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

14. září 2026 aktualizováno: 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.

Přehled studie

Postavení

Zatím nenabíráme

Podmínky

Spinální svalová atrofie

Typ studie

Pozorovací

Zápis (Odhadovaný)

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní kontakt

Jméno: Novartis Pharmaceuticals
Telefonní číslo: +41613241111
E-mail: novartis.email@novartis.com

Studijní záloha kontaktů

Jméno: Novartis Pharmaceuticals

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

Dítě
Dospělý
Starší dospělý

Přijímá zdravé dobrovolníky

Metoda odběru vzorků

Vzorek nepravděpodobnosti

Studijní populace

SMA patients administered onasemnogene abeparvovec by intrathecal injection in Japan.

Popis

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).

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

Počet skupin / kohort

Kohorty a intervence

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

Co je měření studie?

Primární výstupní opatření

Měření výsledku	Popis opatření	Časové okno
Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs) Časové okno: Up to approximately 4 years and 5 months		Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs Časové okno: 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 Časové okno: 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 Časové okno: 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 Časové okno: 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 Časové okno: 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

Sekundární výstupní opatření

Měření výsledku	Popis opatření	Časové okno
Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score Časové okno: 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 Časové okno: 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 Časové okno: 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 Časové okno: 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 Časové okno: 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 Časové okno: 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 Časové okno: 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

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

Novartis Pharmaceuticals

Vyšetřovatelé

Ředitel studie: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Odhadovaný)

30. října 2026

Primární dokončení (Odhadovaný)

31. března 2031

Dokončení studie (Odhadovaný)

31. března 2031

Termíny zápisu do studia

První předloženo

11. srpna 2026

První předloženo, které splnilo kritéria kontroly kvality