Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.
- Trial name
- NCT05036512
- Testing
- GBT021601
- Where
- 4 sites in United States, Australia
- Volunteers
- 129 people, healthy volunteers welcome
- Sponsor
- Pfizer
Sickle Cell Disease
Pilot Study on the Effects of Intravenous Ketamine on Acute Pain Crisis in Patients With Sickle Cell Disease
The purpose of this pilot study is to provide a preliminary assessment of the feasibility and efficacy of intravenous ketamine in controlling pain in patients with sickle cell disease (who are admitted to the hospital with…
- Trial name
- NCT00252122
- Testing
- Ketamine
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's Hospital of Philadelphia
Sickle Cell Disease
Re-Aiming at Hydroxyurea Adherence for Sickle Cell With mHealth
National Institutes of Health (NIH)/National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend that hydroxyurea be offered to symptomatic adults and all children with sickle cell disease (SCD) (HbSS and HbSβ0-thal…
- Trial name
- NCT03344900
- Where
- 2 sites in United States
- Volunteers
- 100 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease (SCD)
Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients
The purpose of this study was to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 2 to <18 years with a history of Vaso-Occlusive Crisis (VOC) with or without…
- Trial name
- NCT03474965
- Testing
- Crizanlizumab
- Where
- 35 sites in United States, Belgium and 12 more
- Volunteers
- 117 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease
This research study is designed to investigate a new potential medicine for mobilizing stem cells and apheresis collection in patients with Sickle Cell Disease. MGTA-145, the new potential medicine, will be given with plerixafor.
- Trial name
- NCT05445128
- Testing
- MGTA-145, Plerixafor
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- Ensoma
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
Hemoglobinopathies
Prevalence and Hematological Characteristics of Hemoglobinopathies
This study aimed to determine the prevalence and hematological characteristics of hemoglobinopathies in Mardan and surrounding districts of Khyber Pakhtunkhwa (KPK), Pakistan.
- Trial name
- NCT07180836
- Testing
- No Intervention (Observational Study)
- Where
- Pakistan
- Volunteers
- 839 people
- Sponsor
- Bacha Khan Medical College
Sickle Cell Disease, Cognitive Ability, General
Improving Parental Psychosocial Functioning and Early Developmental Outcomes in Children With Sickle Cell Disease
There are all significant risk factors for poor early cognitive development and, as such, neurocognitive deficits have been demonstrated in pre-school children with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Cognitive Ability, General, Psychological Stress, Parenting
- Trial name
- NCT02394899
- Testing
- Play therapy, Problem solving skills
- Where
- Jamaica
- Volunteers
- 64 people
- Sponsor
- The University of The West Indies
Sickle Cell Disease
Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions
The objective of this proposal is to test the feasibility of red blood cell (RBC) rejuvenation to chronic transfusion in sickle cell disease (SCD) and the potential benefit of RBC rejuvenation in this population to determine if a…
- Trial name
- NCT02731157
- Testing
- Rejuvesol, Blood transfusion
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Duke University
Sickle Cell Anemia
Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia
A total of fifty severely affected patients with homozygous sickle cell disease or other sickling disorders (e.g. B negative or B positive Thalassemia/Sickle) who are greater than 18 years of age will be eligible for treatment.
- Trial name
- NCT00001197
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Evaluation of Nocturnal Enuresis and Barriers to Treatment Among Pediatric Patients With Sickle Cell Disease
Pediatric patients with sickle cell disease are at greater risk for exhibiting nocturnal enuresis (bedwetting) compared to the general population.
- Trial name
- NCT01959958
- Testing
- Questionnaire/Interview
- Where
- United States
- Volunteers
- 250 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease, Leg Ulcers
Resolution of Sickle Cell Leg Ulcers With Voxelotor
This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease.
- Trial name
- RESOLVE (NCT05561140)
- Testing
- Voxelotor Oral Tablet
- Where
- 23 sites in Brazil, Kenya and 1 more
- Volunteers
- 88 people
- Sponsor
- Pfizer
Data Collection, Sickle Cell Disease
Data Collection For Patients With Sickle Cell Disease
To create a database of clinical information, participant /disease characteristics, and treatment history collected from patients with Sickle Cell Disease.
- Trial name
- NCT07911618
- Testing
- data collection
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- M.D. Anderson Cancer Center
Sickle Cell Disorders
GenoMed4ALL: Improving SCD Classification and Prognosis by AI
GenoMed4All 'Genomics and Personalized Medicine for all though Artificial Intelligence in Haematological Diseases' aims to advance on individual SCD patients' disease characterisation and to improve the monitoring of patients'…
- Trial name
- GenoMed4ALL (NCT06019208)
- Where
- 5 sites in France, Italy and 2 more
- Volunteers
- 1,000 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Pulmonary Hypertension, Lung Disease
Hospital-Based Program for Treatment of Severe Cardiopulmonary Disease With Inhaled Nitric Oxide
The purpose of this program is to evaluate the logistic issues and patient requirements for chronic pulsed INOmax delivery in ambulatory, home-care patients.
- Conditions
- Pulmonary Hypertension, Lung Disease, Sickle Cell Disease, Cardiac Transplant
- Trial name
- NCT00041574
- Testing
- Inhaled Nitric Oxide
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Mallinckrodt
Sickle Cell Disease, Kidney Diseases, Chronic
The Predictive Capacity of Machine Learning Models for Progressive Kidney Disease in Individuals With Sickle Cell Anemia
This is a multicenter prospective, longitudinal cohort study which will evaluate the predictive capacity of machine learning (ML) models for progression of CKD in eligible patients for a minimum of 12 months and potentially for…
- Trial name
- PREMIER (NCT05214105)
- Testing
- Biospecimen/DNA collection and analysis
- Where
- 3 sites in United States
- Volunteers
- 400 people
- Sponsor
- University of Tennessee
Sickle Cell Disease, Adverse Childhood Experiences
Early Life Exposures Among Children With Sickle Cell Disease
This study is being conducted to determine the relationship between early childhood exposures, such as Adverse Childhood Experiences, Social Determinants of Health and nutrition/breastfeeding, among children with sickle cell…
- Conditions
- Sickle Cell Disease, Adverse Childhood Experiences, Breastfeeding
- Trial name
- NCT05377372
- Testing
- Breastfeeding support group, Observation
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Alabama at Birmingham
Sickle Cell Disease
Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD
Study participants will undergo up to four periods of voxelotor administered orally at progressively higher dose levels from 1500 mg until either a maximum tolerated dose (MTD) or 3000 mg/day dose is reached, whichever occurs…
- Trial name
- NCT04247594
- Testing
- Voxelotor
- Where
- 6 sites in United Kingdom
- Volunteers
- 6 people
- Sponsor
- Pfizer
Sickle Cell Disease
Novel Dose Escalation to Predict Treatment With Hydroxyurea
Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, chronic anemia (a decrease in the number of red blood cells), and damage to body…
- Trial name
- NDEPTH (NCT02042222)
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Baylor College of Medicine
Sickle Cell Disease
Evaluation of Sickle Cell Liver Disease
Background: Sickle cell disease changes the shape of red cells. This makes them more likely to break down as they get stuck in small blood vessels.
- Trial name
- NCT01950429
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)