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The Validation of the CAP-PRI (Chronic Acquired Polyneuropathy-Patient Reported Index) in Patients with Acquired and Idiopathic Polyneuropathy
Rochester, MN
The primary objective of this study is to validate the chronic acquired polyneuropathy-patient reported index (CAP-PRI) in subjects with acquired and idiopathic polyneuropathy.
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A Non-interventional Cohort Safety Study of Patients With hATTR-PN
Rochester, MN
The purpose of this study with the Health Authorities from the United States (US), Canada and Europe, is to collect long-term safety information (including any potential side effects) for the drug TEGSEDI in patients with hATTR-PN under real-world conditions.
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The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
Rochester, MN
The purpose of this study is to evaluate the safety and effectiveness of long-term dosing with (ALN-TTR02) patisiran in patients who have familial transthyretin mediated amyloidosis with polyneuropathy, and have completed a prior clinical study on patisiran.
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Efficacy and Safety of ISIS-TTR Rx in Familial Amyloid Polyneuropathy
Rochester, MN
The purpose of this study is to evaluate the efficacy and safety of ISIS-TTR Rx given for 65 weeks in patients with Familial Amyloid Polyneuropathy
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Open-Label Extension Assessing Long Term Safety and Efficacy of ISIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
Rochester, MN
This study evaluates the safety and tolerability of extended dosing with ISIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.
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NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Rochester, MN
Evaluating the efficacy of ION-682884 after administration for 65 weeks, as compared to the historical control of the placebo cohort (inotersen) in the NEURO-TTR trial, based on the change from Baseline in serum TTR concentration, mNIS+7 and in the Norfolk Quality of Life.
Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) in patients with hATTR. To evaluate the efficacy of ION-682884, as compared to the placebo cohort in the NEURO-TTR trial, based on the change from Baseline in the following measures:
- Neuropathy Symptom and Change Score (NSC)
- Physical component summary (PCS) score of 36-Item Short ...
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A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Scottsdale/Phoenix, AZ
The purpose of this study is to evaluate the effectiveness and safety of AKCEA-TTR-LRx after administration for 65 weeks to patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN), as compared to the NEURO-TTR trial (NCT01737398).
For more information, please visit http://www.neuro-ttransform.com/.
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ATTR Expanded Access Program (EAP) by Ionis
Rochester, MN
The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).