Clinical trials: Lymphatic Malformation
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
Hemophilia B, Allergic Reactions
Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B
Retrospective, multicenter study in patients with hemophilia B. This study will be placed at 20 to 50 hemophilia treatment centers in North America and the EU, and is designed to estimate the frequency of Class II and III…
- Trial name
- NCT00195221
- Where
- 38 sites in United States, Austria and 7 more
- Volunteers
- 166 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Beta Thalassemia Major
Effect of Spirulina on Serum Hyaluronic Acid in Beta Thalassemic Children With Hepatitis C
Thalassemics can develop liver fibrosis because of iron overload and hepatitis C infection. The latter is the main risk factor for liver fibrosis in transfusion dependent thalassemics.
- Trial name
- NCT02744547
- Testing
- Spirulina
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Tanta University
Hemoglobinopathies
Prevalence and Hematological Characteristics of Hemoglobinopathies
This study aimed to determine the prevalence and hematological characteristics of hemoglobinopathies in Mardan and surrounding districts of Khyber Pakhtunkhwa (KPK), Pakistan.
- Trial name
- NCT07180836
- Testing
- No Intervention (Observational Study)
- Where
- Pakistan
- Volunteers
- 839 people
- Sponsor
- Bacha Khan Medical College
Congenital Bleeding Disorder, Haemophilia A
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who…
- Trial name
- pathfinder™5 (NCT01731600)
- Testing
- turoctocog alfa pegol
- Where
- 51 sites in United States, Brazil and 14 more
- Volunteers
- 68 people
- Sponsor
- Novo Nordisk A/S
Leukemia, Myelodysplastic Syndromes
Combination Chemotherapy With or Without Filgrastim and/or Tretinoin in Treating Patients With Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Colony-stimulating factors such as filgrastim may increase the number of immune cells found in bone marrow…
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00005863
- Testing
- filgrastim, cytarabine, daunorubicin hydrochloride
- Where
- United Kingdom
- Sponsor
- Medical Research Council
Sickle Cell Disease
Sickle-cell Disease Registry of the GPOH
Sickle cell disease is one of the most common hereditary diseases. Most severe complications can be avoided if the disease is detected early and treated appropriately.
- Trial name
- SichReg (NCT03327428)
- Where
- Germany
- Volunteers
- 1,000 people
- Sponsor
- University Hospital Heidelberg
Venous Malformation, Lymphatic Malformation
Weekly Sirolimus Therapy
In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months.
- Trial name
- NCT04861064
- Testing
- Sirolimus
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Medical University of South Carolina
Beta-Thalassemia
Thrombophilia In Beta Thalassemia
β-thalassemia disease is one of the most common congenital hemolytic anemia commonly found in the malarial belt areas including the Mediterranean, the Middle East, Africa, Southeast Asian countries, and China.
- Trial name
- NCT04219449
- Testing
- PT, Protein C, Platelet aggregation by ADP and arachidonic acid
- Volunteers
- 100 people
- Sponsor
- Assiut University
Neutropenia, Sickle Cell Anemia
Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders
OBJECTIVES: I. Determine the efficacy of bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders. II.
- Conditions
- Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
- Trial name
- NCT00005893
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Fairview University Medical Center
GM2 Gangliosidosis, Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study…
- Trial name
- PRISMA (NCT07399704)
- Testing
- AZ-3102
- Where
- 3 sites in Brazil
- Volunteers
- 21 people
- Sponsor
- Azafaros B.V.
Hemophilia A
An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
This is an observational study of children with mild, moderate, or severe hemophilia A who are receiving damoctocog alfa pegol, and are between 7 to <12 years of age at the time of enrolment.
- Trial name
- HEM-POWR Kids (NCT07088458)
- Testing
- Damoctocog alfa pegol (Jivi, Bay94-9027)
- Where
- 12 sites in United States, Brazil and 9 more
- Volunteers
- 40 people
- Sponsor
- Bayer
Beta-thalassemia, Myelodysplastic Syndromes
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions.
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan
- Trial name
- NCT00061763
- Testing
- Deferasirox
- Where
- 6 sites in United States
- Volunteers
- 175 people
- Sponsor
- Novartis Pharmaceuticals
Constitutional Mismatch Repair Deficiency Syndrome, Hematopoietic and Lymphoid Cell Neoplasm
Testing the Combination of Two Immunotherapy Drugs (Nivolumab and Ipilimumab) in Children, Adolescent, and Young Adult Patients With Relapsed/Refractory Cancers That Have an Increased Number of Genetic Changes, The 3CI Study
This phase Ib trial investigates the side effects of the combination of nivolumab and ipilimumab, and to see how well they work in treating patients with cancers that have come back (relapsed) or does not respond to treatment…
- Conditions
- Constitutional Mismatch Repair Deficiency Syndrome, Hematopoietic and Lymphoid Cell Neoplasm, Lynch Syndrome, Recurrent Lymphoma
- Trial name
- NCT04500548
- Testing
- Biospecimen Collection, Ipilimumab, Nivolumab
- Where
- 9 sites in United States, Canada
- Sponsor
- National Cancer Institute (NCI)
Hemophilia, Arthropathy of Knee
Evaluation of Static Postural Balance in Children With Hemophilia and Its Relationship With Joint Health
Hemophilia is a rare hematological disorder characterized by a partial or complete deficiency of clotting factor VIII or IX.Bleeding disorders mainly affect the musculoskeletal system.
- Conditions
- Hemophilia, Arthropathy of Knee, Arthropathy of Ankle or Foot
- Trial name
- NCT04406519
- Where
- Turkey (Türkiye)
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Necmettin Erbakan University
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Fludarabine, Cyclophosphamide, and Total-Body Irradiation Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients Who Are Undergoing a Donor Umbilical Cord Blood Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00255684
- Testing
- graft-versus-tumor induction therapy, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Rochester
Breast Cancer, Kidney Cancer
Combination Chemotherapy and Total-Body Irradiation Before Donor Umbilical Cord Blood Transplant in Treating Patients With Advanced Hematologic Cancer, Metastatic Breast Cancer, or Kidney Cancer
RATIONALE: Giving low doses of chemotherapy and radiation therapy before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Breast Cancer, Kidney Cancer, Leukemia, Lymphoma
- Trial name
- NCT00365287
- Testing
- anti-thymocyte globulin, cyclophosphamide, cyclosporine
- Volunteers
- 148 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Shwachman Syndrome, Fanconi's Anemia
Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin level in patients with inherited bone marrow failure…
- Conditions
- Shwachman Syndrome, Fanconi's Anemia, Dyskeratosis Congenita, Thrombocytopenia
- Trial name
- NCT00004787
- Testing
- filgrastim
- Volunteers
- 20 people
- Sponsor
- National Center for Research Resources (NCRR)
Wound; Rupture, Surgery, Cesarean Section, Postpartum Hemorrhage
HEMOLEVEN® Expanded Access Program Prevention of Surgical/Postpartum Hemorrhage Severe Inherited Factor XI Deficiency
The objective of the Expanded Access Program is to provide HEMOLEVEN, a replacement coagulation factor XI, to patients with severe inherited factor XI deficiency where, in the opinion of the treating physician, the benefits of…
- Conditions
- Wound; Rupture, Surgery, Cesarean Section, Postpartum Hemorrhage, Surgery
- Trial name
- EAP (NCT01701947)
- Testing
- Hemoleven
- Where
- United States
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Thalassemia Major
The Effect of N-acetylcysteine on Oxidative Stress Status and Iron Overload in Thalassemia Major
The effect of N-acetylcysteine as antioxidant and its effect on pretransfusion hemoglobin and iron overload in patients with thalassemia were compared to patients who didn't receive n-acetylcysteine after 3 months of study…
- Trial name
- NCT04260516
- Testing
- N-acetyl cysteine
- Where
- Egypt
- Volunteers
- 100 people
- Sponsor
- Beni-Suef University
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