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A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Children With Gaucher Disease Under 5 Years Old

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ClinicalTrials.gov Identifier: NCT04721366
Recruitment Status : Recruiting
First Posted : January 22, 2021
Last Update Posted : April 2, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda

Brief Summary:

The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants with Gaucher disease that are under 5 years old. Symptoms will be checked with blood tests.

Another aim of the study is to check if there are any side effects from VPRIV.

The study will take place in the United States.

After the study doctor has checked that each child can take part in this study, part of each participant's medical record will be recorded. Then, information from each participant's ongoing treatment with VPRIV, which will follow standard patient care, will be recorded. In this study, standard patient care means that the participants will receive VPRIV as they usually would from their doctor. Also, no extra tests will be carried out.


Condition or disease Intervention/treatment
Gaucher Disease Other: Standard of Care

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Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Gaucher Disease During Infancy and Early Childhood and Experience With Enzyme Replacement Therapy (ERT) Using Velaglucerase Alfa (VPRIV): A Combined Retrospective and Prospective Cohort Study
Actual Study Start Date : January 8, 2021
Estimated Primary Completion Date : January 2, 2023
Estimated Study Completion Date : January 2, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gaucher Disease

Group/Cohort Intervention/treatment
Standard of Care (SoC)
Neonatal and pediatric participants who has been on ERT (VPRIV) will be assessed up to 36 months from the time of treatment initiation as per SOC.
Other: Standard of Care
Neonatal and pediatric participants who has been on ERT (VPRIV) will be assessed as per SOC.
Other Name: SOC




Primary Outcome Measures :
  1. Change From Baseline in Hemoglobin (Hb) Level [ Time Frame: From start of ERT initiation up to 5 years of age ]
    Increase hemoglobin levels up to 11.0 gram per deciliter (g/dL) will be assessed.

  2. Percent Change From Baseline in Platelet Count Increase [ Time Frame: From start of ERT initiation up to 5 years of age ]
    Percent change from baseline for platelet count increase will be assessed.

  3. Percent Change From Baseline in Liver Volume [ Time Frame: From start of ERT initiation up to 5 years of age ]
    Percent change from baseline in liver volume will be assessed.

  4. Percent Change From Baseline for Spleen Volume [ Time Frame: From start of ERT initiation up to 5 years of age ]
    Percent change from baseline for spleen volume will be assessed.

  5. Percentage of Participants With Growth Normalization [ Time Frame: From start of ERT initiation up to 5 years of age ]
    Percentage of participants with growth normalization will be assessed.

  6. Percentage of Participants With Improvement in Bone Disease [ Time Frame: From start of ERT initiation up to 5 years of age ]
    Percentage of participants with improvement in bone disease will be assessed.

  7. Percentage of Participants With Improvement in Thrombocytopenia [ Time Frame: From start of ERT initiation up to 5 years of age ]
    Percentage of participants with improvement in thrombocytopenia will be assessed.


Secondary Outcome Measures :
  1. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: From start of ERT initiation up to 5 years of age ]
    An adverse event (AE) is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. AEs include SAEs, non-serious AEs.



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Neonatal and pediatric participants included have a diagnosis of GD type I and III and are currently being treated with another ERT (VPRIV) up to 36 months from the time of treatment initiation.
Criteria

Inclusion Criteria:

  • The participant's caregiver is able and willing to provide informed consent.
  • The participant is male or female younger than or equal to 3 year of age at initial visit.
  • The participant has received and confirmed a current diagnosis of GD type 1 or type 3 (biochemically and/or genetically).
  • The participant has been receiving intravenous (IV) Velaglucerase alfa 60 units per kilogram (U/kg) every other week (EOW) ERT treatment for GD.
  • In the opinion of the investigator, the participant's caregiver is capable of understanding and complying with protocol requirements.
  • The participant's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures.

Exclusion Criteria:

  • The participant is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in conduct of this study (e.g., child, sibling) or may consent under duress.
  • The participant is judged by the investigator as being ineligible for any other reason.

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): NCT04721366


Contacts
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Contact: Takeda Contact +1 866 842 5335 ClinicalTransparency@takeda.com

Locations
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United States, Virginia
Lysosomal & Rare Disorders Research & Treatment Center Recruiting
Fairfax, Virginia, United States, 22030
Contact: Site Contact    703-261-6220    info@ldrtc.org   
Principal Investigator: Ozlem Goker-Alpan         
Sponsors and Collaborators
Takeda
Investigators
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Study Director: Study Director Takeda
Additional Information:
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Responsible Party: Takeda
ClinicalTrials.gov Identifier: NCT04721366    
Other Study ID Numbers: MACS-2020-052801
First Posted: January 22, 2021    Key Record Dates
Last Update Posted: April 2, 2021
Last Verified: March 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
URL: https://vivli.org/ourmember/takeda/

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders