Clinical trials: Hemolytic Disease of the Newborn
Beta-Thalassemia
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
The main purpose of this study is to evaluate the efficacy of 3 multiple doses of VIT-2763 as measured by the reduction in red blood cell (RBC) transfusion burden from Week 13 to Week 24, to identify the most efficacious and safe…
- Trial name
- NCT04938635
- Testing
- VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID
- Where
- 7 sites in United States, Bulgaria and 1 more
- Sponsor
- Vifor (International) Inc.
Priapism, Anemia, Sickle Cell
Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence
Priapism, a prolonged erection of the penis, is a medical issue that often affects men with sickle cell disease. The purpose of this study is to collect demographic and clinical information on priapism by interviewing men with…
- Trial name
- NCT00300235
- Where
- 27 sites in United States
- Volunteers
- 1,464 people
- Sponsor
- University of Texas Southwestern Medical Center
Thrombosis
Sickle Cell Trait and the Risk of Venous Thromboembolism
The purpose of this trial is to investigate D-Dimer levels, a surrogate marker of venous thromboembolism, in pregnant/postpartum white women as compared to pregnant/postpartum black women, and pregnant/postpartum women with…
- Trial name
- SCT&DVT (NCT01148940)
- Where
- United States
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
The goal of this clinical trial is to learn whether a single dose of buccal buprenorphine (BELBUCA) is safe when used in place and/or with full agonist opioids that would otherwise be started during a sickle cell pain crisis…
- Conditions
- Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children
- Trial name
- NCT07799571
- Testing
- Buprenorphine Buccal Film [Belbuca]
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Natasha Archer
Beta-Thalassemia
A Long-term Follow-up Study in Participants Who Received CS-101
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-06 (NCT06024876),No investigational drug product will be administered in the study.
- Trial name
- NCT06685536
- Testing
- Safety and efficacy assessments
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Thalassemia
L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension
This study compares L-arginine Versus Sildenafil as treatment for pulmonary hypertension in Children with Beta Thalassemia
- Trial name
- NCT03402191
- Testing
- L-arginine, Sildenafil
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Sherief Abd-Elsalam
Liver Iron Overload
Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.
- Trial name
- NCT01033747
- Testing
- Deferasirox
- Where
- 4 sites in Italy
- Volunteers
- 70 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Vaso-occlusive Pain Episodes
Virtual Reality As Adjunct Therapy for Vaso-Occlusive Pain
The goal of this randomized control clinical trial is to learn if virtual reality can be used to treat sickle cell pain in children.
- Trial name
- NCT06773715
- Testing
- KindVR Aqua Program
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Louisiana State University Health Sciences Center in New Orleans
Sickle Cell Disease
Osteopathic Manipulation in the Management of Pain Associated With Sickel Cell Disease
Background: Sickle cell disease is the most common monogenic disease in the world caused by a mutation in the β-globin gene which creates abnormal hemoglobin called HbS.
- Trial name
- OSTEODREP (NCT05632289)
- Testing
- osteopathic manipulative therapy
- Where
- France
- Volunteers
- 37 people
- Sponsor
- College Osteopathique de Provence Aix-Marseille
Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA)
Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders
The purpose of this study is to study the efficacy of Traditional Chinese Medicine (TCM) on anaemic and cytopenic haematological disorders including myelodysplastic syndrome (MDS), aplastic anaemia (AA), myelofibrosis (MF) and…
- Conditions
- Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA), Myelofibrosis (MF), Thalassemia Intermedia
- Trial name
- NCT01224496
- Testing
- Chinese herbal concoction twice a day for 6 months
- Where
- Singapore
- Volunteers
- 38 people
- Sponsor
- Singapore General Hospital
Sickle Cell Disease
Acupuncture for Pain in Sickle Cell Disease
Sickle cell disease (SCD) is the most common genetic disorder in the United States affecting approximately 100,000 individuals primarily of African ancestry. Pain is the most common complication of SCD.
- Trial name
- NCT04122378
- Testing
- acupuncture
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Deepika Darbari
Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD
Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses
This is an open label, single arm, and multicenter study. The study will include the following phases. A screening phase which lasts for 4 weeks to determine patient eligibility.
- Trial name
- NCT01948817
- Testing
- Deferasirox
- Sponsor
- Novartis Pharmaceuticals
Thalassemia Major
Observing the Changes of Endocrine and Metabolism in Patients With Thalassemia Major
Patient with thalassemia major have many endocrine diseases, such as hypopituitarism, hypothyroidism, hypoparathyroidis, osteoporosis, and etc.. These problems may be due to anemia itself or related to iron deposition.
- Trial name
- NCT03951818
- Where
- Taiwan
- Volunteers
- 100 people
- Sponsor
- National Taiwan University Hospital
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Hepatitis C, Thalassemia
Evaluating the Safety of Two Medications to Treat Hepatitis C in People With Thalassemia (The HepC Study)
Hepatitis C is one of the most common causes of long-term liver disease in the United States. Ribavirin and peginterferon alfa-2a are two medications that are used to treat hepatitis C infection.
- Trial name
- HepC (NCT00502788)
- Testing
- Peginterferon Alfa-2a and Ribavirin, Ribavirin
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Carelon Research
Sickle Cell Disease, Pain
Psychometric Evaluation of the IPPAQ in Pediatric Patients With Sickle Cell Disease Hospitalized With Vasoocclusive Pain
Historically, sickle cell disease has not been viewed in the chronic pain paradigm because of its recurrent nature. Patients with sickle cell disease may be hospitalized for extended periods of time.
- Trial name
- NCT01058473
- Where
- United States
- Volunteers
- 159 people
- Sponsor
- Connecticut Children's Medical Center
Beta Thalassemia Intermedia
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.
- Trial name
- NCT04059406
- Testing
- sapablursen
- Where
- 19 sites in Australia, Greece and 3 more
- Volunteers
- 29 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Sickle Cell Disease, Sickle Cell Anemia
Alendronate for Osteonecrosis in Adults With Sickle Cell Disease
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Osteonecrosis, Ischemic Necrosis
- Trial name
- NCT06016634
- Testing
- Alendronate Sodium
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, Davis
Sickle Cell Disease
A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease
The purpose of this study is to determine whether ticagrelor is effective in reducing the number of days of pain, intensity of pain, and reducing the use of analgesics due to sickle cell disease
- Trial name
- Hestia2 (NCT02482298)
- Testing
- Ticagrelor
- Where
- 20 sites in United States, Egypt and 6 more
- Volunteers
- 87 people
- Sponsor
- AstraZeneca
To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease, Anemia, Sickle Cell
To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
This is an open-label study to understand the safety and tolerability of AXA4010, a novel composition of amino acids in adult and adolescent subjects with sickle cell disease over 12 weeks.
- Conditions
- To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease, Anemia, Sickle Cell, Sickle Cell Anemia, Sickle Cell Disorders
- Trial name
- NCT04134299
- Testing
- AXA4010
- Where
- 4 sites in United States
- Volunteers
- 9 people
- Sponsor
- Axcella Health, Inc