Clinical trials: Lymphatic Malformation
Acute Chest Syndrome, Sickle Cell Disease
Feasibility Study of Unfractionated Heparin in Acute Chest Syndrome
The purpose of this study is to determine the feasibility of performing a larger multicenter phase III trial to assess the effects of unfractionated heparin (UFH) in acute chest syndrome (ACS).
- Trial name
- NCT02098993
- Testing
- Unfractionated heparin
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Craig Seaman
Hemophilia A
Pharmacokinetic Study of ADVATE 3000 IU in Previously Treated Patients With Severe Hemophilia A
The objective of this clinical study is to compare the pharmacokinetic parameters of 3000 IU Advate using one 3000 IU potency vial dissolved in 5 mL diluent with that of 3000 IU Advate using two vials of 1500 IU potency dissolved…
- Trial name
- NCT00916032
- Testing
- Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]
- Where
- 4 sites in Bulgaria, Russia
- Volunteers
- 29 people
- Sponsor
- Baxalta now part of Shire
Sickle Cell Anemia, Children, Only
Shared-Decision Making for Hydroxyurea
The goal of the study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that takes into account medical evidence and parent…
- Trial name
- ENGAGE-HU (NCT03442114)
- Testing
- Hydroxyurea SDM Toolkit, Clinician Pocket Guide
- Where
- 11 sites in United States
- Volunteers
- 176 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Myeloproliferative Disorders, Leukemia
Umbilical Cord Blood (UCB) Transplant, Fludarabine, Melphalan, and Anti-thymocyte Globulin (ATG) in Treating Patients With Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy before a donor umbilical cord blood transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00827099
- Testing
- anti-thymocyte globulin, fludarabine phosphate, Melphalan
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Northside Hospital, Inc.
Sickle Cell Crisis
Ketamine for Acute Painful Crisis in Sickle Cell Disease Patients
Investigators hypothesize that administration of ketamine for pain relief in sickle cell patients with vaso-occlusive crisis early on will lead to a more rapid improvement in pain score and less narcotic requirement.
- Trial name
- NCT03431285
- Testing
- Morphine Group, Ketamine Group, standard IV hydration
- Where
- Saudi Arabia
- Volunteers
- 278 people
- Sponsor
- Imam Abdulrahman Bin Faisal University
Hemophilia B
Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B
The primary objective of the study is to evaluate the safety of Recombinant Human Coagulation Factor IX Fc Fusion Protein (rFIXFc) in previously treated pediatric subjects with hemophilia B.
- Trial name
- Kids B-LONG (NCT01440946)
- Testing
- rFIXFc, FIX
- Where
- 15 sites in United States, Australia and 4 more
- Volunteers
- 30 people
- Sponsor
- Bioverativ Therapeutics Inc.
Anemia, Sickle Cell, Sickle Cell Disease
Preventing Sickle Cell Kidney Disease
Untreated hypertension and renal injury are risk factors for increased morbidity and mortality in sickle cell disease, yet early markers of progressive disease have not been identified and therapies to prevent the development of…
- Conditions
- Anemia, Sickle Cell, Sickle Cell Disease, Kidney Disease, Hypertension
- Trial name
- NCT02373241
- Testing
- Losartan
- Where
- United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Dyskeratosis Congenita, Aplastic Anemia
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who…
- Trial name
- NCT02162420
- Testing
- Alemtuzumab, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 61 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
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
Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm
Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH treatment trial studies how well ensartinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with ALK or ROS1 genomic alterations that have come back…
- Conditions
- Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
- Trial name
- NCT03213652
- Testing
- Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan
- Where
- 126 sites in United States, Australia and 2 more
- Volunteers
- 13 people
- Sponsor
- National Cancer Institute (NCI)
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Dyskeratosis Congenita, Aplastic Anemia
Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Transplantation with stem cells is a standard therapy in many centers around the world. Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other cancers, aplastic anemia and other non-malignant…
- Trial name
- NCT00455312
- Testing
- Campath 1H, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sickle Cell Trait
Increasing Documentation and Disclosure of Sickle Cell Trait Status: An Implementation Science Approach
The hemoglobinopathy newborn screen (NBS) performed on all neonates in the U.S. allows for early life-saving medical care for infants with sickle cell disease (SCD), an autosomal recessive genetic disorder.
- Trial name
- NCT05387564
- Testing
- SCT Documentation and Disclosure Toolkit (SCT-DD)
- Where
- United States
- Volunteers
- 114 people
- Sponsor
- Nemours Children's Clinic
Leukemia, Lymphoma
Combination Chemotherapy, Donor Stem Cell Transplant, Tacrolimus, Mycophenolate Mofetil, and Cyclophosphamide in Treating Patients With Hematologic Cancer
RATIONALE: Giving chemotherapy, such as fludarabine, busulfan, and cyclophosphamide, before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells and helps stop the patient's immune system from…
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT00782379
- Testing
- busulfan, cyclophosphamide, fludarabine phosphate
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Northside Hospital, Inc.
Leukemia, Myelodysplastic Syndromes
Donor Stem Cell Transplant in Treating Patients With Myeloid Cancer or Other Disease
RATIONALE: Giving total-body irradiation and chemotherapy, such as fludarabine and thiotepa, before a donor stem cell transplant helps stop the growth of cancer or abnormal cells.
- Trial name
- NCT00392782
- Testing
- anti-thymocyte globulin, fludarabine phosphate, thiotepa
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Leukemia, Lymphoma
Donor Stem Cell Transplant in Treating Patients With Relapsed Hematologic Malignancies or Secondary Myelodysplasia Previously Treated With High-Dose Chemotherapy and Autologous Stem Cell Transplant
RATIONALE: Giving chemotherapy, such as busulfan and fludarabine phosphate, before a peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT01118013
- Testing
- anti-thymocyte globulin, donor lymphocytes, filgrastim
- Where
- 10 sites in United States
- Volunteers
- 6 people
- Sponsor
- Alliance for Clinical Trials in Oncology
Hemophilia B
Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.
- Trial name
- NCT01335061
- Testing
- Nonacog alfa
- Where
- 17 sites in Bulgaria, Canada and 7 more
- Volunteers
- 25 people
- Sponsor
- Pfizer
Leukemia, Myelodysplastic Syndrome
Bortezomib and Vorinostat in Treating Patients With High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia
RATIONALE: Bortezomib and vorinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving bortezomib together with vorinostat may kill more cancer cells.
- Trial name
- NCT00818649
- Testing
- bortezomib, vorinostat
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma
Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well erdafitinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with FGFR mutations that have spread to other places in the body and…
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma
- Trial name
- NCT03210714
- Testing
- Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan
- Where
- 116 sites in United States, Puerto Rico
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)