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 - NEURO – TTRANSFORM

Ionis Pharmaceuticals, Inc.0 个研究点目标入组 140 人开始时间: 2019年12月3日最近更新: 2020年11月30日

研究概览

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•1. Aged 18 to 82 years at the time of informed consent
•2. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal
•3. 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
•4. Diagnosis of hereditary transthyretin-mediated polyneuropathy as defined by meeting all 3 of the following:
•Stage 1 or Stage 2 FAP
•Documented genetic mutation in the TTR gene
•Symptoms and signs consistent with polyneuropathy as measured by NIS score = 10 and = 130
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 86
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 54

•1. 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
•2. Karnofsky performance status = 50
•3. Other causes of sensorimotor or autonomic neuropathy (e.g., autoimmune disease), including uncontrolled diabetes
•4. Prior liver transplant or anticipated liver transplant within 1-yr of Screening
•5. New York Heart Association (NYHA) functional classification of = 3
•6. Acute coronary syndrome or major surgery within 3 months of Screening
•7. Other types of amyloidosis
•8. 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
•9. 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 2 weeks prior to Study Day 1
•10. Previous treatment with TegsediTM (Inotersen) or OnpattroTM (patisiran), or other oligonucleotide or RNA therapeutic (including siRNA)

发起方