Clinical trials: Lymphatic Malformation
Haemophilia A
Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial
This trial is conducted in Asia, Europe and the United States of America (USA). The aim of the trial is to evaluate safety of immune tolerance induction (ITI) treatment with turoctocog alfa (a recombinant factor VIII) in patients…
- Trial name
- NCT03588741
- Testing
- Turoctocog alfa
- Where
- 14 sites in Austria, Bulgaria and 5 more
- Volunteers
- 1 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
A Study of BAX 888 in Male Adults With Severe Hemophilia A
The main aim of this study is to check if there are side effects from BAX 888 and to determine the dose of BAX 888 for treating severe hemophilia A in male adults.
- Trial name
- NCT03370172
- Testing
- BAX 888
- Where
- 26 sites in United States, Austria and 4 more
- Volunteers
- 4 people
- Sponsor
- Baxalta now part of Shire
Leukemia, Lymphoma
AlloHCT From Matched Unrelated Donors in Pts w/ Advanced Hematologic Malignancies & Disorders
RATIONALE: Giving chemotherapy with or without total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT00547196
- Testing
- filgrastim, Busulfan, Cyclophosphamide
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- City of Hope Medical Center
Niemann-Pick Type C Disease
Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
This study was amended from expanded access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks.
- Trial name
- NCT03643562
- Testing
- Adrabetadex
- Where
- 17 sites in United States
- Volunteers
- 27 people
- Sponsor
- Mandos LLC
Kaposiform Hemangioendothelioma (KHE), Kasabach-Merritt Syndrome
A Study to Compare Vincristine to Sirolimus for Treatment of High Risk Vascular Tumors
In this research study we want to learn more about which treatment works better for patients diagnosed with a vascular tumor called Kaposiform Hemangioendothelioma (KHE) or other high risk vascular tumors such as Tufted Angioma…
- Conditions
- Kaposiform Hemangioendothelioma (KHE), Kasabach-Merritt Syndrome, Tufted Angioma
- Trial name
- NCT02110069
- Testing
- Vincristine, Sirolimus
- Where
- 7 sites in United States
- Volunteers
- 4 people
- Sponsor
- Boston Children's Hospital
IFN-Gamma Therapy, CGD Gene Mutation
Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease
Background: Chronic granulomatous disease (CGD) is an immunodeficiency disease in which white blood cells are unable to kill certain bacteria and fungi.
- Conditions
- IFN-Gamma Therapy, CGD Gene Mutation, CGD Response to IFNg, CGD - Chronic Granulomatous Disease
- Trial name
- NCT01147042
- Testing
- IFN-gamma
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Sickle Cell Disease
Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease
The purpose of this study is to investigate the effect of VIT-2763 on markers of hemolysis (breakdown in red blood cells) in sickle cell disease (SCD).
- Trial name
- ViSionSerenity (NCT04817670)
- Testing
- VIT-2763 120 mg, VIT-2763 360 mg, VIT-2763 240 mg
- Where
- 22 sites in United States, France and 3 more
- Volunteers
- 25 people
- Sponsor
- Vifor (International) Inc.
Sickle Cell Disease, Anti-D Antibodies
RHD Genotype Matched Red Cells for Anti-D
This is a pilot study to evaluate the feasibility and safety of providing RH genotype matched D+ Red Blood Cells (RBCs) to chronically transfused patients with sickle cell disease (SCD) who type D+ but have formed anti-D and are…
- Trial name
- NCT04156906
- Testing
- D+ RH genotype matched red cell units for transfusion
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital of Philadelphia
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Severe Hemophilia A
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at study enrollment from birth to ≤12 months of age with severe hemophilia A…
- Trial name
- HAVEN 7 (NCT04431726)
- Testing
- Emicizumab
- Where
- 32 sites in United States, Australia and 12 more
- Volunteers
- 55 people
- Sponsor
- Hoffmann-La Roche
Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Cyclophosphamide and Busulfan Followed by Donor Stem Cell Transplant in Treating Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome
This trial is studying the side effects and how well giving cyclophosphamide and busulfan followed by donor stem cell transplant works in treating patients with myelofibrosis, acute myeloid leukemia, or myelodysplastic syndrome.
- Conditions
- Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT00445744
- Testing
- cyclophosphamide, busulfan, tacrolimus
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Fred Hutchinson Cancer Center
Renal Angiomyolipoma
Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.
The purpose of this study was to assess the safety and efficacy of everolimus (Afinitor®) in Chinese patients with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC).
- Trial name
- NCT03525834
- Testing
- Everolimus
- Where
- 5 sites in China
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Lymphatic Malformations
OK432 (Picibanil) in the Treatment of Lymphatic Malformations
Standard of care for Lymphatic Malformations has been surgical excision. We have been using OK432/Picibanil (generously supplied by Chugai Pharmaceuticals in Japan) since 1992 with great success for macrocystic disease.
- Trial name
- NCT03427619
- Testing
- OK432
- Where
- 14 sites in United States
- Volunteers
- 275 people, healthy volunteers welcome
- Sponsor
- Richard JH Smith
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
- Trial name
- HOPE-KIDS (NCT02850406)
- Testing
- Voxelotor
- Where
- 20 sites in United States, Lebanon and 1 more
- Volunteers
- 147 people
- Sponsor
- Pfizer
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Ondansetron in Preventing Nausea and Vomiting in Patients Undergoing Stem Cell Transplant
RATIONALE: Ondansetron may help lessen or prevent nausea and vomiting in patients undergoing stem cell transplant. PURPOSE: This phase II trial is studying how well ondansetron works in preventing nausea and vomiting in patients…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT00795769
- Testing
- ondansetron, survey administration, management of therapy complications
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- Fred Hutchinson Cancer Center
Immunodeficiency Syndrome, Severe Aplastic Anemia
Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders
This phase I/II trial studies the side effects of fludarabine phosphate, cyclophosphamide and total-body irradiation followed by donor bone marrow transplant and cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus…
- Conditions
- Immunodeficiency Syndrome, Severe Aplastic Anemia, Genetic Disorder
- Trial name
- NCT00358657
- Testing
- Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Fludarabine Phosphate
- Where
- 3 sites in United States
- Volunteers
- 14 people
- Sponsor
- Fred Hutchinson Cancer Center
Atopic Dermatitis
Study To Evaluate Pf-04965842 In Subjects With Moderate To Severe Atopic Dermatitis
Study B7451006 is a Phase 2b POC study which is planned to assess four PF 04965842 once daily (QD) doses (10, 30, 100, 200 mg) relative to placebo over 12 weeks to characterize the efficacy and safety of PF 04965842 in subjects…
- Trial name
- NCT02780167
- Testing
- PF-04965842
- Where
- 64 sites in United States, Australia and 3 more
- Volunteers
- 269 people
- Sponsor
- Pfizer
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
T-cell Depleted Alternative Donor Transplantation
The primary purpose is to determine the ability of CD34+ selection and T cell depletion using the CliniMACS® device to prevent severe acute graft-versus-host disease (GVHD) in patients receiving a stem cell transplant from an…
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT00968864
- Testing
- CliniMACS® (T cell depletion)
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Wake Forest University Health Sciences
Anemia, Sickle Cell
An Assessment of Prasugrel on Healthy Adults and Sickle Cell Adults
The purpose of this study is to measure the exposure to prasugrel's active metabolite and the pharmacodynamic effects of prasugrel treatment in people with Sickle Cell Disease (SCD).
- Trial name
- NCT01178099
- Testing
- Prasugrel
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Eli Lilly and Company
Acute Undifferentiated Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Bioelectrical Impedance Measurement for Predicting Treatment Outcome in Patients With Newly Diagnosed Acute Leukemia
This clinical trial studies bioelectrical impedance measurement for predicting treatment outcome in patients with newly diagnosed acute leukemia.
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT01869777
- Testing
- bioelectric impedance analysis
- Where
- United States
- Volunteers
- 102 people
- Sponsor
- Wake Forest University Health Sciences