Clinical trials: SCD
Sickle Cell Disease
Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD).
- Trial name
- NCT03263559
- Testing
- Haploidentical Bone Marrow Transplantation, Hydroxyurea, Rabbit-ATG
- Where
- 32 sites in United States
- Volunteers
- 95 people
- Sponsor
- Medical College of Wisconsin
Sickle Cell Disease, Vaso-occlusive Crisis
Steroid Treatment for Sickle Cell Pain Crisis
The painful episode is the most common problem experienced by children with sickle cell disease. Although various treatments are available during painful episodes, the medication most commonly given for pain is a pain medication…
- Trial name
- NCT00263562
- Testing
- Steroid arm
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Baylor College of Medicine
Prematurity, Neurodevelopmental Disorder
Effect of Early L-Carnitine Supplementation on Neurodevelopmental Outcomes in Very Preterm Infants
Preterm infants are vulnerable to brain injury, nutritional deficiencies and poor early growth which places them at increased risk for developmental problems later in life.
- Conditions
- Prematurity, Neurodevelopmental Disorder, Carnitine Deficiency
- Trial name
- NCT01783041
- Testing
- L-carnitine, 5% Dextrose
- Where
- 2 sites in United States
- Volunteers
- 144 people
- Sponsor
- Montefiore Medical Center
Sickle Cell Disease, Vaso-Occlusive Crisis
Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive Crisis
Acute vaso-occlusive episodes (VOEs) in sickle cell disease (SCD) are primarily managed with opioids. Tolerance and hyperalgesia to opioids develops due to N-methyl-D-aspartate (NMDA)-receptor mediated activation of the…
- Trial name
- AKTSS (NCT03296345)
- Testing
- Ketamine
- Where
- United States
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Anemia
Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia
The study assesses the efficacy, safety and tolerability of ACZ885 (canakinumab) in pediatric and young adult patients with sickle cell anemia (SCA).
- Trial name
- NCT02961218
- Testing
- ACZ885
- Where
- 15 sites in United States, Canada and 5 more
- Volunteers
- 49 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)
The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease.
- Trial name
- NCT01794000
- Testing
- Prasugrel
- Where
- 50 sites in United States, Belgium and 12 more
- Volunteers
- 341 people
- Sponsor
- Eli Lilly and Company
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Sickle Cell Disease
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
- Trial name
- NCT05632354
- Testing
- Osivelotor
- Where
- 9 sites in United States, Nigeria
- Volunteers
- 47 people
- Sponsor
- Pfizer
Sickle Cell Disease, COVID-19
COVID-19 Vaccine Response in Sickle Cell Disease
The purpose of this study is to assess the antibody response to COVID-19 vaccination in a cohort of patients with sickle cell disease (SCD) and to assess vaccine and SCD related complications around the time of vaccination.
- Trial name
- NCT05139992
- Testing
- COVID-19 Vaccine
- Where
- 8 sites in United States
- Volunteers
- 59 people
- Sponsor
- ASH Research Collaborative
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
Acute Kidney Injury
Safety and Efficacy of a Selective Cytopheretic Device (SCD) in Pediatric Patients With Acute Kidney Injury (AKI).
The SCD (Selective Cytopheretic Device) is an extracorporeal device used as an adjunct to renal replacement therapy (RRT) to improve the outcomes of pediatric patients with acute kidney injury (AKI).
- Trial name
- SCD-PED-01 (NCT02820350)
- Testing
- SCD-F40
- Where
- 4 sites in United States
- Volunteers
- 19 people
- Sponsor
- CytoPherx, Inc
Nocturnal Enuresis, Anemia, Sickle Cell
Desmopressin for Bedwetting in Children With SCD
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.
- Trial name
- NCT04420585
- Testing
- Desmopressin
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Montefiore Medical Center
Sickle Cell Disease
High Dose Vitamin D for Sickle Cell Disease
Vitamin D deficiency (VDD) is very common among African American adolescents and adults in the US, ten times higher than is seen in Caucasians. VDD is also quite common in sickle cell disease (SCD).
- Trial name
- SCD-VitD (NCT01331148)
- Testing
- Vitamin D
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Wake Forest University Health Sciences
Hemochromatosis, Cerebrovascular Accident
Stroke With Transfusions Changing to Hydroxyurea
The purpose of this study is to compare standard therapy (transfusions and chelation) with alternative therapy (hydroxyurea and phlebotomy) for the prevention of secondary stroke and management of iron overload in children with…
- Conditions
- Hemochromatosis, Cerebrovascular Accident, Anemia, Sickle Cell, Hematologic Diseases
- Trial name
- NCT00122980
- Testing
- Red Cell Transfusions, Iron Chelation, Hydroxyurea
- Where
- 28 sites in United States
- Volunteers
- 134 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease
Voxelotor Neurocognitive Function Study
This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition…
- Trial name
- NCT05228834
- Testing
- Voxelotor Only Product in Oral Dose Form
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Pfizer
Sickle Cell Disease, Thalassemia
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation.
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT00957931
- Testing
- Bone marrow transplantation, Mesenchymal Stromal Cells
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
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 Anemia Crisis
Does IV Acetaminophen Reduce Opioid Requirement in Pediatric Patients With Acute Sickle Cell Crises?
The purpose of this study is to determine whether IV acetaminophen can decrease the need for subsequent opioid administration in the acute management of sickle cell crisis pain in the pediatric emergency room.
- Trial name
- NCT03541980
- Testing
- Acetaminophen, Normal saline
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- Newark Beth Israel Medical Center
Sickle Cell Disease
A Phase 1 Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest Syndrome
Sickle cell disease is a genetic red blood cell disorder characterized by vaso-occlusion from sickling of red blood cells, that can lead to pain or organ complications such as acute chest syndrome.
- Trial name
- NCT02697240
- Testing
- Intravenous citrulline
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Mississippi Medical Center
Sickle Cell Disease (SCD)
An Indian Multi-centric Phase IV Study to Assess the Safety of Crizanlizumab in Sickle Cell Disease Patients
Sickle cell disease (SCD) is a genetic blood disorder. Crizanlizumab is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in patients with SCD aged 16 years and older.
- Trial name
- NCT04662931
- Testing
- crizanlizumab
- Where
- 7 sites in India
- Volunteers
- 140 people
- Sponsor
- Novartis Pharmaceuticals