Clinical trials: Sickle Cell Disorders
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
Gene Editing For Sickle Cell Disease
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a…
- Trial name
- NCT06506461
- Testing
- Plerixafor, Busulfan, Gene-modified CD34+ cells
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
BEATS 2: Music Therapy in Sickle Cell
The purpose of this study is to investigate the effects of the BEATS music therapy program on the self-efficacy, trust, knowledge, and adherence of young adult patients with SCD.
- Trial name
- NCT03233269
- Testing
- Music Therapy
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University Hospitals Cleveland Medical Center
Sickle Cell Disease (SCD)
Integrating Point of Care Testing (POCT) For Newborn Screening and Early Care for Sickle Cell Disease in Yopougon, Côte d'Ivoire
The goal of this clinical trial is to learn if a multifaceted intervention composed of Gazelle-Multispectral sickle cell disease (SCD) point of care testing (POCT) and early initiation of comprehensive SCD care in children with…
- Trial name
- STEP (NCT07488520)
- Testing
- Gazelle-Multispectral point of care testing for newborn sickle cell disease screening; and initiation of early comprehensive clinical interventions
- Where
- 2 sites in Côte d’Ivoire
- Volunteers
- 120 people
- Sponsor
- Swiss Tropical & Public Health Institute
Sickle Cell Disease, Vaso-Occlusive Pain Episode in Sickle Cell Disease
Intranasal Ketamine for Pain Control in Patients with Sickle Cell Disease and Vaso-occlusive Episode (VOE) in the PED
This will be a descriptive cohort study of intranasal ketamine as the initial analgesic for children with sickle cell disease who present to the pediatric emergency department with vaso-occlusive crisis and are awaiting…
- Conditions
- Sickle Cell Disease, Vaso-Occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis, Ketamine
- Trial name
- NCT04150757
- Testing
- Intranasal ketamine
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Abigail Nixon
Graft Failure, Sickle Cell Disease
Allo HSCT for High Risk Hemoglobinopathies
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation…
- Conditions
- Graft Failure, Sickle Cell Disease, Hemoglobinopathies
- Trial name
- NCT06872333
- Testing
- Alemtuzumab, Total Body Irradiation, Cell Infusion
- Where
- United States
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sickle Cell Disease
Best Noninvasive Predictor of Renal Function in Assessing Adult Sickle Nephropathy
Background: Sickle cell disease is a common inherited blood disorder. Kidney disease is a major cause of problems in people with sickle cell disease.
- Trial name
- NCT03958643
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Vaso-occlusive Crisis
Effect of Virtual Reality Technology for Pain Management of Vaso-Occlusive Crisis in Patients With Sickle Cell Disease
Acute vaso-occlusive crisis (VOC) is the most common complication in patients with sickle cell disease (SCD) and pain related to VOC is often inadequately treated.
- Trial name
- NCT03353584
- Testing
- Virtual Reality Therapy
- Where
- 2 sites in United States
- Volunteers
- 76 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease, Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- Trial name
- FLORAL (NCT06609226)
- Testing
- Etavopivat A, Etavopivat B, Etavopivat C
- Where
- 107 sites in United States, Canada and 15 more
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S
Pain, Sickle Cell Disorder
Ketamine Infusion for Acute Sickle Cell Crisis in the Emergency Department
Pain associated with sickle cell disease is a common emergency department visit. It is also frequently associated with a high emergency department recidivism rate for pain control and admissions to the hospital.
- Trial name
- KISS (NCT02417298)
- Testing
- Normal saline, Ketamine
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Billy Sin
Cancer, COPD
SELF-BREATHE RCT for Chronic Breathlessness
A feasibility RCT comprising two groups: 1. Intervention (SELF-BREATHE in addition to standard NHS care) 2. Control group (standard / currently available NHS care)
- Conditions
- Cancer, COPD, Asthma, Bronchiectasis Adult
- Trial name
- NCT04574050
- Testing
- SELF-BREATHE
- Where
- United Kingdom
- Volunteers
- 52 people
- Sponsor
- King's College Hospital NHS Trust
Hemoglobinopathies, Thalassemia
Hemoglobinopathy Nursing Program and Pediatric Nursing Students
Hemoglobinopathies are the most common life threatening, monogenic disorders in the world. The most common causes of hemoglobinopathies are sickle cell disease and thalassemia.
- Conditions
- Hemoglobinopathies, Thalassemia, Sickle Cell Disease, Nurse's Role
- Trial name
- NCT05389891
- Testing
- Hemoglobinopathy Nursing Program
- Where
- Egypt
- Volunteers
- 108 people, healthy volunteers welcome
- Sponsor
- Beni-Suef University
Chronic Pain, Depression, Anxiety
Cognitive Behavioral Therapy and Real-Time Pain Management Intervention for Sickle Cell Via Mobile Applications
The investigators are conducting a comparative effectiveness trial among adult patients with sickle cell disease (SCD) who report chronic pain (N = 350), randomized to receive either mobile phone-delivered computerized cognitive…
- Conditions
- Chronic Pain, Depression, Anxiety, Opioid Use
- Trial name
- CaRISMA (NCT04419168)
- Testing
- cCBT, m-Education
- Where
- 7 sites in United States
- Volunteers
- 359 people
- Sponsor
- University of Pittsburgh
Sickle Cell Disease and Thalassemia
Allograft for Sickle Cell Disease and Thalassemia
The design of the study incorporates the following features: 1. This is a phase II study to determine the safety and therapeutic potential of a new transplant approach (disease-free survival, graft versus myeloma effect) and to…
- Trial name
- NCT02038478
- Testing
- Donor Stem Cell Transplantation
- Where
- United States
- Sponsor
- University of Texas Southwestern Medical Center
Sickle Cell Disease
Crizanlizumab Pregnancy Outcomes Intensive Monitoring
This was a non-interventional study to evaluate pre-specified pregnancy and infant outcomes in women treated with crizanlizumab during pregnancy or within 105 days before the last menstrual period (LMP).
- Trial name
- PRIM (NCT07554547)
- Where
- Switzerland
- Volunteers
- 13 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Acute Chest Syndrome
Use of Macrolides in Acute Chest Syndrome: A Multicenter Retrospective Study
This study looks at whether using macrolide antibiotics is helpful and safe in treating acute chest syndrome, a serious complication in people with sickle cell disease.
- Conditions
- Sickle Cell Disease, Acute Chest Syndrome, Glucose-6-Phosphate Dehydrogenase Deficiency, Anti-Bacterial Agents
- Trial name
- NCT07037706
- Where
- France
- Volunteers
- 250 people
- Sponsor
- Centre Hospitalier de Saint-Denis
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
Pain, Sickle Cell Disease
Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain
The study, known as the Peer suppoRt for adolescents and Emerging adults with Sickle cell pain: promoting ENgagement in Cognitive behavioral thErapy (PRESENCE), aims to determine the effectiveness of digital CBT in reducing pain…
- Trial name
- PRESENCE (NCT06374238)
- Testing
- CBT+ Health coach, CBT w/o Health Coach ( self-guided), Usual Care
- Where
- 14 sites in United States
- Volunteers
- 390 people
- Sponsor
- University of Pittsburgh
Sickle Cell Disease
The Implementation of the Automated Erythrocytapheresis in Egyptian Sickle Cell Disease Center
Improvements of health infrastructure, preventive care and clinical treatment have reduced the morbidity and mortality of sickle cell disease (SCD).
- Trial name
- NCT03903289
- Testing
- Automated red cell exchange, Manual red cell exchange, Simple red cell transfusion
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Ain Shams University
Chronic Pain, Sickle Cell Disease
MBI for Pain and Sleep in Adolescents and Young Adults With Sickle Cell Disease
This study examines the efficacy of a mindfulness-based intervention designed to support pain and sleep management in adolescents and young adults with sickle cell disease.
- Conditions
- Chronic Pain, Sickle Cell Disease, Sleep
- Trial name
- REMedy_RCT (NCT07797582)
- Testing
- Mindfulness-based intervention
- Volunteers
- 48 people
- Sponsor
- University of Illinois Chicago