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AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADOLESCENT PATIENTS WITH PLASMA THERAPY-SENSITIVE ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS)

Objectives: The following trial objectives for adolescent patients (from 12 and up to 18 years of age) with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:* Assess the effect of eculizumab on TMA-Event Free status…

AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADOLESCENT PATIENTS WITH PLASMA THERAPY-RESISTANT ATYPICAL HEMOLYTIC-UREMIC SYNDROME (AHUS)

Objectives: The following trial objectives for adult patients with plasma therapy-resistantAtypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:• Assess the effect of eculizumab to reduce thrombotic microangiopathy (TMA) asindicated by…

AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADULT PATIENTS WITH PLASMA THERAPY-RESISTANT ATYPICAL HEMOLYTIC-UREMIC SYNDROME (AHUS)

Objectives: The following trial objectives for adult patients with plasma therapy-resistantAtypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:• Assess the effect of eculizumab to reduce thrombotic microangiopathy (TMA) asindicated by…

An Open-label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients with Atypical Hemolytic-Uremic Syndrome

Assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.

An Open-Label, Multi-Center Clinical Trial of Eculizumab in Adult Patients with Atypical Hemolytic-Uremic Syndrome

Assess the efficacy of eculizumab in adult patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.

An Open-Label, Multi-Center Controlled Clinical Trial of Eculizumab
in Adult Patients with Plasma Therapy-Sensitive Atypical Hemolytic-Uremic Syndrome (AHUS)

Objectives: The following trial objectives for adult patients with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS) are to:Primary:* Assess the effect of eculizumab on TMA-Event Free status defined as the absence of [1] decrease in…

Coversin in paroxysmal nocturnal haemoglobinuria (PNH) in patients with resistance to eculizumab due to complement C5 polymorphisms (VIP study 578)

To determine the safety and efficacy of coversin in the treatment of patients with PNH resistant to eculizumab.

A PIVOTAL, OPEN-LABEL, MULTICENTER STUDY TO ASSESS THE EFFICACY AND SAFETY OF BIVV009 IN PATIENTS WITH PRIMARY COLD AGGLUTININ DISEASE WHO HAVE A RECENT HISTORY OF BLOOD TRANSFUSION

Part APrimary Objective:• The primary objective of Part A is to determine whether BIVV009 administration results in a >= 2 g/dL increase in hemoglobin (Hgb) levels or increases Hgb to >= 12 g/dL and obviates the need for blood…

An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria

The objectives of the study are to assess safety and tolerability, pharmacokinetics (PK), and biological activity (including efficacy and pharmacodynamics [PD]) of multiple subcutaneous doses of pegcetacoplan in pediatric patients with paroxysmal…

An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase II and Phase III studies with iptacopan

This study has been transitioned to CTIS with ID 2023-509843-28-00 check the CTIS register for the current data. The purpose of this study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to…

rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

* The purpose of this study is to to determine the safety profile of long-termCoversin treatment.* To observe the long term safety and efficacy of Coversin over periods in excess of 6 months* To assess the long term patient acceptability of Coversin…

A PHASE 3, OPEN-LABEL STUDY OF ALXN1210
IN CHILDREN AND ADOLESCENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)

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