Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
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|ClinicalTrials.gov Identifier: NCT03862807|
Recruitment Status : Recruiting
First Posted : March 5, 2019
Last Update Posted : April 16, 2019
|Condition or disease||Intervention/treatment||Phase|
|Amyloidosis, Familial Transthyretin Amyloidosis||Drug: Patisiran||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) With Disease Progression Post-Orthotopic Liver Transplant|
|Actual Study Start Date :||March 27, 2019|
|Estimated Primary Completion Date :||January 14, 2021|
|Estimated Study Completion Date :||January 14, 2021|
Participants will receive patisiran during the Treatment Period.
Patisiran will be administered by intravenous (IV) infusion.
Other Name: ALN-TTR02
- Average of Month 6 and Month 12 Percentage Reduction from Baseline in Serum Transthyretin (TTR) [ Time Frame: Baseline, Months 6 and 12 ]
- Change from Baseline in the Neuropathy Impairment Score (NIS) at Month 12 [ Time Frame: Baseline, Month 12 ]The NIS is a composite neurologic impairment score that assesses motor weakness (NIS-W), sensation (NIS-S) and reflexes (NIS-R) by physical exam. The minimum and maximum values are 0 and 244, respectively. A higher score indicates a worse outcome.
- Change from Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Score at Month 12 [ Time Frame: Baseline, Month 12 ]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.
- Change from Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Month 12 [ Time Frame: Baseline, Month 12 ]The R-ODS is comprised of a 24-item linearly weighted scale that specifically captures activity and social participation limitations. The minimum and maximum values are 0 and 48, respectively. A higher score indicates a better outcome.
- Change from Baseline in the Composite Autonomic Symptom Score (COMPASS-31) at Month 12 [ Time Frame: Baseline, Month 12 ]The COMPASS-31 questionnaire is a measure of autonomic neuropathy symptoms. The questions evaluate 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.
- Change from Baseline in the Modified Body Mass Index (mBMI) at Month 12 [ Time Frame: Baseline, Month 12 ]
- Percentage of Participants With Adverse Events [ Time Frame: From baseline to end of study at Month 13 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03862807
|Contact: Alnylam Clinical Trial Information Line||1-877-ALNYLAMfirstname.lastname@example.org|
|Contact: Alnylam Clinical Trial Information Lineemail@example.com|
|Clinical Trial Site||Recruiting|
|Study Director:||Jing Jing Wang, MD||Alnylam Pharmaceuticals|