Clinical trials: Anemia, Neonatal
Sickle Cell Disease
A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease
Majority of patients who are eligible for allogeneic HSCT for cure of severe sickle cell disease lack a matched family donor. This study aims for cure of sickle cell disease by performing unrelated donor (outside family)…
- Trial name
- NCT01279616
- Testing
- Fludarabine monophosphate, Rituximab, Busulfan
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Nationwide Children's Hospital
Hemolytic Disease of the Fetus and Newborn
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Pregnant Women at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (HDFN)
The purpose of this study is to evaluate the safety in mother and neonate/infant of M281 administered to pregnant women who are at high risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (EOS-HDFN).
- Trial name
- NCT03842189
- Testing
- M281
- Where
- 19 sites in United States, Australia and 7 more
- Volunteers
- 25 people
- Sponsor
- Janssen Research & Development, LLC
Fanconi Anemia
Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
RATIONALE: Giving chemotherapy, such as cyclophosphamide and fludarabine, before a donor stem cell transplant helps to remove the patient's cells to allow for the transplant cells to take and grow.
- Trial name
- NCT00630253
- Testing
- Anti-Thymocyte Globulin, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease
Treatment of Sickle Cell Anemia With Stem Cell Transplant
This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor.
- Conditions
- Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease, Sickle Cell-β0-thalassemia
- Trial name
- NCT01350232
- Testing
- Fludarabine, Cytarabine, Cellular Infusions
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Sickle Cell Disease
A GBT021601 ADME Microtracer Study in Healthy Volunteers
An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.
- Trial name
- NCT05718687
- Testing
- GBT021601
- Where
- Netherlands
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Pfizer
Non Malignant Disorders, Immunodeficiencies
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients < 21…
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies
- Trial name
- NCT00744692
- Testing
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Duke University
Sickle Cell Disease, Sickle Cell Anemia
Sickle Cell Disease (SCD) Bone Pain Study
A prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Low Bone Density, Osteoporosis
- Trial name
- NCT05283148
- Testing
- Dual-energy X-ray absorptiometry, Vertebral fracture analysis, Adult Sickle Cell Quality of Life Measurement System pain impact questionnaire
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- University of California, Davis
Sickle Cell Disease
Preventing Acute Chest Syndrome by Transfusion Feasibility Study
Acute chest syndrome (ACS) is similar to severe pneumonia and is a common cause of hospitalizations for people with sickle cell disease (SCD). Blood transfusions are one treatment option for ACS.
- Trial name
- PROACTIVE (NCT00951808)
- Testing
- Single blood transfusion, Standard care
- Where
- 24 sites in United States
- Volunteers
- 237 people
- Sponsor
- Carelon Research
Sickle Cell Disease (SCD)
Pharmacokinetics and Pharmacodynamics Study of SEG101 (Crizanlizumab) in Sickle Cell Disease (SCD) Patients With Vaso- Occlusive Crisis (VOC)
The purpose of the CSEG101A2202 study was to characterize the Pharmacokinetic (PK) and Pharmacodynamic (PD) of SEG101/crizanlizumab and to evaluate the safety and efficacy of SEG101/crizanlizumab in sickle cell disease (SCD)…
- Trial name
- NCT03264989
- Testing
- crizanlizumab
- Where
- 12 sites in United States
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Anemia, Sickle Cell
Daily Vitamin D for Sickle-cell Respiratory Complications
This study aims to answer the question whether daily oral vitamin D supplementation can reduce the risk of respiratory or lung complications in children and adolescents with sickle cell disease.
- Conditions
- Sickle Cell Disease, Anemia, Sickle Cell, Anemia, Hemolytic, Congenital, Respiratory Tract Diseases
- Trial name
- ViDAS-2 (NCT04170348)
- Testing
- Daily oral vitamin D3, 3,333 IU, Monthly oral vitamin D3, 100,000 IU
- Where
- United States
- Volunteers
- 58 people
- Sponsor
- Columbia University
Sickle Cell Anemia
Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies
This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or…
- Trial name
- NCT03020615
- Testing
- Hydroxyurea
- Where
- 4 sites in United States
- Volunteers
- 58 people
- Sponsor
- St. Jude Children's Research Hospital
Malaria, Vivax, G6PD Deficiency
Assessing a Risk Model for G6PD Deficiency
A clinical study designed to develop and inform an individual risk of hemolysis model based on individual red blood cell G6PD levels.
- Trial name
- NCT03337152
- Testing
- primaquine, chloroquine + primaquine
- Where
- Thailand
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- PATH
Thalassemia
Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Thalassemia intermedia (TI) is an inherited blood disorder that can cause anemia due to low levels of hemoglobin. Decitabine is a medication that may be effective at increasing hemoglobin levels.
- Trial name
- NCT00661726
- Testing
- Decitabine (USAN, INN)
- Where
- 3 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- Carelon Research
G6PD Deficiency
Validation of Diagnostics to Identify Glucose-6-phosphate Dehydrogenase Activity in the US
The primary objective of this study is to assess the accuracy of the SD Biosensor STANDARD G6PD Analyzer in measuring G6PD activity when used by trained health care workers.
- Trial name
- NCT04010695
- Testing
- SD Biosensor G6PD Analyzer, Pointe Scientific Test Kit, HemoCue System
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- PATH
Anemia, Sickle Cell, Sickle Cell Disease
Hydroxyurea and EPO in Sickle Cell Disease
The proposed study is a Phase 1/2 multi-center study evaluating the safety and efficacy of erythropoietin (EPO) in combination with hydroxyurea in the treatment of chronic anemia in patients with sickle cell disease (SCD).
- Trial name
- ACHiEvE-SCD (NCT05451940)
- Testing
- Hydroxyurea, Epoetin Alfa-BioSimilar
- Where
- 2 sites in United States, Nigeria
- Volunteers
- 17 people
- Sponsor
- Julia Xu
Sickle Cell Disease
Music Therapy in Sickle Cell Pain Mixed Methods Study
The purpose of this research study is to: 1. Investigate the effects of a single 20-minute music therapy intervention with a music therapist on the pain intensity, pain relief, and mood of adult patients with sickle cell disease…
- Trial name
- NCT02270060
- Testing
- Music Therapy, Music Listening
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University Hospitals Cleveland Medical Center
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia
Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
Due to an overall and disease free survival of 85% to 100%, allogeneic blood or bone marrow stem cell transplantation using an HLA matched sibling donor is the therapy of choice for patients with severe aplastic anemia (SAA).
- Conditions
- Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
- Trial name
- NCT00244010
- Testing
- Allogeneic stem cell transplant
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- St. Jude Children's Research Hospital
Thalassemia
Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO
Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells.
- Trial name
- THALLO (NCT00578292)
- Testing
- Busulfan, Fludarabine, Campath 1H
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Baylor College of Medicine
Anemia, Sickle Cell, Pain
Intranasal Fentanyl for Initial Treatment of a Vaso-occlusive Crisis
The purpose of this study is to determine if intranasal fentanyl can decrease the pain of patients with sickle cell disease who present to the pediatric emergency department with a vaso-occlusive crisis.
- Trial name
- NCT01482091
- Testing
- Fentanyl Citrate, Normal Saline
- Where
- United States
- Volunteers
- 124 people
- Sponsor
- Montefiore Medical Center
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited