|- candidate number||17556|
|- NTR Number||NTR4548|
|- ISRCTN||ISRCTN no longer applicable|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||23-apr-2014|
|- Secondary IDs||NL38989.041.12 |
|- Public Title||Joint bleeds in Von Willebrand disease: Impact on joint integrity, -function and daily life.|
|- Scientific Title||Arthropathy in patients with congenital severe and moderate severe Von Willebrand disease|
|- ACRONYM||Willebrand Artropathy Study|
|- hypothesis||the severity and impact of arthropathy in patients with VWD could be significant|
|- Healt Condition(s) or Problem(s) studied||Von Willebrand Disease, Joint|
|- Inclusion criteria||Patients with moderate or severe VWD treated with coagulation factor or desmopressin for 1 or more joint bleeds as documented in their MF.
A similar size control group of age (< 2 years difference), FVIII and sex matched moderate or severe VWD patients who participated in the WiN study, indicated that they would like to participate in future studies, and did not report treatment for 1 or more joint bleeds and also did not have joint bleeds recorded in their MF.
Also eligible for the control group are moderate or severe VWD patients who did not participate in the WiN study and are currently treated at the haemophilia treatment centres in the Netherlands who have not been treated with coagulation factor or desmopressin for 1 or more joint bleeds and without joint bleeds treatment documented in their MF.
|- Exclusion criteria||Inability of the patient or the patients parents to give informed consent|
A recent joint bleed without complete recovery
Restricted motion of an ankle, knee or elbow joint for another medical reason
No medical file available
|- mec approval received||yes|
|- multicenter trial||yes|
|- Type||2 or more arms, non-randomized|
|- planned startdate ||12-aug-2013|
|- planned closingdate||30-jan-2015|
|- Target number of participants||100|
|- Interventions||assessment of joint damage, joint pain and related functional and social impairment by 4 questionnaires for adults, 1 for children, 1 physical joint examination, joint X-rays and 1 functional test. The physical joint examination and functional test will be scored by an experienced physiotherapist using the Haemophilia Joint Health Score (HJHS). The functional test consists of a figure 8 walking test. X-rays will be taken of affected and contralateral joints in all participants aged>12 years and of the same joint in a matched control patient. |
|- Primary outcome||Existence of arthropathy: the number and percentage of patients with arthropathy among the different patient groups.
Severity of arthropathy: separate and cumulative scores of HJHS and Pettersson.
Impact of arthropathy on physical functioning and participation: time to complete the figure 8 walking test, separate and cumulative scores of (Ped)HAL and IPA questionnaires.
Impact on quality of life: separate and cumulative scores of D-AIMS2-affect and MPQ-DLV questionnaires.
|- Secondary outcome||Influence of the severity of the coagulation defect on the existence, severity and impact of arthropathy
Number and sites of affected joints
Quantitative use of desmopressin and coagulation factors in relation to the existence of arthropathy
Prophylactic use of coagulation factors in relation to the existence of arthropathy
|- Timepoints||one assessment no follow up assessments planned|
|- Trial web site|
|- status||inclusion stopped: follow-up|
|- CONTACT FOR PUBLIC QUERIES|| K.P.M. Galen, van|
|- CONTACT for SCIENTIFIC QUERIES|| K.P.M. Galen, van|
|- Sponsor/Initiator ||University Medical Center Utrecht (UMCU)|
(Source(s) of Monetary or Material Support)
|- Brief summary||Cross sectional multicenter cohort study that investigates the prevalence, severity and impact of arthropathy in patients with moderate and severe Von Willebrand disease and documented treatment of joint bleeds with desmopressin or clotting factor concentrate compared to moderate and severe VWD patients without such documented joint bleed treatment.
|- Main changes (audit trail)|
|- RECORD||23-apr-2014 - 26-mei-2014|