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ATTR Expanded Access Program (EAP) by Ionis

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. Identifier: NCT03400098
Expanded Access Status : Approved for marketing
First Posted : January 17, 2018
Last Update Posted : November 12, 2018
Clinigen Group PLC
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

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.

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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)

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

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

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

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 identifier (NCT number): NCT03400098

  Show 19 Study Locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Clinigen Group PLC
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Study Director: Eugene Schneider, MD Vice President, Clinical Development, Ionis

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Responsible Party: Ionis Pharmaceuticals, Inc. Identifier: NCT03400098     History of Changes
Other Study ID Numbers: ISIS 420915-CS5
First Posted: January 17, 2018    Key Record Dates
Last Update Posted: November 12, 2018
Last Verified: November 2018

Additional relevant MeSH terms:
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Amyloidosis, Familial
Proteostasis Deficiencies
Metabolic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn