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A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

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: NCT04201418
Recruitment Status : Completed
First Posted : December 17, 2019
Last Update Posted : June 6, 2022
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Study Description
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Brief Summary:
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.

Condition or disease Intervention/treatment
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis Polyneuropathy Drug: Patisiran

Study Design
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Study Type : Observational
Actual Enrollment : 67 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation
Actual Study Start Date : December 18, 2019
Actual Primary Completion Date : May 24, 2022
Actual Study Completion Date : May 24, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis
Drug Information available for: Patisiran

Groups and Cohorts
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Group/Cohort Intervention/treatment
Patisiran Prospective Cohort
Patients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy.
 Drug: Patisiran
Patisiran-lipid complex injection, for intravenous use
Other Names:
  • ONPATTRO
  • ALN-TTR02
Patisiran Mixed Cohort
Patients who are currently on commercial patisiran therapy for less than 12 months at study enrollment.
 Drug: Patisiran
Patisiran-lipid complex injection, for intravenous use
Other Names:
  • ONPATTRO
  • ALN-TTR02
Patisiran Retrospective Cohort
Patients who have been on commercial patisiran therapy for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.
 Drug: Patisiran
Patisiran-lipid complex injection, for intravenous use
Other Names:
  • ONPATTRO
  • ALN-TTR02


Outcome Measures
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Primary Outcome Measures :
  1. Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline [ Time Frame: Baseline, Month 12 ]
    PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden.


Eligibility Criteria
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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.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation
Criteria

Inclusion Criteria:

  • Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation
  • PND score of I-IIIB at baseline.

  • Exposure to commercial patisiran in one of the 3 cohorts:

    • Prospective Cohort: Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran.
    • Mixed cohort: Currently on commercial patisiran therapy for less than 12 months at study enrollment.
    • Retrospective cohort: Exposed to commercial patisiran treatment for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.

Exclusion Criteria:

  • New York Heart Association (NYHA) heart failure classification ≥3
  • Karnofsky Performance Status (KPS) <60%
  • Unstable congestive heart failure (CHF)
  • Known primary amyloidosis (AL) or leptomeningeal amyloidosis
  • Prior major organ transplant
  • Previously received patisiran
  • Previous treatment with a TTR silencing therapy
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): NCT04201418


Locations
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United States, California
Clinical Trial Site
Los Angeles, California, United States, 90095
Clinical Trial Site
San Diego, California, United States, 92103
United States, Colorado
Clinical Trial Site
Aurora, Colorado, United States, 80045
United States, Connecticut
Clinical Trial Site
Farmington, Connecticut, United States, 06030
United States, Florida
Clinical Trial Site
Jacksonville, Florida, United States, 32224
Clinical Trial Site
Tampa, Florida, United States, 33609
United States, Georgia
Clinical Trial Site
Braselton, Georgia, United States, 30517
Clinical Trial Site
Macon, Georgia, United States, 31201
United States, Illinois
Clinical Trial Site
Chicago, Illinois, United States, 60611
Clinical Trial Site
Chicago, Illinois, United States, 60612
United States, Massachusetts
Clinical Trial Site
Boston, Massachusetts, United States, 02111
United States, Michigan
Clinical Trial Site
Detroit, Michigan, United States, 48202
United States, Mississippi
Clinical Trial Site
Jackson, Mississippi, United States, 39216
United States, Missouri
Clinical Trial Site
Kansas City, Missouri, United States, 64111
United States, Nebraska
Clinical Trial Site
Omaha, Nebraska, United States, 68198
United States, North Carolina
Clinical Trial Site
Chapel Hill, North Carolina, United States, 27599
Clinical Trial Site
Charlotte, North Carolina, United States, 28207
United States, Ohio
Clinical Trial Site
Columbus, Ohio, United States, 43210
United States, Pennsylvania
Clinical Trial Site
Allentown, Pennsylvania, United States, 18103
Clinical Trial Site
Bethlehem, Pennsylvania, United States, 18015
Clinical Trial Site
Lancaster, Pennsylvania, United States, 17602
Clinical Trial Site
Pittsburgh, Pennsylvania, United States, 15212
Clinical Trial Site
Pittsburgh, Pennsylvania, United States, 15232
United States, Tennessee
Clinical Trial Site
Germantown, Tennessee, United States, 38138
United States, Texas
Clinical Trial Site
Austin, Texas, United States, 78756
Clinical Trial Site
San Antonio, Texas, United States, 78229
United States, Wisconsin
Clinical Trial Site
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals
More Information
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04201418    
Other Study ID Numbers: ALN-TTR02-012
First Posted: December 17, 2019    Key Record Dates
Last Update Posted: June 6, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Alnylam Pharmaceuticals:
Valine to isoleucine substitution at position 122
V122I
Threonine to alanine substitution at position 60
T60A
Familial Amyloid Polyneuropathies
ATTR
Transthyretin amyloidosis
Transthyretin
Amyloidosis
TTR-mediated Amyloidosis
RNAi therapeutic
FAP
Polyneuropathies
Amyloid neuropathies
 Amyloid neuropathies, familial
Amyloidosis, familial
Peripheral nervous system diseases
Nervous system diseases
TTR
Neuromuscular diseases
Proteostasis deficiencies
Metabolic diseases
Heredodegenerative disorders, nervous system
Neurodegenerative diseases
Genetic diseases, inborn
Metabolism, inborn errors
ATTRv
Additional relevant MeSH terms:
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Polyneuropathies
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
 Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases