A Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Study of a Study Medicine for Hereditary Transthyretin Amyloid Polyneuropathy

Age
18 years - 82 years
Gender
All
participants needed
140
Location
1
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A Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Brief description of study

A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. For those with : a. Stage 1 or Stage 2 according to the Familial Amyloid Polyneuropathy (FAP) or Coutinho Stage b. Documented genetic mutation in the TTR gene c. Symptoms and signs consistent with neuropathy associated with transthyretin amyloidosis

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Transthyretin-Mediated Amyloid Polyneuropathy,neuropathy,
  • Age: 18 years - 82 years
  • Gender: All

Male or Female Age 18-82 a. Stage 1 or Stage 2 according to the Familial Amyloid Polyneuropathy (FAP) or Coutinho Stage b. Documented genetic mutation in the TTR gene c. Symptoms and signs consistent with neuropathy associated with, transthyretin.

The purpose of this research study is to learn about the effects and safety of a study medicine in adults with hereditary transthyretin-mediated amyloid polyneuropathy. This is a nerve problem caused by a change (mutation) in the transthyretin (TTR) gene. The study is for people with Stage 1 or Stage 2 disease.

Participants will receive the study medicine. The study team will compare results between participants receiving different study treatment assignments.

Who can participate:
Adults with hereditary transthyretin-mediated amyloid polyneuropathy may be able to participate if they:

  • Are 18–82 years old
  • Have Stage 1 or Stage 2 disease based on the Familial Amyloid Polyneuropathy (FAP) or Coutinho stage
  • Have a confirmed mutation in the TTR gene
  • Have symptoms and signs of nerve damage (neuropathy) linked to transthyretin amyloidosis

Updated on 04 Aug 2024. Study ID: 834496

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