Clinical trials: Sickle Cell Disorders
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
Healthy Volunteers, Sickle Cell Disease
A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants
Novo Nordisk is developing a new study medicine, Etavopivat, to treat individuals with sickle cell disease (SCD). The purpose of the study is to determine the effect of Etavopivat on the electrical activity of the heart in…
- Conditions
- Healthy Volunteers, Sickle Cell Disease, Thalassemia
- Trial name
- NCT07023029
- Testing
- Etavopivat, Moxifloxacin
- Where
- United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Anemia, Sickle Cell
Trial of Oral Glutamine in Patients With Sickle Cell Anemia
Children with sickle cell anemia (SCA) seem to have higher energy needs than children who do not have the disease. This may be the reason why children and teenagers with sickle cell anemia tend to be smaller, weigh less, and have…
- Trial name
- NCT00131508
- Testing
- Glutamine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- St. Jude Children's Research Hospital
Patients With Thalassemia Intermedia,, Congenital Dyserythropoietic Anemia Type I
GDF 15 in Sickle Cell Disease and Hereditary Spherocytosis
Patients with thalassemia intermedia, congenital dyserythropoietic anemia type I , and sideroblastic anemia were found to express very high levels of serum GDF15, and this contributed to the inappropriate suppression of hepcidin…
- Trial name
- GDF 15 (NCT01201135)
- Volunteers
- 80 people
- Sponsor
- Wolfson Medical Center
Sickle Cell Disease, Thalassemia
Effects of Transfusion of Older Stored Red Cells
The purpose of this study is to determine the effects of transfusion of fresh and stored blood on patients. The investigators hope to test: whether a similar effect (older stored blood is associated with worse outcomes) is seen…
- Trial name
- NCT01534676
- Testing
- Transfusion, Blood, Chelation therapy
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Columbia 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)
Anemia, Sickle Cell, Sickle Cell Anemia
Phase 2 Study of MP4CO to Treat Vaso-occlusive Sickle Crisis
Sickle Cell disease is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body.
- Conditions
- Anemia, Sickle Cell, Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Disorders
- Trial name
- NCT01925001
- Testing
- MP4CO, Sodium chloride solution
- Where
- 14 sites in Bahrain, Belgium and 7 more
- Sponsor
- Sangart
Sickle Cell Disease
Epidemiology and Pathophysiological Mechanisms of HTAP in SS and SC Children in Martinique and Guadeloupe.
pulmonary arterial hypertension (PAH) has been reported with a prevalence of approximately 30% in adult sickle cell disease (SCD) patients, with an increased mortality in SCD patients with PAH, compared with those without PAH.
- Trial name
- SAPOTILLE (NCT03368261)
- Testing
- HTAP/ clinical complications in the sickle cell disease
- Where
- Martinique
- Volunteers
- 185 people
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Sickle Cell Disease, Vaso-occlusive Crisis
Outcomes of Vaso-occlusive Crisis in Pediatric Sickle Cell Disease
This study aims to assess the incidence and clinical outcomes of vaso-occlusive crises in pediatric patients with sickle cell disease. Data will be collected and analyzed to identify risk factors and related complications.
- Trial name
- NCT07200869
- Testing
- No intervention
- Where
- Egypt
- Volunteers
- 25 people
- Sponsor
- Assiut University
Brain Development Abnormality, Sickle Cell Disease
Brain Structure and Neurocognitive Development in Sickle Cell Disease; a Longitudinal Cohort Study (BRICK Study)
Sickle cell disease (SCD) is an autosomal recessive red blood cell blood disorder. One especially vital organ affected in SCD is the brain.
- Trial name
- BRICK (NCT05564845)
- Testing
- MR scan of the brain, Blood work, Neurological examination
- Where
- 2 sites in Netherlands
- Volunteers
- 84 people
- Sponsor
- Dr. Marjon H. Cnossen MD PhD
Sickle Cell Disease
Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease
The purpose of this study is to develop a safe and curative stem cell transplant approach to treating sickle cell disease by assessing the safety of haploidentical hematopoietic stem cell transplantation using αβ+ T-cell…
- Trial name
- NCT04207320
- Testing
- αβ+ T-cell depletion with Miltenyi CliniMACS system
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of Chicago
Sickle Cell Disease
Safety Evaluation of DREPADOM - Home Care Services and Hospitalizations for Sickle Cell Disease Patients
The objective of this study is to evaluate the incidence of ACS within the DREPADOM system and compare it to expected incidences of ACS (historic cohorts of PRESEV1 and PRESEV2)
- Trial name
- DREPADOM (NCT05649280)
- Where
- France
- Volunteers
- 100 people
- Sponsor
- Soutien aux Actions contre les Maladies du Globule Rouge
Pulmonary Hypertension, Sickle Cell Disease
Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
The primary hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease…
- Conditions
- Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
- Trial name
- NCT01048905
- Testing
- L-Glutamine
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
Development and Adaptation of I-STRONG for SCD
This study develops and tests the feasibility and acceptability of an adapted intervention, Integrative Strong Body and Mind Training (I-STRONG), in adolescents with pain from sickle cell disease.
- Trial name
- NCT06110754
- Testing
- I-STRONG for SCD
- Where
- 2 sites in United States
- Volunteers
- 45 people
- Sponsor
- Emory University
Organ Transplant, Cancer
ORCHARD- Optimising Home Assessment of Rural Patients
This project assesses feasibility of providing medically vulnerable rural patients with Medical-Self-Assessment-Boxes containing equipment to use at home during telephone and video consultations (telemedicine) with GPs and other…
- Conditions
- Organ Transplant, Cancer, Inborn Errors of Metabolism, Immunosuppression
- Trial name
- ORCHARD (NCT05732922)
- Testing
- A sphygmanometer, a digital thermometer, a pulse oximeter and a peak flow meter will be given to participants to use during their telemedicine consultations with health care providers.
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- University of Aberdeen
Sickle Cell Disease, Sickle Cell Hemoglobin C
Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study
The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among…
- Conditions
- Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
- Trial name
- NCT04678037
- Testing
- PROs assessment using sickle cell disease mobile app (SCD-app)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Sickle Cell Disease
Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor
With recent advances in gene editing, gene therapy is becoming a viable curative treatment option for sickle cell disease. In order to do genetic manipulation, investigators need to collect hematopoietic stem cells from patients…
- Trial name
- NCT04817345
- Testing
- Plerixafor
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease, Asthma
Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial
Acute and chronic pulmonary complications with concomitant inflammatory response are a leading cause of morbidity and mortality in children with sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Asthma, Acute Chest Syndrome
- Trial name
- NCT02187445
- Testing
- Budesonide inhalation suspension
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Vanderbilt University
Endocrine Disease, Infertility
Evaluation of Women With Endocrine and Reproductive-Related Conditions
This study was designed to allow inpatient and outpatient evaluation of women with a variety of reproductive and endocrine-related disorders for purposes of research and physician education.
- Conditions
- Endocrine Disease, Infertility, Leiomyoma, Endometriosis
- Trial name
- NCT00001850
- Where
- United States
- Volunteers
- 833 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease
The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD).
- Trial name
- Crescent (NCT01476696)
- Testing
- Prasugrel
- Where
- 9 sites in United States
- Volunteers
- 33 people
- Sponsor
- Eli Lilly and Company
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