- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03400098
ATTR Expanded Access Program (EAP) by Ionis
August 1, 2019 updated by: Ionis Pharmaceuticals, Inc.
Expanded Access Program for Inotersen (ISIS 420915) in Patients With Hereditary Transthyretin Amyloidosis (hATTR)
The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).
Study Overview
Status
Approved for marketing
Conditions
Intervention / Treatment
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
Expanded Access Type
- Treatment IND/Protocol
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
N/A
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Registration Dates
First Submitted
January 8, 2018
First Submitted That Met QC Criteria
January 8, 2018
First Posted (Actual)
January 17, 2018
Study Record Updates
Last Update Posted (Actual)
August 5, 2019
Last Update Submitted That Met QC Criteria
August 1, 2019
Last Verified
August 1, 2019
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ISIS 420915-CS5
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Amyloidosis, Hereditary
-
University of PennsylvaniaFox Chase Cancer CenterCompletedCardiovascular Diseases | Cancer | Hereditary Cancer | Hereditary Cardiac AmyloidosisUnited States
-
University of PennsylvaniaAstraZeneca; Ionis Pharmaceuticals, Inc.Active, not recruitingHereditary Amyloidosis, Transthyretin-RelatedUnited States
-
University of Sao Paulo General HospitalRecruitingMicrobial Colonization | Amyloidosis, Hereditary, Transthyretin-RelatedBrazil
-
Alnylam PharmaceuticalsNot yet recruitingHereditary Amyloidosis, Transthyretin-Related | Asymptomatic Carrier StateFrance
-
Akcea TherapeuticsRecruitingHereditary Transthyretin Amyloidosis With PolyneuropthyUnited States, Canada
-
Virginia Commonwealth UniversityNot yet recruitingAmyloidosis, HereditaryUnited States
-
Akcea TherapeuticsUnited BioSource, LLCRecruitingHereditary Transthyretin Amyloidosis With PolyneuropthyUnited States, France, Greece, Italy, Spain, Canada, Germany, Portugal
-
PfizerCompletedPolyneuropathy | Hereditary Transthyretin Amyloidosis (ATTRv)Spain
-
Alnylam PharmaceuticalsNo longer availableAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies
-
Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid PolyneuropathiesUnited States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
Clinical Trials on Inotersen
-
Akcea TherapeuticsRecruitingPregnancy | Hereditary Transthyretin-mediated Amyloidosis With PolyneropathyUnited States
-
Brigham and Women's HospitalUnknown
-
Ionis Pharmaceuticals, Inc.CompletedAmyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | TransthyretinUnited States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
-
Ionis Pharmaceuticals, Inc.CompletedAmyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | TransthyretinNew Zealand, United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
-
Ionis Pharmaceuticals, Inc.CompletedHereditary Transthyretin-Mediated Amyloid PolyneuropathyUnited States, Spain, Taiwan, Italy, Germany, Argentina, Australia, Brazil, Canada, Cyprus, France, Greece, New Zealand, Portugal, Sweden, Turkey