Clinical Trial
Study Details
Status
RECRUITING
Study ID #
NCT04306510
Study Start
Primary Completion
Study Completion
Enrollment
75
Study Type
Interventional
Phase
Phase 4
Interventions
Blood samples for anti-drug antibodies

Patients taking TEGSEDI as prescribed by their physician per the product label, patients will have additional blood samples for anti-drug antibodies at various time-points as indicated in the event of hypersensitivity reactions.

Primary Outcomes
Measure
To characterize AEs occurring within one day of TEGSEDI administration to adult patients with hATTR-PN.
Description

All treatment emergent AEs potentially related to TEGSEDI will be summarized.

Timeframe
1 year minimum to 2 years maximum
Summary

The objective of the study is to characterize adverse events (AEs) occurring within one day of TEGSEDI administration to adult patients with hATTR-PN overall and in individual patients with respect to time course of AE onset, vital sign changes, preventive measures, treatment required, risk factors, and subsequent adverse outcomes.

Conditions
Hereditary Transthyretin Amyloidosis With Polyneuropthy
Study Contact
Name
Ionis Pharmaceuticals
Role
Contact
Phone
(844) 742-0280
Email
ionisNCT04306510study@clinicaltrialmedia.com
Facility

Study Center
Rosedale, NY 11422
United States

Facility

Study Center
Toronto ON M3K0A6
Canada

Eligibility Criteria

Inclusion Criteria:

1. Satisfy one of the following:

1. US Patients: Adult patients (≥ 18 years old) diagnosed with hATTR-PN and prescribed TEGSEDI according to the USPI
2. Canadian Patients: Adult patients (≥ 18 years old) diagnosed with stage 1 or stage 2 hATTR PN and prescribed TEGSEDI according to the CPM
2. Must have given written informed consent for participation in this study
3. Must provide access to their previous medical records
4. Are about to initiate or have recently initiated treatment with TEGSEDI and have not received more than 9 doses in total
5. Be willing to complete required testing and report any AEs and/or changes in medications
6. Satisfy one of the following:

1. Females: Non-pregnant and non-lactating; abstinent, or if engaged in sexual relations of childbearing potential, patient is using an acceptable contraceptive method from time of signing the informed consent form (ICF) until 13 weeks after the last dose of TEGSEDI administration
2. Males: Abstinent or if engaged in sexual relations with a female of childbearing potential, patient is utilizing an acceptable contraceptive method from the time of signing the ICF until 13 weeks after the last dose of TEGSEDI administration

Exclusion Criteria:

* None

Eligibility Minimum Age
18 Years
Sexes Eligible for Study
ALL
Eligibility Std Ages
Adult
Older Adult
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