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Trial record 46 of 365 for:    transthyretin

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)

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ClinicalTrials.gov Identifier: NCT02510261
Recruitment Status : Active, not recruiting
First Posted : July 29, 2015
Last Update Posted : September 10, 2019
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE July 16, 2015
First Posted Date  ICMJE July 29, 2015
Last Update Posted Date September 10, 2019
Study Start Date  ICMJE July 2015
Estimated Primary Completion Date August 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 27, 2015)
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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02510261 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 27, 2015)
  • 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 ]
  • 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 ]
  • Assessment of changes from baseline in neurologic impairment assessed using NIS+7 associated with long-term dosing of patisiran [ Time Frame: baseline, week 52 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • Assessment of changes from baseline in serum TTR lowering associated with long-term dosing of patisiran [ Time Frame: baseline, week 52 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • Assessment of changes from baseline in motor function assessed by grip strength test with long-term dosing of patisiran [ Time Frame: baseline, week 52 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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)
Official Title  ICMJE 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
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Amyloidosis
Intervention  ICMJE Drug: Patisiran (ALN-TTR02)
administered by intravenous (IV) infusion
Study Arms  ICMJE Experimental: Patisiran (ALN-TTR02)
Intervention: Drug: Patisiran (ALN-TTR02)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: October 5, 2018)
211
Original Estimated Enrollment  ICMJE
 (submitted: July 27, 2015)
228
Estimated Study Completion Date  ICMJE August 2022
Estimated Primary Completion Date August 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 85 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Australia,   Brazil,   Bulgaria,   Canada,   Cyprus,   France,   Germany,   Italy,   Japan,   Korea, Republic of,   Malaysia,   Mexico,   Netherlands,   Portugal,   Spain,   Sweden,   Taiwan,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02510261
Other Study ID Numbers  ICMJE ALN-TTR02-006
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Alnylam Pharmaceuticals
Study Sponsor  ICMJE Alnylam Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Jing Jing Wang, MD Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date September 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP