Trial · NCT01617967
Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
NCT01617967, Phase 2, Completed, Alnylam Pharmaceuticals; results first posted 2018-11-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis. Trials, record NCT01617967. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01617967
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
- NCT number
- NCT01617967
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 29 participants, actual
- Lead sponsor
- Alnylam Pharmaceuticals
- Conditions
- TTR-mediated Amyloidosis
- Interventions
- Patisiran
- First posted
- 2012-06-13
- Last updated
- 2024-04-19
- Other registries' IDs
- eudract:2012-000467-24
- Results first posted
- 2018-11-21
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Up to 56 days post first dose | The number of participants experiencing at least one adverse event (AE), at least one serious adverse event (SAE) and study drug discontinuation (due to any reason). |
| Secondary | Percentage Change From Baseline in Serum Transthyretin (TTR) Protein | Baseline to Day 21/28 and Day 42/56 depending on dosing regimen (Q3W/Q4W) | Percentage change of TTR relative to pretreatment/baseline levels is reported. For arms with a dosing regimen of Q4W TTR protein samples were measured on Days 28 and 56. For the arms with a dosing regimen of Q3W TTR protein samples were measured on Days 21 and 42. |
| Secondary | Pharmacokinetic Parameters of Patisiran - Area Under the Concentration Curve From Time 0 to Last Measurable Time Point (AUC0-last) | Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W) | Pharmacokinetic profiles for patisiran (ALN-TTR02) were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21. |
| Secondary | Pharmacokinetic Parameters of Patisiran - Maximum Observed Plasma Concentration (Cmax) | Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W) | Pharmacokinetic profiles for patisiran (ALN-TTR02) were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21. |
| Secondary | Pharmacokinetic Parameters of Patisiran - Beta Elimination Half-life (t1/2 Beta) | Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency | Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21. |
| Secondary | Pharmacokinetic Parameters of Patisiran - Systemic Clearance (CL) | Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency | Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21. |
| Secondary | Pharmacokinetic Parameters of Patisiran - Apparent Volume of Distribution at Steady State (Vss) | Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency | Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21. |
| Secondary | Pharmacokinetic Parameters of Patisiran - Renal Clearance (CLR) | Predose (within 1 h of planned dosing start) and post-infusion at 0-6 h (pooled) on Day 0 and Day 21/28 depending on dosing frequency | Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Up to 56 days post first dose | Intent-to-treat (ITT) population included all participants, who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage Change From Baseline in Serum Transthyretin (TTR) Protein | Baseline to Day 21/28 and Day 42/56 depending on dosing regimen (Q3W/Q4W) | ITT population included all participants, who received at least 1 dose of study drug. | Percentage | MEAN |
| SECONDARY | Pharmacokinetic Parameters of Patisiran - Area Under the Concentration Curve From Time 0 to Last Measurable Time Point (AUC0-last) | Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W) | The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen. | ng*h/mL | MEAN |
| SECONDARY | Pharmacokinetic Parameters of Patisiran - Maximum Observed Plasma Concentration (Cmax) | Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W) | The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen. | ng/mL | MEAN |
| SECONDARY | Pharmacokinetic Parameters of Patisiran - Beta Elimination Half-life (t1/2 Beta) | Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency | The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen. | hour (h) | MEAN |
| SECONDARY | Pharmacokinetic Parameters of Patisiran - Systemic Clearance (CL) | Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency | The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen. | L/h/kg | MEAN |
| SECONDARY | Pharmacokinetic Parameters of Patisiran - Apparent Volume of Distribution at Steady State (Vss) | Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency | The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen. | L/kg | MEAN |
| SECONDARY | Pharmacokinetic Parameters of Patisiran - Renal Clearance (CLR) | Predose (within 1 h of planned dosing start) and post-infusion at 0-6 h (pooled) on Day 0 and Day 21/28 depending on dosing frequency | The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen. | mL/h/kg | MEAN |
Source ClinicalTrials.gov
Results by group90
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Patisiran 0.010 mg/kg Q4W | At Least 1 AE | 1 | Participants | |
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Patisiran 0.050 mg/kg Q4W | At Least 1 AE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Patisiran 0.150 mg/kg Q4W | At Least 1 AE | 2 | Participants | |
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Patisiran 0.300 mg/kg Q4W | At Least 1 AE | 7 | Participants | |
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Patisiran 0.300 mg/kg Q3W | At Least 1 AE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Patisiran 0.300 mg/kg Q3W Alternative | At Least 1 AE | 7 | Participants | |
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Patisiran 0.010 mg/kg Q4W | At Least 1 SAE | 0 | Participants | |
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Patisiran 0.050 mg/kg Q4W | At Least 1 SAE | 0 | Participants | |
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Patisiran 0.150 mg/kg Q4W | At Least 1 SAE | 0 | Participants | |
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation | Patisiran 0.300 mg/kg Q4W | At Least 1 SAE | 1 | Participants |
1 to 10 of 90Source ClinicalTrials.gov
See all 90 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Patisiran 0.010 mg/kg Q4W | 4 | |
| Overall Study | STARTED | Patisiran 0.050 mg/kg Q4W | 3 | |
| Overall Study | STARTED | Patisiran 0.150 mg/kg Q4W | 3 | |
| Overall Study | STARTED | Patisiran 0.300 mg/kg Q4W | 7 | |
| Overall Study | STARTED | Patisiran 0.300 mg/kg Q3W | 3 | |
| Overall Study | STARTED | Patisiran 0.300 mg/kg Q3W Alternative | 9 | |
| Overall Study | COMPLETED | Patisiran 0.010 mg/kg Q4W | 3 | |
| Overall Study | COMPLETED | Patisiran 0.050 mg/kg Q4W | 3 | |
| Overall Study | COMPLETED | Patisiran 0.150 mg/kg Q4W | 3 | |
| Overall Study | COMPLETED | Patisiran 0.300 mg/kg Q4W | 6 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events294
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 | Patisiran 0.010 mg/kg Q4W | 0 | 4 | |
| other | Anaemia | Blood and lymphatic system disorders | Patisiran 0.050 mg/kg Q4W | 0 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | Patisiran 0.150 mg/kg Q4W | 0 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | Patisiran 0.300 mg/kg Q4W | 0 | 7 | |
| other | Anaemia | Blood and lymphatic system disorders | Patisiran 0.300 mg/kg Q3W | 0 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | Patisiran 0.300 mg/kg Q3W Alternative | 1 | 9 | |
| other | Leukocytosis | Blood and lymphatic system disorders | Patisiran 0.010 mg/kg Q4W | 0 | 4 | |
| other | Leukocytosis | Blood and lymphatic system disorders | Patisiran 0.050 mg/kg Q4W | 0 | 3 | |
| other | Leukocytosis | Blood and lymphatic system disorders | Patisiran 0.150 mg/kg Q4W | 0 | 3 | |
| other | Leukocytosis | Blood and lymphatic system disorders | Patisiran 0.300 mg/kg Q4W | 1 | 7 |
1 to 10 of 294Source ClinicalTrials.gov
See all 294 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Patisiran 0.010 mg/kg Q4W | 0 | 4 |
| Total, all-cause mortality | Patisiran 0.050 mg/kg Q4W | 0 | 3 |
| Total, all-cause mortality | Patisiran 0.150 mg/kg Q4W | 0 | 3 |
| Total, all-cause mortality | Patisiran 0.300 mg/kg Q4W | 0 | 7 |
| Total, all-cause mortality | Patisiran 0.300 mg/kg Q3W | 0 | 3 |
| Total, all-cause mortality | Patisiran 0.300 mg/kg Q3W Alternative | 0 | 9 |
| Total, other adverse events | Patisiran 0.010 mg/kg Q4W | 1 | 4 |
| Total, other adverse events | Patisiran 0.050 mg/kg Q4W | 3 | 3 |
| Total, other adverse events | Patisiran 0.150 mg/kg Q4W | 2 | 3 |
| Total, other adverse events | Patisiran 0.300 mg/kg Q4W | 7 | 7 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 26338094 | DERIVED |
Source PubMed
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/NCT01617967
GET https://conductscience.org/api/trials/v1/NCT01617967/results?page=2