An Open-label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

The primary endpoints (PEP) are change from Baseline (Index Study Baseline and separately open-label extension [OLE] Baseline) in the following measures: • Platelet count, renal function and transaminases • Adverse events (AE) • Use of concomitant medications • Vital signs and weight • Physical examination findings • Clinical laboratory tests • Electrocardiogram (ECG) parameters, The primary endpoints (PEP) are change from Baseline (Index Study Baseline and separately open-label extension [OLE] Baseline) in the following measures: • Thyroid panel tests • Coagulation tests • Inflammatory panel tests • Complement and immunogenicity tests

The secondary endpoints are Change from Baseline (Index Study Baseline and separately OLE Baseline) in the following measures: • Neuropathy Impairment Score (NIS) • Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire, The secondary endpoints are Change from Baseline (Index Study Baseline and separately OLE Baseline) in the following measures: Neuropathy Symptom and Change score (NSC) • Serum Transthyretin (TTR) concentration • Physical Component Summary score (PCS) of 36-Item Short Form Survey (SF-36), The secondary endpoints are Change from Baseline (Index Study Baseline and separately OLE Baseline) in the following measures: • Polyneuropathy disability score (PND) • Modified body mass index (mBMI) • Composite Autonomic Symptom Score-31 (COMPASS-31) • 5 Level EQ-5D (EQ-5D-5L)

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