Clinical trials: Lymphatic Abnormalities, open to volunteers
Alpha-Thalassemia, Alpha Thalassemia Major
International Registry of Patients With Alpha Thalassemia
This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of…
- Conditions
- Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor
- Trial name
- ATM Registry (NCT04872179)
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- University of California, San Francisco
Hemophilia
Feasibility and Safety of Blood-Flow-Restriction Training in Patients With Hemophilia
In the course of a cross-over study design, 12 patients with hemophilic arthropathy will perform a training load on the knee extensor muscles.
- Trial name
- NCT07259356
- Testing
- Control, BFR Exercise (50% of the LOP) while Leg Press Exercise (4 Sets, All out)
- Where
- Germany
- Volunteers
- 12 people
- Sponsor
- University Hospital, Bonn
Hereditary Hemorrhagic Telangiectasia
Cardiac Evaluation in Hereditary Hemorrhagic Telangiectasia
Hereditary haemorrhagic telangiectasia (HHT) is a rare autosomal dominant genetic disease associated with mutations in genes encoding proteins of the transforming growth factor β (TGF-β) family, i.e.
- Trial name
- CARDI-HHT (NCT07101575)
- Testing
- Transthoracic echocardiography
- Volunteers
- 380 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Thrombocytopenia, Neonatal Thrombocytopenia
Neonatal Thrombocytopenia in the NICU
This prospective cohort study aims to determine the frequency, severity, and clinical outcome of thrombocytopenia among neonates admitted to the neonatal intensive care unit.
- Trial name
- NCT07882342
- Volunteers
- 120 people
- Sponsor
- Assiut University
Hemophilia A
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients.
- Trial name
- NCT06238908
- Testing
- NGGT003
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
MRI, Hemophilia
MRI Role in Knee Hemophilic Arthopathy
The aim of this study is to assess role of MRI in detecting synovial, cartilaginous , osseous abnormalities ، bleeding inside knee joint and to use a system for assessing HA as support for therapeutic regimes and for monitoring…
- Conditions
- MRI, Hemophilia, Arthropathy Hemophilic, Arthropathy of Knee
- Trial name
- NCT07233122
- Volunteers
- 95 people
- Sponsor
- Assiut University
Hemophilia B
Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
- Trial name
- IX-TEND 3003 (NCT05962398)
- Testing
- AAV5-hFIXco-Padua
- Where
- 28 sites in United States, Belgium and 6 more
- Volunteers
- 56 people
- Sponsor
- CSL Behring
Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA)
Lymphatic Anomalies Registry for the Assessment of Outcome Data
Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the relative benefit of medical…
- Conditions
- Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA), Central Conducting Lymphatic Anomaly, CLOVES Syndrome
- Trial name
- NCT02399527
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Boston Children's Hospital
Hemophilia A
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion.
- Trial name
- GENEr8-LTE (NCT05768386)
- Where
- 41 sites in United States, Australia and 10 more
- Volunteers
- 172 people
- Sponsor
- BioMarin Pharmaceutical
Vascular Anomaly, Generalized Lymphatic Anomaly
Identification of Biomarkers for Patients with Vascular Anomalies
The study will use blood (serum and plasma) and tissue obtained from participants undergoing prescribed surgical resection of vascular anomalies of interest proposed in this study.
- Conditions
- Vascular Anomaly, Generalized Lymphatic Anomaly, Kaposiform Hemangioendothelioma, Kaposiform Lymphangiomatosis
- Trial name
- NCT03001180
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly deoxyribonucleic acid (DNA)) from the patient to treat his…
- Trial name
- NCT07708350
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Daniel Bauer
Hemophilia
The World Federation of Hemophilia Gene Therapy Registry
The aim of the WFH GTR is to provide a database in which long-term data on PWH who receive gene therapy from around the world, will be collected and housed.
- Trial name
- WFH GTR (NCT04883710)
- Testing
- Gene therapy
- Volunteers
- 5,000 people
- Sponsor
- World Federation of Hemophilia
Neonatal Sepsis, Neutropenia
G-CSF In Neonatal Sepsis With Neutropenia
This study will evaluate whether adding G-CSF to standard treatment improves outcomes in newborns (0-28 days) with sepsis and neutropenia.
- Trial name
- NCT07888829
- Testing
- Granulocyte-colony stimulating factor (G-CSF), Standard Sepsis Management
- Where
- Pakistan
- Volunteers
- 52 people
- Sponsor
- University of Health Sciences Lahore
Ehlers-Danlos Syndrome
Registry of Ehlers-Danlos Syndrome
RED is a retrospective and prospective registry, finalized for care and research purposes. It is articulated in main sections - strongly related and mutually dependent on each other - corresponding to different data domains…
- Trial name
- RED (NCT04133272)
- Where
- Italy
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Luca Sangiorgi
Cavernous Malformation, Cerebral
Daratumumab for Familial Cerebral Cavernous Malformations: A Single-Arm Safety and Efficacy Study
Cerebral cavernous malformation (CCM) is a common vascular abnormality of the brain, affecting 0.1%-0.5% of people. It often causes recurrent brain hemorrhages, epilepsy, and neurological impairments, with surgery being the main…
- Trial name
- CCM-DS01 (NCT07026604)
- Testing
- Daratumumab
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Beijing Tiantan Hospital
Hemolytic Disease of the Newborn, Fetal Anemia
Neonatal Outcomes on Direct Fetal Intravenous Immunoglobulin During Intrauterine Transfusion for Rhesus Isoimmunization
The goal of this clinical trial is to learn if giving intravenous immunoglobulin directly to the fetus during intrauterine transfusion can improve newborn outcomes in pregnancies affected by rhesus isoimmunization.
- Conditions
- Hemolytic Disease of the Newborn, Fetal Anemia, Rhesus Isoimmunization, Erythroblastosis Fetalis Due to RH Antibodies
- Trial name
- NCT07885592
- Testing
- Intrauterine Transfusion, Intravenous immunoglobulin (IVIG)
- Where
- India
- Volunteers
- 30 people
- Sponsor
- Post Graduate Institute of Medical Education and Research, Chandigarh
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.
Chronic Respiratory Diseases, Cystic Fibrosis
Breath Analysis and Arterial Stiffness in Patients With Respiratory Diseases
Assessment of cardiovascular disorders using echocardiography and arterial stiffness; comparative noninvasive assessment of volatile organic compound (eVOC) exhale breath patterns in patients with different chronic respiratory…
- Conditions
- Chronic Respiratory Diseases, Cystic Fibrosis, Lymphangioleiomyomatosis, Hypersensitivity Pneumonitis
- Trial name
- NCT05727852
- Testing
- Breath test using the Compact PTR-TOF-MS, Portable cardiac monitor "CardioQvark", Assessment of arterial stiffness using the VaSera VS-1500N
- Where
- Russia
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- I.M. Sechenov First Moscow State Medical University
Hereditary Hemorrhagic Telangiectasia, Pulmonary Arterial Hypertension
DIAG723 in Adults With Hereditary Hemorrhagic Telangiectasia
This is a Phase 1/2, randomized, double-blind, placebo-controlled, first-in-human study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of subcutaneously administered DIAG723 in adult patients with…
- Trial name
- DIAMOND (NCT07623525)
- Testing
- DIAG723
- Where
- 11 sites in Australia, Canada and 1 more
- Volunteers
- 93 people
- Sponsor
- Diagonal Therapeutics, Inc.