Clinical trials: Sickle Cell Disorders
Sickle Cell Disease, Hemolytic Anemia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Background: Sickle cell disease (SCD) is a disorder that causes episodes of acute pain and progressive organ damage. Ways to manage SCD have evolved slowly. Treatments do not always work.
- Trial name
- NCT04610866
- Testing
- Mitapivat
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Implication of the Oxydative Stress in the Pathophysiology of Sickle Cell Anemia:
Despite important advances in the current understanding of sickle cell vaso-occlusion, the basis of its control and prevention remain partially unknown.
- Trial name
- STRESS (NCT03368924)
- Testing
- SCA patients (SS genotype)
- Where
- Guadeloupe
- Volunteers
- 38 people
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Hemoglobin SC Disease, Sickle Cell Anemia
A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders
Sickle cell anemia is a genetic disorder that results from a single nucleotide substitution in codon 6 of the beta-globin gene which, in the homozygous state, produces an abnormal hemoglobin that is prone to polymer formation…
- Trial name
- NCT00005783
- Testing
- Recombinant-methionyl human stem cell factor
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sickle Cell Disease, Vaso-occlusive Crisis
IMPACTS Trial: Investigation of the Modulation of Phospholipase in Acute Chest Syndrome
The study will be conducted at 15-20 US centers in a randomized, placebo-controlled, double-blind fashion. Enrollees will be hospitalized sickle cell disease (SCD) patients at-risk for acute chest syndrome (ACS) based on the…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Acute Chest Syndrome
- Trial name
- NCT00434473
- Testing
- A-001
- Where
- 6 sites in United States
- Volunteers
- 30 people
- Sponsor
- Anthera Pharmaceuticals
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Anorexia, Thalassemia
Clinical Diagnosis of Teenagers Admitted to Pediatric Departments
This study intends to analyze the clinical characteristics of teenagers admitted to pediatric wards due to organic illness and non organic diagnosis, including patients suffering from chronic diseases and comorbidity.
- Conditions
- Anorexia, Thalassemia, Sickle Cell Anemia, Chronic Renal Failure
- Trial name
- NCT00735540
- Testing
- Observational
- Where
- Israel
- Volunteers
- 1,389 people
- Sponsor
- HaEmek Medical Center, Israel
Chest Pain, Sickle Cell Anemia
Effects of Nitric Oxide and Nitroglycerin in Patients With Sickle Cell Anemia
Sickle cell anemia is the most common genetic disease affecting African-Americans. About 1 in every 1000 African-Americans has the disease and 1 in every 12 carry the genes that could be passed on to their children.
- Trial name
- NCT00001716
- Testing
- Nitric Oxide, Nitroglycerin
- Where
- United States
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- National Institutes of Health Clinical Center (CC)
Sickle Cell Disease
Managed Access Programs for SEG101, Crizanlizumab
The purpose of this registration is to list Managed Access Programs (MAPs) related to SEG101, Crizanlizumab
- Trial name
- NCT03720626
- Testing
- crizanlizumab
- Sponsor
- Novartis Pharmaceuticals
Chronic Pain, Sickle Cell Disease
Focused Ultrasound Neuromodulation for Chronic Pain
This study will evaluate the feasibility, tolerability, and preliminary analgesic effects of individualized Precision Confidence Mapping-guided low-intensity focused ultrasound neuromodulation in adults with chronic pain.
- Conditions
- Chronic Pain, Sickle Cell Disease, Chronic Pelvic Pain Syndrome
- Trial name
- NCT07910396
- Testing
- Focused PCM-Guided CMN LIFU, Unfocused Control LIFU
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- University of Minnesota
Healthy Subjects, Sickle Cell Disease
A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease
The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of GBT440 compared with placebo in healthy subjects and subjects with sickle cell disease (SCD).
- Trial name
- NCT02285088
- Testing
- GBT440
- Where
- United Kingdom
- Volunteers
- 133 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics
Sickle Cell Disease
Pain Management in Children and Young Adults With Sickle Cell Disease
This is a phase II double-blind placebo-controlled clinical trial evaluating the effect of gabapentin when added to standard pain management for patients with sickle cell disease experiencing acute pain crisis in the ambulatory…
- Trial name
- NCT01954927
- Testing
- Gabapentin
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- St. Jude Children's Research Hospital
Stroke
Hydroxyurea to Prevent Stroke in Children With Sickle Cell Anemia and Elevated TCD Flow Velocity
The purpose of this study is to assess prospectively the efficacy of hydroxyurea therapy in the setting of cerebrovascular disease, manifest as conditional or abnormal transcranial doppler ultrasonography (TCD) flow velocities…
- Trial name
- NCT00402480
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Duke University
Acute Chest Syndrome, Sickle Cell Anemia
Awake Prone Positioning for Severe Acute Chest Syndrome
Acute chest syndrome (ACS) is the leading cause of admission to intensive care and the leading cause of death in patients with sickle cell disease.
- Trial name
- PRONE-ACS (NCT06698120)
- Testing
- Awake prone positioning (APP)
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Sickle Cell Anemia
A Low-Interventional Study of an Electronic Sickle Cell Disease Patient Reported Outcomes in Sickle Cell Participants
The purpose of this study is to learn more about painful crisis in people with Sickle Cell Disease. For this reason, Pfizer is conducting a study to understand how people with Sickle Cell Disease feel on a daily basis, treat…
- Trial name
- NCT06503458
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Sanguine Biosciences
Neutropenia, Sickle Cell Disease
Decitabine for High-Risk Sickle Cell Disease
Background: In sickle cell disease (SCD), the proteins in the red blood cells that carry oxygen do not behave normally. In parts of the body where there are low levels of oxygen or where oxygen is used more, the sickle hemoglobin…
- Trial name
- NCT01375608
- Testing
- Decitabine
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
A Study of IMR-687 in Subjects With Sickle Cell Disease
A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
- Trial name
- NCT04474314
- Testing
- IMR-687
- Where
- 49 sites in United States, Ghana and 11 more
- Volunteers
- 115 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Sickle Cell Anemia
Phase III Randomized Study of Poloxamer 188 for Vaso-Occlusive Crisis of Sickle Cell Disease
OBJECTIVES: I. Assess the efficacy of poloxamer 188 in reducing the duration of painful vaso-occlusive crisis in patients with sickle cell disease. II.
- Trial name
- NCT00004408
- Testing
- poloxamer 188
- Volunteers
- 300 people
- Sponsor
- Mast Therapeutics, Inc.
Sickle Cell Disease, Macula Thickening
Evolution of Sickle Cell Maculopathy
To investigate retrospectively the longitudinal evolution of vascular defects in patients with typical or atypical forms of SCM.
- Conditions
- Sickle Cell Disease, Macula Thickening, Macula Abnormality
- Trial name
- SCMevol (NCT07877753)
- Where
- France
- Volunteers
- 34 people
- Sponsor
- Christophe Orssaud
Sickle Cell Disease, Thalassemia, Beta
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow.
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia
- Trial name
- NCT05736419
- Testing
- Fludarabine, Cyclophosphamide, Tacrolimus
- Where
- 6 sites in United States
- Volunteers
- 24 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Sickle Cell Disease
Safety and Efficacy of Orally Administered NUV001 Nutraceutical Supplement in Sickle Cell Disease Patients
This multicenter, randomized, double-blind, parallel-group, placebo-controlled pilot study evaluated the safety, tolerability, and exploratory efficacy of orally administered NUV001 in adult participants with sickle cell disease…
- Trial name
- NCT05791591
- Testing
- NUV001 - IR, NUV001 - GR
- Where
- 9 sites in India
- Volunteers
- 168 people
- Sponsor
- LGD
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