A Non-interventional Cohort Safety Study of Patients With hATTR-PN

Hereditary Transthyretin Amyloidosis With Polyneuropthy Clinical Trial

Official title:

A Prospective, Non-interventional, Long-term, Multinational Cohort Safety Study of Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (hATTR-PN)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04850105
• Other study ID #: TEG4001
• Secondary ID: EUPAS37728
• Status: Recruiting
• Start date: September 21, 2021
• Est. completion date: March 31, 2036

Study information

• Verified date: April 2024
• Source: Akcea Therapeutics
• Contact: Ionis Pharmaceuticals
• Phone: (844) 915-5145
• Email: ionisNCT04850105study[@]clinicaltrialmedia.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 240
• Est. completion date: March 31, 2036
• Est. primary completion date: March 31, 2036
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Amyloid Neuropathies, Familial
• Amyloidosis
• Hereditary Transthyretin Amyloidosis With Polyneuropthy

Intervention

Other:
• Data Collection
Data on each patient will be collected at study enrollment and at each follow-up visit. No mandatory visits, tests, or assessments are required for this study. All visits will be scheduled and conducted according to the clinical site's normal clinical practice.

Locations

Country	Name	City	State
Canada	Study Center	Toronto
France	Study Center	Lille	Cedex
France	Study Center	Nice	Romaine
France	Study Centre	Saint-Herblain
Germany	Study Center	Heidelberg
Greece	Study Center	Athens
Greece	Study Center	Athens
Greece	Study Center	Heraklion
Italy	Study Center	Bologna
Italy	Study Center	Genova
Italy	Study Center	Lagosanto	Roma
Italy	Study Center	Messina
Italy	Study Center	Milano
Italy	Study Center	Napoli
Italy	Study Center	Pavia
Italy	Study Center	Roma
Italy	Study Centre	Roma	Rome
Portugal	Study Center	Lisboa
Spain	Study Centre	Barcelona	Cataluna
Spain	Study Center	Huelva	Andalucia
Spain	Study Center	Madrid
Spain	Study Centre	Madrid	Comunidad De Madrid
Spain	Study Centre	Oviedo	Avenida De Roma
Spain	Study Center	Palma De Mallorca	Islas Baleares
Spain	Study Centre	Villarreal	Barcelona
United States	Study Center	Abington	Pennsylvania
United States	Study Center	Anchorage	Alaska
United States	Study Center	Cleveland	Ohio
United States	Study Center	Dallas	Texas
United States	Study Center	Indianapolis	Indiana
United States	Study Center	Los Angeles	California
United States	Study Center	Los Angeles	California
United States	Study Centre	Louisville	Kentucky
United States	Study Center	Maywood	Illinois
United States	Study Center	New York	New York
United States	Study Center	Norfolk	Virginia
United States	Study Center	Philadelphia	Pennsylvania
United States	Study Center	Rochester	Minnesota
United States	Study Center	Rosedale	New York

Sponsors (2)

Lead Sponsor	Collaborator
Akcea Therapeutics	United BioSource, LLC

Countries where clinical trial is conducted

United States,  Canada,  France,  Germany,  Greece,  Italy,  Portugal,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Further characterization of the long-term safety of TEGSEDI in patients with hATTR-PN under real-world conditions.	Determination of the incidence rate of thrombocytopenia in patients with hATTR-PN treated with TEGSEDI (TEGSEDI-exposed cohort); Comparison of the relative rates of thrombocytopenia in hATTR-PN patients treated with TEGSEDI (TEGSEDI exposed) to hATTR-PN patients unexposed to TEGSEDI (TEGSEDI- unexposed)	10 years
Secondary	Description of the incidence rate of the Adverse Events of Special Interest (AESI) in the TEGSEDI-exposed and TEGSEDI-unexposed patients.	To describe the incidence rate of the following Adverse Events of Special Interest (AESI): severe thrombocytopenia; serious and non-serious bleeding events; glomerulonephritis; composite of stroke and/or cervicocephalic arterial dissection; central nervous system (CNS) vasculitis; ocular toxicity due to vitamin A deficiency	10 years
Secondary	Description of the time to onset of Adverse Events of Special Interest (AESI) in the TEGSEDI-exposed and TEGSEDI-unexposed patients.	To describe the time to onset of the following Adverse Events of Special Interest (AESI): severe thrombocytopenia; serious and non-serious bleeding events; glomerulonephritis; composite of stroke and/or cervicocephalic arterial dissection; central nervous system (CNS) vasculitis; ocular toxicity due to vitamin A deficiency	10 years