Not provided
Not provided
Not provided
Not provided
Not provided
Withdrawal of TEGSEDI from sale was not related to any safety issues.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TEGSEDI | Experimental | Participants received TEGSEDI 284 milligrams (mg), subcutaneously (SC) once weekly, as prescribed by their physician per the product label. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TEGSEDI | Drug | SC injection. |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Incident, Onset and Duration of Treatment Emergent Adverse Events (TEAEs) Occurring Within 24 Hours of Each TEGSEDI Administration | An adverse event (AE) is defined as any unfavorable and unintended sign, symptom, or disease temporally associated with the use of medicinal product, whether or not the AE is considered related to the medicinal product. TEAEs are defined as AEs with an onset date/time on or after the date/time of the first on study administration of TEGSEDI. | Up to 2 years (24 hours post each TEGSEDI injection) |
| Number of Participants With Clinically Significant Changes in Vital Signs | Vital signs including body temperature, heart rate, respiratory rate, and systolic/diastolic blood pressure (BP). | Up to 2 years (24 hours post each TEGSEDI injection) |
| Number of Participants With Clinically Significant Changes in Cytokine Levels and Inflammatory Markers | Cytokines and inflammatory markers including the following parameters: Immunoglobulin (Ig)E, IgG, IgM, C-reactive protein, erythrocyte sedimentation rate, interferon-alpha, interferon beta, chemokines (macrophage inflammatory protein-1a and membrane cofactor protein-1, granulocyte-macrophage colony-stimulating factor, Interleukin (IL)-1alpha (α), IL-1 beta (β), IL-6, IL-8, IL-12, and tumor necrosis factor-α. | Up to 2 years (24 hours post each TEGSEDI injection) |
Not provided
Not provided
Inclusion Criteria:
Satisfy one of the following:
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:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Study Center | Rosedale | New York | 11422 | United States | ||
| Study Center |
A total of 8 participants with polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) were screened for the study, of which 7 participants were treated with TEGSEDI. The 8th participant was enrolled after the sponsor had notified the sites about the study close-out activities, therefore only 7 participants were included in the study.
Participants took part at 2 clinical sites in the United States of America (USA) and Canada from 21 January 2021 to 20 March 2024.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | TEGSEDI | Participants received TEGSEDI 284 milligrams (mg), subcutaneously (SC) once weekly, as prescribed by their physician per the product label. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
The safety set included all participants who received at least 1 dose of TEGSEDI while enrolled in this study protocol.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | TEGSEDI | Participants with hATTR-PN received TEGSEDI 284 mg, SC once weekly, as prescribed by their physician per the product label. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Incident, Onset and Duration of Treatment Emergent Adverse Events (TEAEs) Occurring Within 24 Hours of Each TEGSEDI Administration | An adverse event (AE) is defined as any unfavorable and unintended sign, symptom, or disease temporally associated with the use of medicinal product, whether or not the AE is considered related to the medicinal product. TEAEs are defined as AEs with an onset date/time on or after the date/time of the first on study administration of TEGSEDI. | The evaluable set included participants in the safety set having at least 1 post-dose assessment. | Posted | Count of Participants | Participants | No | Up to 2 years (24 hours post each TEGSEDI injection) |
|
Up to 2 years (24 hours post each TEGSEDI injection)
The evaluable set included participants in the safety set having at least 1 post-dose assessment.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | TEGSEDI | Participants with hATTR-PN received TEGSEDI 284 mg, SC once weekly, as prescribed by their physician per the product label. |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Chills | General disorders | MedDRA25.0 | Systematic Assessment |
This study was subject to limitations due to the rarity of hATTR-PN, which limits the enrollment of potentially eligible patients. Additionally, the availability of concurrent clinical trials and newer therapies targeting the same patient population further complicated efforts to achieve the planned enrollment.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Ionis Pharmaceuticals, Inc. | Ionis Pharmaceuticals, Inc. | 760-603-2346 | globalregulatoryaffairs@ionis.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Oct 6, 2023 | Mar 20, 2025 | Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| C567782 | Amyloidosis, Hereditary, Transthyretin-Related |
| D000686 | Amyloidosis |
| ID | Term |
|---|---|
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000629536 | Inotersen |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Toronto |
| Ontario |
| M3K0A6 |
| Canada |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
|
|
| Primary | Number of Participants With Clinically Significant Changes in Vital Signs | Vital signs including body temperature, heart rate, respiratory rate, and systolic/diastolic blood pressure (BP). | The evaluable set included participants in the safety set having at least 1 post-dose assessment. | Posted | Count of Participants | Participants | No | Up to 2 years (24 hours post each TEGSEDI injection) |
|
|
|
| Primary | Number of Participants With Clinically Significant Changes in Cytokine Levels and Inflammatory Markers | Cytokines and inflammatory markers including the following parameters: Immunoglobulin (Ig)E, IgG, IgM, C-reactive protein, erythrocyte sedimentation rate, interferon-alpha, interferon beta, chemokines (macrophage inflammatory protein-1a and membrane cofactor protein-1, granulocyte-macrophage colony-stimulating factor, Interleukin (IL)-1alpha (α), IL-1 beta (β), IL-6, IL-8, IL-12, and tumor necrosis factor-α. | The evaluable set included participants in the safety set having at least 1 post-dose assessment. | Posted | Count of Participants | Participants | No | Up to 2 years (24 hours post each TEGSEDI injection) |
|
|
|
| 0 |
| 7 |
| 0 |
| 7 |
| 6 |
| 7 |
| Influenza like illness | General disorders | MedDRA25.0 | Systematic Assessment |
|
| Injection site discomfort | General disorders | MedDRA25.0 | Systematic Assessment |
|
| Injection site pain | General disorders | MedDRA25.0 | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA25.0 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA25.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA25.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA25.0 | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA25.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA25.0 | Systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA25.0 | Systematic Assessment |
|
Not provided