A Phase 3 Global, Open-Label, Randomized Study to Evaluat... | EligiMed

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

CompletadoFase 3ClinicalTrials.gov

ID: NCT04136184Tipo: INTERVENTIONALInicio: 11 de dic de 2019Fin estimado: 12 de jul de 2023

Traducción no disponible, mostrando original

Resumen

The main objective of this study was to evaluate the efficacy of eplontersen as compared with the historical control of the placebo cohort in the NEURO-TTR trial (NCT01737398/2012-001831-30), in subjects with hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN). For more information, please visit http://www.neuro-ttransform.com/.

Descripción detallada

This study was a multicenter, open-label study in up to 168 participants, who were randomized to receive subcutaneous (SC) injections of either eplontersen once every 4 weeks or inotersen once a week. Participants also received daily supplemental doses of the recommended daily allowance of vitamin A. Participants included in the inotersen reference arm crossed over to eplontersen at Week 37 after completing the Week 35 assessments.

Elegibilidad

Edad mínima: 18 YearsEdad máxima: 82 YearsSexo: ALL

Criterios de inclusión

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 participant or the participantss 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 Neuropathy Impairment Score (NIS) ≥ 10 and ≤ 130

Criterios de exclusión

Clinically-significant (CS) abnormalities in medical history, screening laboratory results, physical or physical examination that would render a participants 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 participant unsuitable for inclusion, or could interfere with the participant 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)

Intervenciones

drug

Inotersen

Inotersen by subcutaneous injection

drug

Eplontersen

Eplontersen by subcutaneous injection

Ubicaciones

Hospital Italiano de Buenos Aires

Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina

Hospital El Cruce

San Juan Bautista, Buenos Aires, Argentina

STAT Research

Buenos Aires, Argentina

Instituto Fleni

Buenos Aires, Argentina

Patrocinadores

PrincipalIonis Pharmaceuticals, Inc.

Aviso: La información de este ensayo proviene de fuentes públicas y tiene carácter exclusivamente informativo. No constituye asesoramiento médico, regulatorio ni de ningún otro tipo. Las traducciones son automatizadas y no poseen validez oficial. Consultá siempre con un profesional de la salud. Ver Términos de Uso.