Clinical trial NCT04306510: A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PN
Official title: A Phase 4 Safety Study Assessing the Adverse Events Occurring Within One Day of TEGSEDI Administration in Patients With Polyneuropathy of Hereditary Transthyretin-mediated Amyloidosis (hATTR-PN)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Hereditary Transthyretin Amyloidosis With Polyneuropthy
Keywords: Hereditary Transthyretin Amyloidosis
hATTR-PN
hATTR
Amyloidosis
Lead sponsor: Akcea Therapeutics
Sponsor class: INDUSTRY
First posted: 2020-03-13
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With Incident, Onset and Duration of Treatment Emergent Adverse Events (TEAEs) Occurring Within 24 Hours of Each TEGSEDI Administration
timeFrame: Up to 2 years (24 hours post each TEGSEDI injection)
measure: Number of Participants With Clinically Significant Changes in Vital Signs
timeFrame: Up to 2 years (24 hours post each TEGSEDI injection)
measure: Number of Participants With Clinically Significant Changes in Cytokine Levels and Inflammatory Markers
timeFrame: Up to 2 years (24 hours post each TEGSEDI injection)
Secondary outcomes:
Other outcomes:
Locations:
city: Rosedale
country: United States
facility: Study Center
state: New York
city: Toronto
country: Canada
facility: Study Center
state: Ontario
Study officials:
References: