Clinical trial NCT06883240: An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
Official title: A Multicenter Non-Interventional Study Evaluating Bleeds and Health-Related Quality Of Life in Patients With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Von Willebrand Disease, Type 3
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2025-03-19
Enrollment: 40
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This study will observe approximately 40 participants with Type 3 VWD (aged 2 years and older) that are currently being treated with SOC prophylactic therapy.

Brief summary: This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants with Type 3 von Willebrand disease (VWD) receiving prophylactic therapy per local standard of care (SOC) over an observation period of at least 24 weeks.


Eligibility criteria: Inclusion Criteria:

* Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
* Adequate hematologic, hepatic, and renal function
* Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) and anticipation to remain on the same regimen during the study
* For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements

Exclusion Criteria:

* Inherited or acquired bleeding disorder other than Congenital Type 3 VWD
* History of gastrointestinal bleeding within 18 months prior to enrollment, or any previous diagnosis of angiodysplasia
* History of intracranial hemorrhage
* Previous or current treatment for thromboembolic disease or signs of thromboembolic disease
* Other conditions (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis
* History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
* Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy

Primary outcomes:
measure: Annualized Bleed Rate (ABR) for Treated Bleeds
timeFrame: From Baseline to at least 24 weeks
Secondary outcomes:
measure: ABR for All Bleeds
timeFrame: From Baseline to at least 24 weeks
measure: ABR for Treated Spontaneous Bleeds
timeFrame: From Baseline to at least 24 weeks
measure: ABR for Treated Joint Bleeds
timeFrame: From Baseline to at least 24 weeks
measure: Incidence and Severity of Adverse Events, with Severity Determined According to the World Health Organization (WHO) Toxicity Grading Scale
timeFrame: From Baseline until study completion (at least 24 weeks)
Other outcomes:
Locations:
city: Leuven
country: Belgium
facility: UZ Leuven Gasthuisberg
city: Medellin
country: Colombia
facility: IPS SURA Industriales Medellín
city: Roma
country: Italy
facility: Universita' Degli Studi La Sapienza-Ist.Di Ematologia
state: Lazio
city: Firenze
country: Italy
facility: AOU Careggi
state: Toscana
city: Fukuoka
country: Japan
facility: Kurume University Hospital
city: Madrid
country: Spain
facility: Hospital Universitario la Paz
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: London
country: United Kingdom
facility: Great Ormond Street Hospital
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: