RECRUITING

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The main aim of the study is to check effectiveness, side effects, and tolerability of vonicog alfa (recombinant von Willebrand factor \[rVWF\]), with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric participants (less than (\<)18 years of age) with severe hereditary von Willebrand disease (VWD). The participants will be treated with vonicog alfa for 12-18 months. Their von Willebrand Disease will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be followed up at clinics or over telephone calls.

Official Title

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease

Quick Facts

Study Start:2017-11-06

Study Completion:2026-03-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT02932618

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 17 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
* Diagnosis of severe von Willebrand disease (VWD) (defined as von Willebrand factor: ristocetin cofactor \[VWF:RCo\] less than \[\<\] 20 percent \[%\]): * Type 1 (VWF:RCo \<20 International Units per deciliter \[IU/dL\]); or * Type 2A (VWF:RCo \<20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (Factor VIII coagulation activity \[FVIII:C\] \<10 % and historically documented genetics), Type 2M; or * Type 3 (VWF:Ag less than or equal to \[=\<\] 3 IU/dL). * Age 0 to \<18 years at the time of Screening. * The participant has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent. * If female of childbearing potential, participant presents with a negative serum pregnancy test. * If applicable, participant agrees to employ adequate birth control measures for the duration of the study. * The participant and/or the legally authorized representative are willing and able to comply with the requirements of the protocol, which should also be confirmed based on a pre-screening evaluation held between the Investigator and the Sponsor, to ensure no eminent risk is present that could challenge the participants compliance with the study requirements. * Unable to tolerate are inadequately responsive to, or not a good candidate for 1-deamino-8-D-arginine vasopressin (DDAVP). Examples of participants who are not good candidates for DDAVP include participants with type 2B or type 3 VWD. * The participant has had a minimum of 1 documented bleed requiring VWF coagulation factor replacement therapy (i.e. treatment with a VWF product) during the previous 12 months prior to enrollment and overall historically 3 or more exposure days (EDs) to VWF replacement therapy.	* Diagnosis of pseudo-VWD or another hereditary or acquired coagulation disorder (eg, qualitative and quantitative platelet disorders or elevated prothrombin time \[PT\]/international normalized ratio \[INR\] greater than \[\>\] 1.4). * History or presence of a VWF inhibitor at Screening. * History or presence of a Factor VIII (FVIII) inhibitor with a titer greater than or equal \[\>=\] 0.4 Bethesda units (BU) (by Nijmegen assay) or \>=0.6 BU (by Bethesda assay). * Documented history of a VWF: RCo half-life \<6 hours. * Known hypersensitivity to any of the components of the study drug, such as mouse or hamster proteins. * Medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis/asthma, food allergies, or animal allergies. * Medical history of a thromboembolic event. * Human immunodeficiency virus (HIV) positive, with an absolute CD4 count \<200/ cubic millimeter (mm\^3). * In the judgment of the Investigator, the participant has another clinically significant concomitant disease (e.g. uncontrolled hypertension, cancer) that may pose additional risks for the participant. * Diagnosis of significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) of 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child B or C. * Diagnosis of renal disease, with a serum creatinine level \>=2.5 milligram per deciliter (mg/dL). * Immunomodulatory drug treatment other than anti-retroviral chemotherapy (e.g. α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 milligram per day \[mg/day\] (excluding topical treatment \[e.g. ointments, nasal sprays\]), within 30 days prior to signing the informed consent (or assent, if appropriate). * If female, participant is pregnant or lactating at the time informed consent (or assent, if appropriate) is obtained. * Participant has participated in another clinical study involving an investigational product (IP), other than vonicog alfa with or without ADVATE, or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP other than vonicog alfa or investigational device during the course of this study. * Participant's legal representative is a family member or employee of the Investigator.

Inclusion Criteria

Exclusion Criteria

* Diagnosis of severe von Willebrand disease (VWD) (defined as von Willebrand factor: ristocetin cofactor \[VWF:RCo\] less than \[\<\] 20 percent \[%\]):
* Type 1 (VWF:RCo \<20 International Units per deciliter \[IU/dL\]); or
* Type 2A (VWF:RCo \<20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (Factor VIII coagulation activity \[FVIII:C\] \<10 % and historically documented genetics), Type 2M; or
* Type 3 (VWF:Ag less than or equal to \[=\<\] 3 IU/dL).
* Age 0 to \<18 years at the time of Screening.
* The participant has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent.
* If female of childbearing potential, participant presents with a negative serum pregnancy test.
* If applicable, participant agrees to employ adequate birth control measures for the duration of the study.
* The participant and/or the legally authorized representative are willing and able to comply with the requirements of the protocol, which should also be confirmed based on a pre-screening evaluation held between the Investigator and the Sponsor, to ensure no eminent risk is present that could challenge the participants compliance with the study requirements.
* Unable to tolerate are inadequately responsive to, or not a good candidate for 1-deamino-8-D-arginine vasopressin (DDAVP). Examples of participants who are not good candidates for DDAVP include participants with type 2B or type 3 VWD.
* The participant has had a minimum of 1 documented bleed requiring VWF coagulation factor replacement therapy (i.e. treatment with a VWF product) during the previous 12 months prior to enrollment and overall historically 3 or more exposure days (EDs) to VWF replacement therapy.

* Diagnosis of pseudo-VWD or another hereditary or acquired coagulation disorder (eg, qualitative and quantitative platelet disorders or elevated prothrombin time \[PT\]/international normalized ratio \[INR\] greater than \[\>\] 1.4).
* History or presence of a VWF inhibitor at Screening.
* History or presence of a Factor VIII (FVIII) inhibitor with a titer greater than or equal \[\>=\] 0.4 Bethesda units (BU) (by Nijmegen assay) or \>=0.6 BU (by Bethesda assay).
* Documented history of a VWF: RCo half-life \<6 hours.
* Known hypersensitivity to any of the components of the study drug, such as mouse or hamster proteins.
* Medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis/asthma, food allergies, or animal allergies.
* Medical history of a thromboembolic event.
* Human immunodeficiency virus (HIV) positive, with an absolute CD4 count \<200/ cubic millimeter (mm\^3).
* In the judgment of the Investigator, the participant has another clinically significant concomitant disease (e.g. uncontrolled hypertension, cancer) that may pose additional risks for the participant.
* Diagnosis of significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) of 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child B or C.
* Diagnosis of renal disease, with a serum creatinine level \>=2.5 milligram per deciliter (mg/dL).
* Immunomodulatory drug treatment other than anti-retroviral chemotherapy (e.g. α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 milligram per day \[mg/day\] (excluding topical treatment \[e.g. ointments, nasal sprays\]), within 30 days prior to signing the informed consent (or assent, if appropriate).
* If female, participant is pregnant or lactating at the time informed consent (or assent, if appropriate) is obtained.
* Participant has participated in another clinical study involving an investigational product (IP), other than vonicog alfa with or without ADVATE, or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP other than vonicog alfa or investigational device during the course of this study.
* Participant's legal representative is a family member or employee of the Investigator.

Contacts and Locations

Study Contact

Takeda Contact

CONTACT

1-877-825-3327

medinfoUS@takeda.com

Principal Investigator

Study Director

STUDY_DIRECTOR

Takeda

Study Locations (Sites)

University of Colorado Hemophilia & Thrombosis Center

Aurora, Colorado, 80045

United States

Children's National Medical Center

Washington, District of Columbia, 20010

United States

University of Florida College of Medicine

Jacksonville, Florida, 32610

United States

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61615

United States

Indiana Hemophilia and Thrombosis Center

Indianapolis, Indiana, 46260

United States

University of Nebraska Medical Center

Omaha, Nebraska, 68198

United States

St. Jude Affiliate Clinic at Novant Health

Charlotte, North Carolina, 28204

United States

Comprehensive Cancer Center of Wake Forest Unversity

Winston-Salem, North Carolina, 27157

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229

United States

Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106

United States

Nationwide Children's Hospital

Columbus, Ohio, 43205

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

United States

Medical University of South Carolina

Charleston, South Carolina, 29425

United States

Texas Children's Cancer and Hematology Center

Houston, Texas, 77030

United States

Texas Children's Hospital

Houston, Texas, 77030

United States

Comprehensive Center for Bleeding Disorders

Milwaukee, Wisconsin, 53225

United States

Collaborators and Investigators

Sponsor: Baxalta now part of Shire

Study Director, STUDY_DIRECTOR, Takeda

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2017-11-06

Study Completion Date2026-03-31

Study Record Updates

Study Start Date2017-11-06

Study Completion Date2026-03-31

Terms related to this study

Additional Relevant MeSH Terms

Von Willebrand Disease