Clinical trials: Lymphatic Malformation
Severe Hemophilia A
Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A
A prospective, non-controlled, international, multi-centre phase 3 study to investigate the pharmacokinetics, efficacy, safety, and immunogenicity of Wilate in previously treated children with severe haemophilia A
- Trial name
- NCT03376516
- Testing
- Wilate
- Where
- 3 sites in Russia, Ukraine
- Volunteers
- 11 people
- Sponsor
- Octapharma
Sickle Cell Disease (SCD)
Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease
The primary objective of this study is to assess the safety and tolerability of ALXN1820 SC (subcutaneous) in participants with SCD (Sickle Cell Disease).
- Trial name
- PHOENIX (NCT05565092)
- Testing
- ALXN1820
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Thrombosis
Sickle Cell Trait and the Risk of Venous Thromboembolism
The purpose of this trial is to investigate D-Dimer levels, a surrogate marker of venous thromboembolism, in pregnant/postpartum white women as compared to pregnant/postpartum black women, and pregnant/postpartum women with…
- Trial name
- SCT&DVT (NCT01148940)
- Where
- United States
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Sickle Cell Disease
A Study of the Effect of IW-1701 (Olinciguat), a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD)
The primary objective of the 1701-202 STRONG SCD study is to evaluate the safety and tolerability of different dose levels of IW-1701 compared with placebo when administered daily for approximately 12 weeks to patients with…
- Trial name
- STRONG SCD (NCT03285178)
- Testing
- IW-1701
- Where
- 37 sites in United States, Lebanon and 1 more
- Volunteers
- 88 people
- Sponsor
- Cyclerion Therapeutics
Sickle Cell Disease
Transplantation for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow
The purpose of this study is to learn if it is possible and safe to treat persons with severe sickle cell disease (SCD) by bone marrow transplant (BMT) from human leukocyte antigen (HLA) half-matched related donors.
- Trial name
- TRANSFORM (NCT02757885)
- Testing
- Bone Marrow Transplant (BMT), Hydroxyurea, Thiotepa
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Anemia, Sickle Cell
Effectiveness of Arginine as a Treatment for Sickle Cell Anemia
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited genetic disease that can cause intense pain episodes.
- Trial name
- Arginine (NCT00513617)
- Testing
- Arginine
- Where
- 17 sites in United States
- Volunteers
- 128 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.
- Trial name
- paradigm™6 (NCT02141074)
- Testing
- nonacog beta pegol
- Where
- 93 sites in United States, Algeria and 22 more
- Volunteers
- 54 people
- Sponsor
- Novo Nordisk A/S
Severe Haemophilia A
Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A
To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand…
- Trial name
- NCT01863758
- Testing
- Human-cl rhFVIII
- Where
- 20 sites in Austria, Bulgaria and 6 more
- Volunteers
- 66 people
- Sponsor
- Octapharma
Sickle Cell Disease
Sickle Cell Anemia WE CARE
This mixed-methods study aims to understand the implementation of a previously tested, efficacious social determinants of health (SDoH) screening and referral intervention in the outpatient pediatric hematology setting…
- Trial name
- SCAWECARE (NCT03716726)
- Testing
- WE CARE SDoH Screening Survey, Family Resource Book, Standard of care
- Where
- United States
- Volunteers
- 112 people, healthy volunteers welcome
- Sponsor
- Boston Medical Center
Premature Infant Disease, Transfusion Related Complication
Cord Blood Transfusion In Preterm Neonates (CB-TrIP)
Repeated transfusions have been associated with very poor outcome of preterm infants. Fetal hemoglobin (HbF) and adult Hb (HbA) have different affinity for oxygen.
- Conditions
- Premature Infant Disease, Transfusion Related Complication, Fetal Hemoglobin
- Trial name
- CB-TrIP (NCT03764813)
- Where
- 2 sites in Italy
- Volunteers
- 25 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma
Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial
This phase II pediatric MATCH trial studies how well tipifarnib works in treating patients with solid tumors that have recurred or spread to other places in the body (advanced), lymphoma, or histiocytic disorders, that have a…
- Conditions
- Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma, Recurrent Ectomesenchymoma, Recurrent Ependymoma
- Trial name
- NCT04284774
- Testing
- Tipifarnib
- Where
- 172 sites in United States, Puerto Rico
- Volunteers
- 5 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease
Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor bone marrow transplant helps stop the growth of abnormal cells.
- Trial name
- NCT00489281
- Testing
- Cyclophosphamide, Fludarabine, Mycophenolate mofetil
- Where
- United States
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Acute Myeloid Leukemia (AML) With Multilineage Dysplasia Following Myelodysplastic Syndrome, in Adults, AML (Adult) With 11q23 (MLL) Abnormalities
Decitabine and Midostaurin in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia
This phase 2 study evaluates the sequential combination of decitabine then midostaurin for the treatment of newly-diagnosed acute myeloid leukemia (AML) in older patients.
- Conditions
- Acute Myeloid Leukemia (AML) With Multilineage Dysplasia Following Myelodysplastic Syndrome, in Adults, AML (Adult) With 11q23 (MLL) Abnormalities, AML (Adult) With Del (5q), AML (Adult) With Inv (16) (p13; q22)
- Trial name
- NCT01846624
- Testing
- Decitabine, Midostaurin
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- David Iberri
Sickle Cell Anemia, Stroke
Silent Cerebral Infarct Transfusion Multi-Center Clinical Trial
The goal of this study is to determine the effectiveness of blood transfusion therapy for prevention of silent cerebral infarct (stroke) in children with sickle cell anemia.
- Trial name
- SIT (NCT00072761)
- Testing
- transfusion therapy
- Where
- United States
- Volunteers
- 196 people
- Sponsor
- Washington University School of Medicine
Chronic Myeloproliferative Disorders, Leukemia
Donor Stem Cell Transplant or Donor White Blood Cell Infusions in Treating Patients With Hematologic Cancer
RATIONALE: A peripheral stem cell transplant or an umbilical cord blood transplant from a donor may be able to replace blood-forming cells that were destroyed by chemotherapy or radiation therapy.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00281879
- Testing
- anti-thymocyte globulin, filgrastim, busulfan
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- OHSU Knight Cancer Institute
G6PD Deficiency
Validation of a Diagnostic Test for Glucose-6-phosphate Dehydrogenase (G6PD) Deficiency in Anti-coagulated Blood
The primary objective of the study is to assess the accuracy of the SD Biosensor STANDARD™ point-of-care (POC) G6PD test in measuring G6PD activity and classifying results compared to a reference assay and across repeated…
- Trial name
- NCT04054661
- Testing
- SD Biosensor STANDARD G6PD Test, Pointe Scientific Test Kit, HemoCue System
- Where
- United States
- Volunteers
- 229 people, healthy volunteers welcome
- Sponsor
- PATH
Sickle Cell Disease, Pulmonary Hypertension
Sildenafil Therapy for Pulmonary Hypertension and Sickle Cell Disease
This study will examine whether the drug sildenafil can lower blood pressure in the pulmonary artery (the blood vessel that leads from the heart to the lungs) in patients with sickle cell disease and pulmonary hypertension (high…
- Trial name
- NCT00492531
- Testing
- Sildenafil
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 74 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Anemia, Severe Aplastic Anemia
Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes
RATIONALE: Although used primarily to treat malignant disorders of the blood, allogeneic stem cell transplantation can also cure a variety of non-cancerous, inherited or acquired disorders of the blood.
- Conditions
- Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
- Trial name
- NCT00004143
- Testing
- Campath, Chemo and/or TBI Allo SCT
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- David Rizzieri, MD
Telangiectasia, Hereditary Hemorrhagic
Evaluation of Pazopanib on Bleeding in Subjects With Hereditary Haemorrhagic Telangiectasia
This study will investigate whether pazopanib can reduce epistaxis and improve anaemia in subjects with hereditary haemorrhagic telangiectasia (HHT) at a dose that is well tolerated. The study will have 2 parts.
- Trial name
- NCT02204371
- Testing
- Pazopanib
- Where
- 6 sites in United States, Canada
- Volunteers
- 7 people
- Sponsor
- GlaxoSmithKline
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
AKT Inhibitor MK-2206 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
This phase II trial is studying how well AKT inhibitor MK-2206 works in treating patients with relapsed or refractory acute myeloid leukemia (AML).
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT01253447
- Testing
- Akt inhibitor MK2206, laboratory biomarker analysis
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- National Cancer Institute (NCI)