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

2019-09-16 03:26:45 | BioPortfolio

Summary

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

Study Design

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Intervention

APL-2

Location

Hospital Ampang
Ampang
Selangor
Malaysia
68000

Status

Recruiting

Source

Apellis Pharmaceuticals, Inc.

Results (where available)

View Results

Links

Published on BioPortfolio: 2019-09-16T03:26:45-0400

Clinical Trials [343 Associated Clinical Trials listed on BioPortfolio]

Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).

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 (PN...

Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria

This clinical study is a randomized, open-label, international, multi-center, comparative study of efficacy and safety of BCD-148 and Soliris® in PNH patients. It is planned to investiga...

The Molecular Biology of Paroxysmal Nocturnal Hemoglobinuria (PNH)

This study is designed to better understand the molecular biology of paroxysmal nocturnal hemoglobinuria (PNH) and to determine if prion protein (PrP) functions in long term hematopoietic ...

Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576

rVA576 for patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).

PubMed Articles [265 Associated PubMed Articles listed on BioPortfolio]

Off-Pump Coronary Revascularization Using Bilateral Internal Thoracic Arteries in A Patient with Paroxysmal Nocturnal Hemoglobinuria: A Case Report.

Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-orphan disease. We report the first case in the literature of Off-Pump Coronary Revascularization Using Bilateral Internal Thoracic Arteries in a ...

Gene mutations associated with thrombosis detected by whole-exome sequencing in paroxysmal nocturnal hemoglobinuria.

Thrombosis is a most common and lethal complication of paroxysmal nocturnal hemoglobinuria (PNH), which is a complex progression and its mechanism remains unclear. We tried to explore the possible gen...

Acute myocardial infarction in a Chinese patient with paroxysmal nocturnal hemoglobinuria: A case report.

Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal hematopoietic stem cell disease. Patients with PNH often experience a high incidence (14%-40%) of thrombotic events, which are mainly venous and r...

Outcomes of paroxysmal nocturnal hemoglobinuria in the pediatric age group in a resource-constrained setting.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal stem cell disorder. Eculizumab and bone marrow transplantation are disease-modifying treatments for PNH but may not be readily avail...

Successful cord blood transplantation for a paroxysmal nocturnal hemoglobinuria complicated with Budd-Chiari syndrome and myelodysplastic syndrome.

Medical and Biotech [MESH] Definitions

A condition characterized by the recurrence of HEMOGLOBINURIA caused by intravascular HEMOLYSIS. In cases occurring upon cold exposure (paroxysmal cold hemoglobinuria), usually after infections, there is a circulating antibody which is also a cold hemolysin. In cases occurring during or after sleep (paroxysmal nocturnal hemoglobinuria), the clonal hematopoietic stem cells exhibit a global deficiency of cell membrane proteins.

A parasomnia characterized by paroxysmal episodes of choreoathetotic, ballistic, dystonic movements, and semipurposeful activity. The episodes occur during non-rapid eye movement sleep and typically recur several times per night. (Neurology 1992 Jul;42(7 Suppl 6):61-67; Adams et al., Principles of Neurology, 6th ed, p391)

A disorder characterized by recurrent episodes of paroxysmal brain dysfunction due to a sudden, disorderly, and excessive neuronal discharge. Epilepsy classification systems are generally based upon: (1) clinical features of the seizure episodes (e.g., motor seizure), (2) etiology (e.g., post-traumatic), (3) anatomic site of seizure origin (e.g., frontal lobe seizure), (4) tendency to spread to other structures in the brain, and (5) temporal patterns (e.g., nocturnal epilepsy). (From Adams et al., Principles of Neurology, 6th ed, p313)

Involuntary shock-like contractions, irregular in rhythm and amplitude, followed by relaxation, of a muscle or a group of muscles. This condition may be a feature of some CENTRAL NERVOUS SYSTEM DISEASES; (e.g., EPILEPSY, MYOCLONIC). Nocturnal myoclonus is the principal feature of the NOCTURNAL MYOCLONUS SYNDROME. (From Adams et al., Principles of Neurology, 6th ed, pp102-3).

The presence of free HEMOGLOBIN in the URINE, indicating hemolysis of ERYTHROCYTES within the vascular system. After saturating the hemoglobin-binding proteins (HAPTOGLOBINS), free hemoglobin begins to appear in the urine.

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