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Hemoglobinuria, Paroxysmal Clinical Trials

A listing of Hemoglobinuria, Paroxysmal medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (14) clinical trials

Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Phase N/A

Unrelated Donor Stem Cell Transplantation

The purpose of this study is to provide an opportunity for patients with malignancies or bone marrow failure states who lack a suitable sibling donor to undergo allogeneic hematopoietic progenitor cell transplantation using cells from unrelated individuals or cord blood registries.

Phase N/A

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

Phase N/A

A Phase 3 Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH)

The purpose of this study is to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy of ALXN1210 in pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH).

Phase

A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

The purpose of this study is to evaluate long term safety and efficacy of ACH-0144471 in patients with PNH who have demonstrated clinical benefit from ACH-0144471 in Clinical Study ACH471-100. This study is designed to include up to 12 patients.

Phase

Fludarabine Phosphate Cyclophosphamide Total Body Irradiation and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

PRIMARY OBJECTIVES: I. To evaluate the rate of relapse, defined as recurrence of underlying disease or progression of underlying disease, at 1 year in patients who receive haploidentical peripheral blood stem cells (PBSCs) after reduced intensity conditioning and post-transplant cyclophosphamide. SECONDARY OBJECTIVES: I. To evaluate safety including development of acute ...

Phase

Study of Safety Efficacy Tolerability Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

LNP023 is a novel oral small molecular weight compound, that inhibits alternative complement pathway (AP). Blockade of the AP with oral LNP023 has the potential to prevent both intra - and extravascular hemolysis. This study includes a screening period of up to 68 days, a baseline visit, up to approximately ...

Phase

Unrelated And Partially Matched Related Donor PSCT w/ TCR Depletion for Patients With BMF

This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes. Previously established, disease-specific transplant preparative regimens ...

Phase N/A

Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patients with active PNH who are treatment-naive to complement inhibitor therapy or have not recently received complement inhibitor therapy. The secondary objectives of the study are: To evaluate ...

Phase

Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576

rVA576, a small protein complement C5 inhibitor which prevents the cleavage of C5 by C5 convertase into C5a and C5b, will be used in an open label, non-comparative clinical trial in patients with PNH. Patients will be treated with rVA576 by daily subcutaneous injection in order to determine the safety ...

Phase