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A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients

14 de setembro de 2026 atualizado por: Novartis Pharmaceuticals

Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy)

This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.

This is a non-interventional study and does not impose a therapy protocol, diagnostic/therapeutic procedure, or a visit schedule. Patients will be treated according to the Japan package insert. Data will be collected using a case registration form (CRF) completed by the investigator at each study site.

Visão geral do estudo

Status

Ainda não está recrutando

Condições

Atrofia muscular espinhal

Tipo de estudo

Observacional

Inscrição (Estimado)

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Contato de estudo

Nome: Novartis Pharmaceuticals
Número de telefone: +41613241111
E-mail: novartis.email@novartis.com

Estude backup de contato

Nome: Novartis Pharmaceuticals

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

Filho
Adulto
Adulto mais velho

Aceita Voluntários Saudáveis

Não

Método de amostragem

Amostra Não Probabilística

População do estudo

SMA patients administered onasemnogene abeparvovec by intrathecal injection in Japan.

Descrição

Inclusion criteria:

• All patients treated with onasemnogene abeparvovec.

Exclusion criteria:

• Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

Número de grupos/coortes

Coortes e Intervenções

Grupo / Coorte
Onasemnogene Abeparvovec Intrathecal Group SMA patients treated with onasemnogene abeparvovec intrathecal injection in Japan.

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado	Descrição da medida	Prazo
Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs) Prazo: Up to approximately 4 years and 5 months		Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs Prazo: Up to approximately 4 years and 5 months	Incidence based on the person-year method will be calculated.	Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications Prazo: Up to approximately 4 years and 5 months	Incidence based on the person-year method will be calculated.	Up to approximately 4 years and 5 months
Number of Patients by Time to Onset of Serious Adverse Events and ADRs Prazo: Up to approximately 4 years and 5 months		Up to approximately 4 years and 5 months
Number of Patients by Time to Onset of Serious Adverse Events and ADRs Corresponding to the Safety Specifications Prazo: Up to approximately 4 years and 5 months		Up to approximately 4 years and 5 months
Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications Prazo: Up to approximately 4 years and 5 months	Safety specifications include hepatotoxicity, transient thrombocytopenia, thrombotic microangiopathy, cardiac adverse events, dorsal root ganglia toxicity/peripheral sensory neuropathy, tumorigenicity due to chromosomal integration, and long-term monitoring of gene therapy.	Up to approximately 4 years and 5 months

Medidas de resultados secundários

Medida de resultado	Descrição da medida	Prazo
Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score Prazo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.	Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Change From Baseline in Revised Upper Limb Module (RULM) Score Prazo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	RULM is a tool used to assess upper limb function in SMA patients. It consists of 20 items in total. The first entry item classifies overall functional ability and is not scored. The remaining 19 tasks are scored with 1 task scored as can/cannot (1 is the highest score) and 18 tasks rated on a 3-point scale as follows: 0 - unable to perform the task 1 - able to perform the task with some modification 2 - able to perform the task with no difficulty The total RULM score ranges from 0 to 37 with higher scores indicating better upper limb function.	Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score Prazo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	CHOP-INTEND is a tool used to measure the motor abilities in infants with SMA. It consists of 16 items scored on a scale from 0 (no response/unable to perform the movement) to 4 (complete response/able to perform the task). CHOP-Intend total score ranges from 0 to 64 with higher scores indicating higher motor function.	Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Change From Baseline in Clinical Global Impression-Severity (CGI-S) Score Prazo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	The CGI-S scale is a physician-rated measure used to assess the severity of a patient's illness. Severity of illness is assessed with consideration for the overall clinical experience of patients of the same age. The CGI-S scores range from: 0 = Not evaluated 1 = Normal, not at all ill 2 = Borderline normal and ill 3 = Mildly ill 4 = Moderately ill 5 = Markedly ill 6 = Severely ill 7 = Among the most extremely ill	Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Number of Patients Categorized by Clinical Global Impression-Improvement (CGI-I) Score Prazo: 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	The CGI-I scale is a physician-rated measure used to assess a patient's overall clinical status. The patient's overall clinical status is scored on a 7-point scale of the extent of change relative to before treatment initiation as follows: 1 = Very much improved 2 = Much improved 3 = Minimally improved 4 = No change 5 = Minimally worse 6 = Much worse 7 = Very much worse Number of patients and their 95% confidence intervals (CIs) will be calculated for two categories: improved (CGI-I score: 1 to 3) and unchanged or worsened (CGI-I score: 4 to 7).	6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Number of Patients Achieving Each Developmental Milestone Prazo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	Developmental milestones include: Sitting without support Hands-and-knees crawling Standing with assistance Walking with assistance Standing alone Walking alone	Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Number of Patients Maintaining Each Developmental Milestone Prazo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months		Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Novartis Pharmaceuticals

Investigadores

Diretor de estudo: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Estimado)

30 de outubro de 2026

Conclusão Primária (Estimado)

31 de março de 2031

Conclusão do estudo (Estimado)

31 de março de 2031

Datas de inscrição no estudo

Enviado pela primeira vez

11 de agosto de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ