Clinical trials: Anemia, Neonatal
Sickle Cell Anemia, Sickle - Beta Thalassemia
Hepcidin Levels in Sickle Cell Disease (SCD)
The investigators propose that patients with HbSβ-thalassemia have lower levels of hepcidin and higher levels of GDF-15 than HbSS patients during the non-crisis, "steady states." In addition, the investigators propose that when…
- Trial name
- NCT02258997
- Volunteers
- 42 people
- Sponsor
- Kenneth Ataga, MD
Anemia, Sickle Cell
Gene Transfer for Patients With Sickle Cell Disease
The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and success of gene correction in subjects with sickle cell disease
- Trial name
- NCT02186418
- Testing
- ARU-1801
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Sickle Cell Anemia in Children
PINPOINT: Gaming Technology for SCD Pain
Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet programs to assist SCD adolescents with better identification and communication about pain are lacking.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia in Children, Sickle Cell Thalassemia, Sickle Cell SC Disease
- Trial name
- Pinpoint II (NCT04579926)
- Testing
- Pinpoint app
- Where
- 2 sites in United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Klein Buendel, Inc.
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum
Sickle Cell Disease (SCD)
Self-administered Acupressure in Patients With Sickle Cell Disease
The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupressure analgesia in patients with sickle cell disease (SCD).
- Trial name
- NCT07875777
- Testing
- Verum Acupressure (using AcuWand)
- Where
- 2 sites in United States
- Volunteers
- 300 people
- Sponsor
- University of Cincinnati
Beta Thalassemia Major
the Potential Immunomodulatory Effects of Spirulina on Thalassemic Children
The aim of this study is to evaluate the possible immunologic effects of spirulina in children with beta thalassemia.
- Trial name
- NCT02674607
- Testing
- Spirulina
- Where
- Egypt
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Tanta University
Beta-thalassemia, Myelodysplastic Syndromes
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions.
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan
- Trial name
- NCT00061763
- Testing
- Deferasirox
- Where
- 6 sites in United States
- Volunteers
- 175 people
- Sponsor
- Novartis Pharmaceuticals
Investigation of Platelet Aggregation in Paediatric Patients With Sickle Cell Disease
A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor in Paediatric Patients With Sickle Cell Disease
The purpose of this Phase II dose-ranging study is to investigate pharmacokinetic (PK) and pharmacodynamic (PD) properties of various doses of ticagrelor followed by 4 weeks of twice-daily treatment in paediatric patients with…
- Trial name
- HESTIA 1 (NCT02214121)
- Testing
- Ticagrelor Dose 1a + Dose 2a, Ticagrelor Dose 1b + Dose 2b
- Where
- 18 sites in United States, Canada and 4 more
- Volunteers
- 46 people
- Sponsor
- AstraZeneca
Thalassemia Major
Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation
Hypothesis: Taurine, in combination with standard iron chelation therapy, is more effective than chelation therapy alone in reducing cardiac iron overload, oxidative stress and cardiac damage in β-Thalassemia.
- Trial name
- TICATS (NCT04291352)
- Testing
- Taurine
- Where
- Canada
- Volunteers
- 60 people
- Sponsor
- University Health Network, Toronto
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Transfusion Dependent Thalassemia
Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE)
This is a prospective, multi-center, randomized, crossover trial to evaluate the clinical effectiveness of red blood cells (RBCs) derived from Mirasol-treated whole blood (WB) versus conventional RBCs in transfusion dependent…
- Trial name
- PRAISE (NCT03329404)
- Testing
- Mirasol Red Blood Cells (MIR RBCs), Reference Red Blood Cells (REF RBCs)
- Where
- 9 sites in United States, Israel and 2 more
- Volunteers
- 9 people
- Sponsor
- Terumo BCTbio
Sickle Cell Disease
Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease
The aim of this study is to evaluate the overall safety and feasibility of using haploidentical or one antigen mismatch unrelated hematopoietic stem cell transplant (HSCT) for adult patients with severe sickle cell disease (SCD)…
- Trial name
- NCT02678143
- Testing
- Alemtuzumab, Cyclophosphamide, Mycophenolate mofetil
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Washington University School of Medicine
Non-Neoplastic Hematologic and Lymphocytic Disorder
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases.
- Trial name
- NCT00919503
- Testing
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine
- Where
- 6 sites in United States
- Volunteers
- 98 people
- Sponsor
- Fred Hutchinson Cancer Center
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Acute Chest Syndrome
Utility of the Cardiac Electrical BiomarkerDisease
This project is aiming to identify the diagnostic utility CEB (Cardiac Electrical Biomarker) in patients who are undergoing cardiac investigations.
- Trial name
- VECTRA (NCT03498105)
- Where
- United Kingdom
- Volunteers
- 800 people
- Sponsor
- Joanne Turner
Sickle Cell Disease
Pulmonary Functions in Sickle Cell Disease: Response to Acu-TENS
pulmonary complications are common in sickle cell disease patients. Respiratory training using inspiratory muscle trainer (IMT) is usually a good choice to improve these complications.
- Trial name
- NCT06757829
- Testing
- inspiratory muscle training and acupucnture like transcutanoeus electrical nerve stimulation, inspiratory muscle training
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Cairo University
Beta-Thalassemia Trait, Coronary Artery Disease
B-Thalasaemia Trait and Its Possible Cardioprotective Role
Our purpose is to investigate whether thallasaemia trait offers protection against cardiovascular events and whether this can be applied to specific characteristics of these people ( better lipid profile , better blood pressure…
- Trial name
- NCT02428023
- Where
- Greece
- Volunteers
- 2,500 people, healthy volunteers welcome
- Sponsor
- 404 General Military Hospital, Larissa
Fanconi Anaemia
Non-invasive Characterisation of Oral Carcinomas in Patients With Fanconi Anaemia
Patients with Fanconi anaemia have an increased risk of developing squamous cell carcinoma of the head and neck. In addition, their genetic vulnerability limits the use of genotoxic treatments such as radiotherapy and…
- Trial name
- Fanc-Oral (NCT07242261)
- Testing
- Oral brushing
- Volunteers
- 115 people
- Sponsor
- Institut Jean-Godinot
FANCONI ANEMIA
Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCT
While stem cell transplantation has proven an effective means of treating a wide variety of diseases involving hematopoietic stem cells and their progeny, a shortage of donors has proved a major impediment to the widest…
- Trial name
- RAFHAS (NCT00586274)
- Testing
- CD34 selected haploidentical PBSCT, Fludarabine, T cell infusion
- Where
- 2 sites in United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Fanconi's Anemia
Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia
OBJECTIVES: I. Determine the safety of transferring the Fanconi anemia complementation group C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconi's anemia, complementation group…
- Trial name
- NCT00005896
- Testing
- filgrastim, Autologous stem cells transduced with FACC retroviral vector, Autologous Stem Cell Transplantation
- Volunteers
- 6 people
- Sponsor
- University of Minnesota