Trial · NCT02175004
Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
NCT02175004, Phase 3, Completed, Ionis Pharmaceuticals, Inc.; results first posted 2023-02-09
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP). Trials, record NCT02175004. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02175004
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
- NCT number
- NCT02175004
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 135 participants, actual
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
- Interventions
- Inotersen
- First posted
- 2014-06-26
- Last updated
- 2023-11-18
- Other registries' IDs
- eudract:2013-004561-13
- Results first posted
- 2023-02-09
Source ClinicalTrials.gov
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug | 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 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. |
| Primary | Percentage of Participants With Change From Baseline in Vital Signs | 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. |
| Primary | Percentage of Participants With Change From Baseline in Weight | 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. |
| Primary | Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Test Values | 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\^9/L to 400×10\^9/L. |
| Primary | Percentage of Participants With Change From Baseline in QT Interval Corrected Using Fridericia's Formula (QTcF) as Determined by Electrocardiogram (ECG) | 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. |
| Primary | Percentage of Participants Using Concomitant Medication for Nervous and Cardiovascular System Disorders | 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. |
| Primary | Percentage of Participants With Change From Baseline in Ophthalmic Examination as Assessed by Visual Acuity Changes | From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks) | |
| Primary | Percentage of Participants With Change From Baseline in Light Detection Ability Measured by Electroretinography | Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156 | |
| Secondary | Change From Baseline in the Modified Neuropathy Impairment Score (mNIS)+7 Composite Score at Weeks 78 and 156 | 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. |
| Secondary | Change From Baseline in the mNIS +7 Component: Heart Rate to Deep Breathing Score at Weeks 78 and 156 | 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. |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 registered outcomesOutcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug | From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks) | SS included all enrolled participants who received at least 1 injection of inotersen in CS3. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Change From Baseline in Vital Signs | From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks) | SS included all enrolled participants who received at least 1 injection of inotersen in CS3. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Change From Baseline in Weight | From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks) | SS included all enrolled participants who received at least 1 injection of inotersen in CS3. Overall number analyzed are the number of participants available for analyses. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Test Values | From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks) | SS included all enrolled participants who received at least 1 injection of inotersen in CS3. Number analyzed is the number of participants with data available for analysis for the given category. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Change From Baseline in QT Interval Corrected Using Fridericia's Formula (QTcF) as Determined by Electrocardiogram (ECG) | From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks) | SS included all enrolled participants who received at least 1 injection of inotersen in CS3. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants Using Concomitant Medication for Nervous and Cardiovascular System Disorders | From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks) | SS included all enrolled participants who received at least 1 injection of inotersen in CS3. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Change From Baseline in Ophthalmic Examination as Assessed by Visual Acuity Changes | From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks) | SS included all enrolled participants who received at least 1 injection of inotersen in CS3. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Change From Baseline in Light Detection Ability Measured by Electroretinography | Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156 | SS included all enrolled participants who received at least 1 injection of inotersen in CS3. Overall number analyzed are the number of participants available for analyses. Number analyzed is the number of participants with data available for analysis at the given time point. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in the Modified Neuropathy Impairment Score (mNIS)+7 Composite Score at Weeks 78 and 156 | Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156 | Full Analysis Set (FAS) included all enrolled participants who received at least 1 injection of inotersen in this study (CS3) and who had at least 1 post-baseline efficacy assessment for the mNIS+7 score or Norfolk quality of life-diabetic neuropathy (QoL-DN) questionnaire total score collected after CS3 Study Day 1. Number analyzed is the number of participants with data available for analysis at the given time point. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the mNIS +7 Component: Heart Rate to Deep Breathing Score at Weeks 78 and 156 | Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156 | FAS included all enrolled participants who received at least 1 injection of inotersen in this study (CS3) and who had at least 1 post-baseline efficacy assessment for the mNIS+7 score or Norfolk QoL-DN questionnaire total score collected after CS3 Study Day 1. Number analyzed is the number of participants with data available for analysis at the given time point. | score on a scale | MEAN |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group188
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug | Previous Placebo-Inotersen 300 mg | TEAEs | 100 | percentage of participants | |
| Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug | Previous Inotersen-Inotersen 300 mg | TEAEs | 96.5 | percentage of participants | |
| Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug | Previous Placebo-Inotersen 300 mg | Serious TEAEs | 36.0 | percentage of participants | |
| Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug | Previous Inotersen-Inotersen 300 mg | Serious TEAEs | 54.1 | percentage of participants | |
| Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug | Previous Placebo-Inotersen 300 mg | TEAEs Related to Study Drug | 82.0 | percentage of participants | |
| Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug | Previous Inotersen-Inotersen 300 mg | TEAEs Related to Study Drug | 69.4 | percentage of participants | |
| Percentage of Participants With Change From Baseline in Vital Signs | Previous Placebo-Inotersen 300 mg | Systolic Blood Pressure: <90 millimeters of mercury (mmHg) | 12.0 | percentage of participants | |
| Percentage of Participants With Change From Baseline in Vital Signs | Previous Inotersen-Inotersen 300 mg | Systolic Blood Pressure: <90 millimeters of mercury (mmHg) | 18.8 | percentage of participants | |
| Percentage of Participants With Change From Baseline in Vital Signs | Previous Placebo-Inotersen 300 mg | Systolic Blood Pressure: >140 mmHg | 32.0 | percentage of participants | |
| Percentage of Participants With Change From Baseline in Vital Signs | Previous Inotersen-Inotersen 300 mg | Systolic Blood Pressure: >140 mmHg | 41.2 | percentage of participants |
1 to 10 of 188Source ClinicalTrials.gov
See all 188 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Previous Placebo-Inotersen 300 mg | 50 | |
| Overall Study | STARTED | Previous Inotersen-Inotersen 300 mg | 85 | |
| Overall Study | COMPLETED | Previous Placebo-Inotersen 300 mg | 9 | |
| Overall Study | COMPLETED | Previous Inotersen-Inotersen 300 mg | 9 | |
| Overall Study | NOT COMPLETED | Previous Placebo-Inotersen 300 mg | 41 | |
| Overall Study | NOT COMPLETED | Previous Inotersen-Inotersen 300 mg | 76 |
Source ClinicalTrials.gov
Adverse events344
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Nausea | Gastrointestinal disorders | Previous Placebo-Inotersen 300 mg | 8 | 50 | |
| other | Nausea | Gastrointestinal disorders | Previous Inotersen-Inotersen 300 mg | 25 | 85 | |
| other | Diarrhoea | Gastrointestinal disorders | Previous Placebo-Inotersen 300 mg | 13 | 50 | |
| other | Diarrhoea | Gastrointestinal disorders | Previous Inotersen-Inotersen 300 mg | 21 | 85 | |
| other | Fatigue | General disorders | Previous Placebo-Inotersen 300 mg | 7 | 50 | |
| other | Fatigue | General disorders | Previous Inotersen-Inotersen 300 mg | 20 | 85 | |
| other | Urinary tract infection | Infections and infestations | Previous Placebo-Inotersen 300 mg | 13 | 50 | |
| other | Urinary tract infection | Infections and infestations | Previous Inotersen-Inotersen 300 mg | 18 | 85 | |
| other | Fall | Injury, poisoning and procedural complications | Previous Placebo-Inotersen 300 mg | 11 | 50 | |
| other | Fall | Injury, poisoning and procedural complications | Previous Inotersen-Inotersen 300 mg | 18 | 85 |
1 to 10 of 344Source ClinicalTrials.gov
See all 344 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Previous Placebo-Inotersen 300 mg | 2 | 50 |
| Total, all-cause mortality | Previous Inotersen-Inotersen 300 mg | 15 | 85 |
| Total, other adverse events | Previous Placebo-Inotersen 300 mg | 50 | 50 |
| Total, other adverse events | Previous Inotersen-Inotersen 300 mg | 80 | 85 |
| Total, serious adverse events | Previous Placebo-Inotersen 300 mg | 18 | 50 |
| Total, serious adverse events | Previous Inotersen-Inotersen 300 mg | 46 | 85 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Previous Inotersen-Inotersen 300 mg vs Previous Placebo-Inotersen 300 mg: deaths from any cause | Trial comparisons |
| Previous Inotersen-Inotersen 300 mg vs Previous Placebo-Inotersen 300 mg: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02175004
GET https://conductscience.org/api/trials/v1/NCT02175004/registered?page=2