Clinical trials: Lymphatic Abnormalities
Hemophilia A
SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
The purpose of the aPCC-emicizumab safety study is to investigate the hemostatic efficacy as measured by thrombin generation, of a low personalized dose of aPCC (FEIBA) in children and adults with hemophilia A and inhibitors on…
- Trial name
- SAFE (NCT04563520)
- Testing
- Emicizumab, FEIBA, rFVIIa
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Emory University
Sickle Cell Disease
A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases
The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled, parallel groups study aimed to evaluate the safety, tolerability and the efficacy of riociguat compared with placebo in patients with…
- Trial name
- NCT02633397
- Testing
- Riociguat
- Where
- 20 sites in United States
- Volunteers
- 130 people
- Sponsor
- Mark Gladwin
Leukemia, Juvenile Myelomonocytic, JMML
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML).
- Conditions
- Leukemia, Juvenile Myelomonocytic, JMML, JCML, Neurofibromatosis 1
- Trial name
- NCT05849662
- Testing
- Trametinib, Azacitidine, Fludarabine
- Where
- 21 sites in United States
- Volunteers
- 58 people
- Sponsor
- Therapeutic Advances in Childhood Leukemia Consortium
Hemophilia A
Drug Use Investigation of Kovaltry in Hemophilia A Patients
The objective in this study is collecting post-marketing information on the safety and efficacy of Kovaltry under the routine clinical practice.
- Trial name
- 18706 (NCT02941783)
- Testing
- Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973)
- Volunteers
- 230 people
- Sponsor
- Bayer
Neimann-Pick Disease
Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1
Niemann-Pick disease type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence.
- Trial name
- NCT02124083
- Testing
- Vorinostat
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Red Blood Cells, Schistocytes
ERYthrocyte Morphology Using Flow Imaging on ImageSTREAM Cytometer
Erythrocyte morphology analysis is a key step in the diagnosis flowchart of anemia. It is performed on a peripheral blood smear after May Grümwald Giemsa staining.
- Conditions
- Red Blood Cells, Schistocytes, Artificial Intelligence
- Trial name
- ERYSTREAM (NCT05282888)
- Testing
- blood sample
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Centre Hospitalier Universitaire, Amiens
Non-Hodgkin's Lymphoma, Hodgkin's Lymphoma
Dasatinib for Immune Modulation After Donor Stem Cell Transplant for Hematologic Malignancies
This study uses a drug called dasatinib to produce an anti-cancer effect called large granular lymphocyte cellular expansion. Large granular lymphocytes are blood cells known as natural killer cells that remove cancer cells.
- Conditions
- Non-Hodgkin's Lymphoma, Hodgkin's Lymphoma, Myeloid Leukemia, Multiple Myeloma
- Trial name
- NCT01643603
- Testing
- Dasatinib, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Sickle Cell Disease
Ibuprofen and Opioid (Morphine or Diamorphine) for Acute Pain in Sickle Cell Disease - Sickle With Ibuprofen & Morphine
The use of oral ibuprofen combined with Opioid (Morphine or Diamorphine) administered through patient controlled analgesia (PCA) will be clinically effective for acute pain crisis in adults with sickle cell disease (SCD).
- Trial name
- SWIM (NCT00880373)
- Testing
- Ibuprofen, Diamorphine or Morphine
- Where
- 2 sites in United Kingdom
- Volunteers
- 320 people
- Sponsor
- London North West Healthcare NHS Trust
Thalassemia, Iron Overload
Inhibitory Effect of a Polyphenol Supplement on Dietary Iron Absorption in Adults with Thalassemia
Genetic disorders, such as thalassemia, can lead to iron overload and severe adverse health outcomes. In iron-loading thalassemia, iron overload is due to increased iron absorption.
- Trial name
- NCT05326503
- Testing
- Meal matrix with polyphenol supplement (PPS), No meal matrix with PPS
- Where
- Thailand
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Swiss Federal Institute of Technology
Hemophilia A
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis.
- Trial name
- NCT06145373
- Testing
- Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 3 sites in United States, Taiwan
- Volunteers
- 20 people
- Sponsor
- Sanofi
Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell…
- Conditions
- Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT03047746
- Testing
- CliniMACs
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's Hospital of Philadelphia
Thalassemia
Effect of Exercise on Body Composition and Bone Health in Patients With Thalassemia
The goal of this clinical trial is to determine if a weight bearing exercise intervention can improve body composition and bone health in adolescents and adults with Thalassemia.
- Trial name
- NCT07064941
- Testing
- Exercise
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- University of California, San Francisco
Hemophilia A
Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq
Uncontrolled, multi-centre, non-interventional study with a prospective and a retrospective cohort, to evaluate the efficacy of Wilate or Nuwiq in achieving complete or partial immune tolerance induction (ITI) success in severe…
- Trial name
- PREVAIL (NCT03344003)
- Testing
- Wilate or Nuwiq
- Where
- 3 sites in Canada
- Volunteers
- 14 people
- Sponsor
- Octapharma
Fanconi Anemia, Anemia, Hypoplastic, Congenital
Prenatal Transplantation for Fetuses With Fanconi Anemia
The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed with Fanconi anemia (FA) during pregnancy.
- Conditions
- Fanconi Anemia, Anemia, Hypoplastic, Congenital, Congenital Bone Marrow Failure Syndromes, Bone Marrow Failure Disorders
- Trial name
- NCT07408583
- Testing
- IUHSCT for FA-affected fetuses
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Agnieszka Czechowicz
Anemia, Sickle Cell
A Socio-ecological Approach for Improving Self-management in Adolescents With SCD
The goal of this clinical trial is to evaluate the impact of SCThrive (a behavioral self-management intervention) on patient activation, self-management behaviors, daily functioning, and emergency room visits in 260 adolescents…
- Trial name
- SC-Thrive (NCT06290401)
- Testing
- SCThrive, SCHealthED
- Where
- 4 sites in United States
- Volunteers
- 310 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Alpha Thalassemia Hemoglobin H Constant Spring, Hemoglobinopathies
Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease
The purpose of this study is to evaluate the safety and efficacy of RM-004 for Hemoglobin H-Constant Spring disease.
- Conditions
- Alpha Thalassemia Hemoglobin H Constant Spring, Hemoglobinopathies, Hereditary Diseases
- Trial name
- NCT06107400
- Testing
- RM-004
- Where
- China
- Volunteers
- 5 people
- Sponsor
- The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Von Willebrand Diseases
Impact of Von Willebrand Factor and Its Multimers on Angiogenesis
The study aims to study the expression of numerous proteins involved in angiogenesis in 70 patients with von Willebrand disease in order to try to identify markers of interest.
- Trial name
- WILLANGIO (NCT04810702)
- Testing
- no intervention
- Where
- France
- Volunteers
- 109 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Fanconi Anemia
Hematopoietic Stem Cell Transplant for Fanconi Anemia
The trial proposed is a single arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan…
- Trial name
- FA (NCT01071239)
- Testing
- CliniMACs device, Busulfan, Fludarabine
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Medical College of Wisconsin
Hematologic Diseases, Anaemia, Sickle Cell
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 Years
This will be a first time in human (FTIH) study in sickle cell diseases (SCD) participants. The FTIH study is planned to evaluate the safety, tolerability, and pharmacokinetics of GSK4172239D.
- Trial name
- NCT05660265
- Testing
- GSK4172239D
- Where
- 6 sites in United States
- Volunteers
- 11 people
- Sponsor
- GlaxoSmithKline
Thalassemia Major
To Evaluate Long- Term Safety and Efficacy of Luspatercept
An observational study to evaluate long-term safety and efficacy of luspatercept in subjects with transfusion dependent who received the first dose of luspatercept after its introduction in the clinical practice
- Trial name
- LUSPAREAL001 (NCT06875219)
- Testing
- Luspatercept (ACE-536)
- Where
- Italy
- Volunteers
- 350 people
- Sponsor
- Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia