A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PN

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE4

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-01-21

Study Completion Date

2024-03-20

Brief Summary

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

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Detailed Description

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Conditions

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Hereditary Transthyretin Amyloidosis With Polyneuropthy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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TEGSEDI

Participants received TEGSEDI 284 milligrams (mg), subcutaneously (SC) once weekly, as prescribed by their physician per the product label.

Group Type EXPERIMENTAL

TEGSEDI

Intervention Type DRUG

SC injection.

Interventions

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TEGSEDI

SC injection.

Intervention Type DRUG

Other Intervention Names

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Inotersen

Eligibility Criteria

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Inclusion Criteria

1. Satisfy one of the following:

1. United States (US) Participants: Adult participants (≥ 18 years old) diagnosed with hATTR-PN and prescribed TEGSEDI according to the United States Prescribing Information (USPI).
2. Canadian participants: Adult participants (≥ 18 years old) diagnosed with stage 1 or stage 2 hATTR-PN and prescribed TEGSEDI according to the Canadian Product Monograph (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, participant 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, participant is utilizing an acceptable contraceptive method from the time of signing the ICF until 13 weeks after the last dose of TEGSEDI administration