ATTR Expanded Access Program (EAP) by Ionis | NCT03400098

Studies
ATTR Expanded Access Program (EAP) by Ionis

NCT03400098

Sponsor

Ionis Pharmaceuticals, Inc.

Status

Approved for marketing

Last Update Posted

Aug 5, 2019Actual

Enrollment

Not provided

Phase

Not provided

Conditions

Amyloidosis, Hereditary

Interventions

Inotersen

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

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2019

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

Not provided

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Jan 8, 2018

First Submission Date that Met QC Criteria

Jan 8, 2018

First Posted Date

Jan 17, 2018Actual

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

Aug 1, 2019

Last Update Posted Date

Aug 5, 2019Actual

Description Module

Brief Summary

The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).

Detailed Description

The Program is intended to provided expanded access to Inotersen for eligible patients with hATTR who have limited or no available treatment options.

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Expanded Access Types

Treatment IND/Protocol

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
Inotersen	Drug	Inotersen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Male or female at least 18 years of age with a diagnosis of hATTR
Symptoms consistent with polyneuropathy
Meet Polyneuropathy Disability (PND) Stage I-III requirements

Exclusion Criteria:

Known Primary Amyloidosis, Leptomeningeal Amyloidosis or Monoclonal Gammopathy of Undetermined Significance or Multiple Myeloma
Have inadequate cardiac function
Have low platelet counts
Have inadequate renal function

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

Condition Browse Module

MeSH Terms

ID	Term
D028226	Amyloidosis, Familial

Ancestor Terms

ID	Term
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D008659	Metabolic Diseases

Browse Leaves

Not provided

Browse Branches

Not provided