Trial · NCT01960348
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
NCT01960348, Phase 3, Completed, Alnylam Pharmaceuticals; results first posted 2018-09-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis. Trials, record NCT01960348. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01960348
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
- NCT number
- NCT01960348
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 225 participants, actual
- Lead sponsor
- Alnylam Pharmaceuticals
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
- Interventions
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- First posted
- 2013-10-10
- Last updated
- 2024-04-22
- Other registries' IDs
- eudract:2013-002987-17
- Results first posted
- 2018-09-06
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Modified Neuropathy Impairment Score +7 (mNIS+7) | 18mo | The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in mNIS+7 at 18 months. The mNIS+7 is a composite score that quantitates motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome. |
| Secondary | Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire | 18mo | The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in Norfolk QoL-DN at 18 months. The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that is sensitive to the different features of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome. |
| Secondary | Neurological Impairment Score-Weakness (NIS-W) Score | 18mo | The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in NIS-W at 18 months. NIS-W is a measure of motor strength, comprised of cranial nerve and both upper and lower limb motor assessments. The minimum and maximum values are 0 and 192, respectively. A higher score indicates a worse outcome. |
| Secondary | Rasch-built Overall Disability Scale (R-ODS) Score | 18mo | The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in R-ODS score at 18 months. The R-ODS is comprised of a 24-item linearly weighted scale that specifically captures activity and social participation limitations in patients. The minimum and maximum values are 0 and 48, respectively. A higher score indicates a better outcome. |
| Secondary | Timed 10-meter Walk Test (10-MWT, Gait Speed) | 18mo | The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in 10-MWT at 18 months. Ability to ambulate (gait speed) was assessed through the 10-meter walk test (10-MWT). The walk had to be completed without assistance from another person; ambulatory aids such as canes and walkers were permitted. |
| Secondary | Modified Body Mass Index (mBMI) | 18mo | The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in mBMI at 18 months. The nutritional status of patients was evaluated using the mBMI; calculated as the product of BMI (weight in kilograms divided by the square of height in meters) and serum albumin (g/L). |
| Secondary | Autonomic Symptoms Questionnaire (Composite Autonomic Symptom Score [COMPASS 31]) | 18mo | The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in COMPASS 31 at 18 months. The COMPASS 31 is a measure of autonomic neuropathy symptoms. The questions evaluated 6 autonomic domains (orthostatic intolerance, vasomotor, secretomotor, gastrointestinal, bladder, and pupillomotor). The minimum and maximum values are 0 and 100, respectively. A higher score indicates a worse outcome. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Modified Neuropathy Impairment Score +7 (mNIS+7) | 18mo | Per protocol (PP) population: All randomized participants who received at least 1 dose of patisiran-LNP or placebo, completed baseline and the 18-month mNIS+7 assessments, and did not experience any major protocol deviations that may have impacted the efficacy results. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire | 18mo | Per protocol (PP) population: All randomized participants who received at least 1 dose of patisiran-LNP or placebo, completed baseline and the 18-month Norfolk QoL-DN assessments, and did not experience any major protocol deviations that may have impacted the efficacy results. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Neurological Impairment Score-Weakness (NIS-W) Score | 18mo | Per protocol (PP) population: All randomized participants who received at least 1 dose of patisiran-LNP or placebo, completed baseline and the 18-month NIS-W assessments, and did not experience any major protocol deviations that may have impacted the efficacy results. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Rasch-built Overall Disability Scale (R-ODS) Score | 18mo | Per protocol (PP) population: All randomized participants who received at least 1 dose of patisiran-LNP or placebo, completed baseline and either the 18-month R-ODS assessments, and did not experience any major protocol deviations that may have impacted the efficacy results. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Timed 10-meter Walk Test (10-MWT, Gait Speed) | 18mo | Per protocol (PP) population: All randomized participants who received at least 1 dose of patisiran-LNP or placebo, completed baseline and the 18-month 10-MWT assessments, and did not experience any major protocol deviations that may have impacted the efficacy results. | m/sec | LEAST_SQUARES_MEAN |
| SECONDARY | Modified Body Mass Index (mBMI) | 18mo | Per protocol (PP) population: All randomized participants who received at least 1 dose of patisiran-LNP or placebo, completed baseline and the 18-month mBMI assessments and did not experience any major protocol deviations that may have impacted the results.. | kg/m^2 × albumin g/L | LEAST_SQUARES_MEAN |
| SECONDARY | Autonomic Symptoms Questionnaire (Composite Autonomic Symptom Score [COMPASS 31]) | 18mo | Per protocol (PP) population: All randomized participants who received at least 1 dose of patisiran-LNP or placebo, completed baseline and the 18-month COMPASS 31 assessments, and did not experience any major protocol deviations that may have impacted the efficacy results. | score on a scale | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Modified Neuropathy Impairment Score +7 (mNIS+7) | Patisiran (ALN-TTR02) | -6.03 | Standard Error 1.739 | score on a scale | |
| Modified Neuropathy Impairment Score +7 (mNIS+7) | Placebo | 27.96 | Standard Error 2.602 | score on a scale | |
| Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire | Patisiran (ALN-TTR02) | -6.7 | Standard Error 1.77 | score on a scale | |
| Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire | Placebo | 14.4 | Standard Error 2.73 | score on a scale | |
| Neurological Impairment Score-Weakness (NIS-W) Score | Patisiran (ALN-TTR02) | 0.05 | Standard Error 1.306 | score on a scale | |
| Neurological Impairment Score-Weakness (NIS-W) Score | Placebo | 17.93 | Standard Error 1.959 | score on a scale | |
| Rasch-built Overall Disability Scale (R-ODS) Score | Patisiran (ALN-TTR02) | 0.0 | Standard Error 0.59 | score on a scale | |
| Rasch-built Overall Disability Scale (R-ODS) Score | Placebo | -8.9 | Standard Error 0.88 | score on a scale | |
| Timed 10-meter Walk Test (10-MWT, Gait Speed) | Patisiran (ALN-TTR02) | 0.077 | Standard Error 0.0242 | m/sec | |
| Timed 10-meter Walk Test (10-MWT, Gait Speed) | Placebo | -0.235 | Standard Error 0.0358 | m/sec |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Patisiran (ALN-TTR02) | 148 | |
| Overall Study | STARTED | Placebo | 77 | |
| Overall Study | COMPLETED | Patisiran (ALN-TTR02) | 138 | |
| Overall Study | COMPLETED | Placebo | 55 | |
| Overall Study | NOT COMPLETED | Patisiran (ALN-TTR02) | 10 | |
| Overall Study | NOT COMPLETED | Placebo | 22 |
Source ClinicalTrials.gov
Adverse events372
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 | Diarrhoea | Gastrointestinal disorders | Patisiran (ALN-TTR02) | 50 | 148 | 156 |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 28 | 77 | 94 |
| other | Oedema peripheral | General disorders | Patisiran (ALN-TTR02) | 43 | 148 | 68 |
| other | Oedema peripheral | General disorders | Placebo | 17 | 77 | 35 |
| other | Infusion related reaction | Immune system disorders | Patisiran (ALN-TTR02) | 28 | 148 | 145 |
| other | Infusion related reaction | Immune system disorders | Placebo | 7 | 77 | 79 |
| other | Fall | Injury, poisoning and procedural complications | Patisiran (ALN-TTR02) | 25 | 148 | 47 |
| other | Fall | Injury, poisoning and procedural complications | Placebo | 22 | 77 | 42 |
| other | Constipation | Gastrointestinal disorders | Patisiran (ALN-TTR02) | 22 | 148 | 29 |
| other | Constipation | Gastrointestinal disorders | Placebo | 12 | 77 | 17 |
1 to 10 of 372Source ClinicalTrials.gov
See all 372 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Patisiran (ALN-TTR02) | 7 | 148 |
| Total, all-cause mortality | Placebo | 6 | 77 |
| Total, other adverse events | Patisiran (ALN-TTR02) | 143 | 148 |
| Total, other adverse events | Placebo | 75 | 77 |
| Total, serious adverse events | Patisiran (ALN-TTR02) | 54 | 148 |
| Total, serious adverse events | Placebo | 31 | 77 |
Source ClinicalTrials.gov
Papers7
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 |
|---|---|
| Patisiran (ALN-TTR02) vs Placebo: deaths from any cause | Trial comparisons |
| Patisiran (ALN-TTR02) 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/NCT01960348
GET https://conductscience.org/api/trials/v1/NCT01960348/events?page=2