Clinical trials: Sickle Cell Anaemia
Sickle Cell Disease, Graft Versus Host Disease
Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease
To assess the tolerability of the costimulation blocking agent abatacept (CTLA4-Ig) when added to the standard graft versus host disease (GVHD) prophylaxis regimen of a calcineurin inhibitor and methotrexate in patients receiving…
- Trial name
- NCT02867800
- Testing
- Diphenhydramine, Acetaminophen, Methylprednisolone
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Monica Bhatia
Sickle Cell Disease, Thalassemia
Multi-Center Study of Iron Overload: Pilot Study
The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions…
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- MCSIO (NCT01114776)
- Where
- 3 sites in United States, Germany and 1 more
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem…
- Trial name
- NCT06287099
- Testing
- BRL-101
- Volunteers
- 5 people
- Sponsor
- Bioray Laboratories
Sickle Cell Disease
Improving Self-Management in Adolescents With Sickle Cell Disease
The objective of this study is to determine the feasibility and acceptability of SCThrive, a an innovative, technology-enhanced, group self-management intervention that uses a mixed in-person and online format and supported by a…
- Trial name
- SCThrive (NCT02851615)
- Testing
- SCThrive Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
- Trial name
- HOPE-KIDS (NCT02850406)
- Testing
- Voxelotor
- Where
- 20 sites in United States, Lebanon and 1 more
- Volunteers
- 147 people
- Sponsor
- Pfizer
Sickle Cell Disease, Virtual Reality
Role of Virtual Reality (VR) in Patients With Sickle Cell Disease (SCD)
Patients with sickle cell disease (SCD) and cancer often have complicated courses while hospitalized and often deal with pain, anxiety and depression.
- Conditions
- Sickle Cell Disease, Virtual Reality, Anxiety, Depression
- Trial name
- NCT03387033
- Testing
- Relaxation response and virtual reality (VR) session
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Duke University
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, 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
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
Sickle Cell Disease (SCD)
Exploratory Study on Global Reflexology in Sickle Cell Disease
Sickle cell disease (or sickle cell anemia) is the most common genetic disease in France with 586 children screened in 2019. This chronic disease is characterized by the presence of abnormal Hemoglobin (Hb) S and a deformation of…
- Trial name
- REFLEXODREPANO (NCT06619197)
- Testing
- Global reflexology
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Hospices Civils de Lyon
Cardiogenic Shock, Malignant Arrhythmias
The Caribbean Registry of Extracorporeal Membrane Oxygenation (ECMO) From the University Hospital in Martinique
The project's main goal is to collect baseline clinical and procedural data as well as to assess clinical outcomes for all patients undergoing VV, VA or VAV ECMO implantation in the French West Indies and Guiana.
- Conditions
- Cardiogenic Shock, Malignant Arrhythmias, Ischemic or Valvular Heart Failure, Drepanocytosis and Thoracic Syndrome
- Trial name
- NCT07351435
- Volunteers
- 1,200 people
- Sponsor
- University of Genova
Sickle Cell Disease
Quantitative MRI for Patients With Sickle Cell Disease Undergoing Hematopoietic Cell Transplant
The primary purpose of this research is to determine if it is feasible to perform serial magnetic resonance imaging (MRI) to evaluate the amount of bone marrow cells (also called cellularity) and iron stores before and after bone…
- Trial name
- NCT03458897
- Testing
- serial functional magnetic resonance imaging (MRI)
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of Michigan
Sickle Cell Disease, Sickle Cell Anaemia
Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype
SIL-8301 (senicapoc) is being developed for the chronic treatment of patients with sickle cell disease in both adults and children.
- Conditions
- Sickle Cell Disease, Sickle Cell Anaemia, Sickle Cell Anemia
- Trial name
- RESCUE (NCT07282210)
- Testing
- Senicapoc
- Where
- 4 sites in Canada
- Volunteers
- 65 people
- Sponsor
- Biossil Inc.
Sickle Cell Disease
A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of…
- Trial name
- HIBISCUS (NCT04624659)
- Testing
- Etavopivat Tablets Low dose, Etavopivat Tablets High dose, Etavopivat Tablets
- Where
- 142 sites in United States, Canada and 15 more
- Volunteers
- 450 people
- Sponsor
- Forma Therapeutics, Inc.
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.
Sickle Cell Crisis, Functional Abdominal Pain
Music Therapy: Sickle Cell and Pain Crisis
This study will assess whether participants who receive music therapy during inpatient care report a decrease in pain and whether music therapy influences factors such as family satisfaction.
- Conditions
- Sickle Cell Crisis, Functional Abdominal Pain, Patients Followed by the Pain Team
- Trial name
- NCT03901560
- Testing
- Music Therapy
- Volunteers
- 150 people
- Sponsor
- University of Oklahoma
Sickle Cell Disease, Chronic Kidney Diseases
Peer i-Coaching for Activated Self-Management Optimization in Adolescents and Young Adults With Chronic Conditions
The purpose of this study is to test the efficacy of a peer support coaching intervention to improve activated chronic illness self-management versus an attention control group in 225 adolescents and young adults with childhood…
- Conditions
- Sickle Cell Disease, Chronic Kidney Diseases, Systemic Lupus Erythematosus, Childhood Cancer
- Trial name
- PiCASO (NCT03938324)
- Testing
- PiCASO Intervention Group
- Where
- United States
- Volunteers
- 222 people
- Sponsor
- Duke University
Sickle Cell Anemia, Sickle Cell Disease
Sickle Cell Disease - Stroke Prevention in Nigeria Trial
Given large absolute numbers of individuals with sickle cell disease in Nigeria, hydroxyurea therapy for all individuals with sickle cell disease may not be initially feasible; however, a targeted strategy of hydroxyurea use for…
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Stroke
- Trial name
- SPIN (NCT01801423)
- Testing
- Hydroxyurea
- Where
- Nigeria
- Volunteers
- 29 people
- Sponsor
- Vanderbilt University Medical Center