Clinical trials: Sickle Cells Disease
Pulmonary Hypertension, Sickle Cell Anemia
Secondary Pulmonary Hypertension in Adults With Sickle Cell Anemia
The purpose of this study is to determine how often people with sickle cell anemia develop pulmonary hypertension a serious disease in which blood pressure in the artery to the lungs is elevated.
- Conditions
- Pulmonary Hypertension, Sickle Cell Anemia, Sickle Cell Disease
- Trial name
- NCT00011648
- Where
- 2 sites in United States
- Volunteers
- 986 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell, Diabetes
Developing the Family Map: Looking at Communal Coping
Background: Knowing one s family medical history is a part of staying healthy. Some health risks run in families, and knowing these risks can promote more healthy behavior.
- Conditions
- Sickle Cell, Diabetes, Cancer, Cardiovascular Disease
- Trial name
- NCT01633021
- Where
- 3 sites in United States
- Volunteers
- 1,061 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Sickle Cell Disease
Pathophysiology of Acute Pain in Patients With Sickle Cell Disease
Background: Sickle Cell Disease (SCD) is a blood disorder that occurs mainly in people of African descent. Researchers want to learn more about the painful attacks and complications associated with SCD.
- Trial name
- NCT03049475
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Anemia, Sickle Cell Thalassemia
Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen; this in turn can injure organs including the heart, lungs, and kidneys.
- Conditions
- Sickle Cell Anemia, Sickle Cell Thalassemia, Sickle Cell Pain, Hbss
- Trial name
- NCT05675436
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
- Conditions
- Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia
- Trial name
- NCT05777993
- Testing
- Mitapivat
- Where
- 23 sites in Belgium, Brazil and 6 more
- Volunteers
- 55 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
The main purpose of this study is to compare the effect of tebapivat versus placebo on anemia and to detect a dose-response for hemoglobin (Hb) response in participants with SCD.
- Trial name
- NCT06924970
- Testing
- Tebapivat
- Where
- 20 sites in United States, Belgium and 5 more
- Volunteers
- 59 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease
Open-Label Extension Study to Pioneer Study 6058-SCD-101
This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder…
- Trial name
- NCT07401823
- Testing
- Pociredir
- Where
- 8 sites in United States
- Volunteers
- 1 people
- Sponsor
- Fulcrum Therapeutics
Sickle Cell Disease
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.
- Trial name
- NCT07656415
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 159 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512.
- Trial name
- NCT06546670
- Testing
- ITU512
- Where
- 14 sites in United States, Brazil and 1 more
- Volunteers
- 161 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Hydroxyurea Optimisation in Sickle Cell Disease
The wide interindividual variability in clinical response to hydroxyurea therapy in the management of sickle cell disease has limited its use.
- Trial name
- PHOENIX-NG (NCT07543289)
- Testing
- Hydroxyurea (HU)
- Where
- Nigeria
- Volunteers
- 100 people
- Sponsor
- Ochuko Orherhe
Sickle Cell Disease
Blood Collection for Research Related to Certain Diseases Involving Blood Vessels
The collection of human specimens from both patients and healthy volunteers is necessary for the development of laboratory assays to further basic and clinical research studies.
- Trial name
- NCT00047996
- Where
- 2 sites in United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Trait, Sickle Cell Disease
Blood Sampling for Research Related to Sickle Cell Disease
This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease.
- Conditions
- Sickle Cell Trait, Sickle Cell Disease, Sickle Cell Anemia
- Trial name
- NCT00542230
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sickle Cell Disease
Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell…
- Trial name
- NCT03279094
- Testing
- Hematopoietic stem cell transplantation
- Sponsor
- City of Hope Medical Center
Sickle Cell Disease
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to…
- Trial name
- NCT05031780
- Testing
- Mitapivat
- Where
- 91 sites in United States, Belgium and 14 more
- Volunteers
- 286 people
- Sponsor
- Agios Pharmaceuticals, Inc.
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
Sickle Cell Disease, Sickle Cell Trait
CHOICES3: Sickle Cell Disease Parenting CHOICES
The study will use web-based data collection (SCKnowIQ) and intervention delivery strategies enhanced by nudges and tailored boosters in a sample of 430 adult men and women, aged 18-45 yr with SCD (Sickle Cell Disease) or SCT…
- Trial name
- CHOICES3 (NCT05292781)
- Testing
- CHOICES, eBook
- Where
- United States
- Volunteers
- 506 people
- Sponsor
- University of Florida
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
The primary objective of this multicenter observational registry is to characterize the clinical, epidemiological, and biological profiles of individuals of Middle Eastern, North African, and Levantine ancestry, including…
- Conditions
- Antiphospholipid (aPL)-Positive, Rheumatic Arthritis, Celiac Disease, Focal Segmental Glomerulosclerosis (FSGS)
- Trial name
- MERIT (NCT07892651)
- Where
- Jordan
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Axiom Bioregistry
Mortality in Sickle Cell, Sickle Cell Cardiopulmonary Complications
Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies
Background: People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers want to learn how and why this happens.
- Conditions
- Mortality in Sickle Cell, Sickle Cell Cardiopulmonary Complications, Sickle Cell Organ Damage, Sickle Cell Life Expectancy and Risk Factors for Early Death
- Trial name
- NCT05213572
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Trans-auricular Nerve Stimulation as an Innovative Approach to the Treatment of Pain in Pediatric Patients Suffering From Sickle Cell Disease
The goal of this study is to evaluate the effects of transauricular vagus and trigeminal nerve stimulation on the treatment of pain in children with Sickle Cell Disease.
- Trial name
- NCT07090668
- Testing
- tAN device
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Emory University
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris