Clinical trials: Hemoglobinopathies
Sickle Cell Disease
A Phase 1 Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest Syndrome
Sickle cell disease is a genetic red blood cell disorder characterized by vaso-occlusion from sickling of red blood cells, that can lead to pain or organ complications such as acute chest syndrome.
- Trial name
- NCT02697240
- Testing
- Intravenous citrulline
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Mississippi Medical Center
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
Sickle Cell Disease
A Study to Learn About Sickle Cell Disease In Adult Patients
The purpose of this clinical trial is to evaluate the performance of the sickle cell disease (SCD) electronic diary in people with SCD who are on treatment that will change SCD and those not on such a treatment.
- Trial name
- NCT05407805
- Testing
- Electronic Diary
- Where
- 4 sites in United States
- Volunteers
- 98 people
- Sponsor
- Pfizer
Sickle Cell Disease
Measurement of Cerebral Blood Flow Using Transcranial Doppler Ultrasound in Children With Sickle Cell Disease
Sickle cell disease (SCD) affects haemoglobin - the molecule in blood cells which carries oxygen. It causes red blood cells to become abnormal crescent (or sickle)- shaped.
- Trial name
- NCT02090881
- Testing
- Ultrasound scan
- Where
- United Kingdom
- Volunteers
- 25 people
- Sponsor
- Imperial College Healthcare NHS Trust
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
Investigation of Platelet Aggregation in Paediatric Patients With Sickle Cell Disease
A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor in Paediatric Patients With Sickle Cell Disease
The purpose of this Phase II dose-ranging study is to investigate pharmacokinetic (PK) and pharmacodynamic (PD) properties of various doses of ticagrelor followed by 4 weeks of twice-daily treatment in paediatric patients with…
- Trial name
- HESTIA 1 (NCT02214121)
- Testing
- Ticagrelor Dose 1a + Dose 2a, Ticagrelor Dose 1b + Dose 2b
- Where
- 18 sites in United States, Canada and 4 more
- Volunteers
- 46 people
- Sponsor
- AstraZeneca
Thalassemia
Zinc & Bone Health in Thalassemia: The Think Zinc Study
The purpose of this study is to test whether zinc can improve bone health in young patients with thalassemia.
- Trial name
- ThinkZn (NCT00459732)
- Testing
- Zinc
- Where
- 3 sites in United States
- Volunteers
- 45 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
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
Sickle Cell Anemia
Evaluation of the AMICUS RBCx System in Sickle Cell Patients
The purpose of this study is to evaluate the performance of the AMICUS Red Blood Cell Exchange (RBCx) System (Exchange and Depletion/Exchange procedures) in patients with sickle cell disease.
- Trial name
- NCT02372877
- Testing
- Amicus Red Cell Exchange in SCD patients
- Where
- 6 sites in United States
- Volunteers
- 83 people
- Sponsor
- Fenwal, Inc.
Sickle Cell Disease
Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features.
- Trial name
- STRIDE (NCT01565616)
- Testing
- Conditioning Regimen with Bone Marrow Transplant
- Where
- 8 sites in United States
- Volunteers
- 22 people
- Sponsor
- Emory University
Sickle Cell Disease, Pulmonary Hypertension
Sildenafil Therapy for Pulmonary Hypertension and Sickle Cell Disease
This study will examine whether the drug sildenafil can lower blood pressure in the pulmonary artery (the blood vessel that leads from the heart to the lungs) in patients with sickle cell disease and pulmonary hypertension (high…
- Trial name
- NCT00492531
- Testing
- Sildenafil
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 74 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Beta-thalassemia, Iron Overload
Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.
- Trial name
- NCT00447694
- Testing
- Deferasirox
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Novartis
Sickle Cell Disease, Thalassemia
Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere
Primary Objective 1. Define the pharmacokinetics of liquid-formulated HU in infants (9 months to <2 years) 2. Assess the relative bioavailability of HU "sprinkles" compared to capsules in children and adolescents (≥2 to 18…
- Trial name
- NCT03825341
- Testing
- Hydroxyurea, Hydroxyurea Oral Capsule
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Safety and Efficacy of Sodium Nitrite in Sickle Cell Disease
This study will determine if administration of sodium nitrite is safe and can improve small vessel blood flow and tissue oxygenation when given as an additional treatment in patients with acute vaso-occlusive crisis (pain crisis)…
- Trial name
- NCT01033227
- Testing
- sodium nitrite injection, usp
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital Los Angeles
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
Sickle Cell Disease, Thalassemia
A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen
Hypothesis 1: A novel nonmyeloablative condition regimen will be safe and efficacious in producing stable donor chimerism and cure of severe hemoglobinopathy.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemoglobinopathies
- Trial name
- NCT00427661
- Testing
- Busulfan; Fludarabine; cyclosporine A and MMF
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Pittsburgh
Sickle Cell Disease
Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises
The purpose of this study was to determine whether the investigational drug SelG1 when given to sickle cell disease patients either taking or not taking hydroxyurea was effective in preventing or reducing the occurrence of pain…
- Trial name
- SUSTAIN (NCT01895361)
- Testing
- SelG1
- Where
- 56 sites in United States, Brazil and 1 more
- Volunteers
- 198 people
- Sponsor
- Reprixys Pharmaceutical Corporation
Sickle Cell Disease
Regional Anesthesia for Sickle Cell Crisis Using Ultrasound in The Emergency Department: Phase I
The hypothesis of this study is: Femoral nerve blocks can feasibly be performed on patients with Sickle Cell Disease and painful crisis in the Emergency Department.
- Trial name
- RESCUED (NCT01256281)
- Testing
- Femoral Nerve Block
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Sickle Cell Disease, Thalassemia
Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.
- Trial name
- NCT02504619
- Testing
- CordIn
- Where
- 3 sites in United States, France
- Volunteers
- 1 people
- Sponsor
- Gamida Cell ltd