Nicht-interventionelle Studie (Anwendungsbeobachtung) NIS-Nr.: 645
Studiencode: COAV101A1DE01
Allgemeine Angaben
Institution: |
Novartis Pharma GmbH |
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Titel der NIS: |
Routine data collection and evaluations of onasemnogene abeparvovec in Germany |
Ziel der NIS: |
The objective of this study is to evaluate the overall effectiveness and safety in patients with spinal muscular atrophy (SMA) treated with gene therapy Zolgensma® (onasemnogene abeparvovec) compared to Spinraza® (nusinersen). Non-interventional, non-randomized data collection using secondary data from the SMArtCARE registry. Based on SMA incidence information derived from the results of pilot new-born screening in Germany, the study is anticipated to enroll up to 599 pa-tients, which will be included both retrospectively and prospectively from the initiation of the SMArtCARE registry in July 2018 to the time of data cut for final analysis on December 31, 2026.
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Ort der Durchführung: |
multinational |
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Patientenanzahl insgesamt: |
599 |
Anzahl Ärzte insgesamt: |
25 |
Patientenanzahl in Deutschland: |
450 |
Ärzte in Deutschland: |
20 |
Weitere Angaben und Unterlagen
Anzeige am: |
28.01.2022 |
Geplanter Beginn: |
01.02.2022 |
Geplantes Ende: |
31.12.2026 |
Angaben zu den beobachteten Arzneimitteln, soweit angezeigt
Arzneimittelbezeichnung |
INN |
Zulassungs-Nr. |
ATC-Code |
Substanzklasse |
Zolgensma |
onasemnogene abeparvovec |
EU/1/20/1443/001 - 037 |
M09AX09 |
Gentherapeutika |
Spinraza |
nusinersen |
EU/1/17/1188/001 |
M09AX07 |
Antisense-Oligonukleotide |
Verfügbare Dokumente
Art des Dokuments |
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Beobachtungsplan, NIS 645 |
pdf 16MB |