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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 (7) clinical trials

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, 13 weeks of ...

Phase

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

The purpose of this study is to determine the safety and effectiveness of ACH-0144471 in currently untreated patients with PNH. This study is designed to include 4 to 12 patients.

Phase

Proof of Concept Study to Assess the Efficacy Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria

To determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with 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

A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to Eculizumab

The purpose of this study is to determine the effectiveness of ACH-0144471 in improving anemia, as measured by increased blood hemoglobin, when given with eculizumab (a drug commonly used for treatment of PNH) for 24 weeks in patients with PNH.

Phase

Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms

Coversin, 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 and proven resistance to eculizumab due to C5 polymorphisms. Patients will be treated with Coversin by ...

Phase