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ATTR Expanded Access Program (EAP) by Ionis

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ClinicalTrials.gov Identifier: NCT03400098
Expanded Access Status : Approved for marketing
First Posted : January 17, 2018
Last Update Posted : August 5, 2019
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

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

Condition or disease Intervention/treatment
Amyloidosis, Hereditary Drug: Inotersen

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.
Study Design
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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Expanded Access Program for Inotersen (ISIS 420915) in Patients With Hereditary Transthyretin Amyloidosis (hATTR)

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Amyloidosis
Drug Information available for: Inotersen

Interventions
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Intervention Details:
  • Drug: Inotersen
    Inotersen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm
Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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
Contacts and Locations
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No Contacts or Locations Provided
More Information
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03400098    
Other Study ID Numbers: ISIS 420915-CS5
First Posted: January 17, 2018    Key Record Dates
Last Update Posted: August 5, 2019
Last Verified: August 2019
Additional relevant MeSH terms:
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Amyloidosis, Familial
Amyloidosis
Proteostasis Deficiencies
 Metabolic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn