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Активно (без набиране) Фаза 3 NCT05881408

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

Фаза 3 – широко изпитване преди одобрение
Заболявания: Duchenne Muscular Dystrophy

Спонсор: Sarepta Therapeutics, Inc.

Налично на: БГ
Обобщение
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
Кой може да участва
Inclusion Criteria: * Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. * Cohort 1 only: Non-ambulatory per protocol-specified criteria. * Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to \<18 years of age at the time of Screening. * Ability to cooperate with motor assessment testing. * Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight). * Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements. * A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or 9. Exclusion Criteria: * Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits. * Abnormality in protocol-specified diagnostic evaluations or laboratory tests. * Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer. Other inclusion or exclusion criteria could apply.
Места на провеждане 14
Австралия (1)
The Children's Hospital at Westmead
Westmead , New South Wales
Белгия (1)
Universitair Ziekenhuis Gent
Ghent , Oost-Vlaanderen
Канада (1)
The Children's Hospital of Eastern Ontario
Ottawa , Ontario
Германия (1)
LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt
München , Bavaria
Hong Kong (1)
Hong Kong Children's Hospital
Hong Kong
Израел (1)
Institute of Neruology, Schneider Children's Medical Center of Israel
Petah Tikva
Италия (1)
U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
Genova
Япония (1)
National Hospital Organization Osaka Toneyama Medical Center
Toyonaka-shi , Osaka
Южна Корея (1)
Pusan National University Yangsan Hospital
Yangsan , Gyeongsangnam-do
Испания (1)
Hospital Sant Joan de Deu
Esplugues de Llobregat , Barcelona
Швеция (1)
Sahlgrenska Universitetssjukhuset
Gothenburg
Taiwan (1)
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City
Великобритания (1)
Great Ormond Street Hospital for Children Foundation Trust
London , Greater London
САЩ (1)
Arkansas Children's Hospital
Little Rock , Arkansas
Технически детайли
Статус
Активно (без набиране)
Фаза
Фаза 3
Вид изследване
INTERVENTIONAL
Пол
Само мъже
Здрави доброволци
Не
Начална дата
31.05.2023
Крайна дата
30.06.2028
Регистрационен номер
NCT05881408
Източник
anzctr
Запитване за медицински туризъм

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