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ATTR Expanded Access Program (EAP) by Ionis

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ClinicalTrials.gov Identifier: NCT03400098
Recruitment Status : Available
First Posted : January 17, 2018
Last Update Posted : April 25, 2018
Sponsor:
Collaborator:
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.

Study Type : Expanded Access
  Treatment IND/Protocol
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

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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

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 ClinicalTrials.gov identifier (NCT number): NCT03400098


Contacts
Contact: VP, Clinical Development 1-833-288-7327 ATTREAP@ionisph.com

  Show 25 Study Locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Clinigen Group PLC
Investigators
Study Director: Eugene Schneider, MD Vice President, Clinical Development, Ionis

Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03400098     History of Changes
Other Study ID Numbers: ISIS 420915-CS5
First Posted: January 17, 2018    Key Record Dates
Last Update Posted: April 25, 2018
Last Verified: April 2018

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