Active clinical trials for "Hemoglobinuria"

The purpose of this study is to enable continued access to zilucoplan (RA101495) for patients with paroxysmal nocturnal hemoglobinuria (PNH) after they complete a zilucoplan clinical study.

The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria (PNH). The secondary objectives of the study are: To evaluate the long-term effect of REGN3918 on intravascular hemolysis To assess the concentrations of total REGN3918 in serum To evaluate the occurrence of the immunogenicity of REGN3918

The purpose of this research study is to understand the safety, pharmacokinetics and pharmacodynamics of a single dose of TT30 (ALXN1102 and ALXN1103 formulations) when given IV (through a vein) or SC (under the skin) to patients with PNH.

Patients with diseases requiring complement inhibition who have previously taken part in Akari clinical trials and who wish to continue to receive rVA576 (Coversin) after their active participation in the parent trial has completed and patients treated under compassionate use or named patient arrangements who wish to continue on rVA576 (Coversin) therapy.

The trial is the first human trial. The safety, tolerability, PK and PD of MY008211A Tablets will be evaluated in healthy subjects.

This is a single-center, randomized, double-blind, placebo-controlled, dose-escalation, and multiple-dose study to evaluate safety, tolerability, PK and PD of MY008211A Tablets in healthy subjects.

This was a single-center, single-dose, open-label clinical study. 12 subjects were randomly assigned in a 1:1 ratio to one of the following dosing sequences (sequence 1: AB; Sequence 2: BA). Each dosing sequence consisted of two cycles, one dose per cycle, with a 5-day washout period between doses.

The objectives of the study are to assess the safety, tolerability, preliminary efficacy and PK of multiple subcutaneous (SC) doses of pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who have not received treatment with eculizumab in the past. An exploratory objective of the study is to assess the pharmacodynamics (PD) of multiple SC doses of pegcetacoplan when administered to PNH patients.

This study will be the initial exploration of pegcetacoplan in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of pegcetacoplan will guide decisions to further develop the drug.

The primary objective of this study is to evaluate pharmacokinetics (PK) of ravulizumab administered subcutaneously via an on-body delivery system (OBDS) compared with intravenously administered ravulizumab in adult participants with PNH who are clinically stable on eculizumab for at least 6 months.