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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

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

Pilot Study to Assess Safety Preliminary Efficacy and Pharmacokinetics of S.C. APL-2 in PNH Subjects

This is a Phase Ib, open-label, multiple ascending dose, pilot study in patients with PNH who have not received eculizumab (Soliris) in the past. Two cohorts of 3 subjects are planned for evaluation. Safety will be assessed throughout the study; serial blood and urine samples will be collected for these ...

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

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

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

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

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

Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNH

Evaluation of the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria

Phase