NCT03400098

Brief Summary

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

Trial Health

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 8, 2018

Completed
9 days until next milestone

First Posted

Study publicly available on registry

January 17, 2018

Completed
Last Updated

August 5, 2019

Status Verified

August 1, 2019

First QC Date

January 8, 2018

Last Update Submit

August 1, 2019

Conditions

Amyloidosis, Hereditary

Interventions

InotersenDRUG

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

Eligibility Criteria

Age18 Years+
Sexall
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Amyloidosis, Familial

Interventions

Inotersen

Condition Hierarchy (Ancestors)

Metabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesAmyloidosisProteostasis Deficiencies

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 8, 2018

First Posted

January 17, 2018

Last Updated

August 5, 2019

Record last verified: 2019-08