Clinical trials: Lymphatic Malformation, active
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
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
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
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 Disease
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).
- Trial name
- PRECISE (NCT06923111)
- Testing
- Xromi
- Where
- 12 sites in Germany, United Kingdom
- Volunteers
- 180 people
- Sponsor
- Nova Laboratories Limited
Atrial Fibrillation, Thalassemia
Atrial Fibrillation in Beta-Thalassemia
The study aims to evaluate the clinical, laboratory and instrumental differences that exist between beta-thalassemia patients with atrial fibrillation and those not affected by arrhythmia.
- Trial name
- NCT05508932
- Where
- Italy
- Volunteers
- 350 people
- Sponsor
- University Hospital of Ferrara
Sickle Cell Disease
A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke
The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke.
- Trial name
- HIBISCUS 3 (NCT05953584)
- Testing
- Etavopivat, Hydroxyurea
- Where
- 9 sites in India, Nigeria and 1 more
- Volunteers
- 27 people
- Sponsor
- Forma Therapeutics, Inc.
Sickle Cell Disease (SCD), Invasive Bacterial Infections
Epidemiology of Invasive Bacterial Infections in Children With Sickle Cell Disease in France Between 2020 and 2025
Children with sickle cell disease are at high risk of invasive bacterial infections, which may lead to serious complications. Preventive measures, including antibiotic prophylaxis and vaccination, have changed the epidemiology of…
- Trial name
- DREPIBAC (NCT07639008)
- Where
- 31 sites in France
- Volunteers
- 350 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Fetal Conditions, Maternal; Procedure
A Prospective Study on the Role of Karl Storz Curved and Straight Fetoscopes (11508AAK and 11506AAK) for Fetoscopic Intrauterine Procedures
In this research study, the investigators want to learn more about the role of new innovative surgical devices, the Karl Storz Curved and Straight Fetoscopes for in-utero surgery.
- Conditions
- Fetal Conditions, Maternal; Procedure, Pregnancy Related, Twin to Twin Transfusion Syndrome
- Trial name
- NCT06056635
- Testing
- Karl Storz Curved Scope, Karl Storz Straight Scope
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Boston Children's Hospital
PIK3CA-Related Overgrowth Spectrum (PROS), Lymphatic Malformations
A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study)
This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, zovegalisib (RLY-2608), in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations…
- Conditions
- PIK3CA-Related Overgrowth Spectrum (PROS), Lymphatic Malformations, Vascular Malformations, PIK3CA Mutation
- Trial name
- NCT06789913
- Testing
- RLY-2608
- Where
- 40 sites in United States, Australia and 7 more
- Volunteers
- 277 people
- Sponsor
- Relay Therapeutics, Inc.
Telangiectasia, Hereditary Hemorrhagic
Molecular Studies on Hereditary Haemorrhagic Telangiectasia Families
This study will examine genes involved in the vascular dysplasia Hereditary haemorrhagic telangiectasia i(HHT)
- Trial name
- NCT00230620
- Where
- United Kingdom
- Volunteers
- 1,000 people
- Sponsor
- Imperial College London
Factor VIII Deficiency, Congenital
Hemlibra in Mild Hemophilia A
This is a single arm, phase 4, prospective, open-label, United States single-center study to determine the hemostatic characteristics of Hemlibra (emicizumab) as measured by coagulation laboratory parameters in the mild…
- Trial name
- NCT04567511
- Testing
- Emicizumab
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Indiana Hemophilia &Thrombosis Center, Inc.
Sickle Cell Disease
Open-Label Extension Study to Pioneer Study 6058-SCD-101
This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder…
- Trial name
- NCT07401823
- Testing
- Pociredir
- Where
- 8 sites in United States
- Volunteers
- 50 people
- Sponsor
- Fulcrum Therapeutics
Fracture, Lengthening; Leg
Pediatric Orthopaedic Implant Safety & Efficacy
Implant devices are important tools - their use is essential across a number of orthopaedic indications, including hip conditions, trauma and limb deformity.
- Conditions
- Fracture, Lengthening; Leg, Orthopedic Disorder, Deformity of Limb
- Trial name
- Global POISE (NCT05361980)
- Testing
- Pediatric orthopaedic implant
- Where
- Canada
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- University of British Columbia
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
Hemophilia A or B
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
A study to evaluate the safety of Hympavzi under the actual use in patients with congenital hemophilia who do not have inhibitors.
- Trial name
- HIZ (NCT07161687)
- Testing
- Marstacimab
- Where
- Japan
- Volunteers
- 50 people
- Sponsor
- Pfizer
Bone Marrow Failure Syndromes, Erythrocyte Disorder
Investigation of the Genetics of Hematologic Diseases
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases.
- Conditions
- Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis
- Trial name
- NCT02720679
- Where
- United States
- Volunteers
- 1,716 people
- Sponsor
- St. Jude Children's Research Hospital