Clinical trials: Lymphatic Abnormalities
Hemophilia A
Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A
The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts.
- Trial name
- NCT00157040
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Baxalta now part of Shire
Hemolytic Disease of Newborn, Neonatal Hyperbilirubinemia
Safe Threshold to Discontinue Phototherapy in Hemolytic Disease of Newborn
We hypothesized that adopting a lower rather than a higher threshold for phototherapy discontinuation will be associated with reduced rates of rebound hyperbilirubinemia in term and late preterm neonates with hemolytic disease of…
- Trial name
- NCT04218318
- Testing
- phototherapy
- Where
- Saudi Arabia
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- Ministry of Health, Saudi Arabia
Anemia, Sickle Cell
Effectiveness of Arginine as a Treatment for Sickle Cell Anemia
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited genetic disease that can cause intense pain episodes.
- Trial name
- Arginine (NCT00513617)
- Testing
- Arginine
- Where
- 17 sites in United States
- Volunteers
- 128 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Chronic Granulomatous Disease
Role of Interferon-gamma 1-b (IFN-γ) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease
The overall goal of the study is to investigate the functional, biochemical, and gene expression effects of Interferon-gamma 1-b (IFN-γ) on the neutrophils of patients with Chronic Granulomatous Disease (CGD).
- Trial name
- NCT03548818
- Testing
- Interferon Gamma-1B
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Colorado, Denver
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Fludarabine and Busulfan Followed by Donor Peripheral Stem Cell Transplant and Antithymocyte Globulin, Tacrolimus, and Methotrexate in Treating Patients With Myeloid Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and busulfan, before a donor peripheral stem cell transplant helps stop the growth of abnormal and cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00346359
- Testing
- anti-thymocyte globulin, busulfan, fludarabine phosphate
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
Beta-Thalassemia
Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood…
- Trial name
- NCT00061750
- Testing
- ICL670, deferoxamine
- Where
- 7 sites in United States
- Volunteers
- 595 people
- Sponsor
- Novartis Pharmaceuticals
Hemophilia A
Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A
This study is a multi-center, open-label, single-arm trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (≥12 years old) with severe hemophilia A who…
- Trial name
- NCT03815318
- Testing
- Recombinant Human Coagulation FVIII
- Where
- China
- Volunteers
- 73 people
- Sponsor
- Sinocelltech Ltd.
Sickle Cell Disease
Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
The use of analgesics can lead to cases of drug abuse and dependence. It can also cause pseudo-addiction in patients suffering from pain.
- Trial name
- PHEDRE (NCT02580565)
- Testing
- telephone interview
- Where
- France
- Volunteers
- 1,004 people
- Sponsor
- Nantes University Hospital
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care…
- Trial name
- NCT06192576
- Testing
- Olipudase alfa
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Sanofi
Sickle Cell Anemia
Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia
The purpose of this study is to use comprehensive exercise testing to examine longitudinal changes in exercise capacity over a 2 year period in children and young adults with sickle cell anemia.
- Trial name
- NCT01558076
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Hemophilia A
Individualizing Hemophilia Prophylaxis Using Thromboelastography
Currently dosing for prophylaxis is not individualized, and the general approach is to use a dose of 25-40 units/kg given 3 times per week or every other day.
- Trial name
- NCT02582060
- Testing
- Thromboelastography/Thromboelastometry-Guided Treatment Regimen
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Children's Hospital Los Angeles
Sickle Cell Disease
Study of Blood Platelets in Sickle Cell Disease
We are studying if sickle cell disease blood platelets are larger than normal and how they may cause obstruction of blood vessels.
- Trial name
- NCT00226083
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Rockefeller University
Sickle Cell Disease
Spectralis HRA+OCT Imaging of the Retina With Autofluorescence in Sickle Cell Disease
To determine the retinal and choroidal thickness in patients with sickle cell disease compared to age, race matched population without sickle cell disease to allow a better understanding of the clinical manifestations of sickle…
- Trial name
- NCT01123369
- Where
- 2 sites in United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Medical University of South Carolina
Kawasaki Disease
Pharmacokinetics (PK)/Safety Study of Atorvastatin in Children With Kawasaki Disease and Coronary Artery Abnormalities
Kawasaki disease (KD) is the leading cause of acquired heart disease in children in the developed world. Despite available treatment, 25% of children in San Diego County appropriately treated for KD develop coronary artery…
- Trial name
- NCT01431105
- Testing
- Atorvastatin
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- University of California, San Diego
Von Willebrand Disease, Type 3
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been…
- Trial name
- WILL-EMI (NCT06998524)
- Testing
- Emicizumab, von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates
- Where
- 29 sites in United States, Belgium and 12 more
- Volunteers
- 75 people
- Sponsor
- Hoffmann-La Roche
22q11.2 Deletion Syndrome
Understanding of Psychotic Disorders in Children With 22q11.2DS
The study PremiCeS22 will investigate the prodromal signals at the onset of psychotic disorders of children with 22q11.2 deletion syndrome
- Trial name
- PremiCeS22 (NCT04639388)
- Testing
- neuropsychological testing, questionnaires and experimental tasks
- Where
- France
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Hôpital le Vinatier
Hemophilia A
Hemophilia Inhibitor Previously Untreated Patient Study
Hemophilia A is a congenital bleeding disorder caused by deficiency of factor VIII (FVIII) and is treated by replacement therapy with FVIII concentrate.
- Trial name
- HIPS (NCT01652027)
- Testing
- FVIII concentrate
- Where
- 17 sites in United States, Austria and 3 more
- Volunteers
- 25 people
- Sponsor
- The University of Texas Health Science Center, Houston
Thalassemia Major
Long Term Outcomes in β Thalassemia Major
Beta thalassemia (β-thalassemia) is the most common genetic disease worldwide. Individuals with thalassemia are born with a defect in hemoglobin.
- Trial name
- NCT02307786
- Where
- United States
- Volunteers
- 176 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Mevalonate Kinase Deficiency
Geranylgeraniol Supplementation in Patients With Mevalonate Kinase Deficiency
Mevalonate kinase deficiency (HyperIgD syndrome, HIDS) is an inborn error of immunity caused by a block in the mevalonate pathway.
- Trial name
- NCT06497829
- Testing
- geranylgeraniol
- Where
- Czechia
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- University Hospital, Motol
Cyanotic Congenital Heart Disease, Anemia
Erythropoietin to Prevent Unnecessary Transfusions In Patients With Cyanotic CHD - A Prospective Control Trial
Cyanotic congenital cardiac patients require higher hemoglobin concentrations (red blood cell levels) for optimal oxygen delivery to the body.
- Conditions
- Cyanotic Congenital Heart Disease, Anemia, Cyanosis, Congenital Heart Disease
- Trial name
- NCT02564796
- Testing
- Epoetin Alfa and Iron, Iron
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of California, San Diego
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