Trial · NCT02510261
The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)
NCT02510261, Phase 3, Completed, Alnylam Pharmaceuticals; results first posted 2023-12-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran). Trials, record NCT02510261. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02510261
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)
- NCT number
- NCT02510261
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 211 participants, actual
- Lead sponsor
- Alnylam Pharmaceuticals
- Conditions
- Amyloidosis
- Interventions
- Patisiran
- First posted
- 2015-07-29
- Last updated
- 2023-12-06
- Other registries' IDs
- eudract:2014-003877-40
- Results first posted
- 2023-12-06
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 Adverse Events (AEs) Leading to Study Discontinuation | First dose up to 28 days after last dose of study drug (approximately 5.6 years) | AE is any untoward medical occurrence in a participant or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. |
| Secondary | Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5 | Baseline, Year 5 | The NIS assessment is a 244-point composite measure of neurologic impairment which includes a physical exam of lower limbs, upper limbs, and cranial nerves to assess the components: motor strength/weakness (NIS-W), reflexes (NIS-R), and sensation (NIS-S). NIS total score is obtained by combining all the component scores, ranging from 0 to 244. Higher scores represent a greater severity of disease. A positive change from baseline indicates the worsening of neuropathy. |
| Secondary | Change From Baseline in the Total Modified NIS (mNIS +7) Composite Score At Year 3 | Baseline, Year 3 | The mNIS+7 is a composite measure of neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs, and cranial nerves to assess motor strength/weakness (192 points), reflexes (20 points), electrophysiologic measurement of small and large nerve fiber function (10 points), sensory testing (80 points), and postural blood pressure (2 points). The total mNIS+7 composite score is obtained by combining all the component scores, ranging from 0 (no impairment) to 304 (maximum impairment). A negative change from baseline indicates an improvement in neuropathy. |
| Secondary | Change From Baseline in the NIS+7 Total Score at Week 52 | Baseline, Week 52 | The NIS+7 provides additional, objective measures of nerve fibre function and autonomic nerve function in participants with diabetic neuropathy. The NIS+7 includes the full NIS, sum of 5 nerve conduction studies (NCS) (Sural sensory nerve action potential \[SNAP\], tibial motor nerve distal latency, peroneal compound motor action potential \[CMAP\], motor nerve conduction velocity, motor nerve distal latency), vibration detection threshold, and pulse rate response to deep breathing. The total NIS+7 score is obtained by combining all the component scores, ranging from 0 (no impairment) to 270 points (maximum impairment). A positive change from baseline indicates worsening. |
| Secondary | Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Total Score at Year 5 | Baseline, Year 5 | The Norfolk QoL-DN questionnaire is a standardized 47-item patient-reported outcomes measure, sensitive to the perception of the effects of diabetic neuropathy by the participant. The scores range from -4 (best possible QOL) to 136 (worst possible QOL). A negative change from baseline represents improved QOL. |
| Secondary | Change From Baseline in the EuroQOL-5 Dimensions-5 Levels (EQ-5D-5L) Index Score at Year 5 | Baseline, Year 5 | The EQ-5D-5L is a patient-reported measure of QoL based on 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The overall score is rated on a scale from 0 (worst) to 1 (no impairment). Higher scores indicate a higher QoL. A negative change from baseline indicates worsening of QoL. |
| Secondary | Change From Baseline in the EuroQoL Visual Analogue Scale (EQ-VAS) Score at Year 5 | Baseline, Year 5 | EQ-VAS measures the participant's self-rated health on a vertical scale evaluated on a scale of 0 ("worst health you can imagine") to 100 ("best health you can imagine"). Higher scores indicate a higher QOL. A negative change from baseline indicates worsening of QoL. |
| Secondary | Change From Baseline in the Composite Autonomic Symptom Score (COMPASS 31) Total Score at Week 52 | Baseline, Week 52 | COMPASS 31 questionnaire measures autonomic symptoms in participants with neuropathy. The questionnaire consists of 31 clinically selected questions evaluating 6 autonomic domains (orthostatic intolerance, secretomotor, gastrointestinal, bladder, and pupillomotor). COMPASS 31 is measured on a scale from 0 to 100, with 100 representing maximum impairment. |
| Secondary | Change From Baseline in the Modified Body Mass Index (mBMI) at Year 5 | Baseline, Year 5 | Nutritional status of participants was evaluated using the mBMI, calculated as BMI (kilograms per square meter \[kg/m\^2\]) multiplied by the concentration of serum albumin (grams per liter \[g/L\]). A positive change from baseline indicates improvement in nutritional status. |
| Secondary | Change From Baseline in the Rasch-built Overall Disability Scale (R-ODS) at Year 5 | Baseline, Year 5 | The R-ODS is a 24-item patient-reported questionnaire that specifically captures activity and social participation limitations. It measures the level of disability on a scale of 0 (worst) to 48 (best, no limitations), higher score indicates a better outcome. A negative change from baseline indicates worsening of disability. |
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 Adverse Events (AEs) Leading to Study Discontinuation | First dose up to 28 days after last dose of study drug (approximately 5.6 years) | Safety analysis set included all the enrolled participants who received at least 1 dose of patisiran in this study. Percentages are rounded off to the nearest decimal point. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5 | Baseline, Year 5 | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the Total Modified NIS (mNIS +7) Composite Score At Year 3 | Baseline, Year 3 | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the NIS+7 Total Score at Week 52 | Baseline, Week 52 | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Total Score at Year 5 | Baseline, Year 5 | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the EuroQOL-5 Dimensions-5 Levels (EQ-5D-5L) Index Score at Year 5 | Baseline, Year 5 | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the EuroQoL Visual Analogue Scale (EQ-VAS) Score at Year 5 | Baseline, Year 5 | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the Composite Autonomic Symptom Score (COMPASS 31) Total Score at Week 52 | Baseline, Week 52 | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in the Modified Body Mass Index (mBMI) at Year 5 | Baseline, Year 5 | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | kg.g/m^2.L | MEAN |
| SECONDARY | Change From Baseline in the Rasch-built Overall Disability Scale (R-ODS) at Year 5 | Baseline, Year 5 | Full analysis set included all participants who were enrolled in this study. 'Overall number of participants analyzed' indicates the number of participants with data available for outcome measure analysis. 'Number analyzed' indicates the number of participants with data available for analysis at specified timepoint. | score on a scale | MEAN |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group180
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 Adverse Events (AEs) Leading to Study Discontinuation | Prior Placebo Group of Study 004 | 49.0 | percentage of participants | ||
| Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation | Prior Patisiran Group of Study 004 | 16.8 | percentage of participants | ||
| Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation | Prior Patisiran Group of Study 003 | 0.0 | percentage of participants | ||
| Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5 | Prior Placebo Group of Study 004 | Baseline | 81.47 | Standard Deviation 41.674 | score on a scale |
| Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5 | Prior Patisiran Group of Study 004 | Baseline | 62.26 | Standard Deviation 37.875 | score on a scale |
| Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5 | Prior Patisiran Group of Study 003 | Baseline | 35.48 | Standard Deviation 28.686 | score on a scale |
| Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5 | Prior Placebo Group of Study 004 | Change From Baseline at Year 5 | 11.45 | Standard Deviation 16.566 | score on a scale |
| Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5 | Prior Patisiran Group of Study 004 | Change From Baseline at Year 5 | 10.72 | Standard Deviation 13.654 | score on a scale |
| Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5 | Prior Patisiran Group of Study 003 | Change From Baseline at Year 5 | 11.18 | Standard Deviation 17.554 | score on a scale |
| Change From Baseline in the Total Modified NIS (mNIS +7) Composite Score At Year 3 | Prior Placebo Group of Study 004 | Baseline | 101.07 | Standard Error 43.774 | score on a scale |
1 to 10 of 180Source ClinicalTrials.gov
See all 180 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Prior Placebo Group of Study 004 | 49 | |
| Overall Study | STARTED | Prior Patisiran Group of Study 004 | 137 | |
| Overall Study | STARTED | Prior Patisiran Group of Study 003 | 25 | |
| Overall Study | Full Analysis Set | Prior Placebo Group of Study 004 | 49 | |
| Overall Study | Full Analysis Set | Prior Patisiran Group of Study 004 | 137 | |
| Overall Study | Full Analysis Set | Prior Patisiran Group of Study 003 | 25 | |
| Overall Study | Safety Analysis Set | Prior Placebo Group of Study 004 | 49 | |
| Overall Study | Safety Analysis Set | Prior Patisiran Group of Study 004 | 137 | |
| Overall Study | Safety Analysis Set | Prior Patisiran Group of Study 003 | 25 | |
| Overall Study | Pharmacodynamic (PD) Analysis Set | Prior Placebo Group of Study 004 | 40 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events993
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 | Prior Placebo Group of Study 004 | 7 | 49 | |
| other | Anaemia | Blood and lymphatic system disorders | Prior Patisiran Group of Study 004 | 10 | 137 | |
| other | Anaemia | Blood and lymphatic system disorders | Prior Patisiran Group of Study 003 | 2 | 25 | |
| other | Arrhythmia | Cardiac disorders | Prior Placebo Group of Study 004 | 3 | 49 | |
| other | Arrhythmia | Cardiac disorders | Prior Patisiran Group of Study 004 | 1 | 137 | |
| other | Arrhythmia | Cardiac disorders | Prior Patisiran Group of Study 003 | 0 | 25 | |
| other | Atrial Fibrillation | Cardiac disorders | Prior Placebo Group of Study 004 | 2 | 49 | |
| other | Atrial Fibrillation | Cardiac disorders | Prior Patisiran Group of Study 004 | 14 | 137 | |
| other | Atrial Fibrillation | Cardiac disorders | Prior Patisiran Group of Study 003 | 0 | 25 | |
| other | Atrial Flutter | Cardiac disorders | Prior Placebo Group of Study 004 | 1 | 49 |
1 to 10 of 993Source ClinicalTrials.gov
See all 993 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Prior Placebo Group of Study 004 | 19 | 49 |
| Total, all-cause mortality | Prior Patisiran Group of Study 004 | 21 | 137 |
| Total, all-cause mortality | Prior Patisiran Group of Study 003 | 1 | 25 |
| Total, other adverse events | Prior Placebo Group of Study 004 | 47 | 49 |
| Total, other adverse events | Prior Patisiran Group of Study 004 | 135 | 137 |
| Total, other adverse events | Prior Patisiran Group of Study 003 | 25 | 25 |
| Total, serious adverse events | Prior Placebo Group of Study 004 | 39 | 49 |
| Total, serious adverse events | Prior Patisiran Group of Study 004 | 82 | 137 |
| Total, serious adverse events | Prior Patisiran Group of Study 003 | 12 | 25 |
Source ClinicalTrials.gov
Papers3
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT02510261
GET https://conductscience.org/api/trials/v1/NCT02510261/registered?page=2