Back to resultsA Clinical Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Patients With hATTR-PN
Primary Purpose
Hereditary Transthyretin Amyloidosis With Polyneuropthy
Status
Recruiting
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Blood samples for immunoglobulins
Blood samples for inflammatory markers
Blood samples for anti-drug antibodies
Sponsored by
About this trial
This is an interventional other trial for Hereditary Transthyretin Amyloidosis With Polyneuropthy focused on measuring Hereditary Transthyretin Amyloidosis, hATTR-PN, hATTR, Amyloidosis
Eligibility Criteria
Inclusion Criteria:
Satisfy one of the following:
- US Patients: Adult patients (≥ 18 years old) diagnosed with hATTR-PN and prescribed TEGSEDI according to the USPI
- Canadian Patients: Adult patients (≥ 18 years old) diagnosed with stage 1 or stage 2 hATTR PN and prescribed TEGSEDI according to the 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:
- Females: Non-pregnant and non-lactating; abstinent, or if engaged in sexual relations of childbearing potential, patient 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
- Males: Abstinent or if engaged in sexual relations with a female of childbearing potential, patient 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
Sites / Locations
- Study CenterRecruiting
- Study CenterRecruiting
- Study CenterRecruiting
- Study CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Group 1
Arm Description
TEGSEDI
Outcomes
Primary Outcome Measures
To characterize AEs occurring within one day of TEGSEDI administration to adult patients with hATTR-PN.
All treatment emergent AEs potentially related to TEGSEDI will be summarized.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04306510
Brief Title
A Clinical Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Patients 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
2. Study Status
Record Verification Date
December 2022
Overall Recruitment Status
Recruiting
Study Start Date
January 21, 2021 (Actual)
Primary Completion Date
March 15, 2025 (Anticipated)
Study Completion Date
March 15, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Akcea Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
5. Study Description
Brief Summary
The objective of the study is to characterize adverse events (AEs) occurring within one day of TEGSEDI administration to adult patients with hATTR-PN overall and in individual patients with respect to time course of AE onset, vital sign changes, preventive measures, treatment required, risk factors, and subsequent adverse outcomes.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Transthyretin Amyloidosis With Polyneuropthy
Keywords
Hereditary Transthyretin Amyloidosis, hATTR-PN, hATTR, Amyloidosis
7. Study Design
Primary Purpose
Other
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
75 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Group 1
Arm Type
Other
Arm Description
TEGSEDI
Intervention Type
Procedure
Intervention Name(s)
Blood samples for immunoglobulins
Intervention Description
Following an injection of TEGSEDI as prescribed by their physician per the product label, patients will have additional blood tests at 1, 2, 4, 8 and 24 hours post injection for immunoglubulins at a clinic visit every 4 months for up to 2 years.
Intervention Type
Procedure
Intervention Name(s)
Blood samples for inflammatory markers
Intervention Description
Following an injection of TEGSEDI as prescribed by their physician per the product label, patients will have additional blood tests at 1, 2, 4, 8 and 24 hours post injection for inflammatory markers at a clinic visit every 4 months for up to 2 years.
Intervention Type
Procedure
Intervention Name(s)
Blood samples for anti-drug antibodies
Intervention Description
Patients taking TEGSEDI as prescribed by their physician per the product label, patients will have additional blood samples for anti-drug antibodies at various time-points as indicated in the event of hypersensitivity reactions.
Primary Outcome Measure Information:
Title
To characterize AEs occurring within one day of TEGSEDI administration to adult patients with hATTR-PN.
Description
All treatment emergent AEs potentially related to TEGSEDI will be summarized.
Time Frame
1 year minimum to 2 years maximum
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Satisfy one of the following:
US Patients: Adult patients (≥ 18 years old) diagnosed with hATTR-PN and prescribed TEGSEDI according to the USPI
Canadian Patients: Adult patients (≥ 18 years old) diagnosed with stage 1 or stage 2 hATTR PN and prescribed TEGSEDI according to the 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:
Females: Non-pregnant and non-lactating; abstinent, or if engaged in sexual relations of childbearing potential, patient 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
Males: Abstinent or if engaged in sexual relations with a female of childbearing potential, patient 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Ionis Pharmaceuticals
Phone
(844) 742-0280
Email
ionisNCT04306510study@clinicaltrialmedia.com
Facility Information:
Facility Name
Study Center
City
Rosedale
State/Province
New York
ZIP/Postal Code
11422
Country
United States
Individual Site Status
Recruiting
Facility Name
Study Center
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Name
Study Center
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
Study Center
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M3K0A6
Country
Canada
Individual Site Status
Recruiting
12. IPD Sharing Statement
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A Clinical Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Patients With hATTR-PN
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