Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Type 3 Von Willebrand International Registries Inhibitor Prospective Study (3WINTERS-IPS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02460458
Recruitment Status : Unknown
Verified April 2016 by Fondazione Angelo Bianchi Bonomi.
Recruitment status was:  Active, not recruiting
First Posted : June 2, 2015
Results First Posted : April 19, 2016
Last Update Posted : May 19, 2016
Sponsor:
Collaborator:
Sintesi Research Srl
Information provided by (Responsible Party):
Fondazione Angelo Bianchi Bonomi

Study Type Observational
Study Design Observational Model: Cohort
Condition Type 3 Von Willebrand's Disease
Enrollment 266
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Type 3 VWD
Hide Arm/Group Description Diagnosis of Type 3 von Willebrand Disease
Period Title: Retrospective Phase
Started 266
Completed 266
Not Completed 0
Period Title: Confirmatory Phase
Started 266
Completed 0 [1]
Not Completed 266
[1]
Information not yet available.
Period Title: Prospective Phase
Started 0 [1]
Completed 0 [1]
Not Completed 0
[1]
Information not yet available.
Arm/Group Title Type 3 VWD
Hide Arm/Group Description Diagnosis of Type 3 von Willebrand Disease
Overall Number of Baseline Participants 266
Hide Baseline Analysis Population Description
The study population should include a cohort of at least 250 patients with diagnosis of Type 3 Von Willebrand Disease enrolled using homogeneous and standardized criteria.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 266 participants
<=18 years
76
  28.6%
Between 18 and 65 years
179
  67.3%
>=65 years
11
   4.1%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 266 participants
30.13534  (18.22409)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 266 participants
Female
156
  58.6%
Male
110
  41.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 266 participants
Netherlands 9
Sweden 3
Hungary 16
Finland 9
Italy 60
United Kingdom 8
France 13
Germany 18
Iran, Islamic Republic of 120
Spain 10
1.Primary Outcome
Title Bleeding Severity Score (BSS)
Hide Description The range of measurement is from -1 to +4 for each symptom considered (12 symptoms, total range from -12 to 48). For each symptom: 0=no symptom, 1=referred by the patient, 2=brought to medical attention, 3=major intervention. For spontaneous hemorrhagic symptoms, scores equal or greater than two require that the patient has specifically addressed that hemorrhagic symptom with a physician, whatever has been the diagnosis and therapy subsequently proposed. Score 0 and 1 are attributed to symptoms referred by the patient, hence without any precise medical intervention. Score 0 is for negligible or absent symptoms; 1 otherwise. For surgical procedures, it is considered important to differentiate between patients that have never bled because they never underwent surgery and those that did not bled after a surgery. These latter receive a negative score, indicating that the probability of VWD diminishes if you don’t bleed after surgery.
Time Frame 24 months (retrospective phase)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Type 3 VWD
Hide Arm/Group Description:
Diagnosis of Type 3 von Willebrand Disease
Overall Number of Participants Analyzed 266
Median (Full Range)
Unit of Measure: Score on a scale
12
(1 to 33)
2.Primary Outcome
Title General Laboratory Tests for VWD3 Diagnosis (Composite)
Hide Description Hemoglobin: (mmol/L), HT(%), MVC (fl); Leucocytes: (E9/L); Neutrophil (%); Basophil (%); Eosinophil (%); Lymphocyte (%); Platelet count: (E9/L), MPV (fl); Prothrombin Time (sec); PTT (sec); PTT mix 50:50 (sec); Ferritin (ug/l); Bleeding Time (min:sec); Closure Time (Sec); Collagen/ADP (sec); Collagen/Epinephrine (sec); FVIII:C (IU/mL); VWF:RCo (IU/mL); VWF:Ag (IU/mL).
Time Frame 36 months (retrospective + confirmatory phase)
Outcome Measure Data Not Reported
3.Primary Outcome
Title Test for Anti-VWF Antibodies
Hide Description Evaluation of the titre of Anti-VWF Antibodies through Bethesda test (BU).
Time Frame 36 months (retrospective + confirmatory phase)
Outcome Measure Data Not Reported
4.Primary Outcome
Title Molecular Diagnosis of VWF in DNA
Hide Description Evaluation of the presence of VWF gene defects (confirmation or screening for the first time).
Time Frame 36 months (retrospective + confirmatory phase)
Outcome Measure Data Not Reported
5.Primary Outcome
Title Previous Use of Blood Products
Hide Description Record of any product used in the previous 24 months (collected type of blood products/VWF concentrate, year of first exposure, units used).
Time Frame 24 months (retrospective)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Type 3 VWD
Hide Arm/Group Description:
Diagnosis of Type 3 von Willebrand Disease
Overall Number of Participants Analyzed 266
Measure Type: Number
Unit of Measure: participants
Packed red cells 24
Cryoprecipitates 123
Fresh frozen plasma 10
Platelet concentrates 1
6.Primary Outcome
Title Allergic Reactions During Use of VWF-containing Concentrates
Hide Description Record of any allergic and anaphilactic reactions occurred in the past due to the use of any VWF concentrate and the date of onset.
Time Frame 24 months (retrospective)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Type 3 VWD
Hide Arm/Group Description:
Diagnosis of Type 3 von Willebrand Disease
Overall Number of Participants Analyzed 266
Measure Type: Number
Unit of Measure: participants
41
7.Primary Outcome
Title Record of Bleeding Episodes
Hide Description Bleeding: severity, start date, stop date; Treatment: Product name, start date, stop date, Total IU, Total ED.
Time Frame 24 months (prospective phase)
Outcome Measure Data Not Reported
8.Primary Outcome
Title Adverse Events
Hide Description Record of all adverse events occurred during the prospective phase of the study.
Time Frame 24 months (prospective phase)
Outcome Measure Data Not Reported
9.Primary Outcome
Title Type of VWF/FVIII-containing Concentrates in Use
Hide Description Record of any VWF/FVIII-containing concentrates used and currently in use, including the current schedule type of treatment.
Time Frame 24 months (prospective phase)
Outcome Measure Data Not Reported
Time Frame Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
Adverse Event Reporting Description For AE associated with treatments, Investigators are encouraged to contact the manufacturer / regulatory authorities to report it, and to share a copy of any form submitted with Sponsor. An Independent Data Monitoring Committee (DMC) will be appointed for data monitoring on safety.
 
Arm/Group Title Type 3 VWD
Hide Arm/Group Description Diagnosis of Type 3 von Willebrand Disease
All-Cause Mortality
Type 3 VWD
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Type 3 VWD
Affected / at Risk (%)
Total   0/266 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Type 3 VWD
Affected / at Risk (%)
Total   0/266 (0.00%) 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
 
Results Point of Contact
Name/Title: Prof. Augusto B. Federici
Organization: Hematology and Transfusion Medicine, L. Sacco University Hospital Department of Clinical & Community Sciences, University of Milan, Via Pace, 9 20122 Milan, Italy
Phone: +39-0250319895
Publications:
von Willebrand EA. (1926) Hereditär Pseudohemofili. Finska Läkaresällskapets Handlingar 68, 87-112.
Federici AB, Bucciarelli P, Castaman G et al. (2007) Incidence and determinants of bleeding in different types of von Willebrand disease:results of the first prospective multicenter study on 814 Italian patients Blood 110, 713
Abuzenadah AM, Gursel T, Ingerslev J et al. (1999) Mutational analysis of the von Willebrand factor gene in 27 families from Turkey with von Willebrand disease Thromb Haemost 82 (Suppl), 283
Castaman G, Giacomelli SH, Coppola A et al. (2008) Molecular bases of type 3 von Willebrand disease in Italy: report on 12 families Blood Transfus Suppl 3, 44
Gazda H, Budde U, Krey S et al. (1997) Delta C in exon 18 of the von Willebrand factor gene is the most common mutation in patients with severe von Willebrand disease type 3 in Poland Blood 90 (Suppl 1), 94b
Titapiwatanakun R, Guenther JC, Asmann YW et al. (2007) Novel Mutations in Types 2 & 3 von Willebrand Disease and Correlation with von Willebrand Factor Multimer Patterns. Blood 110, 2136
Montgomery RR, Jozwiak MA, Hutter JJ et al. (1999) A homozygous variant of the von Willebrand factor (VWF) that fails to c-terminal dimerize resulting in loss of VWF multimers larger than dimer Blood 94 (Suppl 1), 443a
Schneppenheim R, Budde U, Drewke E et al. (1999) Cysteine mutations of von Willebrand factor correlate with different types of von Willebrand disease Thromb Haemost 82 (Suppl), 283
Enayat MS, Guilliatt AM, Surdhar GK et al. (2001) Identification of five novel mutations in families with type 3 von Willebrand's disease Thromb Haemost 86 (Suppl), P1810
Federici AB, Gianniello F, Canciani MT et al. (2005) Secondary long-term prophylaxis in severe patients with von Willebrand disease: an Italian cohort study Blood 106, 507a
Responsible Party: Fondazione Angelo Bianchi Bonomi
ClinicalTrials.gov Identifier: NCT02460458     History of Changes
Other Study ID Numbers: ABB-11-01
First Submitted: May 27, 2015
First Posted: June 2, 2015
Results First Submitted: February 3, 2016
Results First Posted: April 19, 2016
Last Update Posted: May 19, 2016