Clinical trials: Lymphatic Malformation
Sickle Cell Disease
Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease
Background: Sickle cell disease (SCD) is a blood disease. The drug hydroxyurea (HU) is approved to prevent pain crises in people with SCD. Researchers want to see how higher doses of HU affect the blood.
- Trial name
- NCT02225132
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Niemann-Pick Disease, Type C1
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
Background: For people who have Niemann-Pick disease, type C1 (NPC1), cholesterol and other fats have trouble moving out of liver and other tissue cells. This makes the cells sick.
- Trial name
- NCT03887533
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Hemophilia A
A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A
The goal of this study is to gather more information on safety and efficacy of Kovaltry for the prevention and treatment of bleeds in Chinese children, adolescents/adults with severe hemophilia A.
- Trial name
- NCT04565236
- Testing
- Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 1, Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 2, Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 3
- Where
- 10 sites in China
- Volunteers
- 45 people
- Sponsor
- Bayer
Severe Haemophilia A
Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
The rationale of this study is to further fine-tune and individualize prophylactic treatment of patients with severe Haemophilia A with the goal of keeping the trough FVIII level above 1% between doses.
- Trial name
- NCT02256917
- Testing
- Human cl rhFVIII
- Where
- 30 sites in United States, Canada and 7 more
- Volunteers
- 58 people
- Sponsor
- Octapharma
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia
Alvocidib, Cytarabine, and Mitoxantrone in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia
This phase II trial is studying the side effects and how well giving alvocidib together with cytarabine and mitoxantrone works in treating patients with newly diagnosed acute myeloid leukemia.
- Conditions
- Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
- Trial name
- NCT00407966
- Testing
- alvocidib, cytarabine, mitoxantrone hydrochloride
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- National Cancer Institute (NCI)
Hematopoietic and Lymphatic System Neoplasm, Recurrent Ependymoma
Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial
This phase II pediatric MATCH treatment trial studies how well selpercatinib works in treating patients with solid tumors that may have spread from where they first started to nearby tissue, lymph nodes, or distant parts of the…
- Conditions
- Hematopoietic and Lymphatic System Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma
- Trial name
- NCT04320888
- Testing
- Computed Tomography, Magnetic Resonance Imaging, Positron Emission Tomography
- Where
- 174 sites in United States, Australia and 1 more
- Volunteers
- 1 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease
Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease
The purpose of this study is to develop a safe and curative stem cell transplant approach to treating sickle cell disease by assessing the safety of haploidentical hematopoietic stem cell transplantation using αβ+ T-cell…
- Trial name
- NCT04207320
- Testing
- αβ+ T-cell depletion with Miltenyi CliniMACS system
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of Chicago
Hemophilia A or B
PF-06741086 Long-term Treatment in Severe Hemophilia
This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study.
- Trial name
- NCT03363321
- Testing
- PF-06741086
- Where
- 8 sites in United States, Brazil and 5 more
- Volunteers
- 20 people
- Sponsor
- Pfizer
Chronic Myeloproliferative Disorders, Leukemia
Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer Who Are Undergoing Donor Stem Cell Transplant
RATIONALE: Giving low doses of chemotherapy, monoclonal antibodies, and radiation therapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00691015
- Testing
- rituximab, busulfan, carmustine
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Thalassemia
Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO
Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells.
- Trial name
- THALLO (NCT00578292)
- Testing
- Busulfan, Fludarabine, Campath 1H
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Baylor College of Medicine
Iron Overload, Sickle Cell Disease
Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias
This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body.
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Trial name
- FIRST (NCT02041299)
- Testing
- Deferiprone, Deferoxamine
- Where
- 33 sites in United States, Brazil and 6 more
- Volunteers
- 230 people
- Sponsor
- ApoPharma
Microcystic Lymphatic Malformation
Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations
This study evaluates the safety and efficacy of PTX-022 (sirolimus) Topical Gel 3.9% w/w in the treatment of Microcystic Lymphatic Malformations. The participant will receive 3 months of PTX-022 treatment by the end of the study.
- Trial name
- NCT05050149
- Testing
- PTX-022
- Where
- 7 sites in United States
- Volunteers
- 12 people
- Sponsor
- Palvella Therapeutics, Inc.
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Lithium Carbonate in Treating Patients With Acute Intestinal Graft-Versus-Host-Disease (GVHD) After Donor Stem Cell Transplant
RATIONALE: Lithium carbonate may be an effective treatment for intestinal graft-versus-host disease caused by a donor stem cell transplant.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- GVHD (NCT00408681)
- Testing
- lithium carbonate, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Fred Hutchinson Cancer Center
Graft Versus Host Disease, Leukemia
Busulfan, Cyclophosphamide, & Antithymocyte Globulin Followed by Stem Cell Transplant in Treating Hematologic Cancer
RATIONALE: Giving chemotherapy before a donor bone marrow transplant or peripheral stem cell transplant helps stop the growth of cancer cells and helps stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00611351
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of Nebraska
Hemophilia A
Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII
The purpose of this research study is to determine the effectiveness, safety, and pharmacokinetics (PK) of moroctocog alfa (AF-CC) in previously treated subjects, who are younger than 6 years of age, with severe or moderately…
- Trial name
- NCT00543439
- Testing
- Moroctocog alfa (AF-CC)
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 66 people
- Sponsor
- Pfizer
Sickle Cell Disease
A Dose-Finding Study of AG-348 in Sickle Cell Disease
Background: Sickle Cell Disease (SCD) is an inherited blood disorder. People with SCD have abnormal hemoglobin in their red blood cells.
- Trial name
- NCT04000165
- Testing
- AG-348
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Sickle Cell Disease
Age of Blood in Sickle Cell Transfusion
The Investigators hypothesize that older red cell units trigger phagocytosis and activation of circulating macrophages with a downstream immunomodulatory cascade and release of excess Non Transferrin Bound Iron(NTBI) that leads…
- Trial name
- NCT03704922
- Testing
- Transfusion
- Where
- 3 sites in United States
- Volunteers
- 26 people
- Sponsor
- University of North Carolina, Chapel Hill
Hereditary Hemorrhagic Telangiectasia (HHT)
Topical Bevacizumab for the Management of Recurrent Epistaxis in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
Terence M. Davidson, MD is conducting a research study to find out more about the topical application of Avastin (proper chemical name is bevacizumab) in the treatment of epistaxis (nose bleeding) in patients with Hereditary…
- Trial name
- NCT01397695
- Testing
- Bevacizumab
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of California, San Diego
Sickle Cell Disease
A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)
Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).
- Trial name
- NCT03401112
- Testing
- IMR-687
- Where
- 13 sites in United States, United Kingdom
- Volunteers
- 100 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Severe Hemophilia A
Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A
The primary objectives of this study are: to evaluate the safety and tolerability of rFVIIIFc administered as a prophylaxis (Arm 1), weekly (Arm 2), on-demand (Arm 3), and surgical treatment regimen; to evaluate the efficacy of…
- Trial name
- NCT01181128
- Testing
- Factor VIII (rFVIIIFc), Advate®
- Where
- 64 sites in United States, Australia and 17 more
- Volunteers
- 165 people
- Sponsor
- Bioverativ Therapeutics Inc.