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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 (17) 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

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

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

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

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

Study to Assess Safety Efficacy Pharmacokinetics and Pharmacodynamics of RO7112689 in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria

This is a Phase I/II, first-in-human study consisting of 3 sequential parts and an open-label extension (OLE) in which the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single-doses of RO7112689 will be evaluated in healthy volunteers (HV) during Part I, and safety, tolerability, PK, and PD of RO7112689 will ...

Phase

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

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

A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria

Subjects will be randomised in a 1:1 ratio to either treatment sequence. Subjects randomly assigned to treatment with SB12 or Soliris will receive 600 mg of eculizumab IV every week for first 4 weeks (initial phase) and 900 mg for the fifth week, followed by 900 mg every 2 weeks ...

Phase