Trial Outcomes & Findings for Type 3 Von Willebrand International Registries Inhibitor Prospective Study (NCT NCT02460458)
NCT ID: NCT02460458
Last Updated: 2024-12-27
Results Overview
Multimer analysis of Von Willebrand Factor (VWF) was carried out by electrophoresis of blood samples collected by investigational sites. The number of patients belonging of each multimer profile group (1 - Homozygotes / 2 - Only Protomers / 3 - 2-4 Bands) was calculated. The qualitative evaluation of VWF multimers is part of the diagnostic process of VWD3.
Recruitment status
COMPLETED
Target enrollment
265 participants
Primary outcome timeframe
12 months (confirmatory phase)
Results posted on
2024-12-27
Participant Flow
Recruitment Period: November 2012 - March 2015
Participant milestones
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3). In the Retrospective Phase, the retrospective data of the participants were collected and analyzed, and a blood sample was drawn. During the Confirmatory Phase, local VWD3 diagnosis was verified using centralized tests. Participants with a confirmed case of VWD3 were then prospectively observed for two years (First Prospective Phase). After a Confirmation of the Clinical Phase Data, the participants started a Second Prospective Phase, being observed for two additional years.
|
|---|---|
|
Retrospective Phase
STARTED
|
265
|
|
Retrospective Phase
COMPLETED
|
265
|
|
Retrospective Phase
NOT COMPLETED
|
0
|
|
Confirmatory Phase
STARTED
|
265
|
|
Confirmatory Phase
COMPLETED
|
210
|
|
Confirmatory Phase
NOT COMPLETED
|
55
|
|
First Prospective Phase
STARTED
|
210
|
|
First Prospective Phase
COMPLETED
|
126
|
|
First Prospective Phase
NOT COMPLETED
|
84
|
|
Confirmation of Clinical Phase Data
STARTED
|
126
|
|
Confirmation of Clinical Phase Data
COMPLETED
|
92
|
|
Confirmation of Clinical Phase Data
NOT COMPLETED
|
34
|
Reasons for withdrawal
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3). In the Retrospective Phase, the retrospective data of the participants were collected and analyzed, and a blood sample was drawn. During the Confirmatory Phase, local VWD3 diagnosis was verified using centralized tests. Participants with a confirmed case of VWD3 were then prospectively observed for two years (First Prospective Phase). After a Confirmation of the Clinical Phase Data, the participants started a Second Prospective Phase, being observed for two additional years.
|
|---|---|
|
Confirmatory Phase
Patients excluded basing on phenotype central analysis
|
31
|
|
Confirmatory Phase
Lack of sample to analyse
|
19
|
|
Confirmatory Phase
Patients excluded due to lack of central characterized VWD mutations
|
5
|
|
First Prospective Phase
Lost to Follow-up
|
84
|
|
Confirmation of Clinical Phase Data
Lost to Follow-up
|
34
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=265 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Age, Categorical
<=18 years
|
75 Participants
n=265 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
179 Participants
n=265 Participants
|
|
Age, Categorical
>=65 years
|
11 Participants
n=265 Participants
|
|
Age, Continuous
|
30.19 years
STANDARD_DEVIATION 18.22 • n=265 Participants
|
|
Sex: Female, Male
Female
|
157 Participants
n=265 Participants
|
|
Sex: Female, Male
Male
|
108 Participants
n=265 Participants
|
|
Region of Enrollment
Netherlands
|
9 participants
n=265 Participants
|
|
Region of Enrollment
Sweden
|
3 participants
n=265 Participants
|
|
Region of Enrollment
Hungary
|
16 participants
n=265 Participants
|
|
Region of Enrollment
Finland
|
9 participants
n=265 Participants
|
|
Region of Enrollment
Italy
|
60 participants
n=265 Participants
|
|
Region of Enrollment
United Kingdom
|
8 participants
n=265 Participants
|
|
Region of Enrollment
France
|
13 participants
n=265 Participants
|
|
Region of Enrollment
Germany
|
18 participants
n=265 Participants
|
|
Region of Enrollment
Iran, Islamic Republic of
|
119 participants
n=265 Participants
|
|
Region of Enrollment
Spain
|
10 participants
n=265 Participants
|
|
MCMDM-1 Bleeding Score System Retrospective Data
|
15.14 units on a scale
n=249 Participants • The MCMDM-1 Bleeding Score System Retrospective Data was collected for 249 / 265 patients since historical data about bleedings was not fully available at the investigational sites for the remaining 16 patients.
|
PRIMARY outcome
Timeframe: 12 months (confirmatory phase)Population: Factor VIII (FVIII) Procoagulant Activity (FVIII:C) was centrally obtained from samples collected from investigational sites.
Measurement of the Factor VIII (FVIII) Procoagulant Activity (FVIII:C) in the blood through one-stage clotting test. Only patients with FVIII:C less or equal to 5 IU/dL were considered for the analysis.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=183 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Centralized Factor VIII (FVIII) Procoagulant Activity (FVIII:C) Laboratory Test for Type 3 Von Willebrand's Disease (VWD3) Diagnosis
|
2.42 IU/dL
Standard Deviation 0.88
|
PRIMARY outcome
Timeframe: 12 months (confirmatory phase)Population: Von Willebrand Factor (VWF) protein blood levels were centrally obtained from samples collected from investigational sites.
Measurement of the amount of Von Willebrand Factor (VWF) protein in the blood through Von Willebrand Factor Antigen (VWF:Ag) test. Only patients with VWF:Ag less or equal to 5 IU/dL were considered for the analysis.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=209 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Centralized Von Willebrand Factor Antigen (VWF:Ag) Laboratory Test for Type 3 Von Willebrand's Disease (VWD3) Diagnosis
|
1.01 IU/dL
Standard Deviation 1.03
|
PRIMARY outcome
Timeframe: 12 months (confirmatory phase)Population: Factor VIII (FVIII) Amidolytic Activity (FVIII:Am) in the blood was centrally obtained from samples collected from investigational sites.
Measurement of Factor VIII (FVIII) Amidolytic Activity (FVIII:Am) in the blood through chromogenic test. Only patients with FVIII:Am less or equal to 5 IU/dL were considered for the analysis.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=191 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Centralized Factor VIII (FVIII) Amidolytic Activity (FVIII:Am) Laboratory Test for Type 3 Von Willebrand's Disease (VWD3) Diagnosis
|
1.54 IU/dL
Standard Deviation 0.91
|
PRIMARY outcome
Timeframe: 12 months (confirmatory phase)Population: Factor VIII (FVIII) protein blood levels were centrally obtained from samples collected from investigational sites.
Measurement of the amount of Factor VIII (FVIII) protein in the blood through FVIII:Ag test. Only patients with FVIII:Ag less or equal to 5 IU/dL were considered for the analysis.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=140 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Centralized Factor VIII (FVIII) Antigen (FVIII:Ag) Laboratory Test for Type 3 Von Willebrand's Disease (VWD3) Diagnosis
|
3.63 IU/dL
Standard Deviation 0.83
|
PRIMARY outcome
Timeframe: 12 months (confirmatory phase)Population: Von Willebrand Factor (VWF) multimer profiles of patients were centrally obtained from samples collected from investigational sites.
Multimer analysis of Von Willebrand Factor (VWF) was carried out by electrophoresis of blood samples collected by investigational sites. The number of patients belonging of each multimer profile group (1 - Homozygotes / 2 - Only Protomers / 3 - 2-4 Bands) was calculated. The qualitative evaluation of VWF multimers is part of the diagnostic process of VWD3.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=243 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Centralized Von Willebrand Factor (VWF) Multimer Analysis for Type 3 Von Willebrand's Disease (VWD3) Diagnosis
Only Protomers
|
15 Participants
|
|
Centralized Von Willebrand Factor (VWF) Multimer Analysis for Type 3 Von Willebrand's Disease (VWD3) Diagnosis
2 - 4 Bands
|
54 Participants
|
|
Centralized Von Willebrand Factor (VWF) Multimer Analysis for Type 3 Von Willebrand's Disease (VWD3) Diagnosis
Homozygotes
|
174 Participants
|
PRIMARY outcome
Timeframe: 12 months (confirmatory phase)Population: Von Willebrand Factor (VWF) Propeptide blood levels were centrally obtained from samples collected from investigational sites.
Measurement of Von Willebrand Factor (VWF) Propeptide levels in the blood through VWF Propeptide test. This test has been performed according to the most recent methods and the results are important to characterize the molecular aspects of VWD patients.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=238 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Centralized Von Willebrand Factor (VWF) Propeptide Laboratory Test for Type 3 Von Willebrand's Disease (VWD3) Diagnosis
|
6.44 IU/dL
Standard Deviation 13.98
|
PRIMARY outcome
Timeframe: 12 months (confirmatory phase)Population: Samples were not available for 34 patients out of 265
Evaluation of the presence of Von Willebrand Factor (VWF) gene defects (confirmation or screening for the first time).
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=231 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Centralized Molecular Type 3 Von Willebrand's Disease (VWD3) Diagnosis Through DNA Analysis
Analyzed participants with confirmed VWD3 diagnosis
|
219 Participants
|
|
Centralized Molecular Type 3 Von Willebrand's Disease (VWD3) Diagnosis Through DNA Analysis
Analyzed participants without confirmed VWD3 diagnosis
|
12 Participants
|
PRIMARY outcome
Timeframe: 24 months (first prospective phase) + 24 months (second prospective phase)Population: Patients that have competed both prospective phases.
Record of all bleedings occurred during the prospective phase of the study.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=92 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Record of Bleeding Episodes
|
713 bleeding episodes
|
PRIMARY outcome
Timeframe: 24 months (first prospective phase) + 24 months (second prospective phase)Population: Patients that have competed both prospective phases.
Record of all adverse events occurred during the prospective phase of the study.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=92 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Adverse Events
|
47 AE
|
PRIMARY outcome
Timeframe: 24 months (first prospective phase) + 24 months (second prospective phase)Population: Patients that have competed both prospective phases.
Record of any Von Willebrand Factor / Factor VIII (VWF/FVIII)-containing concentrates used and currently in use, including the current schedule type of treatment.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=92 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Type of Von Willebrand Factor / Factor VIII (VWF/FVIII)-Containing Concentrates in Use
Recombinant Activated Factor FVIII
|
1 participants
|
|
Type of Von Willebrand Factor / Factor VIII (VWF/FVIII)-Containing Concentrates in Use
Fanhdi
|
1 participants
|
|
Type of Von Willebrand Factor / Factor VIII (VWF/FVIII)-Containing Concentrates in Use
Voncento/Haemate P
|
33 participants
|
|
Type of Von Willebrand Factor / Factor VIII (VWF/FVIII)-Containing Concentrates in Use
Wilate/Wilfactin (Wilfact)
|
5 participants
|
|
Type of Von Willebrand Factor / Factor VIII (VWF/FVIII)-Containing Concentrates in Use
Aryoseven
|
1 participants
|
SECONDARY outcome
Timeframe: 24 months (retrospective phase)Record of any allergic and anaphylactic reactions occurred in the past due to the use of any Von Willebrand Factor (VWF) concentrate and the date of onset.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=265 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Patients Experiencing Allergic Reactions During Use of Von Willebrand Factor (VWF)-Containing Concentrates
|
41 Participants
|
SECONDARY outcome
Timeframe: 24 months (retrospective phase)Record of any product used during the retrospective phase (collected type of blood products/Von Willebrand Factor (VWF) concentrate, year of first exposure, units used).
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=265 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Number of Participants With Previous Use of Blood Products
Fresh frozen plasma
|
10 Participants
|
|
Number of Participants With Previous Use of Blood Products
Platelet concentrates
|
1 Participants
|
|
Number of Participants With Previous Use of Blood Products
Packed red cells
|
24 Participants
|
|
Number of Participants With Previous Use of Blood Products
Cryoprecipitates
|
123 Participants
|
SECONDARY outcome
Timeframe: 24 months (retrospective phase)Evaluation of the titre of Anti-Von Willebrand Factor (anti-VWF) Antibodies through Bethesda Test.
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=265 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Number of Patients With Available Local Laboratory Test for Anti-Von Willebrand Factor (Anti-VWF) Antibodies
|
4 Participants
|
SECONDARY outcome
Timeframe: 24 months (retrospective phase)Number of patients for who the following tests have been performed: Hemoglobin (mmol/L), Hemagglutination Titer (HT) (%), Mean Corpuscular Volume (MVC) (fl), Leucocytes (E9/L), Neutrophils (%), Basophils (%), Eosinophils (%), Lymphocytes (%), Platelet Count (E9/L), Mean Platelet Volume (MPV) (fl), Prothrombin Time (sec), Partial Thromboplastin Time (PTT) (sec), Partial Thromboplastin Time Mix 50:50 (PTT mix 50:50) (sec), Ferritin (ug/l), Bleeding Time (min:sec), Closure Time (sec), Collagen/ADP (sec), Collagen/Epinephrine (sec); Factor VIII Procoagulant Activity (FVIII:C) (IU/mL), Von Willebrand Factor Ristocetin Cofactor (VWF:RCo) (IU/mL), Won Willebrand Factor Antigen (VWF:Ag) (IU/mL).
Outcome measures
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=265 Participants
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Local Laboratory Tests for Type 3 Von Willebrand's Disease (VWD3) Diagnosis (Composite)
|
265 Participants
|
Adverse Events
Type 3 Von Willebrand's Disease (VWD3)
Serious events: 5 serious events
Other events: 28 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=265 participants at risk
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
Reproductive system and breast disorders
Post-partum bleeding in uterus
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Gastrointestinal disorders
Gastro-Intestinal bleeding
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Nervous system disorders
Intracranial bleeding
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Blood and lymphatic system disorders
Anaemia
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Nervous system disorders
Headache righr hemisphere
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
Other adverse events
| Measure |
Type 3 Von Willebrand's Disease (VWD3)
n=265 participants at risk
Patients with diagnosis of Type 3 Von Willebrand's Disease (VWD3)
|
|---|---|
|
General disorders
Abdomen and flank pain
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
General disorders
Abdominal pain
|
1.5%
4/265 • Number of events 4 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Gastrointestinal disorders
Diarrhea
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Respiratory, thoracic and mediastinal disorders
Common cold
|
3.8%
10/265 • Number of events 23 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Immune system disorders
Allergy
|
0.38%
1/265 • Number of events 4 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrea
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Musculoskeletal and connective tissue disorders
Arthrosis in right ankle
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Musculoskeletal and connective tissue disorders
Ankle trauma
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Metabolism and nutrition disorders
B12 deficiency
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Skin and subcutaneous tissue disorders
Foot burn
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Eye disorders
Conjunctivitis
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Musculoskeletal and connective tissue disorders
Capsulitis in finger (right hand)
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
General disorders
Fall
|
0.75%
2/265 • Number of events 2 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Blood and lymphatic system disorders
Epistaxis
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
General disorders
Fever
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Gastrointestinal disorders
Gastroenteritis
|
1.1%
3/265 • Number of events 3 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Skin and subcutaneous tissue disorders
Hematoma
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Gastrointestinal disorders
Hypogastric pain
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Vascular disorders
Hypertension
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Metabolism and nutrition disorders
Iron deficiency
|
0.75%
2/265 • Number of events 2 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Respiratory, thoracic and mediastinal disorders
Pharingitis
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
General disorders
Pelvic pain
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Respiratory, thoracic and mediastinal disorders
Sinusitis
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Musculoskeletal and connective tissue disorders
Right knee pain
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Musculoskeletal and connective tissue disorders
Tooth fracture
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Surgical and medical procedures
Tooth extraction
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Injury, poisoning and procedural complications
Trauma of elbow
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Injury, poisoning and procedural complications
Trauma of gum
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Injury, poisoning and procedural complications
Twisting ankle
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Reproductive system and breast disorders
Vulvovaginal pain
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Gastrointestinal disorders
Vomiting
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract infection
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
General disorders
Flashing after receiving VWD Concentrate
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Reproductive system and breast disorders
In Vitro Fertilization
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
|
Skin and subcutaneous tissue disorders
Sensitiveness
|
0.38%
1/265 • Number of events 1 • Data about adverse events occurring to participants will be collected throughout the whole study period (retrospective phase + prospective phase).
For adverse events 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.
|
Additional Information
Prof. Augusto B. Federici
Hematology and Transfusion Medicine, L. Sacco University Hospital, University of Milan, Via G. B. Grassi, 74 20157 Milan, Italy
Phone: +39-0250319895
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER