Clinical trials: Lymphatic Malformation
Leukemia
Chemotherapy, Total-Body Irradiation, Donor Natural Killer Cell Infusion, Aldesleukin, and UCB Transplant in Treating Patients With Relapsed or Refractory AML
RATIONALE: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells.
- Trial name
- NCT00871689
- Testing
- aldesleukin, cyclophosphamide, fludarabine phosphate
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Primary Immunodeficiency, Agammaglobulinemia
Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)
The purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID).
- Conditions
- Primary Immunodeficiency, Agammaglobulinemia, Hypogammaglobulinemia, Antibody Deficiency
- Trial name
- NCT01581593
- Testing
- Kedrion IVIG 10%
- Where
- 15 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Kedrion S.p.A.
Graft Versus Host Disease, Leukemia
Alemtuzumab, Busulfan, and Cyclophosphamide Followed By a Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Monoclonal antibodies, such as alemtuzumab, can find cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells.
- Conditions
- Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Trial name
- NCT00555048
- Testing
- alemtuzumab, busulfan, cyclophosphamide
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Fred Hutchinson Cancer Center
Lysosomal Acid Lipase Deficiency
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.
- Trial name
- NCT02193867
- Testing
- Sebelipase Alfa
- Where
- 5 sites in United States, Finland and 2 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Sickle Cell Disease
Integration of mHEALTH Into the Care of Patients With Sickle Cell Disease to Increase Hydroxyurea Utilization
This project proposes to develop, test and evaluate targeted interventions to improve clinical provider prescribing of and patient adherence to hydroxyurea (HU).
- Trial name
- NCT04080167
- Testing
- InCharge Health mobile application, HU Toolbox mobile application
- Where
- 7 sites in United States
- Volunteers
- 382 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Hemophilia B
BeneFIX Drug Use Results Survey [All-Case Surveillance]
The survey is intended to investigate the following matters, etc. under the actual use status after marketing in all patients who are administered this drug for a certain period of time after the launch. 1.
- Trial name
- NCT01154231
- Testing
- Nonacog Alfa (Genetical Recombination)
- Where
- Japan
- Volunteers
- 314 people
- Sponsor
- Pfizer
Adult Acute Erythroid Leukemia (M6), Adult Acute Megakaryoblastic Leukemia (M7)
CPX-351 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome
This phase 2 clinical trial studies how well CPX-351 (liposomal cytarabine-daunorubicin) works in treating patients with relapsed or refractory acute myeloid leukemia or myelodysplastic syndrome.
- Conditions
- Adult Acute Erythroid Leukemia (M6), Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia and Acute Monocytic Leukemia (M5)
- Trial name
- NCT02019069
- Testing
- liposomal cytarabine-daunorubicin CPX-351
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Rondeep Brar
Anemia, Sickle Cell, Transfusion Hemosiderosis
Clinical Importance of Treating Iron Overload in Sickle Cell Disease
Hypothesis: The investigators suspect that significant degrees of iron overload in subjects with SCD result in decreased red cell survival, abnormal endothelial function and markedly dysregulated autonomic function.
- Trial name
- NCT00981370
- Testing
- deferasirox
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Children's Hospital Los Angeles
Primary Immune Deficiency Disease
Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects
This is a multi-center, open-label study to assess the efficacy and safety of Flebogamma 5% DIF in the pediatric population.
- Trial name
- NCT00634569
- Testing
- Flebogamma 5% DIF
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Instituto Grifols, S.A.
Leukemia
Prophylactic White Cell Transfusions Versus Therapeutic White Cell Transfusions in Patients With Leukemia
Patients with leukemia often have low white blood cell counts after chemotherapy, which puts them at greater risk for infection.
- Trial name
- NCT01204788
- Testing
- Prophylactic White Cell Transfusion, Therapeutic White Cell Transfusion
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- M.D. Anderson Cancer Center
Sickle Cell Disease
A Rehabilitation Program in Children With Sickle Cell Disease and Cognitive Deficits: a Pilot Study
The overall goal of this project is to determine the feasibility of conducting a cognitive intervention for children with sickle cell disease.
- Trial name
- NCT00895154
- Testing
- General Tutoring, Memory Training
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
Dasatinib, Cytarabine, and Idarubicin in Treating Patients With High-Risk Acute Myeloid Leukemia
This phase I/II trial studies the side effects and best dose of dasatinib when given together with cytarabine and idarubicin hydrochloride and to see how well they work in treating patients with acute myeloid leukemia that is…
- 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
- NCT01876953
- Testing
- cytarabine, idarubicin, dasatinib
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- City of Hope Medical Center
Hemophilia A
A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A
The main aim of the study is to determine how well Adynovate works to decrease bleeding in previously treated Chinese men and boys with severe hemophilia A when given prophylactically.
- Trial name
- NCT05707351
- Testing
- Adynovate
- Where
- 12 sites in China
- Volunteers
- 37 people
- Sponsor
- Takeda
Congenital Bleeding Disorder, Haemophilia A
Research Study to Look at Side Effects During Regular Injection With Factor VIII Medicine Named Turoctocog Alfa for a 8 Weeks Period
This study will test the well-known medicine turoctocog alfa for any side effects. The purpose is to test turoctocog alfa for any side effects in the Indian population. The participants will get turoctocog alfa.
- Trial name
- guardian 10 (NCT03449342)
- Testing
- turoctocog alfa
- Where
- 9 sites in India
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Chronic Iron Overload
Pharmacokinetic Study of Deferiprone in Paediatric Patients
Deferiprone (DFP) is the most extensively studied oral iron chelator to date. It has been authorised in Europe in 1999 for the treatment of iron overload in patients with beta-thalassaemia major when DFO is contraindicated or…
- Trial name
- DEEP-1 (NCT01740713)
- Testing
- Deferiprone, dose level 1, Deferiprone, dose level 2, Deferiprone, dose level 3
- Where
- 7 sites in Cyprus, Egypt and 1 more
- Volunteers
- 23 people
- Sponsor
- Consorzio per Valutazioni Biologiche e Farmacologiche
Sickle Cell Disease
Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause organ damage, stroke, and intense pain episodes.
- Trial name
- SCURT (NCT00745420)
- Testing
- Hematopoietic Stem Cell Transplantation
- Where
- 19 sites in United States
- Volunteers
- 30 people
- Sponsor
- Medical College of Wisconsin
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773
This trial is conducted in Asia, Europe, Japan, North America and South Africa. The aim is to evaluate the safety and efficacy of nonacog beta pegol (NNC-0156-0000-0009) after long-term exposure in patients with haemophilia B.
- Trial name
- paradigm™ 4 (NCT01395810)
- Testing
- nonacog beta pegol
- Where
- 49 sites in United States, Austria and 16 more
- Volunteers
- 71 people
- Sponsor
- Novo Nordisk A/S
Acute Myeloid Leukemia, Myelodysplastic Syndrome
Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
This randomized phase II/III trial studies how well azacitidine with or without nivolumab or midostaurin, or decitabine and cytarabine alone work in treating older patients with newly diagnosed acute myeloid leukemia or high-risk…
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndrome, Myelodysplastic Syndrome/Acute Myeloid Leukemia
- Trial name
- NCT03092674
- Testing
- Azacitidine, Cytarabine, Decitabine
- Where
- 506 sites in United States
- Volunteers
- 76 people
- Sponsor
- National Cancer Institute (NCI)
Common Variable Immunodeficiency, Agammaglobulinemia
Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
The objective of this study is to assess the efficacy and safety of Vivaglobin in previously untreated patients (PUPs) with primary immunodeficiency (PID) over a 25-week observation period.
- Trial name
- NCT00520494
- Testing
- Vivaglobin
- Where
- 6 sites in Canada, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- CSL Behring
Hemophilia A
Survey of Inhibitors in Plasma-Product Exposed Toddlers
The primary objective of the study is to assess the immunogenicity of VWF/FVIII and of rFVIII concentrates by determining the frequency of inhibitor development in previously untreated patients (PUPs) or minimally blood…
- Trial name
- SIPPET (NCT01064284)
- Testing
- PLASMA DERIVED Factor VIII, Recombinant FVIII
- Where
- 48 sites in United States, Argentina and 12 more
- Volunteers
- 303 people
- Sponsor
- Fondazione Angelo Bianchi Bonomi