Clinical trial NCT04850105: A Non-interventional Cohort Safety Study of Patients With hATTR-PN
Official title: A Prospective, Non-interventional, Long-term, Multinational Cohort Safety Study of Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (hATTR-PN)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hereditary Transthyretin Amyloidosis With Polyneuropthy
Keywords: Hereditary Transthyretin Amyloidosis
hATTR-PN
hATTR
Amyloidosis
Lead sponsor: Akcea Therapeutics
Sponsor class: INDUSTRY
Collaborators: name: United BioSource, LLC
First posted: 2021-04-20
Enrollment: 240
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The TEGSEDI-exposed cohort will consist of patients diagnosed with hATTR-PN who are receiving TEGSEDI. Data from the TEGSEDI-exposed cohort will be compared to data collected prospectively from a TEGSEDI-unexposed cohort which will consist of patients diagnosed with hATTR-PN who have not taken any dose of TEGSEDI within 25 weeks prior to enrollment and are eligible for TEGSEDI treatment per applicable product label.

Patients in Europe, US, and Canada will be enrolled from centers that manage patients with hATTR-PN.

Brief summary: This is a prospective, non-interventional, Long-term, multinational cohort safety study of patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN). The overarching goal of this study is to further characterize the long-term safety of TEGSEDI (inotersen) in patients with hATTR-PN under real-world conditions.

Detailed description: Study Rationale:

hATTR-PN is an inherited, progressive, fatal disease caused by misfolded transthyretin (TTR) proteins that accumulate as amyloid fibrils predominantly in the peripheral nerves, heart, gastrointestinal tract, and other organs. hATTR-PN is a rare disease and there are no large epidemiological studies that reliably provide an indication of its prevalence. The worldwide distribution is unequal, with higher rates in Portugal, Japan, Brazil, Northern Sweden, and the US. Current estimates suggest there may be 10,000 afflicted patients worldwide.

TEGSEDI (inotersen) is an antisense oligonucleotide inhibitor of human TTR protein synthesis. In Europe and Canada, TEGSEDI is indicated for the treatment of Stage 1 or Stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). In the US, TEGSEDI is indicated for treatment of the polyneuropathy of hereditary TTR-mediated amyloidosis in adults. Efficacy has been demonstrated in patients with hATTR-PN, as reflected by a slowing or reversal of disease progression.

Research Question:

The overarching goal of this study is to further characterize the long-term safety of TEGSEDI in patients with hATTR-PN under real-world conditions.

Population:

Patients in Europe, US, and Canada will be enrolled from centers that manage patients with hATTR-PN. Physicians participating in the study will be instructed to invite all patients who meet study eligibility criteria to enroll until the enrollment period is closed.

Eligibility criteria: Inclusion Criteria:

1. Either:

   1. TEGSEDI Exposed Cohort: Patients diagnosed with hATTR-PN who have taken any dose of TEGSEDI within 25 weeks prior to enrollment
   2. TEGSEDI Unexposed Cohort: Patients diagnosed with hATTR-PN who have not taken any dose of TEGSEDI within 25 weeks prior to enrollment and are eligible for TEGSEDI treatment per applicable product label
2. Clinically managed in Canada, Europe, or the US
3. Have provided appropriate written informed consent

Exclusion Criteria:

* None

Primary outcomes:
measure: Further characterization of the long-term safety of TEGSEDI in patients with hATTR-PN under real-world conditions.
timeFrame: 10 years
Secondary outcomes:
measure: Description of the incidence rate of the Adverse Events of Special Interest (AESI) in the TEGSEDI-exposed and TEGSEDI-unexposed patients.
timeFrame: 10 years
measure: Description of the time to onset of Adverse Events of Special Interest (AESI) in the TEGSEDI-exposed and TEGSEDI-unexposed patients.
timeFrame: 10 years
Other outcomes:
Locations:
city: Sofia
country: Bulgaria
facility: Study Centre
city: Égkomi
country: Cyprus
facility: Study Centre
city: Lille
country: France
facility: Study Center
state: Cedex
city: Nice
country: France
facility: Study Center
state: Romaine
city: Saint-Herblain
country: France
facility: Study Centre
city: Heidelberg
country: Germany
facility: Study Center
city: Athens
country: Greece
facility: Study Center
city: Athens
country: Greece
facility: Study Center
city: Heraklion
country: Greece
facility: Study Center
city: Lagosanto
country: Italy
facility: Study Center
state: Roma
city: Roma
country: Italy
facility: Study Centre
state: Rome
city: Bologna
country: Italy
facility: Study Center
city: Genova
country: Italy
facility: Study Center
city: Messina
country: Italy
facility: Study Center
city: Milano
country: Italy
facility: Study Center
city: Napoli
country: Italy
facility: Study Center
city: Pavia
country: Italy
facility: Study Center
city: Roma
country: Italy
facility: Study Center
city: Lisboa
country: Portugal
facility: Study Center
city: Huelva
country: Spain
facility: Study Center
state: Andalucia
city: Oviedo
country: Spain
facility: Study Centre
state: Avenida De Roma
city: Villarreal
country: Spain
facility: Study Centre
state: Barcelona
city: Barcelona
country: Spain
facility: Study Centre
state: Cataluna
city: Madrid
country: Spain
facility: Study Centre
state: Comunidad De Madrid
city: Palma De Mallorca
country: Spain
facility: Study Center
state: Islas Baleares
city: Madrid
country: Spain
facility: Study Center
Study officials:
References: