Trial · NCT01737398
Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
NCT01737398, Phase 2, Phase 3, Completed, Ionis Pharmaceuticals, Inc.; results first posted 2019-01-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy. Trials, record NCT01737398. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01737398
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
- NCT number
- NCT01737398
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 173 participants, actual
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
- Interventions
- Inotersen, Placebo
- First posted
- 2012-11-29
- Last updated
- 2019-07-17
- Results first posted
- 2019-01-23
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline In The Modified Neuropathy Impairment Score (mNIS) +7 Composite Score at Week 66 | Baseline and Week 66 | 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 lower function. |
| Primary | Change From Baseline In The Norfolk Quality Of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66 | Baseline and Week 66 | The Norfolk QoL-DN score is a measure of physical function/large fiber neuropathy, symptoms, activities of daily living, small fiber neuropathy, and autonomic neuropathy. The Norfolk QoL-DN total score has a range of -4 to 136, and a higher Norfolk QoL-DN score indicates poorer QoL. |
| Secondary | Change From Baseline In The Norfolk QoL-DN Questionnaire Symptoms Domain Score at Week 66 | Baseline and Week 66 | The Norfolk QoL-DN symptoms score is a sub-score of the total Norfolk QoL-DN Questionnaire. The Norfolk QoL-DN symptoms domain score has a range of 0-32, and a higher Norfolk QoL-DN score indicates poorer QoL. |
| Secondary | Change From Baseline In The Norfolk QoL-DN Questionnaire Physical Functioning/Large Fiber Neuropathy Domain Score at Week 66 | Baseline and Week 66 | The Norfolk QoL-DN physical functioning/large fiber neuropathy domain score is a sub-score of the total Norfolk QoL-DN Questionnaire. The Norfolk QoL-DN physical function/large fiber neuropathy domain score has a range of -4 to 56, and a higher Norfolk QoL-DN domain score indicates poorer QoL. |
| Secondary | Change From Baseline In Modified Body Mass Index (mBMI) at Week 65 | Baseline and Week 65 | The mBMI is the BMI multiplied by the serum albumin g/L |
| Secondary | Change From Baseline In Body Mass Index (BMI) at Week 65 | Baseline and Week 65 | |
| Secondary | Change From Baseline in Neuropathy Impairment Score (NIS) at Week 66 | Baseline and Week 66 | The NIS score is a measure of neurologic impairment. The NIS Score has a range of 0 to 244 and a higher NIS score indicates lower function. |
| Secondary | Change From Baseline in Modified +7 at Week 66 | Baseline and Week 66 | The Modified +7 score is a version of the NIS score that is a measure of neurologic impairment. The Modified +7 Score has a range of -22.32 to 102.32 and a higher NIS score indicates lower function. |
| Secondary | Change From Baseline in NIS+7 at Week 66 | Baseline and Week 66 | The NIS+7 score is a version of the NIS score that is a measure of neurologic impairment. The NIS+7 Score has a range of -26.04 to 270.04 and a higher NIS score indicates lower function. |
| Secondary | Change From Baseline in Global Longitudinal Strain (GLS) by Echocardiogram (ECHO) at Week 65 in the CM-ECHO Set | Baseline and Week 65 | GLS by ECHO is a measure of cardiac systolic function |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline In The Modified Neuropathy Impairment Score (mNIS) +7 Composite Score at Week 66 | Baseline and Week 66 | The full analysis set included all randomized participants who received at least 1 injection of study drug (inotersen or placebo) and who had a Baseline and at least 1 post-Baseline efficacy assessment for the mNIS+7 score or Norfolk QoL-DN questionnaire total score. Participants analyzed = participants with evaluable data. | Scores on a Scale | MEAN |
| PRIMARY | Change From Baseline In The Norfolk Quality Of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66 | Baseline and Week 66 | The full analysis set included all randomized participants who received at least 1 injection of study drug (inotersen or placebo) and who had a Baseline and at least 1 post-Baseline efficacy assessment for the mNIS+7 score or Norfolk QoL-DN questionnaire total score. Participants analyzed = participants with evaluable data. | Scores on a Scale | MEAN |
| SECONDARY | Change From Baseline In The Norfolk QoL-DN Questionnaire Symptoms Domain Score at Week 66 | Baseline and Week 66 | This endpoint only measured participants with Stage 1 hATTR-PN in Full Analysis Set. Participants analyzed = participants with evaluable data. | Scores on a Scale | MEAN |
| SECONDARY | Change From Baseline In The Norfolk QoL-DN Questionnaire Physical Functioning/Large Fiber Neuropathy Domain Score at Week 66 | Baseline and Week 66 | This endpoints only measured participants who had Stage 2 hATTR-PN in Full Analysis Set. Participants analyzed = participants with evaluable data. | Scores on a Scale | MEAN |
| SECONDARY | Change From Baseline In Modified Body Mass Index (mBMI) at Week 65 | Baseline and Week 65 | The full analysis set included all randomized participants who received at least 1 injection of study drug (inotersen or placebo) and who had a Baseline and at least 1 post-Baseline efficacy assessment for the mNIS+7 score or Norfolk QoL-DN questionnaire total score. Participants analyzed = participants with evaluable data. | kg/m^2*g/L | MEAN |
| SECONDARY | Change From Baseline In Body Mass Index (BMI) at Week 65 | Baseline and Week 65 | The full analysis set included all randomized participants who received at least 1 injection of study drug (inotersen or placebo) and who had a Baseline and at least 1 post-Baseline efficacy assessment for the mNIS+7 score or Norfolk QoL-DN questionnaire total score. Participants analyzed = participants with evaluable data. | kg/m^2 | MEAN |
| SECONDARY | Change From Baseline in Neuropathy Impairment Score (NIS) at Week 66 | Baseline and Week 66 | The full analysis set included all randomized participants who received at least 1 injection of study drug (inotersen or placebo) and who had a Baseline and at least 1 post-Baseline efficacy assessment for the mNIS+7 score or Norfolk QoL-DN questionnaire total score. Participants analyzed = participants with evaluable data. | Scores on a Scale | MEAN |
| SECONDARY | Change From Baseline in Modified +7 at Week 66 | Baseline and Week 66 | The full analysis set included all randomized participants who received at least 1 injection of study drug (inotersen or placebo) and who had a Baseline and at least 1 post-Baseline efficacy assessment for the mNIS+7 score or Norfolk QoL-DN questionnaire total score. Participants analyzed = participants with evaluable data. | Scores on a Scale | MEAN |
| SECONDARY | Change From Baseline in NIS+7 at Week 66 | Baseline and Week 66 | The full analysis set included all randomized participants who received at least 1 injection of study drug (inotersen or placebo) and who had a Baseline and at least 1 post-Baseline efficacy assessment for the mNIS+7 score or Norfolk QoL-DN questionnaire total score. Participants analyzed = participants with evaluable data. | Scores on a Scale | MEAN |
| SECONDARY | Change From Baseline in Global Longitudinal Strain (GLS) by Echocardiogram (ECHO) at Week 65 in the CM-ECHO Set | Baseline and Week 65 | The CM-ECHO Set includes the subset of the Randomized Set who had a diagnosis of TTR cardiomyopathy at study entry but are not in the ECHO subgroup, plus participants who qualified to participate in the ECHO subgroup (whether consented or not). Participants analyzed = participants with evaluable data. | Percent Change | MEAN |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group38
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline In The Modified Neuropathy Impairment Score (mNIS) +7 Composite Score at Week 66 | Inotersen | 4.16 | Standard Deviation 15.672 | Scores on a Scale | |
| Change From Baseline In The Modified Neuropathy Impairment Score (mNIS) +7 Composite Score at Week 66 | Placebo | 23.89 | Standard Deviation 24.190 | Scores on a Scale | |
| Change From Baseline In The Norfolk Quality Of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66 | Inotersen | -0.08 | Standard Deviation 18.967 | Scores on a Scale | |
| Change From Baseline In The Norfolk Quality Of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66 | Placebo | 10.77 | Standard Deviation 21.134 | Scores on a Scale | |
| Change From Baseline In The Norfolk QoL-DN Questionnaire Symptoms Domain Score at Week 66 | Inotersen | -1.40 | Standard Deviation 4.763 | Scores on a Scale | |
| Change From Baseline In The Norfolk QoL-DN Questionnaire Symptoms Domain Score at Week 66 | Placebo | 1.18 | Standard Deviation 5.270 | Scores on a Scale | |
| Change From Baseline In The Norfolk QoL-DN Questionnaire Physical Functioning/Large Fiber Neuropathy Domain Score at Week 66 | Inotersen | 1.05 | Standard Deviation 11.924 | Scores on a Scale | |
| Change From Baseline In The Norfolk QoL-DN Questionnaire Physical Functioning/Large Fiber Neuropathy Domain Score at Week 66 | Placebo | 8.74 | Standard Deviation 9.689 | Scores on a Scale | |
| Change From Baseline In Modified Body Mass Index (mBMI) at Week 65 | Inotersen | -73.32 | Standard Deviation 96.311 | kg/m^2*g/L | |
| Change From Baseline In Modified Body Mass Index (mBMI) at Week 65 | Placebo | -85.21 | Standard Deviation 91.259 | kg/m^2*g/L |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 results by groupParticipant flow10
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Inotersen | 113 | |
| Overall Study | STARTED | Placebo | 60 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Inotersen | 112 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Placebo | 60 | |
| Overall Study | Safety Set | Inotersen | 112 | |
| Overall Study | Safety Set | Placebo | 60 | |
| Overall Study | COMPLETED | Inotersen | 87 | |
| Overall Study | COMPLETED | Placebo | 52 | |
| Overall Study | NOT COMPLETED | Inotersen | 26 | |
| Overall Study | NOT COMPLETED | Placebo | 8 |
Source ClinicalTrials.gov
Adverse events260
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 | Anaemia | Blood and lymphatic system disorders | Inotersen | 15 | 112 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo | 2 | 60 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Inotersen | 14 | 112 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Placebo | 1 | 60 | |
| other | Atrial fibrillation | Cardiac disorders | Inotersen | 6 | 112 | |
| other | Atrial fibrillation | Cardiac disorders | Placebo | 1 | 60 | |
| other | Conjunctival haemorrhage | Eye disorders | Inotersen | 2 | 112 | |
| other | Conjunctival haemorrhage | Eye disorders | Placebo | 3 | 60 | |
| other | Blepharitis | Eye disorders | Inotersen | 2 | 112 | |
| other | Blepharitis | Eye disorders | Placebo | 4 | 60 |
1 to 10 of 260Source ClinicalTrials.gov
See all 260 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Inotersen | 5 | 112 |
| Total, all-cause mortality | Placebo | 0 | 60 |
| Total, other adverse events | Inotersen | 110 | 112 |
| Total, other adverse events | Placebo | 60 | 60 |
| Total, serious adverse events | Inotersen | 36 | 112 |
| Total, serious adverse events | Placebo | 13 | 60 |
Source ClinicalTrials.gov
Papers14
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 |
|---|---|
| Inotersen vs Placebo: deaths from any cause | Trial comparisons |
| Inotersen vs Placebo: 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/NCT01737398
GET https://conductscience.org/api/trials/v1/NCT01737398/results?page=2