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Hereditary Transthyretin-Mediated Amyloid Polyneuropathy| AKCEA-TTR-LRx

NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

This is a multicenter, open-label study in up to 140 participants, who will be randomized to receive subcutaneous (SC) injections of either AKCEA-TTR-LRx once every 4 weeks or inotersen once a week. Participants will also receive daily supplemental doses of the recommended daily allowance of vitamin A. Participants included in the inotersen reference arm will be crossed over to AKCEA-TTR-LRx at Week 37 after completing the Week 35 assessments.

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Purpose of Study

To evaluate the efficacy and safety of AKCEA-TTR-LRx after administration for 65 weeks to patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN), as compared to the NEURO-TTR trial (NCT01737398). For more information, please visit


18 - 82
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Key Inclusion / Exclusion Criteria

Inclusion Criteria:

  • Aged 18 to 82 years at the time of informed consent
  • Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent
  • Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the subject or the subject’s non-pregnant female partner must be using a highly effective contraceptive method
  • Diagnosis of hereditary transthyretin-mediated polyneuropathy as defined by meeting all 3 of the following:
  • Stage 1 or Stage 2 Familial Amyloid Polyneuropathy (FAP) or Coutinho Stage
  • Documented genetic mutation in the TTR gene
  • Symptoms and signs consistent with neuropathy associated with transthyretin amyloidosis, including NIS ≥ 10 and ≤ 130

Exclusion Criteria:

  • Clinically-significant (CS) abnormalities in medical history, screening laboratory results, physical or physical examination that would render a subject unsuitable for inclusion, including but not limited to abnormal safety labs
  • Karnofsky performance status ≤ 50
  • Other causes of sensorimotor or autonomic neuropathy (e.g., autoimmune disease), including uncontrolled diabetes
  • Prior liver transplant or anticipated liver transplant within 1-yr of Screening
  • New York Heart Association (NYHA) functional classification of ≥ 3
  • Acute coronary syndrome within 6 months of screening or major surgery within 3 months of Screening
  • Other types of amyloidosis
  • Have any other conditions, which, in the opinion of the Investigator or Sponsor would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the Study
  • Current treatment with any approved drug for hereditary TTR amyloidosis such as Vyndaqel® / Vyndamax™ (tafamidis), Tegsedi™ (inotersen), Onpattro™ (patisiran), off-label use of diflunisal or doxycycline, and tauroursodeoxycholic acid (TUDCA). If previously treated with Vyndaqel® / Vyndamax™, diflunisal or doxycycline, and TUDCA, must have discontinued treatment for at least 2 weeks prior to Study Day 1
  • Previous treatment with Tegsedi™ (Inotersen) or Onpattro™ (patisiran), or other oligonucleotide or RNA therapeutic (including siRNA)


Hereditary Transthyretin-Mediated Amyloid Polyneuropathy




Phase 3

Clinical Trials ID


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Start Date / End Date:

01 2020 - 01 2024





Full Details:

40 Study Locations

United States







New Zealand





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