ClinicalTrials.gov
ClinicalTrials.gov Menu

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 : Enrolling by invitation
First Posted : July 29, 2015
Last Update Posted : December 11, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 211 participants
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
Estimated Primary Completion Date : August 2022
Estimated Study Completion Date : August 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis

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




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: 52 weeks ]

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, week 52 ]
  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, week 52 ]
  3. Assessment of changes from baseline in neurologic impairment assessed using NIS+7 associated with long-term dosing of patisiran [ Time Frame: baseline, week 52 ]
  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, week 52 ]
  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, week 52 ]
  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, week 52 ]
  7. Assessment of changes from baseline in serum TTR lowering associated with long-term dosing of patisiran [ Time Frame: baseline, week 52 ]
  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, week 52 ]
  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, week 52 ]
  10. Assessment of changes from baseline in motor function assessed by NIS-Weakness (NIS-W) with long-term dosing of patisiran [ Time Frame: baseline, week 52 ]
  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, week 52 ]
  12. Assessment of changes from baseline in motor function assessed by grip strength test with long-term dosing of patisiran [ Time Frame: baseline, week 52 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

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


  Show 51 Study Locations
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Sunita Goyal, MD Alnylam Pharmaceuticals

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02510261     History of Changes
Other Study ID Numbers: ALN-TTR02-006
First Posted: July 29, 2015    Key Record Dates
Last Update Posted: December 11, 2018
Last Verified: December 2018

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
FAP
Familial Amyloid Polyneuropathy
TTR
Transthyretin
Amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Polyneuropathies
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases