TerminatedPhase 4

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

Stopped: Withdrawal of TEGSEDI from sale was not related to any safety issues.

United States, Canada8 participantsStarted 2021-01-21

Plain-language 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.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Satisfy one of the following:
✓. United States (US) Participants: Adult participants (≥ 18 years old) diagnosed with hATTR-PN and prescribed TEGSEDI according to the United States Prescribing Information (USPI).
✓. 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).
✓. Must have given written informed consent for participation in this study.
✓. Must provide access to their previous medical records.
✓. Are about to initiate or have recently initiated treatment with TEGSEDI and have not received more than 9 doses in total.
✓. Be willing to complete required testing and report any AEs and/or changes in medications.
✓. Satisfy one of the following:

What they're measuring

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)

Number of Participants With Clinically Significant Changes in Vital Signs

Timeframe: Up to 2 years (24 hours post each TEGSEDI injection)

Number of Participants With Clinically Significant Changes in Cytokine Levels and Inflammatory Markers

Timeframe: Up to 2 years (24 hours post each TEGSEDI injection)

Trial details

NCT IDNCT04306510

SponsorAkcea Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-03-20

Results submitted2025-03-20

View on ClinicalTrials.gov More Hereditary Transthyretin Amyloidosis With Polyneuropthy trials

Plain-language summary

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Satisfy one of the following:
✓. United States (US) Participants: Adult participants (≥ 18 years old) diagnosed with hATTR-PN and prescribed TEGSEDI according to the United States Prescribing Information (USPI).
✓. 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).
✓. Must have given written informed consent for participation in this study.
✓. Must provide access to their previous medical records.
✓. Are about to initiate or have recently initiated treatment with TEGSEDI and have not received more than 9 doses in total.
✓. Be willing to complete required testing and report any AEs and/or changes in medications.
✓. Satisfy one of the following:

What they're measuring

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)

Number of Participants With Clinically Significant Changes in Vital Signs

Timeframe: Up to 2 years (24 hours post each TEGSEDI injection)

Number of Participants With Clinically Significant Changes in Cytokine Levels and Inflammatory Markers

Timeframe: Up to 2 years (24 hours post each TEGSEDI injection)