Clinical trials: Blood Coagulation Disorders in Children
Hemophilia
Individualized Prophylaxis for Severe Hemophilia A Children
A multi centre two year long term escalating dose tertiary prophylaxis study on the efficacy and cost saving of individualized low dose prophylaxis regimens for boys with severe hemophilia A in China staring with a low dose…
- Trial name
- NCT02116855
- Testing
- according to the efficacy of AJBRs
- Volunteers
- 50 people
- Sponsor
- Beijing Children's Hospital
Treatment of Bleeding, Prophylaxis of Bleeding
A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood
Researchers are looking for a better way to treat hemophilia A. Hemophilia A is a genetic disorder where the body does not create enough of a protein called clotting factor 8 (FVIII) present in the blood.
- Conditions
- Treatment of Bleeding, Prophylaxis of Bleeding, Hemophilia A, Children
- Trial name
- Alfa-PROTECT (NCT05147662)
- Testing
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Where
- 18 sites in United States, Argentina and 5 more
- Volunteers
- 36 people
- Sponsor
- Bayer
Factor X Deficiency
A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children <12 Years
The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months. The secondary objectives of the study are: 1.
- Trial name
- NCT01721681
- Testing
- FACTOR X
- Where
- 3 sites in United Kingdom
- Volunteers
- 9 people
- Sponsor
- Bio Products Laboratory
Immune Thrombocytopenia, Treatment
Anti-CD38 Antibody Treating Children With Primary Immune Thrombocytopenia (ITP)
To evaluate the safety and efficacy of Anti-CD38 Antibody in the treatment of primary immune thrombocytopenia in patients who have failed first-line treatment.
- Trial name
- NCT06838962
- Testing
- Daratumumab Injection
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Prophylaxis, Hemophilia A
Comparison of Prophylaxis and On-demand Treatment in Children With Moderate to Severe Hemophilia A
Comparison of prophylaxis and on-demand treatment in children with moderate to severe hemophilia A. This study determines to compare the efficacy of prophylaxis and on-demand treatment in moderate to severe hemophilia A children…
- Trial name
- NCT02362581
- Testing
- prophylaxis treatment, on demand treatment
- Where
- Thailand
- Volunteers
- 15 people
- Sponsor
- Chulalongkorn University
Thrombotic Thrombocytopenic Purpura (TTP)
A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP)
Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body.
- Trial name
- NCT03393975
- Testing
- TAK-755, Standard of care
- Where
- 33 sites in United States, Austria and 7 more
- Volunteers
- 52 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A
The goal of this study is to gather more information on safety and efficacy of Kovaltry for the prevention and treatment of bleeds in Chinese children, adolescents/adults with severe hemophilia A.
- Trial name
- NCT04565236
- Testing
- Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 1, Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 2, Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 3
- Where
- 10 sites in China
- Volunteers
- 45 people
- Sponsor
- Bayer
Immune Thrombocytopenia
Association of Platelet Parameters With Bleeding Severity in Children With ITP
Patients with severe immune thrombocytopenia (ITP) present with similarly low platelet counts but varying bleeding symptoms, making it difficult to predict the disease course and to decide on an appropriate treatment plan.
- Trial name
- ITP-APPS (NCT03810352)
- Where
- 6 sites in United States
- Volunteers
- 95 people
- Sponsor
- Boston Children's Hospital
Hemophilia A
Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A
Hemophilia A (HA) is a rare congenital bleeding disorder characterized by coagulation factor VIII deficiency. In severe HA, defined as plasma FVIII clotting activity < 1%, bleeding may frequently occur spontaneously, most…
- Trial name
- NCT05248594
- Testing
- HEMLIBRA
- Where
- 10 sites in United States
- Volunteers
- 71 people
- Sponsor
- Montefiore Medical Center
Coagulation Disorder, Cirrhosis, Liver
ADAMTS-13 and Von Willebrand Factor Levels and Activities in Children With Cirrhosis and/or Portal Hypertension
Hemostasis-related disorders are common in cirrhosis and portal hypertension. However, it is not known whether the net effect of changes in hemostasis in the sense of predisposition to hemorrhagic or thrombotic state.
- Trial name
- NCT04267406
- Where
- Turkey (Türkiye)
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Ataturk University
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
- Trial name
- BASIS KIDS (NCT05611801)
- Testing
- marstacimab
- Where
- 63 sites in United States, Argentina and 21 more
- Volunteers
- 100 people
- Sponsor
- Pfizer
Wiskott-Aldrich Syndrome
Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.
The rationale for this retrospective study is to evaluate the efficacy and safety of thrombopoietin-receptor agonist (TPO-RA) romiplostim for reducing thrombocytopenia and bleeding tendency in pediatric participants with…
- Trial name
- NCT04350164
- Testing
- Romiplostim
- Where
- Russia
- Volunteers
- 67 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
IgA Vasculitis, Gastrointestinal Involvement
I-FABP as a Predictor of Gastrointestinal Involvement in Pediatric IgA Vasculitis
This prospective observational study aims to evaluate plasma intestinal fatty acid-binding protein (I-FABP) levels in children with IgA vasculitis and investigate their association with gastrointestinal involvement.
- Trial name
- IFABP-IgAV (NCT07664228)
- Testing
- Plasma I-FABP Measurement
- Where
- Turkey (Türkiye)
- Volunteers
- 100 people
- Sponsor
- Bursa City Hospital
Venous Thromboembolism
Thrombosis Outcomes in Pediatric Venous Thromboembolism
'The TOP Study' is a prospective cohort study whose main objective is to develop better prognostic biomarkers and identify children at risk of adverse thrombotic outcomes very early in the course after an initial venous…
- Trial name
- TOP (NCT03068923)
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- University of Texas Southwestern Medical Center
Haemophilia A
A Study to Investigate the Safety and Efficacy of Optivate® in Children Under 6 Years of Age With Haemophilia A.
The main objectives of this study are: to assess Optivate® consumption (IU/kg consumed per month for prophylactic and on-demand therapy and dose at each bleed). to assess clinical outcome when treating a bleed with Optivate®.
- Trial name
- NCT02246894
- Testing
- Optivate® (Human Coagulation Factor VIII)
- Where
- 5 sites in Poland
- Sponsor
- Bio Products Laboratory
Hemophilia
Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia
The MOrPH study is designed to identify optimal prophylaxis schedules for children with haemophilia. This involves development of combined pharmacokinetic and pharmacodynamic models.
- Trial name
- MOrPH (NCT02585635)
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Neuroscience Research Australia
IgAVN, IgA Vasculitis
Telitacicept in the Treatment of Pediatric IgA Vasculitis-Associated Nephritis
To further evaluate the efficacy and safety of Telitacicept in the treatment of pediatric IgA vasculitis nephritis (IgAVN) within real-world settings, this study employs a multicenter, retrospective and prospective observational…
- Trial name
- NCT07354503
- Testing
- Collect clinical data from the patients., This study exclusively prospectively collected clinical data from the patients and did not involve any therapeutic interventions
- Volunteers
- 300 people
- Sponsor
- The Children's Hospital of Zhejiang University School of Medicine
Hemophilia A, Hemophilia B
The Hemophilia Ultrasound Project
To evaluate the prevalence of subclinical arthropathy in children with severe hemophilia undergoing a prophylaxis regimen and without evidence of target joints, using a validated ultrasound scoring method.
- Trial name
- HUP (NCT02807753)
- Testing
- Ankle and Knee Ultrasound Joint Assessment, Ankle and Knee Magnetic Resonance Imaging
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- University of Miami
Haemophilia
A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children
In the past, due to economic and medical resource constraints, the hemophilia comprehensive care in China was suboptimal. The BCH data of both retrospective and prospective studies reveals that for 4-6y and 6-9y patients with…
- Trial name
- CHIPS (NCT02999308)
- Testing
- Observe the patient's condition and then change the regimen
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Beijing Children's Hospital
Henoch-Schonlein Purpura
Leflunomide for Henoch-Schonlein Purpura
This study conducted a prospective, multicenter, one-arm clinical trial on the combination of leflunomide and steroid therapy for refractory skin Henoch-Schonlein Purpura in children on the basis of ethical principles.
- Trial name
- Lef for HSP (NCT05937880)
- Testing
- Leflunomide
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University