Clinical trials: Sickle Cell Disorder
Sickle Cell Disease, Vaso-occlusive Crisis
Nitrous Oxide Analgesia Vaso-occlusive Crisis
Patients who have sickle cell VOC are usually treated with opioids, such as morphine. However, this current way of treating them has not improved the health, medical outcomes, or rates of hospitalizations.
- Trial name
- NCT01891812
- Testing
- Nitrous oxide 50%
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Columbia University
Bone Avascular Necrosis, Sickle Cell Anemia
Decompression Coring Versus Conservative Therapy in Patients With Avascular Necrosis of the Hip Related to Sickle Cell Disease
OBJECTIVES: I. Phase II trial to determine surgical morbidity of decompression coring, including any adverse events in the perioperative period and the rate of secondary medical or surgical interventions. II.
- Trial name
- NCT00006130
- Testing
- Core decompression
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.
- Trial name
- NCT05036512
- Testing
- GBT021601
- Where
- 4 sites in United States, Australia
- Volunteers
- 129 people, healthy volunteers welcome
- Sponsor
- Pfizer
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML)
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults.
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML)
- Trial name
- NCT02061800
- Testing
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Diane George
Sickle Cell Disease, Sickle Cell Crisis
The Use of Warmed Saline in Vaso-occlusive Episodes
The purpose of this study is to determine if warming the intravenous (IV) fluid given to patients with Sickle Cell Disease who are experiencing painful episodes known as Vaso-Occlusive Episodes; will decrease rates of hospital…
- Trial name
- NCT02316366
- Testing
- Astoflo Plus fluid warmer
- Volunteers
- 80 people
- Sponsor
- Nationwide Children's Hospital
Sickle Cell Trait
Effect of Exercise on Biomarkers in SCT
This study measures the effect of exercise on a variety of biomarkers in blood and urine selected to evaluate the physiological pathways of hemolysis, myolysis, thrombosis, inflammation, and renal function in subjects with sickle…
- Trial name
- NCT04273022
- Testing
- Exercise
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- St. Louis University
Sickle Cell Disease
The Role of Endothelin 1 as a Marker of Renal Impairment in Sickle Cell Disease
Sickle cell disease (SCD) refers to a group of hemoglobinopathies that include mutations in the gene encoding the beta subunit of hemoglobin.
- Trial name
- NCT06158945
- Testing
- urinary endothelin 1
- Where
- Egypt
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Sohag University
Sickle Cell Disease
Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions
The objective of this proposal is to test the feasibility of red blood cell (RBC) rejuvenation to chronic transfusion in sickle cell disease (SCD) and the potential benefit of RBC rejuvenation in this population to determine if a…
- Trial name
- NCT02731157
- Testing
- Rejuvesol, Blood transfusion
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Duke University
Sickle Cell Disease
Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders
The primary objectives of this prospective mixed-method interview study are to use semi-structured interviews in parents of sickle cell disease (SCD) patients to describe parental attitudes of research involving genomic…
- Trial name
- NCT04416178
- Where
- United States
- Volunteers
- 352 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Nano-rheological Biomarkers for Patients With Sickle Cell Disease (SCD) Versus Control Subjects (Other Constitutional Red Blood Cell Diseases and Healthy Subjects)
Numerous pathologies (sickle cell disease, thalassemia, spherocytosis, etc.) lead to changes in the rheological properties of the blood, in particular via alterations in the deformability of red blood cells.
- Trial name
- DREPNANO (NCT05530239)
- Testing
- Blood sample collection
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University Hospital, Grenoble
Anemia; Sickle-Cell, With Crisis, Acute Pain
Ketamine Infusion for Sickle Cell Pain Crisis
The purpose of this study is to prospectively study the efficacy of low dose ketamine infusions in treating patients who are admitted to the hospital with a sickle cell pain crisis.
- Trial name
- NCT04005209
- Testing
- Ketamine, Pain management
- Where
- United States
- Sponsor
- Duke University
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
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Sickle Cell Anemia, Cerebrovascular Accident
Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy
The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD) are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized…
- Trial name
- DREPAGREFFE (NCT01340404)
- Testing
- Stem cell transplantation, Transfusion program
- Where
- France
- Volunteers
- 63 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Hydroxyurea Failure
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human…
- Conditions
- Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies
- Trial name
- NCT05329649
- Testing
- CTX001
- Where
- 7 sites in United States, Germany and 2 more
- Volunteers
- 13 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Neutropenia, Sickle Cell Anemia
Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders
OBJECTIVES: I. Determine the efficacy of bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders. II.
- Conditions
- Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
- Trial name
- NCT00005893
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Fairview University Medical Center
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
Sickle Cell Disease
Improving Emergency Department Management of Adults With Sickle Cell Disease
The objective of this study is to design, implement and test quality improvement measures to improve the care of adults with sickle cell disease in the emergency department.
- Trial name
- NCT01603160
- Testing
- Quality Improvement
- Where
- United States
- Volunteers
- 715 people
- Sponsor
- Duke University
Sickle Cell Disease, Thalassemia
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who…
- Conditions
- Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy
- Trial name
- NCT03367546
- Testing
- Blood and Marrow Transplant
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Hematologic Malignancies, Nonmalignant Diseases
Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Melphalan is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of melphalan in their bodies and if drug…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609827
- Testing
- Melphalan
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
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