Clinical trials: Lymphatic Abnormalities
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment (refractory).
- Trial name
- NCT03190915
- Testing
- Bone Marrow Aspiration and Biopsy, Trametinib
- Where
- 57 sites in United States
- Volunteers
- 10 people
- Sponsor
- National Cancer Institute (NCI)
Lymphangiomatosis, Lymphedema
Characterization of the Pathogenesis of Primary and Secondary Lymphatic Disorders
Background: Lymphatics are a type of vessel, similar to arteries and veins. Lymphatic disorders happen when these vessels don t work properly.
- Conditions
- Lymphangiomatosis, Lymphedema, Lymphangiectasia, Pulmonary Lymphangiectasia
- Trial name
- NCT02156115
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Cardiogenic Shock, Malignant Arrhythmias
The Caribbean Registry of Extracorporeal Membrane Oxygenation (ECMO) From the University Hospital in Martinique
The project's main goal is to collect baseline clinical and procedural data as well as to assess clinical outcomes for all patients undergoing VV, VA or VAV ECMO implantation in the French West Indies and Guiana.
- Conditions
- Cardiogenic Shock, Malignant Arrhythmias, Ischemic or Valvular Heart Failure, Drepanocytosis and Thoracic Syndrome
- Trial name
- NCT07351435
- Volunteers
- 1,200 people
- Sponsor
- University of Genova
Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease
- Trial name
- NCT07082725
- Testing
- Nizubaglustat
- Where
- 34 sites in United States, Argentina and 13 more
- Volunteers
- 72 people
- Sponsor
- Azafaros B.V.
Sickle Cell Disorders
GenoMed4ALL: Improving SCD Classification and Prognosis by AI
GenoMed4All 'Genomics and Personalized Medicine for all though Artificial Intelligence in Haematological Diseases' aims to advance on individual SCD patients' disease characterisation and to improve the monitoring of patients'…
- Trial name
- GenoMed4ALL (NCT06019208)
- Where
- 5 sites in France, Italy and 2 more
- Volunteers
- 1,000 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
B Acute Lymphoblastic Leukemia, B Lymphoblastic Lymphoma
A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia
This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down…
- Conditions
- B Acute Lymphoblastic Leukemia, B Lymphoblastic Lymphoma, Down Syndrome
- Trial name
- NCT03914625
- Testing
- Asparaginase Erwinia chrysanthemi, Blinatumomab, Cyclophosphamide
- Where
- 228 sites in United States, Australia and 3 more
- Volunteers
- 6,720 people
- Sponsor
- National Cancer Institute (NCI)
Chronic Granulomatous Disease (CGD)
Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
Background: X-Linked Chronic Granulomatous Disease (X-CGD) is caused by a gene mutation that makes the immune system to not work properly.
- Trial name
- NCT07113743
- Testing
- Busulfan, Tocilizumab, Eltrombopag
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Sickle Cell Disease
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.
- Trial name
- NCT07656415
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 159 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Gene Mutations, Pseudoxanthoma Elasticum
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to…
- Conditions
- Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
- Trial name
- NCT06462547
- Testing
- INZ-701
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 22 people
- Sponsor
- Inozyme Pharma
Hemophilia A
A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A
The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A.
- Trial name
- Leopold II (NCT01233258)
- Testing
- rFVIII (BAY81-8973) on demand, rFVIII (BAY81-8973) prophylaxis low-dose, rFVIII (BAY81-8973) prophylaxis high-dose
- Where
- 47 sites in United States, Argentina and 16 more
- Volunteers
- 80 people
- Sponsor
- Bayer
Hemophilia A and B
Identification of New Biomarkers in Haemophilic Arthropathy Through Transcriptome Analysis
Chronic arthropathy is one of the most common complications of haemophilia, affecting up to 90 per cent of patients aged 4 and over in at least one joint.
- Trial name
- THEMA (NCT07867821)
- Testing
- Surgery
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Wiskott-Aldrich Syndrome
Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome
The Wiskott-Aldrich Syndrome (WAS) is an inherited disorder that results in defects of the blood and bone marrow. It affects boys because the genetic mistake is carried on the X chromosome.
- Trial name
- NCT01410825
- Testing
- Retrovirus-mediated gene transfer
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- David Williams
Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor
Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Rare Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Bone marrow transplantation may allow doctors to give higher doses of chemotherapy and kill more tumor cells.
- Conditions
- Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Head and Neck Cancer, Kidney Cancer
- Trial name
- NCT00002515
- Testing
- filgrastim, carboplatin, thiotepa
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Glanzmann Thrombasthenia
Anti-αIIbβ3 Immunization in Glanzmann Thrombasthenia: Prevalence and Associated Risk Factors: Thrombasthenia Anti-αIIbβ3 Antibodies Study (TAAS)
This project aim to correlate risk factors (genetic, therapeutic and socio-demographic factors) to anti-αIIbβ3 antibodies formation following blood products transfusion (platelets or packed red cells) or pregnancy in a national…
- Trial name
- TAAS (NCT04595617)
- Testing
- Antibodies screening
- Where
- 7 sites in France
- Volunteers
- 28 people
- Sponsor
- University Hospital, Bordeaux
Cystic Fibrosis, Pulmonary Fibrosis
Role of Genetic Factors in the Development of Lung Disease
This study is designed to evaluate the genetics involved in the development of lung disease by surveying genes involved in the process of breathing and examining the genes in lung cells of patients with lung disease.
- Conditions
- Cystic Fibrosis, Pulmonary Fibrosis, Tuberous Sclerosis, Asthma
- Trial name
- NCT00001532
- Where
- 2 sites in United States
- Volunteers
- 3,500 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Graft Versus Host Disease, Leukemia
Clofarabine, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk and/or Advanced Hematologic Cancer or Other Disease
RATIONALE: Giving chemotherapy, such as clofarabine, melphalan, and thiotepa, before a donor stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00423514
- Testing
- filgrastim, clofarabine, melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
VWD - Von Willebrand's Disease
Update in VWD Laboratory Diagnosis
The present study will focus on the investigation of the global contribution and limitations of the multimeric analysis and mutation analysis in the VWD diagnostic process. 1.
- Trial name
- NCT03896997
- Volunteers
- 100 people
- Sponsor
- Assiut University
Hemophilia B
Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B.
- Trial name
- PUPs B-LONG (NCT02234310)
- Testing
- rFIXFc
- Where
- 27 sites in United States, Australia and 9 more
- Volunteers
- 33 people
- Sponsor
- Bioverativ, a Sanofi company
Sickle Cell Anaemia, Sickle Cell Anemia Crisis
Hypoxic Red Blood Cells in Sickle Cell Anemia
The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia.
- Conditions
- Sickle Cell Anaemia, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
- Trial name
- NCT06743113
- Testing
- Hemanext ONE System, Conventional RBCs
- Where
- 5 sites in United States
- Volunteers
- 48 people
- Sponsor
- Hemanext
Pain, Sickle Cell Disease
Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally
The pathophysiology of sickle cell disease (SCD) manifestations, are complex with interactions of intracellular hemoglobin, membrane and endothelial activation but the hallmark remains recurrent and painful vaso-occlusive…
- Trial name
- COMPARE (NCT01380197)
- Testing
- Morphine, Nubain
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Children's Healthcare of Atlanta
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