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Trial record 8 of 101 for:    Gaucher Disease

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

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ClinicalTrials.gov Identifier: NCT03021941
Recruitment Status : Not yet recruiting
First Posted : January 16, 2017
Last Update Posted : July 4, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

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.


Condition or disease Intervention/treatment Phase
Type 1 Gaucher Disease Drug: Elelyso 60 units/kg Phase 4

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: A Multicenter, Open Label, Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) (Taliglucerase Alfa) In Pediatric Subjects With Type 1 Gaucher Disease
Estimated Study Start Date : July 2018
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2020


Arm Intervention/treatment
Elelyso 60 units/kg
All patients receive 60 units/kg of Elelyso.
Drug: Elelyso 60 units/kg
All patients receive Elelyso 60 units/kg.




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.


Secondary Outcome Measures :
  1. Pharmacodynamics - Spleen volume/size [ Time Frame: 12 Months ]
    Spleen volume/size measured at Baseline and at Month 12.

  2. Pharmacodynamics - Liver volume/size [ Time Frame: 12 Months ]
    Liver volume/size measured at Baseline and at Month 12.

  3. Pharmacodynamics - Growth measurements [ Time Frame: 12 Months ]
    Growth measurements taken at Baseline and at Month 12.

  4. Pharmacodynamics - Hemoglobin and Platelet Counts [ Time Frame: 12 Months ]
    Hemoglobin and platelet counts measured at Baseline and at Month 12.

  5. Safety - Antibody Assessment [ Time Frame: 12 Months ]
    Antibody assessment measured at Month 6 and Month 12.

  6. Safety - Adverse Events related to IV Infusion [ Time Frame: 12 Months ]
    Adverse events related to intravenous infusions of taliglucerase alfa.

  7. Safety - Vital Signs [ Time Frame: 1 Day ]
    Vital signs prospectively collected during the taligluceras alfa infusion associated with PK measurements.



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Ages Eligible for Study:   up to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female pediatric subjects with a diagnosis of Type 1 Gaucher disease (evidence of leukocyte acid-glucosidase activity 30% of the mean of the reference range for healthy persons), and meeting one of three weight categories: less than15 kg; 15 to less than 20 kg or 20-25 kg.
  2. ERT treatment-naïve, or able to perform the PK assessment within the initial 6 months of initial Elelyso treatment (provided the subject was ERT naïve prior to the start of Elelyso).
  3. Have had baseline (ie, prior to the first dose of study medication) PD measurements (spleen volume/size measured by MRI, CT or ultrasound, hemoglobin/platelet counts and growth measures including height and weight), immunogenicity sample collection and Gaucher disease diagnosis history documented in advance of treatment start.
  4. Presence of splenomegaly at baseline defined as spleen volume/size measurement of 5 MN.
  5. Subjects prescribed the nominal dose of 60 units/kg every two weeks and can tolerate an infusion rate of 1 mL/min.
  6. Evidence of a personally signed and dated informed consent document from parent/legal guardian (or adult caregiver) capable of providing informed consent indicating that the subject's parent(s)/legal guardian has been informed of all pertinent aspects of the study before any screening procedures are performed. When age appropriate, written assent must also be obtained.
  7. Have parent/legal guardian (or adult caregiver) capable and willing to comply with scheduled visits, treatment plan, laboratory tests and other study procedures.

Exclusion Criteria:

  1. Evidence or history of clinically significant issue or the presence of a medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator, would interfere with the subject's participation in the study, cause harm to the subject or decrease compliance with the study requirements.
  2. Treatment with an investigational drug within 30 days (or as determined by the local requirement) or 5 half-lives preceding the PK sample collection visit, whichever is longer.
  3. A diagnosis of Type 2 or 3 Gaucher disease, or the presence of neurological signs and symptoms characteristic of Type 2 or 3 Gaucher disease.
  4. Any change during the registry study to the subjects dose of taliglucerase alfa infusion (ie, change from 60 units/kg every two weeks to a different dose of taliglucerase alfa) or a change in infusion duration or rate or a change of ERT medication (ie, switch from taliglucerase alfa to a different ERT).
  5. In the judgment of the Investigator, the subject's vital signs (eg, blood pressure, pulse) prior to infusion on the day of the PK visit indicate that participation in the study would not be in the study candidate's best interest.
  6. A hemoglobin level of <10 g/dL within 30 days of the PK sample visit or on the day of the PK sample visit.
  7. History of sensitivity to heparin or heparin-induced thrombocytopenia. (Note: applies only if heparin lock or flush is to be used on the day of the PK sample visit).
  8. Parents or legal guardians who are investigational site staff members directly involved in the conduct of the study and their family members, children of site staff members otherwise supervised by the Investigator, or subjects who are children of Pfizer employees directly involved in the conduct of the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03021941


Contacts
Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

Locations
United States, Pennsylvania
Children's Hospital of Pittsburgh Not yet recruiting
Pittsburgh, Pennsylvania, United States, 15224
United States, Virginia
O&O Alpan LLC Not yet recruiting
Fairfax, Virginia, United States, 22030
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer

Additional Information:
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT03021941     History of Changes
Other Study ID Numbers: B3031003
First Posted: January 16, 2017    Key Record Dates
Last Update Posted: July 4, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Information relating to our policy on data sharing and the process for requesting data can be found at the following link: http://www.pfizer.com/research/clinical_trials/trial_data_and_results/data_requests
URL: http://

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Pfizer:
Gaucher Disease
Enzyme Replacement Therapy

Additional relevant MeSH terms:
Gaucher Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Sphingolipidoses
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors