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The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02510261
Recruitment Status : Completed
First Posted : July 29, 2015
Last Update Posted : January 20, 2023
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

Condition or disease Intervention/treatment Phase
Amyloidosis Drug: Patisiran (ALN-TTR02) Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 211 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients With Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Patisiran Clinical Study
Study Start Date : July 2015
Actual Primary Completion Date : November 23, 2022
Actual Study Completion Date : November 23, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis
Drug Information available for: Patisiran

Arms and Interventions
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Arm Intervention/treatment
Experimental: Patisiran (ALN-TTR02) Drug: Patisiran (ALN-TTR02)
administered by intravenous (IV) infusion


Outcome Measures
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Primary Outcome Measures :
  1. Safety and tolerability of long-term dosing of patisiran as measured by the proportion of subjects with adverse events leading to discontinuation of study drug [ Time Frame: Up to 5 years ]

Secondary Outcome Measures :
  1. Assessment of changes from baseline in neurologic impairment assessed using the Neuropathy Impairment Score (NIS) associated with long-term dosing of patisiran [ Time Frame: Baseline up to 5 years ]
  2. Assessment of changes from baseline in neurologic impairment assessed using the Modified NIS (mNIS +7) composite score associated with long-term dosing of patisiran [ Time Frame: Baseline up to 3 years ]
  3. Assessment of changes from baseline in neurologic impairment assessed using NIS+7 associated with long-term dosing of patisiran [ Time Frame: Baseline up to 3 years ]
  4. Assessment of changes from baseline in quality of life using the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire associated with long-term dosing of patisiran [ Time Frame: Baseline up to 5 years ]
  5. Assessment of changes from baseline in quality of life using the EuroQOL (EQ-5D) questionnaire associated with long-term dosing of patisiran [ Time Frame: Baseline up to 5 years ]
  6. Assessment of changes from baseline in autonomic function assessed using the Composite Autonomic Symptom Score (COMPASS 31) with long-term dosing of patisiran [ Time Frame: Baseline up to 52 weeks ]
  7. Assessment of changes from baseline in serum TTR lowering associated with long-term dosing of patisiran [ Time Frame: Baseline up to 5 years ]
  8. Assessment of changes from baseline in nutritional status using modified body mass index (mBMI) associated with long-term dosing of patisiran [ Time Frame: Baseline up to 5 years ]
  9. Assessment of changes from baseline in disability reported by patients using the Rasch-built Overall Disability Scale (R-ODS) associated with long-term dosing of patisiran [ Time Frame: Baseline up to 5 years ]
  10. Assessment of changes from baseline in motor function assessed by NIS-Weakness (NIS-W) with long-term dosing of patisiran [ Time Frame: Baseline up to 5 years ]
  11. Assessment of changes from baseline in motor function assessed by timed 10-meter walk test with long-term dosing of patisiran [ Time Frame: Baseline up to 5 years ]
  12. Assessment of changes from baseline in motor function assessed by grip strength test with long-term dosing of patisiran [ Time Frame: Baseline up to 52 weeks ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Have completed a patisiran study (i.e., completed the last efficacy visit in the parent study) and, in the opinion of the investigator, tolerated study drug
  • Be willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent

Exclusion Criteria:

  • Any new or uncontrolled condition that could make the patient unsuitable for participation
Contacts and Locations
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Information from the National Library of Medicine

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): NCT02510261


Locations
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Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02510261    
Other Study ID Numbers: ALN-TTR02-006
First Posted: July 29, 2015    Key Record Dates
Last Update Posted: January 20, 2023
Last Verified: January 2023
Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
FAP
Familial Amyloid Polyneuropathy
 TTR
Transthyretin
Amyloidosis
Additional relevant MeSH terms:
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Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases