Clinical trials: Hemolytic Disease of the Newborn
Sickle Cell Disease, Hemolytic Anemia
Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen.
- Trial name
- NCT07566494
- Testing
- VAS-101
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia
In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)
The investigators aims to evaluate the safety of in utero hematopoietic stem cell transplantation in fetuses with alpha-thalassemia major performed at the time of in utero transfusion of red blood cells.
- Conditions
- Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia, Hemoglobinopathies, Fetal Anemia
- Trial name
- NCT02986698
- Testing
- in utero hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of California, San Francisco
Sickle Cell Disease
Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem…
- Trial name
- NCT06287099
- Testing
- BRL-101
- Volunteers
- 5 people
- Sponsor
- Bioray Laboratories
Anemia, Sickle Cell
A Study of SANGUINATE for the Treatment of Vaso-occlusive Crisis (VOC) in Adult Sickle Cell Disease Patients
Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis who are admitted to the hospital for treatment.
- Trial name
- NCT02672540
- Testing
- SANGUINATE 320 mg/kg, Normal Saline
- Where
- 4 sites in Colombia, Dominican Republic and 2 more
- Volunteers
- 34 people
- Sponsor
- Prolong Pharmaceuticals
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
- Trial name
- HOPE-KIDS (NCT02850406)
- Testing
- Voxelotor
- Where
- 20 sites in United States, Lebanon and 1 more
- Volunteers
- 147 people
- Sponsor
- Pfizer
Beta-Thalassemia
A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-01 (NCT06291961),No investigational drug product will be administered in the study.
- Trial name
- NCT06717932
- Where
- China
- Volunteers
- 8 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
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
Thalassemia Major, Transfusion-dependent Anemia
The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron…
- Trial name
- NCT05462548
- Testing
- Luspatercept Injectable Product
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Sun Yat-sen University
Beta-Thalassemia
To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care
This is a multi-site, prospective, observational study implemented in β-thalassemia treatment centers from 5 countries (Italy, Turkey, Greece, Lebanon, and Thailand).
- Trial name
- NCT02626689
- Testing
- Quality of Life (QOL) questionnaires, Healthcare Resource Utilization
- Where
- 5 sites in Greece, Italy and 3 more
- Volunteers
- 100 people
- Sponsor
- Celgene
Sickle Cell Disease (SCD)
Exploratory Study on Global Reflexology in Sickle Cell Disease
Sickle cell disease (or sickle cell anemia) is the most common genetic disease in France with 586 children screened in 2019. This chronic disease is characterized by the presence of abnormal Hemoglobin (Hb) S and a deformation of…
- Trial name
- REFLEXODREPANO (NCT06619197)
- Testing
- Global reflexology
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Hospices Civils de Lyon
Cardiogenic Shock, Malignant Arrhythmias
The Caribbean Registry of Extracorporeal Membrane Oxygenation (ECMO) From the University Hospital in Martinique
The project's main goal is to collect baseline clinical and procedural data as well as to assess clinical outcomes for all patients undergoing VV, VA or VAV ECMO implantation in the French West Indies and Guiana.
- Conditions
- Cardiogenic Shock, Malignant Arrhythmias, Ischemic or Valvular Heart Failure, Drepanocytosis and Thoracic Syndrome
- Trial name
- NCT07351435
- Volunteers
- 1,200 people
- Sponsor
- University of Genova
Sickle Cell Crisis, Functional Abdominal Pain
Music Therapy: Sickle Cell and Pain Crisis
This study will assess whether participants who receive music therapy during inpatient care report a decrease in pain and whether music therapy influences factors such as family satisfaction.
- Conditions
- Sickle Cell Crisis, Functional Abdominal Pain, Patients Followed by the Pain Team
- Trial name
- NCT03901560
- Testing
- Music Therapy
- Volunteers
- 150 people
- Sponsor
- University of Oklahoma