Clinical trials: Lymphatic Abnormalities
Sickle Cell Anemia, Sickle ß0-Thalassemia
A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for Sickle Cell Anemia or Sickle ß0 Thalassemia as evaluated by the number of occurrences of sickle cell crises.
- Trial name
- NCT01179217
- Testing
- L-glutamine
- Where
- 31 sites in United States
- Volunteers
- 230 people
- Sponsor
- Emmaus Medical, Inc.
Hemophilia A
Study Evaluating The Safety Of Xyntha In Usual Care Settings
The purpose of this study is to collect safety information associated with the use of Xyntha in a usual care setting. Upon meeting eligibility criteria, patients will be required to have approximately 5 study visits over a 2 year…
- Trial name
- NCT00765726
- Testing
- Moroctocog alfa(AF-CC), Blood draw for laboratory testing
- Where
- 6 sites in United States, New Zealand
- Volunteers
- 12 people
- Sponsor
- Pfizer
Hemophilia B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals
Hemophilia A
PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A
An open-label, single-arm, post- authorization pragmatic clinical trial on the safety and efficacy of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in subjects with hemophilia A in usual care settings in China for…
- Trial name
- NCT02492984
- Testing
- Intravenous infusions of Xyntha
- Where
- 15 sites in China
- Volunteers
- 85 people
- Sponsor
- Pfizer
Hemophilia A
Combination Therapy of Low Doses of rFVIIa and FEIBA for Severe Hemophilia A Patients With an Inhibitor to Factor VIII
Patients with severe hemophilia and inhibitors can be treated effectively by Activated Prothrombin Complex Concentrates (APCC, eg. FEIBA) or High dose recombinant factor VIIa (rFVIIa).
- Trial name
- NCT00284193
- Testing
- rFVIIa-FEIBA therapy for hemophilia A inhibitors, FEIBA- Activated Prothrombin Complexes
- Volunteers
- 5 people
- Sponsor
- Sheba Medical Center
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT01419704
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Sponsor
- Talaris Therapeutics Inc.
Hypoxic-Ischemic Encephalopathy, Sepsis
Continous Renal Replacement Therapy With the CARPEDIEM® in a French National Cohort of 25 Neonates and Small Infants
Historically, CKRT and hemodialysis were performed in small infants and newborns with devices developed for adults with high rates of complications and mortality.
- Conditions
- Hypoxic-Ischemic Encephalopathy, Sepsis, Kidney Diseases, Metabolic Disease
- Trial name
- NCT05495607
- Testing
- CARPEDIEN
- Where
- France
- Volunteers
- 25 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia
In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)
The investigators aims to evaluate the safety of in utero hematopoietic stem cell transplantation in fetuses with alpha-thalassemia major performed at the time of in utero transfusion of red blood cells.
- Conditions
- Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia, Hemoglobinopathies, Fetal Anemia
- Trial name
- NCT02986698
- Testing
- in utero hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of California, San Francisco
Leukemia
Combination Chemotherapy With or Without Gemtuzumab in Treating Young Patients With Newly Diagnosed Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00372593
- Testing
- asparaginase, cytarabine, daunorubicin hydrochloride
- Where
- 198 sites in United States, Australia and 4 more
- Volunteers
- 1,070 people
- Sponsor
- Children's Oncology Group
Sickle Cell Disease, Vaso-occlusive Crisis
Effect of Virtual Reality Technology for Pain Management of Vaso-Occlusive Crisis in Patients With Sickle Cell Disease
Acute vaso-occlusive crisis (VOC) is the most common complication in patients with sickle cell disease (SCD) and pain related to VOC is often inadequately treated.
- Trial name
- NCT03353584
- Testing
- Virtual Reality Therapy
- Where
- 2 sites in United States
- Volunteers
- 76 people
- Sponsor
- St. Jude Children's Research Hospital
Kidney Cancer, Leukemia
Voriconazole in Preventing Fungal Infections in Children With Neutropenia After Chemotherapy
RATIONALE: Voriconazole may be effective in preventing systemic fungal infections following chemotherapy. PURPOSE: Phase II trial to study the effectiveness of voriconazole in preventing systemic fungal infections in children who…
- Conditions
- Kidney Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00066599
- Testing
- voriconazole
- Where
- 7 sites in United States
- Sponsor
- National Cancer Institute (NCI)
Hemorrhagic Stroke, Intracerebral, Cerebral Cavernous Malformation
CCM Blood Biomarker Validation Study
Cerebral cavernous malformations (CCMs), one of the most common microvascular malformations in the capillary beds of the brain, are susceptible to hemorrhagic stroke.
- Trial name
- NCT05148663
- Testing
- CCM Hemorrhagic Panel
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Texas Tech University Health Sciences Center, El Paso
Hemophilia A
Study Evaluating ReFacto AF in Severe Hemophilia A
To characterize the safety and efficacy of ReFacto AF in treating acute bleeding episodes during prophylaxis treatment, including neoantigenicity.
- Trial name
- NCT00037544
- Testing
- ReFacto AF
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia A
Covalent Tolerance Induction to Factor VIII-Prediction of Inhibitors in Hemophilia
To correlate the Human Leukocyte Antigen type and genetic defect with hemophilia A.
- Trial name
- NCT00178607
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- The University of Texas Health Science Center, Houston
Sickle Cell Anemia
State Of The Art Functional Imaging In Sickle Cell Disease
Sickle cell anemia (SCA) is a serious blood disease with blood vessel changes leading to brain injury and stroke. Studies show about 11% of patients with SCA will develop obvious stroke before age 20 years, with children less…
- Trial name
- NCT01137721
- Where
- United States
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Drepanocytosis
Study of a Deformability Parameter of Red Blood Cell
Sickle-cell disease is one of the most common severe monogenic disorders in the world, it results in the synthesis of abnormal hemoglobin (HbS) instead of hemoglobin A.
- Trial name
- FITRED (NCT05850156)
- Testing
- basal blood collection, blood collection M6, blood collection M12
- Volunteers
- 130 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Neuropathic Pain, Sickle Cell Disease
Sickle Cell Pain: Intervention With Capsaicin Exposure
This study evaluates the safety and feasibility of using high dose topical capsaicin patches for the treatment of neuropathic pain in pediatric patients with sickle cell disease, as well as the feasibility of using a number of…
- Trial name
- SPICE (NCT03899246)
- Testing
- Eight Percent Topical Capsaicin
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital of Michigan
Metabolism, Inborn Errors, Granulomatous Disease, Chronic
Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders
OBJECTIVES: I. Determine the safety of total body irradiation and fludarabine followed by allogeneic peripheral blood stem cell or bone marrow transplantation in combination with cyclosporine and mycophenolate mofetil for…
- Trial name
- NCT00010361
- Testing
- cyclosporine, fludarabine, mycophenolate mofetil
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Fred Hutchinson Cancer Center
Ehlers-Danlos Syndrome, Vascular Type
SEDVasc (RaDiCo Cohort) (RaDiCo-SEDVasc)
The goal of this observational study is to describe the natural course of vascular Ehlers-Danlos syndrome, in particular the order of appearance of different types of complications (arterial, digestive, pulmonary and uterine).
- Trial name
- NCT05976841
- Where
- 17 sites in France
- Volunteers
- 340 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Hemophilia A
Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving FVIII Prophylaxis
Patients with severe hemophilia A often develop joint complication requiring careful monitoring. This study aimed to compare the effectiveness of standard low dose weight-based FVIII concentrates (CFCs) prophylaxis with…
- Trial name
- HEADUS_SHA8 (NCT06816953)
- Testing
- HEAD-US, Urinary carboxy-terminal cross linked telopeptide of type II collagen, Inflammatory markers
- Where
- Thailand
- Volunteers
- 19 people
- Sponsor
- Chulalongkorn University
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