Clinical trials: Lymphatic Malformation
Iron, Abnormal Blood Level, Other Abnormal Blood Chemistry
Fresh Versus Old Red Blood Cells for Transfusion
Packed red blood cell units destined for transfusion can be stored for up to 42 days prior to transfusion based on Food and Drug Administration (FDA) guidelines.
- Trial name
- NCT01319552
- Testing
- Fresh transfusion, Old transfusion
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Columbia University
Factor X Deficiency
Safety & Efficacy of BPL's High Purity FACTOR X in Treatment of Factor X Deficient Subjects Undergoing Surgery
To primary efficacy variable is to assess the presence or absence of excessive blood loss during and after surgery. The secondary efficacy endpoints are as follows: 1.
- Trial name
- Ten03 (NCT01086852)
- Testing
- FACTOR X
- Where
- 7 sites in United States, Spain and 2 more
- Volunteers
- 4 people
- Sponsor
- Bio Products Laboratory
Sickle Cell Disease (SCD)
Pharmacokinetics and Pharmacodynamics Study of SEG101 (Crizanlizumab) in Sickle Cell Disease (SCD) Patients With Vaso- Occlusive Crisis (VOC)
The purpose of the CSEG101A2202 study was to characterize the Pharmacokinetic (PK) and Pharmacodynamic (PD) of SEG101/crizanlizumab and to evaluate the safety and efficacy of SEG101/crizanlizumab in sickle cell disease (SCD)…
- Trial name
- NCT03264989
- Testing
- crizanlizumab
- Where
- 12 sites in United States
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Metformin+Cytarabine for the Treatment of Relapsed/Refractory AML
The purpose of the study is to determine if metformin in combination with cytarabine is safe and effective. Participants in this research study have acute myeloid leukemia (AML) that has come back after initial treatment or has…
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT01849276
- Testing
- metformin hydrochloride, cytarabine, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Northwestern University
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes.
- Trial name
- NCT03417245
- Testing
- fitusiran, factor concentrates
- Where
- 64 sites in United States, Australia and 17 more
- Volunteers
- 120 people
- Sponsor
- Genzyme, a Sanofi Company
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
Thalassemia Major With Severe Transfusional Iron Overload
Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia
We hypothesize that the combination treatment with deferasirox and deferiprone will be well tolerated and will result in significant improvement in cardiac and liver iron levels.
- Trial name
- NCT01709032
- Testing
- Deferasirox and deferiprone
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- Children's Hospital of Philadelphia
Hemophilia A, Severe Hemophilia A
An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
This multicenter, open-label, phase 3 extension study will investigate the safety and efficacy of rVIII-SingleChain for prophylaxis and on-demand treatment of bleeding episodes in at least 200 previously treated patients (PTPs)…
- Trial name
- NCT02172950
- Testing
- rVIII-SingleChain
- Where
- 64 sites in United States, Australia and 23 more
- Volunteers
- 246 people
- Sponsor
- CSL Behring
Sickle Cell Disease (SCD)
Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients
The purpose of this study was to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 2 to <18 years with a history of Vaso-Occlusive Crisis (VOC) with or without…
- Trial name
- NCT03474965
- Testing
- Crizanlizumab
- Where
- 35 sites in United States, Belgium and 12 more
- Volunteers
- 117 people
- Sponsor
- Novartis Pharmaceuticals
Meningococcal Vaccine
Trial to Describe the Safety, Tolerability, and Immunogenicity of Trumenba When Administered to Immunocompromised Participants ≥10 Years of Age
The aim of this study is to evaluate the safety, tolerability, and immunogenicity of 2 doses of Trumenba® (on a 0- and 6-month schedule) in immunocompromised participants by functionally assessing antibody production in asplenic…
- Trial name
- NCT04893811
- Testing
- Trumenba
- Where
- 14 sites in Czechia, Poland and 1 more
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Pfizer
Lymphoma
A Study of INCB050465 in Relapsed or Refractory Follicular Lymphoma
The purpose of this study is to assess the objective response rate of parsaclisib treatment in participants with relapsed or refractory follicular lymphoma.
- Trial name
- CITADEL-203 (NCT03126019)
- Testing
- Parsaclisib
- Where
- 83 sites in United States, Australia and 11 more
- Volunteers
- 126 people
- Sponsor
- Incyte Corporation
Hemophilia A
Study of PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Male Chinese Subjects With Hemophilia A
An open-label, single dose pharmacokinetic study of Xyntha (Moroctocog Alfa (AF-CC), Recombinant Factor VIII) in male Chinese subjects with hemophilia A
- Trial name
- NCT02461992
- Testing
- Intravenous infusions of Xyntha
- Where
- 2 sites in China
- Volunteers
- 13 people
- Sponsor
- Pfizer
Fibrinogen Deficiency
Human Fibrinogen - Pharmacokinetics
This study evaluated the single-dose pharmacokinetics of human fibrinogen concentrate and clot strength (maximum clot firmness [MCF]) in subjects with congenital fibrinogen deficiency.
- Trial name
- NCT00496262
- Testing
- Human Fibrinogen Concentrate
- Where
- 15 sites in United States, Italy
- Volunteers
- 15 people
- Sponsor
- CSL Behring
DiGeorge Anomaly, Complete DiGeorge Anomaly
Phase I/II Thymus Transplantation With Immunosuppression #950
The study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and…
- Conditions
- DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- #950 (NCT00579527)
- Testing
- Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Neutropenia, Sickle Cell Disease
Decitabine for High-Risk Sickle Cell Disease
Background: In sickle cell disease (SCD), the proteins in the red blood cells that carry oxygen do not behave normally. In parts of the body where there are low levels of oxygen or where oxygen is used more, the sickle hemoglobin…
- Trial name
- NCT01375608
- Testing
- Decitabine
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency
Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases
The primary objective is to demonstrate the bioequivalence of Gammaplex® 10 intravenous immunoglobulin (IGIV) and Gammaplex® 5% IGIV with respect to area under the curve within a 28-day dosing interval (AUC0-28) in a cohort of…
- Conditions
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinaemia, Hyper-IgM Syndrome
- Trial name
- GMX07 (NCT01963143)
- Testing
- Gammaplex (5%), Gammaplex 10
- Where
- 17 sites in United States, Hungary and 1 more
- Volunteers
- 48 people
- Sponsor
- Bio Products Laboratory
Sickle Cell Disease, Iron Overload
Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions.
- Conditions
- Sickle Cell Disease, Iron Overload, Hemolytic Anemia
- Trial name
- NCT00110617
- Testing
- Deferasirox (ICL670), Deferoxamine (DFO)
- Where
- 59 sites in United States, Canada
- Volunteers
- 212 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Anemia, Acute Chest Syndrome
Bi-Level Positive Airway Ventilation for Acute Chest Syndrome
Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by having findings on a chest x-ray and one of the following: chest pain, fever, or trouble breathing.
- Trial name
- NCT01589926
- Testing
- Bi-level positive airway pressure device
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Albert Einstein College of Medicine
Priapism, Anemia, Sickle Cell
Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence
Priapism, a prolonged erection of the penis, is a medical issue that often affects men with sickle cell disease. The purpose of this study is to collect demographic and clinical information on priapism by interviewing men with…
- Trial name
- NCT00300235
- Where
- 27 sites in United States
- Volunteers
- 1,464 people
- Sponsor
- University of Texas Southwestern Medical Center
Hemophilia A
Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment
To evaluate the effect of secondary prophylaxis as compared to episodic treatment on bleeding frequency (number of bleeds per year) and on joint damage.
- Trial name
- SPINART (NCT00623480)
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 38 sites in United States, Argentina and 2 more
- Volunteers
- 84 people
- Sponsor
- Bayer