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To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patisiran Prospective Cohort | Patients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy. |
| |
| Patisiran Mixed Cohort | Patients who are currently on commercial patisiran therapy for less than 12 months at study enrollment. |
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| Patisiran Retrospective Cohort | Patients who have been on commercial patisiran therapy for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Patisiran | Drug | Patisiran-lipid complex injection, for intravenous use |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline | PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden. | Baseline, Month 12 |
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Inclusion Criteria:
Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation
PND score of I-IIIB at baseline.
Exposure to commercial patisiran in one of the 3 cohorts:
Exclusion Criteria:
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Participants with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Los Angeles | California | 90095 | United States | ||
| Clinical Trial Site |
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|
| San Diego |
| California |
| 92103 |
| United States |
| Clinical Trial Site | Aurora | Colorado | 80045 | United States |
| Clinical Trial Site | Farmington | Connecticut | 06030 | United States |
| Clinical Trial Site | Jacksonville | Florida | 32224 | United States |
| Clinical Trial Site | Tampa | Florida | 33609 | United States |
| Clinical Trial Site | Braselton | Georgia | 30517 | United States |
| Clinical Trial Site | Macon | Georgia | 31201 | United States |
| Clinical Trial Site | Chicago | Illinois | 60611 | United States |
| Clinical Trial Site | Chicago | Illinois | 60612 | United States |
| Clinical Trial Site | Boston | Massachusetts | 02111 | United States |
| Clinical Trial Site | Detroit | Michigan | 48202 | United States |
| Clinical Trial Site | Jackson | Mississippi | 39216 | United States |
| Clinical Trial Site | Kansas City | Missouri | 64111 | United States |
| Clinical Trial Site | Omaha | Nebraska | 68198 | United States |
| Clinical Trial Site | Chapel Hill | North Carolina | 27599 | United States |
| Clinical Trial Site | Charlotte | North Carolina | 28207 | United States |
| Clinical Trial Site | Columbus | Ohio | 43210 | United States |
| Clinical Trial Site | Allentown | Pennsylvania | 18103 | United States |
| Clinical Trial Site | Bethlehem | Pennsylvania | 18015 | United States |
| Clinical Trial Site | Lancaster | Pennsylvania | 17602 | United States |
| Clinical Trial Site | Pittsburgh | Pennsylvania | 15212 | United States |
| Clinical Trial Site | Pittsburgh | Pennsylvania | 15232 | United States |
| Clinical Trial Site | Germantown | Tennessee | 38138 | United States |
| Clinical Trial Site | Austin | Texas | 78756 | United States |
| Clinical Trial Site | San Antonio | Texas | 78229 | United States |
| Clinical Trial Site | Milwaukee | Wisconsin | 53226 | United States |
| ID | Term |
|---|---|
| D000686 | Amyloidosis |
| D011115 | Polyneuropathies |
| D028227 | Amyloid Neuropathies, Familial |
| C567782 | Amyloidosis, Hereditary, Transthyretin-Related |
| D017772 | Amyloid Neuropathies |
| D028226 | Amyloidosis, Familial |
| D010523 | Peripheral Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D008661 | Metabolism, Inborn Errors |
| ID | Term |
|---|---|
| D009750 | Nutritional and Metabolic Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000606954 | patisiran |
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