Clinical trials: Lymphatic Malformation
Thalassemia (Transfusion Delendent)
Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia
The CENTAurus trial is a prospective clinical study designed to address systematically some of the relevant endocrine complications in an iron overloaded thalassemic population, primary objective being the assessment of the…
- Trial name
- CENTAurus (NCT02069886)
- Testing
- deferasirox
- Sponsor
- Novartis Pharmaceuticals
Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients.
- Conditions
- Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura, Hemophilia A, Acquired, Disseminated Intravascular Coagulation
- Trial name
- NCT06727669
- Where
- 5 sites in China
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Peking University People's Hospital
Blood Coagulation Disorders, Inherited, Thrombotic Disorder
Evaluation of Hemostasis in Bleeding and Thrombotic Disorders
The objective of this study will be to assess the coagulation system in-vitro in a variety of bleeding and clotting disorders using the ROTEG analyzer and the thrombin generation assay.
- Trial name
- NCT00178594
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- The University of Texas Health Science Center, Houston
Autism Spectrum Disorders
Social Interactions: Ocular Explorations and Pupillometry in Autism
The primary objective of this work will first to characterize in typical childhood, visual exploratory behavior and pupillary response associated with salience of human social stimuli (faces and body movements), and then to…
- Trial name
- ISEOP (NCT01647295)
- Where
- France
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Sickle Cell Disease
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
- Trial name
- NCT05632354
- Testing
- Osivelotor
- Where
- 9 sites in United States, Nigeria
- Volunteers
- 47 people
- Sponsor
- Pfizer
Other Sickle-cell Disorders With Crisis, Unspecified
Quality of Life Study for Sickle Cell Patients Treated With Jobelyn (Sorghum Bicolor Extract)
The purpose of this study is to determine the antioxidant effect of prolonged use of sorghum bicolor (jobelyn) to increase the level of plasma superoxide dismutase and glutathione reductase in patients with sickle cell disease…
- Trial name
- NCT01704794
- Testing
- Folic Acid + Paludrine + Jobelyn (500mg), Folic Acid + Paludrine + Jobelyn (250mg), Folic Acid + Paludrine + Jobelyn (2mg)
- Where
- Nigeria
- Volunteers
- 96 people
- Sponsor
- Lagos State University
Hemophilia
Combination Regimen With Sodium Valproate for Severe Hemophilia: a Single-arm, Phase 1, Pilot Trial.
The goal of this clinical trial is to determine the clinical efficacy and toxic effects of sodium valproate, sirolimus and calcitriol in the treatment of severe haemophilia in participants with severe haemophilia .
- Trial name
- NCT05920512
- Testing
- Sodium valproate extended-release tablets
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Xue-chun Lu
Leukemia
Study of Gemtuzumab Ozogamicin Therapy in DNA Samples From Patients With Acute Myeloid Leukemia Treated on COG-AAML0531
RATIONALE: DNA analysis of blood and tissue samples may help doctors predict how well patients will respond to treatment. It may also help doctors learn more about how gemtuzumab ozogamicin works in the body.
- Trial name
- NCT01407757
- Testing
- DNA analysis, gene expression analysis, laboratory biomarker analysis
- Volunteers
- 1,000 people
- Sponsor
- Children's Oncology Group
Tuberous Sclerosis, Lymphangioleiomyomatosis
TSC Biosample Repository and Natural History Database
The TSC Biosample Repository collects and stores samples of blood, DNA, and tissues that scientists can request to use in their research. The samples we collect are all linked to clinical data in the TSC Natural History Database.
- Trial name
- NCT05676099
- Testing
- Phlebotomy, Buccal (cheek) swab, Genetic Testing
- Where
- 26 sites in United States, Canada
- Volunteers
- 5,000 people
- Sponsor
- National Tuberous Sclerosis Association
Congenital Bleeding Disorder, Haemophilia A
Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia A or B in a Non- Bleeding State
This trial is conducted in Europe. The aim of this trial is to determine the pharmacokinetics of activated recombinant human factor VII (NovoSeven®) in haemophiliac patients in a non-bleeding state.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01562587
- Testing
- activated recombinant human factor VII
- Where
- 8 sites in Germany, Greece and 3 more
- Volunteers
- 18 people
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease
Glutamine Role in Preventing Vaso-occlusive Crisis Among SCD Patients
Prospective phase IV interventional open label randomized controlled trial to assess safety and efficacy of glutamine in preventing vaso-occlusive crisis (VOC) episodes in sickle cell pediatrics and adolescents' patients
- Trial name
- Glu_SCD_Egy (NCT05371184)
- Testing
- L-Glutamine, Oral Powder for Reconstitution, Standard of care
- Where
- 2 sites in Egypt
- Volunteers
- 60 people
- Sponsor
- Ain Shams University
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease.
- Trial name
- HOPE Kids 2 (NCT04218084)
- Testing
- Voxelotor
- Where
- 35 sites in United States, Egypt and 7 more
- Volunteers
- 236 people
- Sponsor
- Pfizer
Hemophilia A
Lentiviral FVIII Gene Therapy
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene…
- Trial name
- NCT03217032
- Testing
- YUVA-GT-F801
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Transfusion-dependent Beta-Thalassemia
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem…
- Trial name
- NCT05860595
- Testing
- KL003 cell injection Drug Product
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
Hemophilia, Hemophilia A
ATHN 8: Previously Untreated Patients (PUPs) Matter Study
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs).
- Conditions
- Hemophilia, Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor
- Trial name
- NCT03818529
- Where
- 27 sites in United States
- Volunteers
- 237 people
- Sponsor
- American Thrombosis and Hemostasis Network
Beta Thalassemia Major
Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia
the aim of this study is to evaluate the effect of Spirulina compared to Amlodipine on cardiac iron overload and cardiac functions in multi-transfused children with beta thalassemia major
- Trial name
- NCT02671695
- Testing
- Spirulina, Amlodipine
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Tanta University
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
AR-42 and Decitabine in Treating Patients With Acute Myeloid Leukemia
This phase I trial studies the side effects and best dose of AR-42 when given together with decitabine in treating patients with acute myeloid leukemia.
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
- Trial name
- NCT01798901
- Testing
- HDAC inhibitor AR-42, decitabine, laboratory biomarker analysis
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Alison Walker
Hemophilia
Assessment of Proprioceptive and Functional Characteristics in Patients With Hemophilia
Analyze and evaluate the skeletal muscle of patients affected with hemophilia in the Region of Murcia. Describe the periarticular muscular deficit of the lower limbs, depending on the degree of injury: acute and chronic, or…
- Trial name
- HE-BALANCE (NCT02165592)
- Where
- Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Universidad Católica San Antonio de Murcia
Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7)
Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia
This research study is studying identification of de novo Fanconi anemia in younger patients with newly diagnosed acute myeloid leukemia.
- Conditions
- Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7), Childhood Acute Minimally Differentiated Myeloid Leukemia (M0), Childhood Acute Monoblastic Leukemia (M5a)
- Trial name
- NCT01146210
- Testing
- laboratory biomarker analysis
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Oncology Group
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.