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30 recruiting studies matching your filters

A Phase I Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life…

Sickle Cell DisordersSickle Cell DiseaseHemoglobin S+2 more

Estimated

~$10 – $5,625

National Heart, Lung, and Blood Institute (NHLBI)NCT05904093

Phase 1

Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of he…

Sickle Cell DiseaseThalassemiaHemoglobinopathy

Estimated

~$10 – $5,625

National Heart, Lung, and Blood Institute (NHLBI)NCT06313398

Phase 1

A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime

The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient f…

Metabolic DisordersHematologic, Immune, or Bone Marrow DisordersHemoglobinopathies+1 more

Estimated

~$10 – $6,250

Washington University School of MedicineNCT00920972

Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Cell Transplant or Gene Therapy

This is a prospective, longitudinal, non-therapeutic study which includes routine assessment for long-term effects, as per FDA guidelines after receipt of an a…

Hemoglobin Disorder

Estimated

~$10 – $750

St. Jude Children's Research HospitalNCT06646640

Phase 2

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Ste…

Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)Paroxysmal Nocturnal Hemoglobinuria (PNH)

Estimated

~$10 – $5,625

National Heart, Lung, and Blood Institute (NHLBI)NCT03520647

Phase 1

Buprenorphine for the Management of Pain in Children With Sickle Cell Disease

The goal of this clinical trial is to learn whether a single dose of buccal buprenorphine (BELBUCA) is safe when used in place and/or with full agonist opioids…

Sickle Cell Disease (SCD)Sickle Cell Disease Vaso-occlusive CrisisSickle Cell Anemia Crisis+4 more

Estimated

~$10 – $7,500

Natasha ArcherNCT07799571

Hematopoietic Cell Transplant and Gene Therapy for Non-Malignant Blood Disorders Biobank Resource (BMT CTN 2402)

A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant…

Aplastic AnemiasHemoglobinopathiesBone Marrow Failure

Estimated

~$10 – $750

Medical College of WisconsinNCT07227155

Phase 3

An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age

The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess wheth…

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Estimated

~$10 – $8,750

Novartis PharmaceuticalsNCT06934967

Prospective Observational Data Collection Study for Validation of a Near-Infrared Spectroscopy-Based Blood Hemoglobin Algorithm in Pediatric Patients Undergoing Cardiac Surgery or Cardiac Catheterization

This study will enroll children and young adults who are scheduled to undergo planned heart surgery or a heart catheterization procedure while under general an…

HemoglobinCerebral OxygenationCongenital Heart Disease

Estimated

~$10 – $750

Boston Children's HospitalNCT07706140

Observational

Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)

This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) in pati…

Hematologic DisordersHemoglobinopathiesImmunodeficiencies

Estimated

~$10 – $6,250

Masonic Cancer Center, University of MinnesotaNCT01666080

Phase 2

Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Non-Malignant Hematological Disorders in Children

This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is s…

Hemoglobinopathy (Disorder)Severe Aplastic AnemiaBone Marrow Failure Syndrome

Estimated

~$10 – $6,250

Johns Hopkins All Children's HospitalNCT04356469

Phase 2

An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria

The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pe…

Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Hemoglobinuria

Estimated

~$10 – $8,750

Apellis Pharmaceuticals, Inc.NCT04901936