Expanded Access Protocol of Patisiran for Patients With H... | NCT02939820

Studies
Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

NCT02939820

Sponsor

Alnylam Pharmaceuticals

Status

Approved for marketing

Last Update Posted

May 20, 2024Actual

Enrollment

Not provided

Phase

Not provided

Conditions

TTR-mediated Amyloidosis
Amyloidosis, Hereditary
Amyloid Neuropathies, Familial
Familial Amyloid Polyneuropathies
Amyloid Neuropathies
Amyloidosis, Hereditary, Transthyretin-Related

Interventions

patisiran (ALN-TTR02)

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Status Module

Record Verification Date

May 2024

Overall Recruitment Status or Expanded Access Status

Approved for marketing

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Oct 18, 2016

First Submission Date that Met QC Criteria

Oct 18, 2016

First Posted Date

Oct 20, 2016Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 16, 2024

Last Update Posted Date

May 20, 2024Actual

Description Module

Brief Summary

The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).

Detailed Description

Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the links provided in More Information.

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

No data available

No data is available for this block.

Biospecimen

No data available

No data is available for this block.

Enrollment

Not provided

Name	Type	Description	Arm Group Labels	Other Names
patisiran (ALN-TTR02)	Drug	patisiran (ALN-TTR02) administered by intravenous (IV) infusion

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Male or female greater than or equal to 18 years of age
Have a diagnosis of hATTR
Meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements
Have adequate complete blood counts, liver function tests and coagulation tests

Exclusion Criteria:

Participated in an interventional hATTR amyloidosis clinical trial involving RNA interference (RNAi) therapeutics within the last 12 months
Are currently eligible to participate in or currently enrolled in an ongoing interventional hATTR amyloidosis clinical trial
Have inadequate cardiac function
Known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis
Have known serious comorbidities or considered unfit for the program by the investigator
Prior or planned liver or heart transplantation

Accepts Healthy Volunteers

Not provided

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Medical Director	Alnylam Pharmaceuticals	Study Director

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
31215818	Derived	Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.

ID	Term
D028226	Amyloidosis, Familial
D028227	Amyloid Neuropathies, Familial
D017772	Amyloid Neuropathies
C567782	Amyloidosis, Hereditary, Transthyretin-Related
D000686	Amyloidosis

ID	Term
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases
D057165	Proteostasis Deficiencies
D020271	Heredodegenerative Disorders, Nervous System
D019636	Neurodegenerative Diseases
D009422	Nervous System Diseases
D010523	Peripheral Nervous System Diseases
D009468	Neuromuscular Diseases