spleen volume/size, hemoglobin/platelet counts and
      height/weight measurements), immunogenicity data and Gaucher disease diagnostic history
      (residual enzyme activity and genotype data) were performed at baseline of the registry study
      and prior to the start of taliglucerase alfa treatment.

      Baseline data from the registry study for PD and immunogenicity testing are defined as PD
      measurements (spleen volume/size, liver volume/size (if available), hemoglobin and platelet
      counts and growth measures), Gaucher disease diagnostic history and immunogenicity samples
      that were collected at entry into the registry study and prior to the start of taliglucerase
      alfa treatment.

      For the secondary PD endpoints, spleen volume/size and liver volume/size (if available) will
      be measured using MRI, CT or ultrasound, whichever is the standard of care according to the
      investigator. The method used to measure spleen and liver volume/size at Baseline will be the
      same one used at Month 12. Change from Baseline and percent change from Baseline at Month 12
      of registry study will be calculated for spleen volume, liver volume (if available),
      hemoglobin and platelet counts, and growth measures (height, weight and Z-scores).

      For each subject enrolled in this study, safety data while on taliglucerase alfa, starting
      from the baseline visit (entry into the registry study) and continuing until 28 days after
      the Month 12 PD data collection visit, will be obtained for reporting.

      Safety assessments for the study will include collection of all adverse event and serious
      adverse event data, including serious hypersensitivity reactions, procedures for
      immunogenicity testing (ie, detection and titers of binding and neutralizing antibodies and
      detection of IgE antibodies) as well as vital signs taken as standard of care during
      infusions. A pre-dose blood sample for testing of antidrug antibodies (ADA) on the day of PK
      Sample Visit will be collected in an effort to assess the impact of immunogenicity on PK.
intervention model: Parallel Assignment
primary purpose: Other
masking: None (Open Label)
measure: Pharmacokinetics - AUCinf
time frame: Up to 6 Months
description: AUCinf of taliglucerase alfa assessed within 6 months from the subject's first dose of taliglucerase alfa.
measure: Pharmacokinetics - AUClast
time frame: Up to 6 Months
description: AUClast of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
measure: Pharmockinetics - Cmax
time frame: Up to 6 Months
description: Cmax of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
measure: Pharmacokinetics - t½
time frame: Up to 6 Months
description: t½ of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
measure: Pharmacokinetics - CL
time frame: Up to 6 Months
description: CL of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
measure: Pharmacokinetics - Vss
time frame: Up to 6 Months
description: Vss of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
measure: Pharmacodynamics - Spleen volume/size
time frame: 12 Months
description: Spleen volume/size measured at Baseline and at Month 12.
measure: Pharmacodynamics - Liver volume/size
time frame: 12 Months
description: Liver volume/size measured at Baseline and at Month 12.
measure: Pharmacodynamics - Growth measurements
time frame: 12 Months
description: Growth measurements taken at Baseline and at Month 12.
measure: Pharmacodynamics - Hemoglobin and Platelet Counts
time frame: 12 Months
description: Hemoglobin and platelet counts measured at Baseline and at Month 12.
measure: Safety - Antibody Assessment
time frame: 12 Months
description: Antibody assessment measured at Month 6 and Month 12.
measure: Safety - Adverse Events related to IV Infusion
time frame: 12 Months
description: Adverse events related to intravenous infusions of taliglucerase alfa.
measure: Safety - Vital Signs
time frame: 1 Day
description: Vital signs prospectively collected during the taligluceras alfa infusion associated with PK measurements.
arm group label: Elelyso 60 units/kg
arm group type: Other
description: All patients receive 60 units/kg of Elelyso.
intervention type: Drug
intervention name: Elelyso 60 units/kg
description: All patients receive Elelyso 60 units/kg.
arm group label: Elelyso 60 units/kg
criteria: 
gender: All
minimum age: N/A
maximum age: 12 Years
healthy volunteers: No
last name: Pfizer CT.gov Call Center
role: Study Director
affiliation: Pfizer
facility: 
country: United States
url: https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=B3031003&StudyName=A+Multicenter%2C+Open+Label%2C+Pharmacokinetics%2C+Pharmacodynamics+And+Safety+Study+Of+Elelyso%28tm%29+%28taliglucerase+Alfa%29+In+Pediatric+Subjects+With+Type+1+Gaucher+Disease
description: To obtain contact information for a study center near you, click here.
responsible party type: Sponsor
mesh term: Gaucher Disease
sharing ipd: Yes
ipd description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
ipd url: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests