Clinical trials: Lymphatic Abnormalities
Niemann-Pick Disease, Type C
Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other parts of the body.
- Trial name
- NCT05588167
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Fetal Hemoglobin, Beta-Thalassemia
Long Term Beta Thalassemia Treatment: Findings From The Extension Period
The project, titled "Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial," aims to compare the efficacy and safety of combination therapy (thalidomide and hydroxyurea) versus…
- Conditions
- Fetal Hemoglobin, Beta-Thalassemia, Hemoglobinopathies
- Trial name
- Thalidomide (NCT06490601)
- Testing
- thalidomide and hydroxyurea, Thalidomide
- Where
- Pakistan
- Volunteers
- 30 people
- Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Hemoglobinopathies, Hemolysis
Clinical and Laboratory Investigation of Humans With Informative Iron or Erythroid Phenotypes
This study will examine blood for factors that may cause or prevent diseases involving iron or red blood cells. Iron is an important nutrient for human health that is needed to produce red blood cells.
- Conditions
- Hemoglobinopathies, Hemolysis, Iron Deficiency and Overload, Anemias
- Trial name
- NCT00102245
- Where
- United States
- Volunteers
- 334 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Anemia, Sickle Cell
Use of Hydroxyurea and Magnesium Pidolate for Treatment of Sickle Cell Disease
The purpose of this study is to estimate the MTD of Mg pidolate in combination with HU in patients with sickle cell disease who have been on a therapeutic dose (15-30 mg/kg/day) of HU for at least 6 months.
- Trial name
- NCT00143572
- Testing
- Magnesium Pidolate, Hydroxyurea
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- St. Jude Children's Research Hospital
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
Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome
Total Marrow Irradiation for Refractory Acute Leukemia
RATIONALE: Giving chemotherapy and total marrow irradiation before a donor umbilical cord blood or hematopoietic stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, Multiple Myeloma
- Trial name
- NCT00686556
- Testing
- cyclophosphamide, cyclosporine, Fludarabine
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Leukemia, Myelodysplastic Syndromes
Donor Umbilical Cord Blood Natural Killer Cells, Aldesleukin and Umbilical Cord Blood Transplant in Patients With Refractory Hematologic Cancers.
RATIONALE: Giving chemotherapy, natural killer cells, aldesleukin, and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of abnormal cells and cancer cells.
- Trial name
- NCT00354172
- Testing
- aldesleukin, filgrastim, natural killer cell (NK) therapy
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Haemophilia, Arthropathy
Biomechanical Determinants and Patterns Associated to the Pathophysiological Cascade of Ankle Arthropathy in Children With Haemophilia: Non-invasive In-vivo Measurement of Foot Joints in Children With Haemophilia During Gait.
Haemophilia is a rare X chromosome-linked coagulation disorder resulting from a congenital deficiency or absence of circulating factor VIII (Haemophilia A) or factor IX (Haemophilia B).As a consequence, patients with haemophilia…
- Trial name
- 3DMKFM (NCT02229331)
- Testing
- Gait analysis
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Hemophilia A
Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery
This study will examine the efficacy and safety of ReFacto AF in patients with severe and moderately severe hemophilia A undergoing elective major surgery when administered by either bolus injections (BI) or continuous infusion…
- Trial name
- NCT00243659
- Testing
- ReFacto AF
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 30 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Mild and Moderate Atopic Dermatitis Patients, Atopic Dermatitis (AD)
Clinical Trial of an Ointment in Patients With Mild and Moderate Atopic Dermatitis
Atopic dermatitis (AD) is a chronic skin disorder, which negatively impacts up to 20% children and 3% adults in the world. The main AD treatments aim to regress clinical manifestations, improve and restore skin lesions.
- Trial name
- NCT07653386
- Testing
- S.formosum-containing ointment, commercial moisturizer
- Where
- Vietnam
- Volunteers
- 90 people
- Sponsor
- VinUniversity
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
Atypical Hemolytic Uremic Syndrome, Membranoproliferative Glomerulonephritis
Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases
This project aims to identify, through RNA-Seq technology, the genetic alterations underlying undiagnosed rare diseases in pediatric and adult patients with early onset and with negative WES.
- Conditions
- Atypical Hemolytic Uremic Syndrome, Membranoproliferative Glomerulonephritis, Autosomal Dominant Polycystic Kidney, Healthy
- Trial name
- ANTHEM (NCT05996731)
- Testing
- Skin biopsy
- Where
- Italy
- Volunteers
- 105 people, healthy volunteers welcome
- Sponsor
- Mario Negri Institute for Pharmacological Research
Brain Tumor, Vascular Lesion
Factor XIII and Pulmonary Embolism in Neurosurgical Patients
Two studies (Gerlach et al. 2000; Gerlach et al. 2002) described the impact of factor XIII on the risk of prospective hemorrhage for patients undergoing craniotomy.
- Conditions
- Brain Tumor, Vascular Lesion, Factor XIII Deficiency, Pulmonary Embolism
- Trial name
- NCT01106937
- Where
- Germany
- Volunteers
- 130 people
- Sponsor
- Ludwig-Maximilians - University of Munich
Sickle Cell Disease
Simvastatin (Zocor) Therapy in Sickle Cell Disease
Recent clinical and experimental data indicate that statins have effects beyond cholesterol lowering that may be beneficial in sickle cell disease by protecting the vascular endothelium.
- Trial name
- NCT00508027
- Testing
- Simvastatin
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Anaemia
Prevention Of Morbidity In Sickle Cell Disease Pilot Phase
The hypothesis is that in sickle cell anaemia, nocturnal oxyhaemoglobin desaturation, is associated with low processing speed index, and this morbidity can be reduced with overnight auto Continuous Positive Airways Pressure…
- Trial name
- NCT00415727
- Testing
- auto Continuous Positive Airways Pressure (CPAP) with oxygen supplementation
- Where
- 2 sites in United Kingdom
- Volunteers
- 22 people
- Sponsor
- Institute of Child Health
Hemoglobin SC Disease
Hydroxyurea and Magnesium Pidolate to Treat People With Hemoglobin Sickle Cell Disease
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes. Hemoglobin SCD (HbSC) is a form of SCD that is characterized by dense red blood cells.
- Trial name
- NCT00532883
- Testing
- Hydroxyurea, Magnesium Pidolate
- Where
- 19 sites in United States
- Volunteers
- 44 people
- Sponsor
- St. Jude Children's Research Hospital
Leukemia, Juvenile Myelomonocytic, JMML
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML).
- Conditions
- Leukemia, Juvenile Myelomonocytic, JMML, JCML, Neurofibromatosis 1
- Trial name
- NCT05849662
- Testing
- Trametinib, Azacitidine, Fludarabine
- Where
- 21 sites in United States
- Volunteers
- 58 people
- Sponsor
- Therapeutic Advances in Childhood Leukemia Consortium
Haemophilia B
A Study to Investigate the Safety and Efficacy of Replenine®-VF in Haemophilia B Patients Under the Age of 6 Years
The objective of this study was to assess the safety and efficacy of Replenine®-VF in children enrolled in the study, under the age of six years, with severe haemophilia B.
- Trial name
- NCT02263469
- Testing
- Replenine®-VF (High Purity Factor IX)
- Where
- 4 sites in Poland, Ukraine
- Sponsor
- Bio Products Laboratory
Focal Segmental Glomerulosclerosis, Minimal Change Disease
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.
- Conditions
- Focal Segmental Glomerulosclerosis, Minimal Change Disease, Immunoglobulin A Nephropathy, IgA Vasculitis
- Trial name
- EPPIK (NCT05003986)
- Testing
- Sparsentan
- Where
- 47 sites in United States, Germany and 6 more
- Volunteers
- 67 people
- Sponsor
- Travere Therapeutics, Inc.
Brain and Central Nervous System Tumors, Kidney Cancer
Early Enteral Tube Feedings in Children Receiving Chemo for AML/MDS & High Risk Solid Tumors
RATIONALE: Tubefeeding may help maintain good nutrition and lessen weight loss in younger patients receiving chemotherapy for cancer.
- Conditions
- Brain and Central Nervous System Tumors, Kidney Cancer, Leukemia, Liver Cancer
- Trial name
- NCT00624962
- Testing
- nutritional intervention, therapeutic nutritional supplementation
- Sponsor
- Vanderbilt University
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