Clinical trials: Lymphatic Malformation
Hemophilia A
A Study of ADVATE in People With Hemophilia A in India
The main aim of this study is to learn more about side effects of Advate when given as standard treatment to people with hemophilia A who have already been treated.
- Trial name
- NCT04985682
- Testing
- ADVATE
- Where
- 5 sites in India
- Volunteers
- 50 people
- Sponsor
- Baxalta now part of Shire
Factor X Deficiency
A Study Investigating Treatment Factor X in People With Factor X Deficiency
The main objective of the study is to assess the pharmacokinetics of FACTOR X after a single dose of 25IU/kg. The secondary objectives of the study are to assess efficacy and safety of FACTOR X in the treatment of bleeding…
- Trial name
- NCT00930176
- Testing
- Human Coagulation FACTOR X
- Where
- 14 sites in United States, Germany and 3 more
- Volunteers
- 16 people
- Sponsor
- Bio Products Laboratory
Factor XIII Deficiency
A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency
Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding.
- Trial name
- NCT00883090
- Testing
- FXIII Concentrate (Human)
- Where
- 6 sites in United States, Spain
- Volunteers
- 15 people
- Sponsor
- CSL Behring
Wiskott-Aldrich Syndrome (WAS)
Gene Therapy for Wiskott-Aldrich Syndrome
This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.
- Trial name
- TIGET-WAS (NCT01515462)
- Testing
- TLT003
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Fondazione Telethon
Sickle Cell Disease, Thalassemia
Effects of Transfusion of Older Stored Red Cells
The purpose of this study is to determine the effects of transfusion of fresh and stored blood on patients. The investigators hope to test: whether a similar effect (older stored blood is associated with worse outcomes) is seen…
- Trial name
- NCT01534676
- Testing
- Transfusion, Blood, Chelation therapy
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Columbia University
Fanconi Anemia
Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100
The purpose of this research study is to determine whether an experimental drug called AMD3100 used in combination with another medication called G-CSF is safe and can help to increase the amount of blood stem cells (called CD34+…
- Trial name
- NCT00479115
- Testing
- AMD3100, AmCell CliniMACs
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Anemia, Sickle Cell
Daily Vitamin D for Sickle-cell Respiratory Complications
This study aims to answer the question whether daily oral vitamin D supplementation can reduce the risk of respiratory or lung complications in children and adolescents with sickle cell disease.
- Conditions
- Sickle Cell Disease, Anemia, Sickle Cell, Anemia, Hemolytic, Congenital, Respiratory Tract Diseases
- Trial name
- ViDAS-2 (NCT04170348)
- Testing
- Daily oral vitamin D3, 3,333 IU, Monthly oral vitamin D3, 100,000 IU
- Where
- United States
- Volunteers
- 58 people
- Sponsor
- Columbia University
Hemophilia
LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures
The purpose of this study, PerSept 3, is to evaluate LR769 for the prevention of excessive bleeding and achievement of hemostasis in congenital hemophilia A or B patients who have inhibitors to Factor VIII or Factor IX , are aged…
- Trial name
- PerSept3 (NCT02548143)
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 9 sites in United States, Mexico and 4 more
- Volunteers
- 12 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Leukemia, Lymphoma
Donor Lymphocyte Infusion in Treating Patients With Recurrent or Persistent Hematologic Cancer After Donor Stem Cell Transplant
RATIONALE: Giving an infusion of donor lymphocytes may be able to kill cancer cells in patients with hematologic cancer that has come back after a donor stem cell transplant.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Trial name
- NCT00534118
- Testing
- donor lymphocytes
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- Roswell Park Cancer Institute
Hereditary Hemorrhagic Telangiectasia
Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept
Hereditary hemorrhagic telangiectasia (HHT) is a hereditary vascular condition characterized by the development of abnormal connections between arteries and veins throughout the body, called vascular malformations.
- Trial name
- NCT01752049
- Testing
- Topical timolol maleate
- Where
- Canada
- Volunteers
- 5 people
- Sponsor
- Unity Health Toronto
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against…
- Conditions
- Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
- Trial name
- HAVEN 6 (NCT04158648)
- Testing
- Emicizumab
- Where
- 22 sites in United States, Belgium and 8 more
- Volunteers
- 73 people
- Sponsor
- Hoffmann-La Roche
Sickle Cell Disease
A Study of IMR-687 in Subjects With Sickle Cell Disease
A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
- Trial name
- NCT04474314
- Testing
- IMR-687
- Where
- 49 sites in United States, Ghana and 11 more
- Volunteers
- 115 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Tumors With Aberrations in ALK, Anaplastic Large Cell Lymphoma
Ceritinib Rare Indications Study in ALK+ Tumors
This is Proof-of-Concept (POC) study to assess the preliminary antitumor activity and safety and tolerablity using ceritinib (LDK378) in the treatment of life threatening tumors that are characterized by ALK genetic alteration…
- Conditions
- Tumors With Aberrations in ALK, Anaplastic Large Cell Lymphoma, Inflammatory Myofibroblastic Tumor, Glioblastoma
- Trial name
- NCT02465528
- Testing
- Ceritinib (LDK378)
- Where
- 14 sites in Czechia, Denmark and 6 more
- Volunteers
- 22 people
- Sponsor
- Novartis Pharmaceuticals
Factor X Deficiency
A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children <12 Years
The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months. The secondary objectives of the study are: 1.
- Trial name
- NCT01721681
- Testing
- FACTOR X
- Where
- 3 sites in United Kingdom
- Volunteers
- 9 people
- Sponsor
- Bio Products Laboratory
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency
Pharmacokinetics (PK) and Safety of Subgam-VF in Primary Immunodeficiency Diseases
The main objective of the study is to determine the pharmacokinetics profile of Subgam-VF. The secondary objectives are to assess the safety of Subgam-VF and refine the dose adjustment coefficient for Subgam-VF needed for…
- Conditions
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinaemia, Hyperimmunoglobulin M Syndrome
- Trial name
- SCIG03 (NCT01884311)
- Testing
- Subgam
- Where
- 16 sites in United States
- Volunteers
- 38 people
- Sponsor
- Bio Products Laboratory
Pseudoxanthoma Elasticum
Magnesium Supplements In The Treatment Of Pseudoxanthoma Elasticum (PXE)
The purpose of this study is to evaluate the effectiveness of magnesium oxide supplements on the reversal of calcium deposits in the skin, and the yellow bumps and folds of skin in subjects with pseudoxanthoma elasticum (PXE).
- Trial name
- NCT01525875
- Testing
- Magnesium Oxide
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Mark Lebwohl
Fanconi Anemia
Stem Cell Transplantation for Fanconi Anemia
The purpose of this study is to determine whether thymic shielding during total body irradiation can be given and whether it will reduce the risk of infections in Fanconi Anemia patients undergoing alternate donor (not a matched…
- Trial name
- NCT00167206
- Testing
- Hematopoietic Stem Cell Transplant, Thymic Shielding During Radiation, Total Body Irradiation
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Hemophilia B
A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
This study will examine the long-term safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children and adults with severe hemophilia B.
- Trial name
- NCT02053792
- Testing
- rIX-FP
- Where
- 42 sites in United States, Australia and 13 more
- Volunteers
- 97 people
- Sponsor
- CSL Behring
Hemophilia A
A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc - Von Willebrand Factor - XTEN Fusion Protein (rFVIIIFc-VWF-XTEN) (BIVV001) in Previously Treated Adults With Severe Hemophilia A (EXTEN-A)
The primary purpose was to assess the safety and tolerability of a single intravenous (IV) administration of BIVV001 in adult previously treated patients (PTPs) with severe hemophilia A.
- Trial name
- NCT03205163
- Testing
- Advate (Low Dose), Advate (High Dose), BIVV001 (Low Dose)
- Where
- 10 sites in United States, Japan
- Volunteers
- 16 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A
Russian Kogenate Pediatric Study
A prospective study to evaluate the effect of rFVIII-FS in different prophylactic regimens on bleeding events frequency and development of arthropathy in Previously Treated and Minimally treated Hemophilia A pediatric population.
- Trial name
- NCT00632814
- Testing
- rFVIII-FS (Kogenate FS, BAY14-2222) 70 IU/kg, dosing once per week, rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg twice per week (30 IU/kg + 40 IU/kg), rFVIII-FS (Kogenate FS, BAY14-2222) 75 IU/kg, dosing three times per week (3 x 25 IU/kg)
- Where
- 4 sites in Russia
- Volunteers
- 32 people
- Sponsor
- Bayer