Clinical trials: Blood Platelets
- Condition Blood Platelets
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Thrombotic Thrombocytopenic Purpura (TTP)
A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body.
- Trial name
- NCT04683003
- Testing
- TAK-755
- Where
- 27 sites in United States, Austria and 9 more
- Volunteers
- 77 people
- Sponsor
- Takeda
Congenital Bleeding Disorder, Congenital FVII Deficiency
Observational Study on the Efficacy and Safety of NovoSeven® During "Real-life" Usage in Germany
This NON INTERVENTIONAL OBSERVATIONAL STUDY is conducted in Europe. The primary aim is to observe the haemostatic efficacy of NovoSeven® treatment during routine practice in German clinics.
- Conditions
- Congenital Bleeding Disorder, Congenital FVII Deficiency, Glanzmann's Disease, Acquired Bleeding Disorder
- Trial name
- WIRK (NCT00697320)
- Testing
- eptacog alfa (activated)
- Where
- Germany
- Volunteers
- 64 people
- Sponsor
- Novo Nordisk A/S
Congenital Thrombotic Thrombocytopenic Purpura
Prospective Psychometric Evaluation Study of a Patient-reported Outcomes (PRO) Instrument for Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP]
The purpose of this study is to assess the psychometric properties of a recently developed congenital thrombotic thrombocytopenic purpura (cTTP)-specific patient-reported outcomes (PRO) instrument.
- Trial name
- NCT03519672
- Testing
- No intervention
- Where
- 4 sites in United States, United Kingdom
- Volunteers
- 41 people
- Sponsor
- Baxalta now part of Shire
Essential Thrombocythemia (ET)
Pediatric Disease Registry in Essential Thrombocythaemia (ET)
The aim of this study is to evaluate the progression of ET in children (aged 6-17years inclusive) over a period of 5 years maximum.
- Trial name
- NCT01198717
- Where
- 32 sites in Czechia, France and 6 more
- Volunteers
- 60 people
- Sponsor
- Shire
Thrombotic Thrombocytopenic Purpura (TTP)
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce…
- Trial name
- NCT07353099
- Testing
- No Intervention
- Where
- 7 sites in Austria, Germany
- Volunteers
- 50 people
- Sponsor
- Takeda
Atypical Hemolytic Uremic Syndrome (aHUS)
Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
The purpose of the study is to assess the efficacy of ravulizumab to control disease activity in children and adolescents with aHUS who have not previously used a complement inhibitor (complement inhibitor treatment-naïve), as…
- Trial name
- NCT03131219
- Testing
- Ravulizumab
- Where
- 18 sites in United States, Belgium and 5 more
- Volunteers
- 34 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Thrombocythemia, Essential
Safety and Pregnancy Outcomes in Thrombocythemia Patients Exposed to XAGRID® (Anagrelide Hydrochloride) Compared to Other Treatments
This is an observational safety study being conducted in Europe comparing patients taking Xagrid to patients taking other cytoreductive treatments.
- Trial name
- NCT00567502
- Where
- 164 sites in Cyprus, Denmark and 12 more
- Volunteers
- 3,647 people
- Sponsor
- Shire
Von Willebrand Disease (VWD)
A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
The main aim of the study is to check effectiveness of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in pediatric and adult participants…
- Trial name
- NCT03879135
- Testing
- rVWF, rFVIII
- Where
- 33 sites in United States, Austria and 7 more
- Volunteers
- 38 people
- Sponsor
- Baxalta now part of Shire
Atypical Hemolytic Uremic Syndrome (aHUS)
The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
Protocol is intended to characterize the overall safety and tolerability of eculizumab in this population.
- Trial name
- NCT01757431
- Testing
- Eculizumab
- Where
- 2 sites in Japan
- Volunteers
- 2 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Purpura, Thrombocytopenic, Idiopathic
A Long-term Safety Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
This was an open-label Phase III extension study to evaluate the long-term safety of eltrombopag in pediatric patients with chronic ITP who previously participated in study TRA115450.
- Trial name
- NCT02201290
- Testing
- Eltrombopag Tablets, Eltrombopag PfOS
- Where
- 3 sites in Russia
- Volunteers
- 9 people
- Sponsor
- Novartis Pharmaceuticals
Hairy Cell Leukemia
US Post-Marketing Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI)
This study is being conducted to satisfy a post-marketing requirement (PMR) to provide evidence characterizing 1) the safety of moxetumomab pasudotox-tdfk in patients who are 65 years of age and older and/or 2) the safety of…
- Trial name
- PROXY (NCT04125290)
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- AstraZeneca
Atypical Hemolytic Uremic Syndrome
aHUS Observational Long Term Follow-Up
There is growing but limited information on the long term clinical status of aHUS patients who have previously received or are continuing to receive treatment with eculizumab.
- Trial name
- LTFU (NCT01522170)
- Where
- 56 sites in United States, Australia and 10 more
- Volunteers
- 94 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Ultomiris-exposed Pregnant/ Postpartum, Pregnancy
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of…
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
- Trial name
- NCT06312644
- Testing
- Ultomiris
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Atypical Hemolytic Uremic Syndrome
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
This was a prospective, open-label study with no participant randomization. Treatment for aHUS was observational and at the discretion of the treating physician.
- Trial name
- EVIDENCE (NCT02614898)
- Testing
- Eculizumab
- Where
- 24 sites in United States, Australia and 2 more
- Volunteers
- 67 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hospitalized Cases of Angioneurotic Oedema, Pneumonia
Drug Utilization and Safety Events Among Children Using Esomeprazole, Other Proton Pump Inhibitors or H2-receptor Antagonists
The purpose of this study is 1. To describe patient characteristics and drug usage among children that are prescribed esomeprazole for the first time and to compare them with patients who are prescribed other proton pump…
- Conditions
- Hospitalized Cases of Angioneurotic Oedema, Pneumonia, Gastroenteritis, Failure to Thrive
- Trial name
- NCT01338363
- Where
- Netherlands
- Volunteers
- 23,470 people
- Sponsor
- AstraZeneca
Purpura, Thrombocytopenic, Idiopathic
Rapid Infusion Of Immune Globulin Intravenous (IGIV) In Patients With ITP
The objective of this study is to determine if the safety and tolerability of Immune Globulin Intravenous (Human), 10% Caprylate/Chromatograph Purified (IGIV-C) is similar when infused at two different infusion rates.
- Trial name
- NCT00220727
- Testing
- Immune Globulin IV [Human], 10% Caprylate/Chromatography Purified
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Grifols Therapeutics LLC
Von Willebrand Disease
Efficacy and Safety of Fanhdi®, a High-purity Von Willebrand Containing FVIII Concentrate, in Pediatric Patients With Von Willebrand Disease
Multicenter, prospective, non-controlled study in a pediatric cohort (<6 years-old) with severe (type 2 or 3) hereditary Von Willebrand Disease (VWD).
- Trial name
- NCT02472665
- Testing
- plasma-derived FVIII/VWF concentrate
- Where
- 4 sites in Spain
- Sponsor
- Grifols Therapeutics LLC
Thrombocytopenia
Efficacy and Safety Study of Platelets Treated for Pathogen Inactivation and Stored for Up to Seven Days
Objective: To determine if platelets treated for pathogen inactivation and stored for 6 to 7 days are safe and effective compared to platelets collected by the same method, stored for the same amount of time and not treated for…
- Trial name
- TESSI (NCT00261924)
- Testing
- Transfusion of Pathogen Inactivated Platelets stored for 6-7 days
- Where
- United Kingdom
- Volunteers
- 211 people
- Sponsor
- Cerus Corporation
Primary Immune Thrombocytopenia (ITP)
A Study of Ianalumab in Addition to Eltrombopag in Pediatric Patients With Primary ITP Who Failed Corticosteroids.
The purpose of this study is to assess the efficacy, safety and pharmacokinetics (PK) of ianalumab (VAY736) in addition to eltrombopag treatment; and to inform the dose of ianalumab in pediatric patients (5 to <18 years of age)…
- Trial name
- VAYHIT-P (NCT07660172)
- Testing
- Ianalumab, Eltrombopag
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Immune Thrombocytopenia
Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia
The purpose of this study is to investigate the efficacy, safety of Hetrombopag in children with previously treated chronic immune thrombocytopenia who are between 6 and 17 years of age. This is a 2 part study.
- Trial name
- NCT04737850
- Testing
- Hetrombopag
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.