18 and older, any sex, with FAP or Familial Amyloid Polyneuropathy. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study DrugPrimary· From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)
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
TEAEs
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
100
Previous Inotersen-Inotersen 300 mg
96.5
Serious TEAEs
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
36.0
Previous Inotersen-Inotersen 300 mg
54.1
TEAEs Related to Study Drug
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
82.0
Previous Inotersen-Inotersen 300 mg
69.4
Percentage of Participants With Change From Baseline in Vital SignsPrimary· From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)
Vital signs included blood pressure, heart rate, respiratory rate, and temperature. Only categories with at least one participant with event are reported.
Systolic Blood Pressure: <90 millimeters of mercury (mmHg)
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
12.0
Previous Inotersen-Inotersen 300 mg
18.8
Systolic Blood Pressure: >140 mmHg
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
32.0
Previous Inotersen-Inotersen 300 mg
41.2
Systolic Blood Pressure: >160 mmHg
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
8.0
Previous Inotersen-Inotersen 300 mg
20.0
Diastolic Blood Pressure: <50 mmHg
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
6.0
Previous Inotersen-Inotersen 300 mg
9.4
Diastolic Blood Pressure: >90 mmHg
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
30.0
Previous Inotersen-Inotersen 300 mg
28.2
Diastolic Blood Pressure: >100 mmHg
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
10.0
Previous Inotersen-Inotersen 300 mg
7.1
Heart Rate: <60 beats per minute (bpm)
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
30.0
Previous Inotersen-Inotersen 300 mg
38.8
Heart Rate: >100 bpm
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
16.0
Previous Inotersen-Inotersen 300 mg
9.4
Percentage of Participants With Change From Baseline in WeightPrimary· From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)
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.
Weight (kg): Decrease of ≥7% From Baseline
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
30.0
Previous Inotersen-Inotersen 300 mg
47.1
Weight (kg): Increase of ≥7% From Baseline
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
24.0
Previous Inotersen-Inotersen 300 mg
11.8
Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Test ValuesPrimary· From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)
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
Confirmed Creatinine Clearance by CKD-EPI <30 ml/min/1.73m^2
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
4.0
Previous Inotersen-Inotersen 300 mg
4.7
Confirmed Urine P/C Ratio >5 × Upper Limit of Normal (ULN)
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
8.0
Previous Inotersen-Inotersen 300 mg
10.6
Confirmed Alanine Aminotransferase (ALT) ≥3 x ULN
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
4.0
Previous Inotersen-Inotersen 300 mg
4.7
Confirmed Value of Platelets <75 × 10^9/L
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
12.0
Previous Inotersen-Inotersen 300 mg
12.9
Percentage of Participants With Change From Baseline in QT Interval Corrected Using Fridericia's Formula (QTcF) as Determined by Electrocardiogram (ECG)Primary· From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)
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.
QTcF >450 ms
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
44.0
Previous Inotersen-Inotersen 300 mg
43.5
QTcF >480 ms
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
20.0
Previous Inotersen-Inotersen 300 mg
23.5
QTcF >500 ms
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
12.0
Previous Inotersen-Inotersen 300 mg
16.5
Percentage of Participants Using Concomitant Medication for Nervous and Cardiovascular System DisordersPrimary· From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)
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.
Nervous System Disorders
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
88.0
Previous Inotersen-Inotersen 300 mg
81.2
Cardiovascular System Disorders
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
68.0
Previous Inotersen-Inotersen 300 mg
75.3
Percentage of Participants With Change From Baseline in Ophthalmic Examination as Assessed by Visual Acuity ChangesPrimary· From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
0.0
Previous Inotersen-Inotersen 300 mg
2.4
Percentage of Participants With Change From Baseline in Light Detection Ability Measured by ElectroretinographyPrimary· Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156
Participants With Change From Baseline at Week 78
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
25.0
Previous Inotersen-Inotersen 300 mg
12.7
Participants With Change From Baseline at Week 156
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
31.3
Previous Inotersen-Inotersen 300 mg
9.1
Change From Baseline in the Modified Neuropathy Impairment Score (mNIS)+7 Composite Score at Weeks 78 and 156Secondary· Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156
The mNIS+7 composite score is a measure of neurologic impairment that evaluates muscle weakness, sensation, reflexes, nerve conduction, and autonomic function. The mNIS+7 Composite Score has a range of -22.32 to 346.32 and a higher mNIS+7 composite score indicates worsening disease. A positive change from Baseline indicates worsening of polyneuropathy impairments. Mixed Effects Model with Repeated Measures (MMRM) was used for the analysis.
Change From Baseline at Week 78
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
33.11
± 28.915
Previous Inotersen-Inotersen 300 mg
10.11
± 18.204
Change From Baseline at Week 156
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
37.34
± 29.030
Previous Inotersen-Inotersen 300 mg
17.21
± 27.307
Change From Baseline in the mNIS +7 Component: Heart Rate to Deep Breathing Score at Weeks 78 and 156Secondary· Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156
Heart rate to deep breathing is a quantitative autonomic test using the CASE IV instrument that measures a participant's change in heart rate after deep breathing. The score of this component ranges from 0 to 3.72 points. Higher scores indicate impairment. MMRM was used for the analysis.
Change From Baseline at Week 78
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
0.23
± 0.977
Previous Inotersen-Inotersen 300 mg
0.11
± 0.761
Change From Baseline at Week 156
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
0.44
± 1.099
Previous Inotersen-Inotersen 300 mg
0.30
± 0.504
Change From Baseline in the mNIS +7 Component: Nerve Conduction Score at Weeks 78 and 156Secondary· Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156
The nerve conduction tests are quantitative tests that measure the conduction attributes of preselected nerves. The score range of this component is 0 to 18.6 points. Higher scores indicate impairment. MMRM was used for the analysis.
Change From Baseline at Week 78
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
1.86
± 2.313
Previous Inotersen-Inotersen 300 mg
0.57
± 1.361
Change From Baseline at Week 156
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
2.05
± 2.351
Previous Inotersen-Inotersen 300 mg
0.77
± 1.724
Change From Baseline in the mNIS +7 Component: Heat-Pain Sensory Score at Weeks 78 and 156Secondary· Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156
The Heat-Pain Sensory test uses the CASE IV instrument to perform standardized psychophysical measurement to determine pain sensory thresholds in response to heat. The maximum score of this component is 0 to 40 points. Higher scores indicate impairment. MMRM was used for the analysis.
Change From Baseline at Week 78
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
2.60
± 8.440
Previous Inotersen-Inotersen 300 mg
2.45
± 7.557
Change From Baseline at Week 156
Group
Value
95% CI
Previous Placebo-Inotersen 300 mg
2.90
± 9.224
Previous Inotersen-Inotersen 300 mg
3.40
± 8.774
Adverse events — posted to ClinicalTrials.gov
Time frame: From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
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· Phase 3
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NCT03702829 — 24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients
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NCT01737398 — Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
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· completed
NCT03400098 — ATTR Expanded Access Program (EAP) by Ionis
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Ionis Pharmaceuticals, Inc.
Last refreshed: 18 November 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02175004.