Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2025-522544-40-00 | EU Trial (CTIS) Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The purpose of this study is to:
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Nucresiran 300 mg | Experimental | Patients will be administered nucresiran 300 mg subcutaneously (SC) once every 6 months (q6M) during the Treatment Period and Treatment Extension Period |
|
| Vutrisiran 25 mg followed by Nucresiran 300 mg | Active Comparator | Patients will be administered vutrisiran 25 mg SC every 3 months (q3M) during the Treatment Period followed by nucresiran 300 mg SC q6M during the Treatment Extension Period |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nucresiran | Drug | Nucresiran 300 mg administered SC q6M |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 9 | The mNIS+7 is a composite score that measures neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs and cranial nerves to assess motor strength/weakness and deep tendon reflexes, electrophysiologic measurement of large nerve fiber function, sensory testing and postural blood pressure. The mNIS+7 is scored from 0 (no impairment) to 304 points (maximum impairment). A higher score indicates a worse outcome. | Baseline and Month 9 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Total Score Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 9 | The Norfolk QoL-DN is a standardized 35-item patient-rated questionnaire used to assess 5 domains: physical function, large fiber neuropathy, activities of daily living, symptoms, small fiber neuropathy, and autonomic neuropathy. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Information Line | Contact | 1-877-ALNYLAM | clinicaltrials@alnylam.com | |
| Clinical Trial Information Line | Contact | 1-877-256-9526 | clinicaltrials@alnylam.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Trial Site | Recruiting | Aurora | Colorado | 80045 | United States | |
| Clinical Trial Site |
Phase 2-4 trials:
Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU.
Access to data may be declined where there is likelihood a patient could be identified or other feasibility issue, where there is a potential conflict of interest, a planned business activities or an actual or potential competitive risk. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Timeframes for data access may vary and can take up to 6 months or more.
Requests for access to data can be submitted via the website www.vivli.org. Questions can also be directed to datasharing@alnylam.com.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Vutrisiran | Drug | Vutrisiran 25 mg administered SC q3M |
|
|
| Baseline and Month 9 |
| Percent Reduction in Serum TTR Levels in the Nucresiran Group Compared to the In-study Vutrisiran Group through Month 9 | Up to Month 9 |
| Change from Baseline in Modified Body Mass Index (mBMI) Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 9 | The mBMI, which is a measure of nutritional status, is calculated as the product of body mass index (BMI) (weight in kilograms divided by the square of height in meters) and serum albumin (g/L) to reflect fluid balance, such as fluid accumulation or dehydration. A negative change from baseline indicates a better outcome. | Baseline and Month 9 |
| Change from Baseline in the mNIS+7 Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 18 | The mNIS+7 is a composite score that measures neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs and cranial nerves to assess motor strength/weakness and deep tendon reflexes, electrophysiologic measurement of large nerve fiber function, sensory testing and postural blood pressure. The mNIS+7 is scored from 0 (no impairment) to 304 points (maximum impairment). A higher score indicates a worse outcome. | Baseline and Month 18 |
| Change from Baseline in Norfolk QoL-DN Total Score Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 18 | The Norfolk QoL-DN is a standardized 35-item patient-rated questionnaire used to assess 5 domains: physical function, large fiber neuropathy, activities of daily living, symptoms, small fiber neuropathy, and autonomic neuropathy. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome. | Baseline and Month 18 |
| Change from Baseline in mBMI Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 18 | The mBMI, which is a measure of nutritional status, is calculated as the product of body mass index (BMI) (weight in kilograms divided by the square of height in meters) and serum albumin (g/L) to reflect fluid balance, such as fluid accumulation or dehydration. A negative change from baseline indicates a better outcome. | Baseline and Month 18 |
| Change from Baseline in Rasch-built Overall Disability Scale (R-ODS) Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 18 | The R-ODS is a patient-reported measure of level of disability on a scale of 0-48, with 0 being the worst and 48 the best (no limitations); scores are based on activities of daily living and social participation. An increase in R-ODS from baseline suggests improvement in disability, and a decrease from baseline suggests worsening of disability. | Baseline and Month 18 |
| Change from Baseline in Timed 10-meter Walk Test (10-MWT) Compared to the External Placebo Group from the APOLLO Study (NCT01960348) at Month 18 | The timed 10-MWT is a measure of ambulatory ability and measures the time (in seconds) that it takes a participant to walk 10 meters (gait speed). An increase in gait speed from baseline represents improvement, and a decrease from baseline represents worsening. | Baseline and Month 18 |
| Percent Reduction in Serum TTR Levels in the Nucresiran Group Compared to the In-study Vutrisiran Group through Month 18 | Up to Month 18 |
| Percent Reduction in Serum TTR Levels in the Nucresiran Group Compared to the In-study Vutrisiran Group at Week 6 | Week 6 |
| Recruiting |
| Chicago |
| Illinois |
| 60611 |
| United States |
| Clinical Trial Site | Recruiting | Baltimore | Maryland | 21287 | United States |
| Clinical Trial Site | Recruiting | Boston | Massachusetts | 02118 | United States |
| Clinical Trial Site | Recruiting | Rochester | Minnesota | 55905 | United States |
| Clinical Trial Site | Recruiting | New York | New York | 10032 | United States |
| Clinical Trial Site | Recruiting | Chapel Hill | North Carolina | 27514 | United States |
| Clinical Trial Site | Recruiting | Dallas | Texas | 75246 | United States |
| Clinical Trial Site | Recruiting | Darlinghurst | 2010 | Australia |
| Clinical Trial Site | Recruiting | São Paulo | 04038-002 | Brazil |
| Clinical Trial Site | Recruiting | Égkomi | 2371 | Cyprus |
| Clinical Trial Site | Recruiting | Créteil | 94000 | France |
| Clinical Trial Site | Recruiting | Le Kremlin-Bicêtre | 94270 | France |
| Clinical Trial Site | Recruiting | Marseille | 13005 | France |
| Clinical Trial Site | Recruiting | Athens | 115 28 | Greece |
| Clinical Trial Site | Recruiting | Heraklion | 715 00 | Greece |
| Clinical Trial Site | Recruiting | Florence | 50134 | Italy |
| Clinical Trial Site | Recruiting | Milan | 20133 | Italy |
| Clinical Trial Site | Recruiting | Pavia | 27100 | Italy |
| Clinical Trial Site | Recruiting | Suita | 565-0871 | Japan |
| Clinical Trial Site | Recruiting | Kuala Lumpur | 59100 | Malaysia |
| Clinical Trial Site | Recruiting | Porto | 4099-001 | Portugal |
| Clinical Trial Site | Recruiting | Seoul | 05030 | South Korea |
| Clinical Trial Site | Recruiting | Seoul | 06351 | South Korea |
| Clinical Trial Site | Recruiting | Stockholm | 113 61 | Sweden |
| Clinical Trial Site | Recruiting | Umeå | 907 37 | Sweden |
| Clinical Trial Site | Recruiting | Taipei | 10002 | Taiwan |
| Clinical Trial Site | Recruiting | Taipei | 112 | Taiwan |
| Clinical Trial Site | Recruiting | Taoyuan City | 333 | Taiwan |
| ID | Term |
|---|---|
| D011115 | Polyneuropathies |
| D000686 | Amyloidosis |
| ID | Term |
|---|---|
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided