Clinical trials: Hemoglobinopathy (Disorder)
Beta Thalassemia
Genetic Variants Affecting the Clinical Severity of Beta Thalassemia
β-thalassemia is one of the most common single gene disorder in Southern China. The phenotypic severity of beta thalassemia widely varies from mild to severe forms.
- Trial name
- NCT04918056
- Testing
- Hematological Analysis and Genetical Analysis
- Where
- China
- Volunteers
- 1,300 people
- Sponsor
- Nanfang Hospital, Southern Medical University
Thalassemia, Iron Overload
Efficacy and Safety of Desferal Versus Osveral in Transfusional Iron Overload
Thalassemic patients often suffer from iron overload due to frequent blood transfusion. Oral iron chelators reduce iron overload in transfusion dependent patients.
- Conditions
- Thalassemia, Iron Overload, Transfusion Related Complications
- Trial name
- NCT01369719
- Testing
- osveral, Desferal
- Where
- Iran
- Volunteers
- 138 people
- Sponsor
- Hormozgan University of Medical Sciences
Thalassemia Intermedia, Thalassemia Major
Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
There are nearly 300,000 patients with severe or intermediate thalassemia in China. Growth retardation is the most significant health issue for children and adolescents with transfusion-dependent thalassemia (TDT), placing a…
- Conditions
- Thalassemia Intermedia, Thalassemia Major, Growth Delay, Puberty, Delayed
- Trial name
- NCT06931912
- Testing
- MENBS clinical interventions
- Where
- China
- Volunteers
- 385 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Sickle Cell Disease
Algorithm for Apherisis Monitoring and Prescription Assistance in Sickle Cell Patients (ALGODREP)
The main objective of this study is to prove the superiority of a procedure which calculates the volume of RBCs to transfuse and the time between apheresis based on this algorithm, compared to the current procedure.
- Trial name
- ALGODREP (NCT04076683)
- Testing
- Algodrep
- Where
- 8 sites in France, Martinique
- Volunteers
- 65 people
- Sponsor
- Etablissement Français du Sang
Sickle Cell Disease, Sickle Cell Anaemia
Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype
SIL-8301 (senicapoc) is being developed for the chronic treatment of patients with sickle cell disease in both adults and children.
- Conditions
- Sickle Cell Disease, Sickle Cell Anaemia, Sickle Cell Anemia
- Trial name
- RESCUE (NCT07282210)
- Testing
- Senicapoc
- Where
- 4 sites in Canada
- Volunteers
- 105 people
- Sponsor
- Biossil Inc.
Sickle Cell Disease
A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of…
- Trial name
- HIBISCUS (NCT04624659)
- Testing
- Etavopivat Tablets Low dose, Etavopivat Tablets High dose, Etavopivat Tablets
- Where
- 142 sites in United States, Canada and 15 more
- Volunteers
- 450 people
- Sponsor
- Forma Therapeutics, Inc.
Sickle Cell Disease
High Dose Vitamin D for Sickle Cell Disease
Vitamin D deficiency (VDD) is very common among African American adolescents and adults in the US, ten times higher than is seen in Caucasians. VDD is also quite common in sickle cell disease (SCD).
- Trial name
- SCD-VitD (NCT01331148)
- Testing
- Vitamin D
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Wake Forest University Health Sciences
Sickle Cell Disease
A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS)…
- Trial name
- NCT04983264
- Testing
- GBT021601
- Where
- 3 sites in United States
- Volunteers
- 6 people
- Sponsor
- Pfizer
Iron, Abnormal Blood Level, Other Abnormal Blood Chemistry
Fresh Versus Old Red Blood Cells for Transfusion
Packed red blood cell units destined for transfusion can be stored for up to 42 days prior to transfusion based on Food and Drug Administration (FDA) guidelines.
- Trial name
- NCT01319552
- Testing
- Fresh transfusion, Old transfusion
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Columbia University
Beta Thalassemia, Hemoglobinopathy
Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)
This 12-month study will evaluate the safety and effectiveness of hydroxyurea in treating beta-thalassemia, a type of anemia caused by defective hemoglobin (the oxygen-carrying pigment in blood).
- Trial name
- NCT00001958
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sickle Cell Anemia
Study of Ventilatory Mechanics in Patients With Sickle Cell Anemia
The Sickle Cell Anemia (SCA) is a recessive genetic condition, monogenic, resulting in defects in the red cell structure. In the investigators' country, this disease affects about 3,000 children each year and is considered one of…
- Trial name
- SCA (NCT02565849)
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Rio de Janeiro State University
Sickle Cell Disease, Anemia
SCD-PROMIS: A Software Platform to Enhance Self-efficacy and Patient-provider Engagement for Patients With Sickle Cell Pain
The overall goal of the project is to reduce pain-related, 30-day readmission rates for sickle cell disease (SCD) patients. The investigators want to see if a mobile phone application (app) can help decrease the need for repeat…
- Conditions
- Sickle Cell Disease, Anemia, Anemia, Hemolytic, Anemia, Sickle Cell
- Trial name
- NCT03006718
- Testing
- PROMIS for Pain Management App
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Julia Finkel
Sickle Cell Disease, Sickle Cell Anemia
Sickle Cell Disease (SCD) Bone Pain Study
A prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Low Bone Density, Osteoporosis
- Trial name
- NCT05283148
- Testing
- Dual-energy X-ray absorptiometry, Vertebral fracture analysis, Adult Sickle Cell Quality of Life Measurement System pain impact questionnaire
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- University of California, Davis
Sickle Cell Disorders
GenoMed4ALL: Improving SCD Classification and Prognosis by AI
GenoMed4All 'Genomics and Personalized Medicine for all though Artificial Intelligence in Haematological Diseases' aims to advance on individual SCD patients' disease characterisation and to improve the monitoring of patients'…
- Trial name
- GenoMed4ALL (NCT06019208)
- Where
- 5 sites in France, Italy and 2 more
- Volunteers
- 1,000 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Sickle Cell Anemia
Adherence to Hydroxyurea in Children With Sickle Cell Anemia
Medication non-adherence is a true public health problem. Despite advancements in the molecular understanding of disease and improvements in therapy, patient health outcomes will not improve unless patients take prescribed…
- Trial name
- NCT00672789
- Testing
- Blood smear education, standard education
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Duke University
Beta-Thalassemia
Choroidal Thickness in Beta-thalassemia Patients
The purpose of this study is to examine choroidal thickness in beta-thalassemia patients and compare it to the one of healthy controls.
- Trial name
- NCT04067258
- Testing
- EDI-OCT
- Where
- Greece
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University Hospital of Patras
Sickle Cell Disease, Sickle Cell-Beta0-Thalassemia
Isoquercetin in Sickle Cell Anemia
This research study is being done to assess the safety and effectiveness of isoquercetin to reduce levels of soluble P-Selectin in patients with sickle cell disease. Isoquercetin is a naturally occurring flavonoid-or vitamin.
- Trial name
- NCT04474626
- Testing
- Isoquercetin
- Volunteers
- healthy volunteers welcome
- Sponsor
- Jeffrey Zwicker, MD
Thalassemia Major, Chronic Periodontitis
Periodontal Therapy in Thalassemia Major Patients
The study aims to evaluate the effect of local non surgical periodontal therapy on the systemic pro-inflammatory markers in the β-thalassemia (TM-β) patients with chronic periodontitis and systemically healthy demographically…
- Trial name
- NCT03311243
- Testing
- Non surgical periodontal therapy, Chemical plaque control
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- King Saud University
Thalassemia
iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations
This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
- Trial name
- NCT03728322
- Testing
- iHSCs treatment group
- Volunteers
- 12 people
- Sponsor
- Allife Medical Science and Technology Co., Ltd.
Sickle Cell Disease
Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study
This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are…
- Trial name
- NCT04657822
- Testing
- Crizanlizumab
- Where
- 31 sites in United States, Belgium and 9 more
- Volunteers
- 130 people
- Sponsor
- Novartis Pharmaceuticals