Clinical trials: Sickle Cell, active
Sickle Cell Disease
Development and Evaluation of an Information Management System and Communication System for Population-wide Point-of-care Infant Sickle Cell Disease Screening
Although over 75% of children with sickle cell disease (SCD) are born in sub-Sahara where the disease highly contributes to under-5 mortality and causes life-long debilitation, evidence-based strategies to control SCD are not…
- Trial name
- SIMCS SCD (NCT06563440)
- Testing
- Digital app and information system
- Where
- 2 sites in United States, Uganda
- Volunteers
- 24,000 people, healthy volunteers welcome
- Sponsor
- Makerere University
Myeloid Chimerism
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some…
- Trial name
- NCT04008368
- Testing
- CliniMACS CD34 Reagent
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Sickle Cell Disease, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
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.
Vaso-Occlusive Pain Episode in Sickle Cell Disease, Sickle Cell Disease (SCD)
Low Dose Bolus Ketamine For Use In Sickle Cell Pain Crisis
The goal of this study is to learn if Ketamine works more efficiently, as compared to Opioids, for Sickle Cell Pain The main questions it aims to answer are: Does Ketamine lower the number of times participants need to be…
- Trial name
- NCT07682662
- Testing
- Standardized Low-dose bolus IV Ketamine within 30 minutes of arrival and every 1 hour as needed for max of 3 doses., Standard Care (in control arm)
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- University of Mississippi Medical Center
Sickle Cell Disease
Early Screening and Treatment of Heart Complication in Sickle Cell Disease
This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.
- Trial name
- NCT07023666
- Testing
- Deferoxamine, Deferasirox, Deferiprone
- Where
- 2 sites in United States
- Volunteers
- 100 people
- Sponsor
- Inova Health Care Services
Sickle Cell Disease, Pulmonary Disease
Cooperative Assessment of Late Effects for SCD Curative Therapies
Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States.
- Conditions
- Sickle Cell Disease, Pulmonary Disease, Renal Disease, Heart Disease
- Trial name
- COALESCE (NCT05153967)
- Where
- 5 sites in United States
- Volunteers
- 377 people
- Sponsor
- Vanderbilt University Medical Center
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Sickle Cell Disease
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).
- Trial name
- PRECISE (NCT06923111)
- Testing
- Xromi
- Where
- 12 sites in Germany, United Kingdom
- Volunteers
- 180 people
- Sponsor
- Nova Laboratories Limited
Sickle Cell Disease
Long-term Endurance Training in Sickle Cell Disease Patients: Impact on Clinical Profile, Physical Fitness, and Quality of Life.
Sickle cell disease (SCD), the most common genetic disease worldwide and in France, is an inherited haemoglobinopathy characterised by chronic haemolytic anaemia, vaso-occlusive crisis (VOC), acute pain, and multi-organ damage.
- Trial name
- EXDRE2 (NCT06823219)
- Testing
- endurance training program
- Where
- France
- Volunteers
- 120 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease (SCD), Vaso-Occlusive Crises
Pleuropulmonary Ultrasound Imaging in Patients With Sickle Cell Disease in a Vaso-occlusive Crisis. "ECHODREP"
Introduction Sickle cell disease is the most common inherited genetic disorder in France and worldwide. Its main acute manifestation is vaso-occlusive crisis, a frequent reason for emergency department visits.
- Conditions
- Sickle Cell Disease (SCD), Vaso-Occlusive Crises, Acute Chest Syndrome
- Trial name
- ECHODREP (NCT07782489)
- Testing
- Pleuropulmonary ultrasound
- Volunteers
- 200 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
Sickle Cell Disease
Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy.
- Trial name
- NCT06155500
- Testing
- OTQ923
- Where
- 3 sites in United States
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly deoxyribonucleic acid (DNA)) from the patient to treat his…
- Trial name
- NCT07708350
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Daniel Bauer
Sickle Cell Disease
Exploring Adherence Monitoring in Sickle Cell Disease
Despite the well-documented benefits of hydroxyurea (HU) therapy in decreasing morbidity and mortality in youth with Sickle cell disease (SCD), pediatric HU adherence rates range as low as 49% and lead to discontinuation of HU…
- Trial name
- NCT03708731
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke
The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke.
- Trial name
- HIBISCUS 3 (NCT05953584)
- Testing
- Etavopivat, Hydroxyurea
- Where
- 9 sites in India, Nigeria and 1 more
- Volunteers
- 27 people
- Sponsor
- Forma Therapeutics, Inc.
Sickle Cell Disease
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).
- Trial name
- NCT04055818
- Testing
- Nicotinamide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- EpiDestiny, Inc.
Vaso-Occlusive Pain Episode in Sickle Cell Disease
Lidocaine for Opioid Sparing in Vaso-occlusive Crisis of Sickle Cell Disease
The purpose of the study is to determine whether adding lidocaine to standard of care in pain management during severe vaso-occlusive crisis has an effect on the cumulative opioid consumption expressed as morphine milligram…
- Trial name
- LidoVOC (NCT07274254)
- Testing
- Lidocain, Standard of care
- Where
- 12 sites in France, Guadeloupe
- Volunteers
- 104 people
- Sponsor
- Nantes University Hospital
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
The goal of this clinical trial is to learn whether a single dose of buccal buprenorphine (BELBUCA) is safe when used in place and/or with full agonist opioids that would otherwise be started during a sickle cell pain crisis…
- Conditions
- Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children
- Trial name
- NCT07799571
- Testing
- Buprenorphine Buccal Film [Belbuca]
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Natasha Archer