Clinical trials: Lymphatic Malformation
Sickle Cell Disease, Cerebral Stroke
Cerebrovascular Reactivity Measurements With High-Density Diffuse Optical Tomography
The purpose of the study protocol is to identify imaging biomarkers for brain tissue under high metabolic stress at risk for permanent injury.
- Trial name
- HDDOT CVR (NCT07776444)
- Testing
- Carbon Dioxide
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Haemophilia B
An Open Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients
To compare the first and second recovery assessments on Replenine®-VF and to evaluate recovery of different batches if patients changed batches during the study.
- Trial name
- NCT02231944
- Testing
- Replenine®-VF (High Purity Factor IX)
- Where
- 7 sites in Poland, United Kingdom
- Sponsor
- Bio Products Laboratory
Congenital Bleeding Disorder, Haemophilia A
Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A
The trial is conducted in Asia, Europe and North America. The aim of the study is to evaluate the safety of administration under the skin of turoctocog alfa pegol (SC N8-GP) in patients with severe haemophilia A.
- Trial name
- alleviate 1 (NCT02994407)
- Testing
- turoctocog alfa pegol
- Where
- 19 sites in United States, Austria and 4 more
- Volunteers
- 50 people
- Sponsor
- Novo Nordisk A/S
Crohn Disease
Study to Evaluate the Efficacy and Safety of PF-06480605 in Adult Participants With Moderate to Severe Crohn's Disease
This Phase 2a, multicenter, randomized, double-blind, placebo-controlled study examines subcutaneous dose of PF-06480605 150 mg administered every 4 weeks in participants with moderate to severe active Crohn's Disease to…
- Trial name
- NCT05471492
- Testing
- Investigational Product
- Sponsor
- Telavant, Inc.
Sickle Cell Disease
School Readiness Intervention for Preschool Children With Sickle Cell Disease
The study participant is being asked to take part in this clinical trial, a type of research study, because the participant is a young child with sickle cell disease or the caregiver of a child with sickle cell disease.
- Trial name
- NCT06367192
- Testing
- The adapted Kids in Transition to School Intervention, Standard school resources
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Sickle Cell Disease Biofluid Chip Technology (SCD BioChip)
'Sickle-shaped' anemia was first clinically described in the US in 1910, and the mutated heritable sickle hemoglobin molecule was identified in 1949.
- Trial name
- NCT02824471
- Testing
- SCD Group
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University Hospitals Cleveland Medical Center
Hemophilia A
Emotions in the Communication and Relationship Styles of Parents With Hemophilic Children.
Severe hemophilia is a rare, inherited, X-linked disorder. Currently, standard therapy involves ev infusion of drugs from a very young age.
- Trial name
- NCT05754619
- Where
- Italy
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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
Sickle Cell Disease, Venous Thromboembolism
Comparison of Thrombin Generation, Platelet Activation and Aggregation Profiles in Patients With Sickle Cell Disease Depending on Their SS or SC Genotype (DrepAgreg)
Sickle cell disease (SCD) is characterized by chronic hemolytic anemia, painful crises called vaso-occlusive crises (VOC) and chronic inflammation.
- Trial name
- DrepAgreg (NCT07852442)
- Where
- 2 sites in France, Guadeloupe
- Volunteers
- 60 people
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Hematologic Malignancies, Nonmalignant Diseases
Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)
Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609814
- Testing
- Clofarabine, Fludarabine Injection
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, San Francisco
Sickle Cell Disease
Pathophysiology of Acute Pain in Patients With Sickle Cell Disease
Background: Sickle Cell Disease (SCD) is a blood disorder that occurs mainly in people of African descent. Researchers want to learn more about the painful attacks and complications associated with SCD.
- Trial name
- NCT03049475
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hemophilia, Upper Extremity
Comparison of Upper Extremity Functional Performance in Hemophilic and Healthy Children
The goal of this observational study is to evaluate the functional performance of the upper extremity joints (scapula, shoulder, elbow, and wrist) in haemophilic children and compare muscle strength and ADL limitations with…
- Conditions
- Hemophilia, Upper Extremity, Strength, Performance
- Trial name
- NCT06826209
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Vaso-Occlusive Crises, Sickle Cell Disease
Evaluation of the Efficacy of Iloprost in the Management of Vaso-occlusive Crises in Adult Patients With Sickle Cell Disease
Sickle cell disease is a severe monogenic genetic disorder caused by an autosomal recessive mutation of the β-globin gene, leading to production of abnormal hemoglobin (HbS).
- Trial name
- PROSTASICKLE (NCT07498309)
- Testing
- Iloprost
- Where
- France
- Volunteers
- 144 people
- Sponsor
- University Hospital, Rouen
Hemophilia A, Hemophilia B
Liver Biopsy Following Gene Therapy For Hemophilia
This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have…
- Trial name
- NCT05932914
- Testing
- Liver Biopsy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Anemia
The Efficacy of Jobelyn (Sorghum Bicolor Extract)in the Treatment of Sickle Cell Anemia
The primary objective is to determine if there is a significant increase in the haematocrit value of patients on Jobelyn and standard therapy compared to those on standard therapy alone.
- Trial name
- NCT01703104
- Testing
- Paludrine + Folic Acid, Paludrine + Folic Acid + Jobelyn
- Where
- Nigeria
- Volunteers
- 150 people
- Sponsor
- Lagos State University
Sickle Cell Disease
Near Infrared Spectroscopy in Sickle Cell Pediatric Patients
Endothelial dysfunction contributes to vaso-occlusion and acute pain in sickle cell disease. Near infrared spectroscopy (NIRS) technology can measure tissue oxygenation and endothelial function.
- Trial name
- NCT04031521
- Testing
- Near infrared spectroscopy
- Where
- United States
- Sponsor
- Children's National Research Institute
Leukemia, Lymphoma
Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Trial name
- NCT00423826
- Testing
- Busulfan, Cytarabine, Fludarabine phosphate
- Where
- United States
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Beta Thalassemia Major
Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth
- Trial name
- NCT03947632
- Testing
- serum ferritin and hemoglobin test
- Volunteers
- 367 people
- Sponsor
- Aga Khan University
Sickle Cell Disease
A GBT021601 ADME Microtracer Study in Healthy Volunteers
An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.
- Trial name
- NCT05718687
- Testing
- GBT021601
- Where
- Netherlands
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Pfizer
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Palifermin in Preventing Chronic Graft-Versus-Host Disease in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer
RATIONALE: Growth factors, such as palifermin, may prevent chronic graft-versus-host disease caused by donor stem cell transplant.
- 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
- NCT01233921
- Testing
- palifermin, flow cytometry, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Martin, Paul
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