Clinical trials: Blood Coagulation Disorders in Children
Haemophilia, Autism Spectrum Disorder
Prevalence of Autistic Spectrum Disorder (ASD) in Children With Haemophilia
Parents of children with haemophilia will be invited to complete 3 questionnaires to look for traits present in ASD. With consent teacher will complete a further 2 questionnaires.
- Conditions
- Haemophilia, Autism Spectrum Disorder, Social Communication Disorder, Attention Deficit Hyperactivity Disorder
- Trial name
- NCT05138224
- Volunteers
- 82 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Hemophilia A, Hemophilia B
Patterns of Hemophilia Care in Assiut Children Patients
Hemophilia A and B are congenital, recessive X-linked disorders caused by lack or deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively.
- Trial name
- NCT05980377
- Volunteers
- 60 people
- Sponsor
- Assiut University
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
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency
A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
The main objective is to determine the efficacy of Gammaplex by measuring the number of serious acute bacterial infections during treatment with Gammaplex over a 12 month period.
- Conditions
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinemia, Hyper-IgM Syndrome
- Trial name
- NCT01289847
- Testing
- Gammaplex
- Where
- 10 sites in United States, Chile and 1 more
- Volunteers
- 25 people
- Sponsor
- Bio Products Laboratory
Hemophilia a, Muscle Strength
Effect of Kinetic Chain Exercises on Muscle Strength, Joint Position Sense and Quality of Life in Children with Haemophilia.
The goal of this clinical trial is to learn if there are effect of close kinetic chain exercises on muscle strength, joint position sense and Quality of life in children with haemophilia? Null hypothesis It will be hypothesized…
- Conditions
- Hemophilia a, Muscle Strength, Joint Position Sense, Quality of Life (QOL)
- Trial name
- NCT06649175
- Testing
- Exercise
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Cairo University
Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplant
A Study Using Real-world Data to Examine Outcomes in Children and Adults Who Develop a Blood Clotting Disorder Called Thrombotic Microangiopathy (TMA) After Undergoing a Hematopoietic Stem Cell Transplant (HSCT).
The purpose of this study was to assess OS outcomes in pediatric (≥ 28 days of age at the time of HSCT-TMA diagnosis) and adult participants diagnosed with HSCT-TMA(referred to as TMA from hereon) within 52 weeks after HSCT and…
- Trial name
- ALX-TMA-502 (NCT07732361)
- Where
- 43 sites in United States, Belgium and 8 more
- Volunteers
- 307 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Chronic Myeloproliferative Disorders, Leukemia
INCB18424 in Treating Young Patients With Relapsed or Refractory Solid Tumor, Leukemia, or Myeloproliferative Disease
RATIONALE: INCB18424 (Ruxolitinib) may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. PURPOSE: This phase 1 clinical trial is studying the side effects and best dose of INCB18424 in…
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT01164163
- Testing
- ruxolitinib phosphate, laboratory biomarker analysis, pharmacological study
- Where
- 24 sites in United States
- Volunteers
- 49 people
- Sponsor
- Children's Oncology Group
Immune Thrombocytopenia
Post IVIG Medication in Children With Immune Thrombocytopenia
This study is a single hospital system, single-arm year-long pilot to evaluate the feasibility of enrolling children with ITP who are receiving IVIG for treatment of disease to a scheduled post-infusion medication for 72 hours…
- Trial name
- NCT04741139
- Testing
- Acetaminophen and Diphenhydramine Only Product
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Baylor College of Medicine
Respiratory Distress Syndrome, Adult
Understanding the Role of Genes and Biomarkers in the Inflammation and Blood Clotting Process in Children With Acute Lung Injury
Acute lung injury (ALI)/Acute Respiratory Distress Syndrome (ARDS) is a severe lung condition that causes respiratory failure. This study will examine if differences in genes and biomarkers involved in the inflammation and blood…
- Trial name
- PALI (NCT00605527)
- Where
- 5 sites in United States
- Volunteers
- 396 people
- Sponsor
- University of California, San Francisco
Immune Thrombocytopenia, Glucocorticoids
Short-course High-dose Prednisone and Dexamethasone in Children With ITP
Comparison of the efficacy and safety of short-course high-dose prednisone and dexamethasone in the treatment of children with newly diagnosed immune thrombocytopenia (ITP)
- Conditions
- Immune Thrombocytopenia, Glucocorticoids, Child, Only
- Trial name
- NCT05522465
- Testing
- Prednisone, Dexamethasone
- Where
- China
- Volunteers
- 608 people
- Sponsor
- Fujian Medical University Union Hospital
Hemophilia
Oral Health Status and Oral Health Related Quality of Life in a Group pf Egyptian Hemophilic Children
aim of the study: 1. to assess, investigate, and compare the oral health status and oral health related quality of life of a group of hemophilic Egyptian children before and after an oral health educational program 2.
- Trial name
- NCT07161596
- Testing
- oral health educational program
- Where
- Egypt
- Volunteers
- 70 people
- Sponsor
- Cairo University
Hemophilia, Upper Extremity
Comparison of Upper Extremity Functional Performance in Hemophilic and Healthy Children
The goal of this observational study is to evaluate the functional performance of the upper extremity joints (scapula, shoulder, elbow, and wrist) in haemophilic children and compare muscle strength and ADL limitations with…
- Conditions
- Hemophilia, Upper Extremity, Strength, Performance
- Trial name
- NCT06826209
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Immune Thrombocytopenia
The Efficacy and Safety of Huaiqihuang Granule in Children With Chronic Primary Immune Thrombocytopenia
This is a multisite, double-blind, randomized, placebo-controlled, and parallel study designed to evaluate the clinical efficacy and safety of Huaiqihuang granule for treatment to children with chronic primary immune…
- Trial name
- ITP (NCT03201848)
- Testing
- Huaiqihuang Granule
- Where
- 14 sites in China
- Volunteers
- 216 people
- Sponsor
- Qidong Gaitianli Medicines Co., Ltd
Immune Thrombocytopenic Purpura, Helicobacter Pylori Infection
Helicobacter Pylori Treatment in Immune Thrombocytopenic Purpura (ITP) Patients
The purpose of this study is to determine whether eradication of Helicobacter pylori infection is effective in the improvement of platelet counts in children and adolescents with chronic ITP.
- Trial name
- NCT01730352
- Testing
- H. pylori triple therapy
- Where
- 4 sites in Brazil
- Volunteers
- 100 people
- Sponsor
- Federal University of São Paulo
Immune Thrombocytopenia
Use of Vitamin D as An Adjuvant Treatment in Children With Chronic Idiopathic Thrombocytopenic Purpura
Immune thrombocytopenia is an autoimmune disease in which platelets are targeted by the host immune system. Platelet depletion occurs when autoantibodies targeting glycoproteins (αIIb-β3 and GPIb) found on the surface of…
- Trial name
- MansouraU (NCT06073925)
- Testing
- VIT D
- Volunteers
- 30 people
- Sponsor
- Mansoura University
Giant Cell Arteritis, Takayasu Arteritis
Biologics in Refractory Vasculitis: A Trial of Biologic Therapy for Refractory Primary Non-ANCA Associated Vasculitis
Vasculitis occur when the body's immune system, rather than protecting the body, attacks blood vessels, causing injury to the vessel and the part of the body it supplies with blood.
- Conditions
- Giant Cell Arteritis, Takayasu Arteritis, Cogan Syndrome, Relapsing Polychondritis
- Trial name
- BIOVAS (NCT05168475)
- Testing
- Rituximab, Infliximab, Tocilizumab
- Where
- 5 sites in United Kingdom
- Volunteers
- 22 people
- Sponsor
- Cambridge University Hospitals NHS Foundation Trust
Idiopathic Thrombocytopenic Purpura
Treatment of Idiopathic Thrombocytopenic Purpura (ITP) With Subcutaneously Administered Anti-D
The purpose of this study is to describe the effect of anti-D on symptoms and platelet count in children suffering from unexplainable low platelet counts, when anti-D is administered as an injection under the skin.
- Trial name
- NCT00128882
- Testing
- Anti-D
- Where
- 17 sites in Denmark
- Volunteers
- 45 people
- Sponsor
- University of Aarhus
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.
Hemophilia A
Outcomes of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast
Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast
- Trial name
- NCT05279924
- Testing
- Prophylaxis with Emicizumab
- Where
- Côte d’Ivoire
- Volunteers
- 33 people
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Idiopathic Thrombocytopenic Purpura (ITP), Immune Thrombocytopenic Purpura (ITP)
Clinical Trial of Rituximab in Children and Adolescents With Chronic Idiopathic Thrombocytopenic Purpura (ITP)
The purpose of the study is to evaluate the safety and efficacy of rituximab in children ages 18 months to 18 years, who have severe, chronic ITP.
- Trial name
- NCT01713738
- Testing
- rituximab
- Where
- 9 sites in United States
- Volunteers
- 36 people
- Sponsor
- Neufeld, Ellis J, MD, PhD