Estudio de Fase I, en 2 Partes, para Evaluar la Seguridad, Tolerabilidad, Farmacocinética y Farmacodinámica de Dosis Únicas Ascendentes de RG6496 Administrado por Vía Intratecal en un Estudio Aleatorizado, Controlado con Placebo, con Cegamiento del Investigador/Participante, con una Extensión Abierta, en Portadores de Expansión del Gen de la Enfermedad de Huntington

ReclutandoFase 1ClinicalTrials.gov

ID: NCT07246941Tipo: INTERVENTIONALInicio: 19 de nov de 2025Fin estimado: 5 de mar de 2029

Resumen

Este es un estudio de primera administración en humanos (FIH, por sus siglas en inglés) de RG6496 que evaluará la seguridad y tolerabilidad de dosis únicas ascendentes de RG6496 administradas a portadores de la expansión del gen de la enfermedad de Huntington (HDGECs, por sus siglas en inglés). El estudio consta de dos partes: Parte 1 \[dosis única ascendente\] seguida de la Parte 2 \[extensión de etiqueta abierta (OLE, por sus siglas en inglés)\].

Elegibilidad

Edad mínima: 25 YearsEdad máxima: 65 YearsSexo: ALL

Criterios de inclusión

Part 1
Confirmation of HDGEC status with cytosine-adenine-guanine (CAG) expansion \> 39.
Confirmation of SNP carrier status of the target SNP
Independence Scale (IS) score of ≥70, total functional capacity (TFC) ≥10, total motor score (TMS) \>6.
Ability to read the words "red," "blue," and "green" and be fluent in the language of the informed consent form (ICF) and the tests used at the study site.
Ability to walk unassisted.
Total body weight \> 40 kilogram (kg) and body mass index (BMI) within the range 18-32 kilogram per square meter (kg/m\^2) (inclusive) at baseline.
Ability to undergo and tolerate MRI scans.
Part 2
Completed the post-dose safety follow-up period in the Part 1 of the study.
In the opinion of the Investigator, the participant has not experienced a worsening in health that precludes their safe continued participation in the study.

Criterios de exclusión

Part 1
Concurrent or planned participation in any interventional clinical study, including current use of an ASO or any HTT-lowering therapy or treatment with investigational therapy within 90 days or 5 drug-elimination half-lives, whichever is longer prior to screening
Pregnant or breastfeeding, or with the intention of becoming pregnant during the study or within the timeframe in which contraception is required
Malignancy within 5 years prior to screening
Planned brain surgery during the study
Positive HIV test, hepatitis B surface antigen and hepatitis B surface antigen at screening
Active psychosis, confusional state, or violent behavior, including aggression that could cause harm to self or others, over the 12 weeks prior to screening.
Current or previous history of a primary independent psychotic disorder.
Scoliosis or spinal deformity or surgery making IT injection not feasible in an outpatient setting
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.
Part 2
Prematurely discontinued from Part 1 for any reason (i.e., before the completion of the postdose safety follow-up period of Part 1).
Pregnant or breastfeeding, or with the intention of becoming pregnant during the study or within the timeframe in which contraception is required.
Concurrent or planned participation in any interventional clinical study, including current use of an ASO or any HTT-lowering therapy
Received any active investigational treatment other than RG6496 during or since completion of Part 1 of the study.
Had confirmed Dose-Limiting Adverse Event (DLAE)(s) in Part 1 of the study.

Intervenciones

drug

RG6496

RG6496 will be administered as per the schedule specified in the respective arms.

drug

Placebo

RG6496 matching placebo will be administered as per the schedule specified in placebo arm.

Ubicaciones

Hospital Britanico de Buenos Aires

Ciudad Autonoma Bs As, Argentina

RECRUITING

Patrocinadores

PrincipalHoffmann-La Roche

Contactos

Reference Study ID Number: BP45378 https://forpatients.roche.com/

CONTACT

global-roche-genentech-trials@gene.com 888-662-6728 (U.S. Only)

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.

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