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

Akcea Therapeutics

Status and phase: Terminated; Phase 4

Conditions: Hereditary Transthyretin Amyloidosis With Polyneuropthy

Treatments: Drug: TEGSEDI

Study type: Interventional

Funder types: Industry

Identifiers

NCT04306510

TEG4004

Details and patient eligibility

About

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.

Enrollment: 8 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Exclusion criteria

• None

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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8 participants in 1 patient group

TEGSEDI

Experimental group

Description:

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

Treatment:

Drug: TEGSEDI