A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects With Deletion- or Nondeletion-type Angelman Syndrome

ReclutandoFase 2ClinicalTrials.gov

ID: NCT07157254Tipo: INTERVENTIONALInicio: 13 de oct de 2025Fin estimado: 1 de ene de 2030

Pediatría

Traducción no disponible, mostrando original

Resumen

The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.

Descripción detallada

This basket study is designed to evaluate safety and efficacy of GTX-102 in participants with Angelman syndrome across genotypes and age groups. The study consists of subprotocols A, B, C and D. All subprotocols are open-label and follow the same design which includes a Screening, Loading and Maintenance period. Subprotocols A, B and C are single arm only. In subprotocol D, participants are randomized 2:1 to a GTX-102 group or a No Treatment group. The No Treatment group follows the same schedule of events as all other groups after completion of the No Treatment period. Participants from all the subprotocols have the option to continue treatment in a long-term extension study following their end of study visit.

Elegibilidad

Edad mínima: 1 YearEdad máxima: 64 YearsSexo: ALL

Criterios de inclusión

Signed informed consent from parent(s) or legal guardian(s)
Males and females of the following ages and genotypes at time of informed consent:
Subprotocol A: ≥ 1 to \< 4 years of age with a genetically confirmed diagnosis of deletion-type Angelman syndrome
Subprotocol B: ≥ 4 to \< 18 years of age with a genetically confirmed diagnosis of UPD/ICD Angelman syndrome
Subprotocol C: ≥ 18 to \< 65 years of age with a genetically confirmed diagnosis of Angelman syndrome, any genotype
Subprotocol D: ≥ 4 to \< 18 years of age with a genetically confirmed diagnosis of mutation-type Angelman syndrome
Weight ≥ 8 kg at Screening Visit
Platelet count, prothrombin time / international normalized ratio, and partial thromboplastin time \< 1.5x the upper limit of normal and platelets \> 75,000 cells/mm3 at the Screening Visit
Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, and all study procedures, including lumbar puncture (LP) procedure, magnetic resonance imaging (MRI) and tolerating anesthesia without intubation
From the time of informed consent through to at least 6 months after the final dose of GTX-102, females of childbearing potential who are sexually active must use highly effective contraception or abstinence. Males are able to participate if they agree to remain abstinent (refrain from heterosexual intercourse) or use acceptable contraceptive methods during the study and for at least 3 months after the final dose of GTX-102

Criterios de exclusión

Concurrent participation in any interventional study
Any change in medications or diet/supplements intended to treat symptoms of Angelman Syndrome (eg, sleeping aids, antiseizure medications, supplements, dietary change including ketogenic or low-glycemic index diet, other) within the month prior to the Screening Visit (excluding weight-based adjustments)
Any condition that creates an increased risk of unsuccessful lumbar puncture
Current or expected concomitant use of drugs that increase the risk of bleeding (eg, heparin, low molecular weight heparin, platelet inhibitors)
Known hypersensitivity to GTX-102 or its excipients or required premedication that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
Presence or history of any condition, lab abnormality, or infection that, in the judgment of the Investigator, would interfere with study participation, pose undue safety risk, or would confound interpretation of results
Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
Use of any investigational product or investigational medical device within 6 months or 5 half-lives prior to the Screening Visit, or any prior use of gene therapy or an ASO regardless of length of time since last use

Intervenciones

other

No intervention

During the no treatment period participants do not receive any study drug

drug

GTX-102

antisense oligonucleotide

Ubicaciones

Clinical Trial Site

Pilar, Buenos Aires, Argentina

NOT_YET_RECRUITING

Patrocinadores

PrincipalUltragenyx Pharmaceutical Inc

Contactos

Patients Contact Trial Recruitment

CONTACT

Trialrecruitment@ultragenyx.com 1-888-756-8657

HCPs Contact: Medical Information

CONTACT

medinfo@ultragenyx.com 1-888-756-8657

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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