Clinical trials: Lymphatic Malformation
Sickle Cell Disease, Sickle Cell Anemia
Decision Aid for Therapeutic Options In Sickle Cell Disease
Sickle cell disease (SCD) is an inherited disorder with chronic multi-system manifestations affecting 100,000 individuals in the US, largely of minority origin and associated with substantial morbidity, premature mortality…
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SS, Hemoglobin SC
- Trial name
- NCT02326597
- Testing
- Decision Aid Tool, Standard Practice
- Where
- United States
- Volunteers
- 134 people, healthy volunteers welcome
- Sponsor
- Emory University
Sickle Cell Anemia, Thalassemia
L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
The purpose of this research is to evaluate the effects of L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia. as evaluated by the number of occurrences of sickle cell crises.
- Trial name
- NCT00125788
- Testing
- L-glutamine
- Where
- 5 sites in United States
- Volunteers
- 70 people
- Sponsor
- Emmaus Medical, Inc.
Non-Neoplastic Hematologic and Lymphocytic Disorder
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases.
- Trial name
- NCT00919503
- Testing
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine
- Where
- 6 sites in United States
- Volunteers
- 98 people
- Sponsor
- Fred Hutchinson Cancer Center
Anemia, Sickle Cell
Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)
Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA.
- Trial name
- NCT00890396
- Testing
- Hydroxyurea
- Where
- 14 sites in United States
- Volunteers
- 163 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hemophilia A
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A
This multicenter, open-label, non-randomized study will assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab administered at a dose of 6 milligrams per kilogram (mg/kg) every 4 weeks in participants…
- Trial name
- HAVEN 4 (NCT03020160)
- Testing
- Emicizumab
- Where
- 17 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- Hoffmann-La Roche
Von Willebrand Disease
Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor (rVWF) in the Treatment of Bleeding Episodes in Von Willebrand Disease (VWD)
The purpose of this Phase 3 study is to assess the pharmacokinetics of rVWF:rFVIII and rVWF, and to assess the safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding events in subjects with severe hereditary von…
- Trial name
- NCT01410227
- Testing
- Recombinant von Willebrand factor (rVWF), Recombinant factor VIIII (rFVIII)
- Where
- 52 sites in United States, Australia and 14 more
- Volunteers
- 49 people
- Sponsor
- Baxalta now part of Shire
Advanced Malignant Neoplasm, Castleman Disease
Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease
This phase I trial studies the side effects and best dose of bevacizumab and temsirolimus alone or in combination with valproic acid or cetuximab in treating patients with a malignancy that has spread to other places in the body…
- Conditions
- Advanced Malignant Neoplasm, Castleman Disease, Digestive System Carcinoma, Erdheim-Chester Disease
- Trial name
- NCT01552434
- Testing
- Bevacizumab, Cetuximab, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 116 people
- Sponsor
- M.D. Anderson Cancer Center
Chronic Granulomatous Disease
Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease
This study is for patients with chronic granulomatous disease (CGD), which is a disorder of the immune system that puts them at risk for severe infections.
- Trial name
- MUNCHR (NCT00578643)
- Testing
- Busulfan, Alemtuzumab, Cyclophosphamide
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Baylor College of Medicine
Sickle Cell Anemia
Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies
This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or…
- Trial name
- NCT03020615
- Testing
- Hydroxyurea
- Where
- 4 sites in United States
- Volunteers
- 58 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease
The purpose of this study is to determine whether ticagrelor is effective in reducing the number of days of pain, intensity of pain, and reducing the use of analgesics due to sickle cell disease
- Trial name
- Hestia2 (NCT02482298)
- Testing
- Ticagrelor
- Where
- 20 sites in United States, Egypt and 6 more
- Volunteers
- 87 people
- Sponsor
- AstraZeneca
Haemophilia A
BAY81-8973 Pediatric Safety and Efficacy Trial
The primary objective was to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.
- Trial name
- NCT01311648
- Testing
- Recombinant Factor VIII (Kovaltry, BAY81-8973)
- Where
- 43 sites in United States, Argentina and 15 more
- Volunteers
- 94 people
- Sponsor
- Bayer
Hemophilia A
Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age
This study will be investigating the safety and efficacy of Xyntha (moroctocog alfa (AF-CC)) in male patients less than 6 years old.
- Trial name
- NCT00759655
- Testing
- Moroctocog alfa
- Volunteers
- 1 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia B
BAX 326 (rFIX) Continuation Study
The purpose of this BAX 326 Continuation Study is to further investigate incremental recovery over time, the hemostatic efficacy, the safety, immunogenicity, and health-related quality of life (HR QoL) of BAX 326 in previously…
- Trial name
- NCT01286779
- Testing
- BAX 326 (Recombinant factor IX)
- Where
- 42 sites in Argentina, Brazil and 15 more
- Volunteers
- 117 people
- Sponsor
- Baxalta now part of Shire
Congenital Hemolytic Anemia, Sickle Cell Disease
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
People with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have been cured with bone marrow transplantation (BMT).
- Trial name
- NCT00061568
- Testing
- Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis
- Where
- United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hemophilia A
An Open-label Safety, Efficacy and Pharmacokinetic Study of a Recombinant FVIII Compared to Recombinant Human Antihemophilic FVIII in Patients With Severe Hemophilia A
This is an open-label, non-randomized, efficacy, safety and pharmacokinetic (PK) study comparing octocog alfa and rVIII-SingleChain.
- Trial name
- NCT01486927
- Testing
- rVIII-SingleChain, Octocog alfa
- Where
- 56 sites in United States, Australia and 18 more
- Volunteers
- 175 people
- Sponsor
- CSL Behring
Leukemia, Lymphoma
Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy before a donor stem cell transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Trial name
- NCT00818961
- Testing
- alemtuzumab, graft-versus-tumor induction therapy, rituximab
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Northside Hospital, Inc.
Hemophilia A
Trial of Rituximab Versus Oral Cyclophosphamide to Eradicate or Suppress Autoimmune Anti-Factor VIII Antibodies in Acquired Hemophilia A
The purpose of this study is to evaluate the rate of response when administering rituximab to suppress or eliminate the anti-body in a patient's blood that inhibits the effectiveness of their factor replacement product compared…
- Trial name
- NCT00306670
- Testing
- Rituxan, prednisone
- Volunteers
- 2 people, healthy volunteers welcome
- Sponsor
- Georgetown University
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Megakaryoblastic Leukemia (M7)
MS-275 and GM-CSF in Treating Patients With Myelodysplastic Syndrome and/or Relapsed or Refractory Acute Myeloid Leukemia or Acute Lymphocytic Leukemia
This phase II trial is studying how well giving MS-275 together with GM-CSF works in treating patients with myelodysplastic syndrome and/or relapsed or refractory acute myeloid leukemia.
- Conditions
- Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a)
- Trial name
- NCT00462605
- Testing
- entinostat, sargramostim
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Cancer Institute (NCI)
Acquired Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with acquired hemophilia A.
- Trial name
- NCT01178294
- Testing
- OBI-1
- Where
- 12 sites in United States, Canada and 2 more
- Volunteers
- 29 people
- Sponsor
- Baxalta now part of Shire
Sickle Cell Disease
Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell Disease
The purpose of the study is to develop patient-provider clinical support tools to improve clinical practice, patient self-management, and disease outcomes in sickle cell disease during transition to adult care.
- Trial name
- iTransition (NCT02200510)
- Testing
- Self-management intervention for Adolescents with SCD, Patient Portal Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati