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13 recruiting studies matching your filters

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β- Transfusion-Dependent Thalassemia

The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusi…

Transfusion-dependent Alpha-ThalassemiaTransfusion-dependent Beta-Thalassemia

Estimated

~$10 – $8,750

Agios Pharmaceuticals, Inc.NCT07506863

Phase 3

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β-Non-Transfusion-Dependent Thalassemia

The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusio…

Non-Transfusion-dependent Alpha-ThalassemiaNon-Transfusion-dependent Beta-Thalassemia

Estimated

~$10 – $8,750

Agios Pharmaceuticals, Inc.NCT07517133

Phase 1

Phase 1/2 Study Evaluating the Safety and Efficacy of Gene Therapy Employing Lentiviral Vector ALS20-transduced Hematopoietic Progenitor Cells in Subjects With Transfusion-dependent-thalassemia

The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem ce…

Beta-Thalassemia

Estimated

~$10 – $6,250

Children's Hospital of PhiladelphiaNCT06364774

Phase 1

Matched Related Donor Non-myeloablative Hematopoietic Cell Transplantation With Alemtuzumab, 400 cGy TBI, and Abatacept for Sickle Cell Disease and Beta-Thalassemia

This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The int…

Sickle Cell DiseaseBeta-thalassemia

Estimated

~$10 – $5,625

National Heart, Lung, and Blood Institute (NHLBI)NCT07599176

Phase 1

T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may…

Sickle Cell AnemiaBeta-thalassemia MajorDiamond-blackfan Anemia

Estimated

~$10 – $7,500

Paul SzabolcsNCT03653338

Phase 2

A Phase 2a Study to Evaluate the Safety and Pharmacokinetics of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be cond…

A Phase 3b, Open-label, Single-arm, Rollover Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants: * Participants receiving l…

Myelodysplastic Syndromes (MDS)Beta-thalassemiaMyeloproliferative Neoplasm(MPN)-Associated Myelofibrosis

Estimated

~$10 – $7,500

CelgeneNCT04064060

A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)

The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in th…

Beta-Thalassemia

Estimated

~$10 – $1,050

Genetix Biotherapeutics Inc.NCT06271512

Phase 2

Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion - NYMC 588

Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the s…

Acute LeukemiaSevere Aplastic AnemiaNon-hodgkin Lymphoma+6 more

Estimated

~$10 – $7,500

Mitchell CairoNCT04099966

A Single-Site Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical resear…

Estimated

~$10 – $1,050

Sanguine BiosciencesNCT05635266

Phase 3

A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will eval…

Beta-ThalassemiaThalassemiaHematologic Diseases+4 more

Estimated

~$10 – $8,750

Vertex Pharmaceuticals IncorporatedNCT05477563

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…

Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more

Estimated

~$10 – $1,125

xCuresNCT06539169