Clinical trials: Lymphatic Malformation
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 Disease
Study of Propranolol as Anti-Adhesive Therapy in Sickle Cell Disease (SCD)
An open label, prospective, randomized cross-over phase II study in up to 60 sickle cell patients who are either homozygous for Hb S or have HbSB0 thalassemia.
- Trial name
- NCT01077921
- Testing
- Propranolol
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Laura M. De Castro, MD
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
Sickle Cell Disease, Sickle Cell Anemia
Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. It results from a single genetic change (mutation) in red blood cells (RBCs). RBCs are the cells that carry oxygen to the body.
- Trial name
- NCT04476277
- Testing
- Biotin label
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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)
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
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission
Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
RATIONALE: Giving chemotherapy before a donor umbilical cord blood transplant (UCBT) helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
- Trial name
- NCT01093586
- Testing
- double-unit umbilical cord blood transplantation, cytogenetic analysis, bone marrow aspiration
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Case Comprehensive Cancer Center
Severe Hemophilia A
Hemophilia Adult Prophylaxis Study: Factor VIII in Severe Hemophilia A
The purpose of this pilot R34 trial is to determine the feasibility of a large single dose Phase III study of hemophilia adult prophylaxis comparing once weekly with thrice-weekly recombinant factor VIII.
- Trial name
- NCT01405742
- Testing
- rF.VIII
- Where
- 6 sites in United States
- Volunteers
- 4 people
- Sponsor
- University of Pittsburgh
Hemophilia A
Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha® In Subjects With Hemophilia A
This study is to describe the safety and efficacy of Xyntha® during the usual care setting.
- Trial name
- NCT01790828
- Testing
- Xyntha : coagulation factor IIIV (recombinant)
- Where
- 4 sites in South Korea
- Volunteers
- 42 people
- Sponsor
- Pfizer
Chronic Iron Overload
Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation
Extend evaluation of deferasirox film-coated tablet (FCT) formulation
- Trial name
- NCT02720536
- Testing
- Deferasirox
- Where
- 14 sites in Austria, Greece and 1 more
- Volunteers
- 53 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials
- Trial name
- 034OLE (NCT03573882)
- Testing
- Voxelotor
- Where
- 60 sites in United States, Canada and 9 more
- Volunteers
- 179 people
- Sponsor
- Pfizer
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Hemophilia
Weight-based Dosing in Hemophilia A
Hemophilia A is an inherited (genetic) disease where a protein, factor VIII (FVIII), which promotes blood clotting is missing or does not work properly. Individuals with hemophilia A are at risk for bleeding.
- Trial name
- NCT02586012
- Testing
- rFVIII
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Craig Seaman
Chronic Myeloproliferative Disorders, Leukemia
Reduced-Intensity Busulfan and Fludarabine With or Without Antithymocyte Globulin Followed by Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease
RATIONALE: Giving low doses of chemotherapy, such as busulfan and fludarabine, before a donor stem cell transplant helps stop the growth of cancer and abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00448201
- Testing
- anti-thymocyte globulin, sargramostim, therapeutic allogeneic lymphocytes
- Where
- United States
- Volunteers
- 71 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Beta-thalassemia, Iron Overload
Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.
- Trial name
- NCT00447694
- Testing
- Deferasirox
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Novartis
Hemophilia B
Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients
The purpose of this pivotal Phase 1/3 study is to determine the pharmacokinetic (PK) parameters, the hemostatic efficacy, and the safety of BAX 326, a recombinant factor IX, in previously treated patients (PTPs) with severe and…
- Trial name
- NCT01174446
- Testing
- BAX 326, BeneFIX
- Where
- 25 sites in Argentina, Brazil and 12 more
- Volunteers
- 86 people
- Sponsor
- Baxalta now part of Shire
Lymphoma, Childhood Lymphoma
Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)
Toxicities related to pediatric cancer treatment can lead to significant illness, organ damage, treatment delays, increased health care cost, and decrease in quality of life.
- Conditions
- Lymphoma, Childhood Lymphoma, Solid Tumor, Childhood Solid Tumor
- Trial name
- UVA-Gen001 (NCT02624388)
- Testing
- Genistein
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Virginia
Iron Overload, Excessive Body Iron Burden
Massive Iron Deposit Assessment
Iron overload is a severe complication of multiple blood transfusions. As the body has no physiologic mechanism for clearing iron, repeated transfusions cause iron accumulation in organs and lead to iron toxicity.
- Trial name
- NCT01572922
- Testing
- R2*-UTE, R2*-GRE, Liver biopsy
- Where
- United States
- Volunteers
- 142 people
- Sponsor
- St. Jude Children's Research Hospital
Niemann-Pick Disease, Type C
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).
- Trial name
- NCT03759639
- Testing
- IB1001
- Where
- 9 sites in United States, Germany and 3 more
- Volunteers
- 33 people
- Sponsor
- IntraBio Inc
Sickle Cell Disease, Thalassemia Major
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD)…
- Trial name
- NCT04523376
- Testing
- CliniMACS
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Timothy Olson