Estudio de fase II, aleatorizado, doble ciego, controlado con placebo, de búsqueda de dosis, para evaluar la seguridad, biomarcadores y eficacia de tominersen en individuos con enfermedad de Huntington prodrómica y manifiesta temprana

Activo, no reclutaFase 2ClinicalTrials.gov

ID: NCT05686551Tipo: INTERVENTIONALInicio: 3 de feb de 2023Fin estimado: 1 de abr de 2027

Resumen

Este estudio evaluará la seguridad, biomarcadores y eficacia de tominersen comparado con placebo en participantes con enfermedad de Huntington (EH) prodrómica y manifiesta temprana.

Elegibilidad

Edad mínima: 25 YearsEdad máxima: 50 YearsSexo: ALL

Criterios de inclusión

DB Period:
HD gene expansion mutation carrier status with a cytosine-adenine-guanine-age product (CAP) score of 400-500 inclusive
Either:
Prodromal HD (defined as Diagnostic Confidence Level (DCL) 2 to 3, Independence Scale (IS) ≥70, and TFC ≥8); Or
Early manifest HD (defined as DCL 4, IS ≥70, and TFC ≥8)
Total body weight \> 40 kilograms (kg) and a body mass index (BMI) within the range of 18-32 kilograms per meter square (kg/m\^2)
Study companion
OLE Period:
Participants must have completed the DB treatment period
Participants must remain in the DB Safety follow-up period until OLE period starts

Criterios de exclusión

DB Period:
Current or previous use of an antisense oligonucleotide (ASO) (including small interfering ribonucleic acid \[RNA\]) or any HTT lowering therapy (including tominersen)
Anti-platelet or anticoagulant therapy within 14 days prior to screening or anticipated use during the study, including, but not limited to, aspirin (unless ≤ 81 milligrams per day \[mg/day\]), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, apixaban, and heparin
History of gene therapy, cell transplantation, or brain surgery
Hydrocephalus
Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug
History of attempted suicide or suicidal ideation with plan (i.e., active suicidal ideation) that required hospital visit and/or change in level of care within 12 months prior to screening
OLE Period:
Early discontinuation from the DB treatment and the safety follow-up (SFU) periods
Pregnant or breastfeeding, or with the intention of becoming pregnant during the study or within the timeframe in which contraception is required
Current or previous use of an ASO other than tominersen (including small interfering RNA) or any other HTT-lowering therapy
Hydrocephalus
Received any active investigational treatment other than tominersen during or since completion of the DB treatment period

Intervenciones

drug

Tominersen

Tominersen will be administered at the dose and schedule specified in the protocol.

drug

Placebo

Matching placebo administered IT, Q16W during the DB period.

Ubicaciones

CINME

Buenos Aires, Argentina

Hospital Ramos Mejía

CABA, Argentina

INEBA

Capital Federal, Argentina

Hospital Britanico de Buenos Aires

Ciudad Autonoma Buenos Aires, Argentina

Patrocinadores

PrincipalHoffmann-La Roche

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