ClinicalTrials.gg
Approved for marketingNCT03400098Updated Aug 5, 2019

ATTR Expanded Access Program (EAP) by Ionis

An expanded access record providing Inotersen in Amyloidosis, Hereditary, sponsored by Ionis Pharmaceuticals, Inc.. Approved for marketing. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-08-05.

Sponsored by Ionis Pharmaceuticals, Inc. · Expanded access

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Study type
Expanded access
Access type
Treatment IND/protocol
Ages
18 Years and older
Sex
All
01

Study summary

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

Read the 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.

02

Conditions studied

  • Amyloidosis, Hereditary

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03

In context

Amyloidosis, Familial

28 studies on the registry are indexed under Amyloidosis, Familial; 10 are open to participants now.

Browse Amyloidosis, Familial studies →

Lead sponsor

Ionis Pharmaceuticals, Inc. is the lead sponsor of 118 studies on the registry; 10 are open to participants now.

Of its 49 completed or terminated interventional studies of FDA-regulated products, 22 (45%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

Exclusion Criteria:

  • Known Primary Amyloidosis, Amyloidosis or Monoclonal Gammopathy of Undetermined Significance or Multiple Myeloma
  • Have inadequate cardiac function
  • Have low counts
  • Have inadequate renal function
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Leptomeningeal disease

Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.

Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

05

Access details

Study type
Expanded access
Access type
Treatment IND/protocol

Available treatment

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

06

Where to request access

No study locations are listed for this record.

07

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 5, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
08

Registry details

Key details

Study ID
NCT03400098
Responsible party
Sponsor
First posted
Jan 17, 2018
Last update
Aug 5, 2019
View the source record on ClinicalTrials.gov ↗

Requesting access

Expanded access is arranged between your doctor and the company. Ask your care team to contact the provider listed on this record.

No contact was published for this record. The registry link below has the sponsor’s details.

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Discussion

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