NCT02175004Phase 3Completed

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Study sponsor: Ionis Pharmaceuticals, Inc. · IONS

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2020-09-11Actual

Enrollment · participants135Actual

Source updated2023-11-18Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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No recorded changes are available for this view and time period. Our history may be incomplete; this does not establish that the trial stayed unchanged.

Study details

Conditions studied

FAP; Familial Amyloid Polyneuropathy; TTR; Transthyretin; Amyloidosis

Treatments or interventions studied

InotersenDrugAlso known as TEGSEDI, IONIS-TTR Rx, ISIS 420915

Primary outcomes · main study measures

Read the study measures

measure: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug; description: An adverse event (AE) is any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE is considered related to the investigational drug product. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. An SAE is any untoward medical occurrence that at any dose that results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, leads to a congenital anomaly/birth defect, or is an important medical event. TEAEs considered related to the study drug as assessed by the Investigator are reported.; timeFrame: From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks); measure: Percentage of Participants With Change From Baseline in Vital Signs; description: Vital signs included blood pressure, heart rate, respiratory rate, and temperature. Only categories with at least one participant with event are reported.; timeFrame: From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks); measure: Percentage of Participants With Change From Baseline in Weight; description: As prespecified in the protocol, percentage of participants with change from baseline in weight is reported in 2 categories, decrease of ≥7% from Baseline and increase of ≥7% from Baseline.; timeFrame: From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks); measure: Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Test Values; description: Clinical laboratory tests included the analysis of chemistry, haematology, and urinalysis. Any value outside the normal range will be flagged for the attention of the investigator who will assess whether or not a flagged value is of clinical significance. Only those categories with at least one participant with event are reported. Normal range of creatinine clearance is 110 to 150 mL/min in males and 100 to 130 mL/min in females. Normal urine protein to creatinine (P/C) ratio= \<0.2. Normal range for Alanine Aminotransferase (ALT) is 4 to 36 units per liter (U/L). Platelets normal range=140×10\^9/L to 400×10\^9/L.; timeFrame: From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks); measure: Percentage of Participants With Change From Baseline in QT Interval Corrected Using Fridericia's Formula (QTcF) as Determined by Electrocardiogram (ECG); description: Normal QTcF at Baseline is defined as ≤450 milliseconds (ms) for males or ≤470 ms for females. Percentage of participants with QT interval outside of normal range are reported.; timeFrame: From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks); measure: Percentage of Participants Using Concomitant Medication for Nervous and Cardiovascular System Disorders; description: A concomitant therapy was any non-protocol-specified drug or substance (including over-the counter medications, herbal medications, and vitamin supplements) administered between signing of informed consent and the final post-treatment visit for treating nervous and cardiovascular system disorders.; timeFrame: From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks); measure: Percentage of Participants With Change From Baseline in Ophthalmic Examination as Assessed by Visual Acuity Changes; timeFrame: From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks); measure: Percentage of Participants With Change From Baseline in Light Detection Ability Measured by Electroretinography; timeFrame: Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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