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

  • STATUS
    Recruiting
  • End date
    Jan 10, 2024
  • participants needed
    140
  • sponsor
    Ionis Pharmaceuticals, Inc.
Updated on 10 July 2021
Investigator
Ionis Pharmaceuticals
Primary Contact
Auckland City Hospital (0.8 mi away) Contact
+58 other location

Summary

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 http://www.neuro-ttransform.com/.

Description

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.

Details
Condition Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy, Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Treatment Inotersen, AKCEA-TTR-LRx
Clinical Study IdentifierNCT04136184
SponsorIonis Pharmaceuticals, Inc.
Last Modified on10 July 2021

Eligibility

Yes No Not Sure

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)
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