Clinical trials: Sickle Cell Disorders, open to volunteers
Sickle Cell Disease (SCD)
Phenotypic and Transcriptomic Description of Megakaryocytes in Sickle Cell Patient
Sickle cell disease is the most common inherited blood disorder in the world. Chronic hemolysis induces platelet activation and chronic inflammation.
- Trial name
- MEGADREP (NCT06887907)
- Where
- France
- Volunteers
- 40 people
- Sponsor
- University Hospital, Toulouse
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 Anemia, Beta-thalassemia Major
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent…
- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Trial name
- NCT03653338
- Testing
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Paul Szabolcs
Sickle Cell Disease, Glomerular Hyperfiltration
Morphine Clearance and Glomerular Filtration in Sickle Cell Patients in Crisis in Intensive Care
Background: Sickle cell disease is a genetic disorder of haemoglobin (which carries oxygen in red blood cells). The shape of sickle cell-patients' red blood cells is abnormal.
- Conditions
- Sickle Cell Disease, Glomerular Hyperfiltration, Sickle Cell Nephropathy, Vaso-Occlusive Crises
- Trial name
- PHEDREA (NCT07246265)
- Testing
- Injection of iohexol for glomerular filtration rate (GFR) measurement
- Where
- 6 sites in France
- Volunteers
- 100 people
- Sponsor
- University Hospital, Tours
Sickle Cell Disease, Executive Dysfunction
Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
The purpose of this study is to determine if patients with sickle cell disease (SCD) can consistently take a drug called Methylphenidate (MPH) daily, once a day for 4 weeks to help with any thinking, attention or schoolwork…
- Conditions
- Sickle Cell Disease, Executive Dysfunction, Cognitive Impairment, Attention Deficit/Hyperactivity Disorder (ADHD)
- Trial name
- NCT07226219
- Testing
- Extended-Release Methylphenidate
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Anemia in Children, Sickle Cell Disease
Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa
Sickle cell anemia (SCA) is among the world's most common and devastating blood disorders, affecting more than 300,000 newborns per year.
- Trial name
- PUSHUP (NCT05285917)
- Testing
- Hydroxyurea
- Where
- Angola
- Volunteers
- 400 people
- Sponsor
- Brown University
Bone Marrow Transplant (BMT)
HPC Offered for PRESERVE Expansion
Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons.
- Trial name
- HOPE (NCT07145411)
- Testing
- Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.
Venous Ulcer, Sickle Cell Ulcer
Fetal Cell Receptors Repertoire
The purpose of this study is to describe the transcriptomic profile of foetal cells in post-partum and more specifically to determine which chemokine receptors are overexpressed in foetal cells in post-partum women with wounds To…
- Conditions
- Venous Ulcer, Sickle Cell Ulcer, Diabetic Ulcer, Post-partum Women
- Trial name
- MoreCCR (NCT06031714)
- Testing
- Saliva sampling, Blood sampling, Interviews
- Where
- France
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Anemia
Serial Assessment of Fertility Experiences
The SAFE study is a long-term research project that watches people with sickle cell anemia (SCA) over time. The main goal is to see how a medicine called hydroxyurea affects their growth, puberty, and ability to have children.
- Trial name
- SAFE (NCT07116772)
- Where
- 3 sites in Dominican Republic, Jamaica and 1 more
- Volunteers
- 250 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Cognitive Impairment
Minocycline In Neurocognitive Outcomes - Sickle Cell Disease
Sickle cell disease (SCD) is a common, inherited blood disorder that primarily affects people of African Ancestry. It has a lot of complications including neurological complications.
- Conditions
- Sickle Cell Disease, Cognitive Impairment, Cognitive Decline, Cognitive Change
- Trial name
- MINO-SCD (NCT05605366)
- Testing
- Minocycline
- Volunteers
- 30 people
- Sponsor
- University of Cincinnati
Sickle Cell Disease
Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare…
- Trial name
- NCT05810181
- Testing
- Interview
- Where
- United States
- Volunteers
- 145 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Hemoglobinopathy, Sickle Cell Disease
A Long-term Follow-up Study in Patients Who Received BEAM-101
This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing…
- Trial name
- NCT07373639
- Testing
- Long-Term Follow-up Study of patients who received BEAM-101
- Where
- 17 sites in United States
- Volunteers
- 50 people
- Sponsor
- Beam Therapeutics Inc.
Hematologic Malignancy, Solid Tumor
Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy
The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular…
- Conditions
- Hematologic Malignancy, Solid Tumor, Sickle Cell Disease, Aplastic Anemia
- Trial name
- NCT06689800
- Testing
- Let's Get REAL
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Washington University School of Medicine
Sickle Cell Crisis
Sickle Cell Improvement: Enhancing Care in the Emergency Department
Sickle cell disease (SCD) is an inherited blood disorder affecting approximately 36,000 children in the United States, approximately 90% of whom are Black.
- Trial name
- SCIENCE (NCT05373771)
- Testing
- Care pathway
- Where
- United States
- Volunteers
- 5,328 people
- Sponsor
- Medical College of Wisconsin
Sickle Cell Disease
Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA)
This study will evaluate the acceptability, feasibility, and preliminary efficacy of a shared decision making intervention for adolescents and young adults (AYAs) with sickle cell disease (SCD).
- Trial name
- EMPOWER-AYA (NCT05383911)
- Testing
- SCD Shared Decision-Making Toolkit for AYAs - including VR, SCD Shared Decision-Making Toolkit for AYAs - without VR
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Nemours Children's Clinic
Sickle Cells Patients
Study of Vitamin B12 Metabolism in Children With Sickle Cell Disease Exposed to MEOPA
Short description of the protocol intended for the lay public. Include a brief statement of the study hypothesis (Limit : 5000 characters) The sickle cells anemia is a monogenic disease linked to the presence of Hemoglobin S due…
- Trial name
- DREPAM (NCT06581900)
- Testing
- Vitamin B12
- Where
- France
- Volunteers
- 29 people
- Sponsor
- CHU de Reims
Sickle Cell Disease
Writing Relaxing Beats in Adolescents Who Have Sickle Cell Disease
This research aims to see if songwriting can help reduce anxiety in adolescents with Sickle Cell Disease. The purpose of the study is to discover if participants find songwriting and playing their songs to be practical and…
- Trial name
- NCT07222475
- Testing
- Songwriting intervention (active music therapy)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Mayo Clinic
Sickle Cell Disease, Vaso-occlusive Crisis
Adaptive Platform Trial for Pain Management in Sickle Cell Vaso-Occlusive Crisis
Sickle cell disease (SCD) is a severe hemoglobinopathy, characterized by recurrent vaso-occlusive crises (VOC) causing intense pain and frequent hospitalizations in adult patients.
- Trial name
- PAMAVOC (NCT07826988)
- Testing
- Group 02 Paracetamol + Morphine + Ketamine, Group 01 Control: Paracetamol + Morphine, Group 03 : Paracetamol + Morphine + Nefopam
- Volunteers
- 400 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients
The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell…
- Trial name
- TRANSPLANTORN2 (NCT06351462)
- Testing
- Spermogram, Hospital Anxiety and Depression Scale (HADS), SF36 Quality of life questionnaire
- Volunteers
- 220 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Pilot Study on the Value of Bedside Pleuropulmonary Ultrasound in Patients With Sickle Cell Disease Presenting With Vaso-occlusive Crisis
Describe the proportion of patients with major sickle cell syndrome in vaso-occlusive crisis presenting at least one pleuropulmonary ultrasound abnormality during one of the ultrasounds performed at D0, between D2 and D5 during…
- Trial name
- DREPP (NCT06755385)
- Testing
- Pleuropulmonary ultrasound
- Volunteers
- 25 people
- Sponsor
- University Hospital, Grenoble