17 results
Frequency of PNH in a selection of patients with DVT. In several publications PNH has been described especially in patients with DVT and younger than 30 years of age and/or with DVT on unusual places.
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…
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…
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…
Assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.
Assess the efficacy of eculizumab in adult patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.
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…
To study the role of GPI-deficiency in susceptibility of hematopoietic stem cells to cytotoxic T lymphocyte (CTL) and Natural Killer (NK) cell mediated lysis in the pathogenesis of PNH.
1.To create insight in current disease burden by creating a descriptive cohort of patients, diagnosed with rare congenital hemolytic anemia. Points of interest are:- Prevalence and incidence of disease- Quality of life- Prevalence and incidence of…
To determine the safety and efficacy of coversin in the treatment of patients with PNH resistant to eculizumab.
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…
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…
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…
To evaluate the prevalence of C-TMA in patients presenting with TMA, either with coexisting conditions or not. Furthermore, (i) the diagnostic performance of an in-house developed ex vivo test, (ii) dynamics of complement measures during follow-up,…
* 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…
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).
1. To assess the effect of increased hematocrit on cerebral perfusion as depicted by CBF and CVR.2. To determine the effect of GBT440 on oxygen saturation of the cerebral vasculature and the oxygen local consumption.3. Determine the dose response…