Clinical trials: Anemia
Sickle Cell Disease
Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell…
- Trial name
- NCT03279094
- Testing
- Hematopoietic stem cell transplantation
- Sponsor
- City of Hope Medical Center
Sickle Cell Disease
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to…
- Trial name
- NCT05031780
- Testing
- Mitapivat
- Where
- 91 sites in United States, Belgium and 14 more
- Volunteers
- 286 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Myelodysplastic Syndromes
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults.
- Trial name
- NCT06303193
- Testing
- pacritinib
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- National Cancer Institute (NCI)
Melanoma, Li-Fraumeni Syndrome
Clinical Genetics Branch Eligibility Screening Survey
Background: Clinical Genetics Branch (CGB) researchers study individuals and populations at high genetic risk of cancer in order to improve our understanding of cancer and to improve cancer care.
- Conditions
- Melanoma, Li-Fraumeni Syndrome, Pulmonary Blastoma, Chordoma
- Trial name
- NCT07005297
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- National Cancer Institute (NCI)
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Severe Aplastic Anemia, Telomere Biology Disorders
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Background: Bone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the diseases and treatments are lacking.
- Conditions
- Severe Aplastic Anemia, Telomere Biology Disorders, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT05012111
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Iron Deficiency Anemia
Effect of Vitamin D Supplementation Added to Iron Therapy in Children With Iron Deficiency Anemia
This randomized controlled trial will evaluate the effect of two vitamin D3 supplementation regimens, administered in addition to oral iron therapy, on hematological and iron status in children aged 6-60 months with confirmed…
- Trial name
- NCT07892170
- Testing
- Elemental iron, Vitamin D3 2000 IU/day, Vitamin D3 400 IU/day
- Where
- Iraq
- Volunteers
- 120 people
- Sponsor
- AlFayhaa General Hospital
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
The primary objective of this multicenter observational registry is to characterize the clinical, epidemiological, and biological profiles of individuals of Middle Eastern, North African, and Levantine ancestry, including…
- Conditions
- Antiphospholipid (aPL)-Positive, Rheumatic Arthritis, Celiac Disease, Focal Segmental Glomerulosclerosis (FSGS)
- Trial name
- MERIT (NCT07892651)
- Where
- Jordan
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Axiom Bioregistry
Sickle Cell Disease
Trans-auricular Nerve Stimulation as an Innovative Approach to the Treatment of Pain in Pediatric Patients Suffering From Sickle Cell Disease
The goal of this study is to evaluate the effects of transauricular vagus and trigeminal nerve stimulation on the treatment of pain in children with Sickle Cell Disease.
- Trial name
- NCT07090668
- Testing
- tAN device
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Emory University
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly deoxyribonucleic acid (DNA)) from the patient to treat his…
- Trial name
- NCT07708350
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Daniel Bauer
Hemolytic Disease of the Newborn, Fetal Anemia
Neonatal Outcomes on Direct Fetal Intravenous Immunoglobulin During Intrauterine Transfusion for Rhesus Isoimmunization
The goal of this clinical trial is to learn if giving intravenous immunoglobulin directly to the fetus during intrauterine transfusion can improve newborn outcomes in pregnancies affected by rhesus isoimmunization.
- Conditions
- Hemolytic Disease of the Newborn, Fetal Anemia, Rhesus Isoimmunization, Erythroblastosis Fetalis Due to RH Antibodies
- Trial name
- NCT07885592
- Testing
- Intrauterine Transfusion, Intravenous immunoglobulin (IVIG)
- Where
- India
- Volunteers
- 30 people
- Sponsor
- Post Graduate Institute of Medical Education and Research, Chandigarh
Aplastic Anaemia
Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA Receiving sUCBT
To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of Aplastic Anemia (AA)
- Trial name
- NCT07885982
- Testing
- Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®)
- Where
- China
- Volunteers
- 59 people
- Sponsor
- Anhui Provincial Hospital
Sickle Cell Disease
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years…
- Trial name
- NCT04774536
- Testing
- CRISPR_SCD001
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Mark Walters, MD
Sickle Cell Disease
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512.
- Trial name
- NCT06546670
- Testing
- ITU512
- Where
- 13 sites in United States, Brazil and 1 more
- Volunteers
- 161 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Hemolytic Anemia, Thalassemia
Native T1 Mapping and ECV in Chronic Hemolytic Anemia
This cross-sectional study aims to evaluate the diagnostic performance of native T1 mapping and extracellular volume fraction for the detection of myocardial iron overload and fibrosis in patients with chronic hemolytic anemia…
- Conditions
- Hemolytic Anemia, Thalassemia, Iron Overload
- Trial name
- NCT07882303
- Volunteers
- 54 people
- Sponsor
- Assiut University
Hematologic Disease and Disorders, Donors
Screening for Hematology Branch Protocols
This study allows the evaluation of subjects in order to determine their ability to safely participate in other active research studies.
- Conditions
- Hematologic Disease and Disorders, Donors, Healthy Volunteer
- Trial name
- NCT00001620
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Anemia in Children
Stroke Prevention With Hydroxyurea Enabled Through Research and Education (SPHERE)
This study will 1) Evaluate the prevalence of elevated (conditional or abnormal) transcranial Doppler (TCD) velocities in a cross-sectional analysis of children with Sickle Cell Anemia (SCA) living in Tanzania; 2) Obtain…
- Trial name
- NCT03948867
- Testing
- Hydroxyurea, Elevated Arm TCD Examination, Normal Arm TCD Examination
- Where
- 2 sites in United States, Tanzania
- Volunteers
- 202 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Severe Aplastic Anemia
A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)
Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant.
- Trial name
- TransIT (NCT05600426)
- Testing
- cyclosporine, Matched Unrelated Donor Hematopoetic Stem Cell Transplant, horse anti-thymocyte globulin (ATG)
- Where
- 52 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- Boston Children's Hospital
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia.
- Trial name
- NCT01174108
- Testing
- Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
- Where
- 2 sites in United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)