Duchenne Musküler Distrofisi (DMD) Olan Katılımcılarda Delandistrogene Moxeparvovec (SRP-9001) Gen Transfer Tedavisinin Güvenliğini ve Etkinliğini Değerlendiren Çalışma

Sade dilde özet

Bu çalışma, Duchenne musküler distrofisi (Duchenne muscular dystrophy) olan çocuklarda Delandistrogene moxeparvovec (SRP-9001) adlı gen transfer tedavisinin güvenliğini ve etkinliğini plaseboya karşı değerlendirmiştir. Çalışmaya, yürüyebilen, 4 ile 8 yaş arasında ve genetik testle DMD tanısı doğrulanmış çocuklar alınmıştır. Faz 3 aşamasında yürütülmüş ve tamamlanmıştır.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

Faz 3 (Phase 3)

Durum

Tamamlandı

Sponsor

Sarepta Therapeutics, Inc.

Müdahale

delandistrogene moxeparvovec, placebo

Başlangıç

27.10.2021

Tahmini bitiş

25.10.2024

Ülkeler

United States, Belgium, Germany, Hong Kong, Italy, Japan, Spain, Taiwan, United Kingdom

İlgili genler

Merkezler

• Little Rock, United States
• La Jolla, United States
• Los Angeles, United States
• Palo Alto, United States
• Sacramento, United States
• Aurora, United States
• Gainesville, United States
• Chicago, United States
• Iowa City, United States
• Baltimore, United States
• Boston, United States
• St Louis, United States
• New York, United States
• Rochester, United States
• Durham, United States
• Columbus, United States
• Portland, United States
• Philadelphia, United States
• Nashville, United States
• Dallas, United States
• Salt Lake City, United States
• Norfolk, United States
• Milwaukee, United States
• Ghent, Belgium
• Bayern, Germany
• Essen, Germany
• Hamburg, Germany
• Kowloon, Hong Kong
• Genoa, Italy
• Milan, Italy
• Roma, Italy
• Kobe, Japan
• Tokyo, Japan
• Tokyo, Japan
• Tokyo, Japan
• Valencia, Spain
• Barcelona, Spain
• Kaohsiung City, Taiwan
• Taipei, Taiwan
• Headington, United Kingdom

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Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

Inclusion Criteria:

* Is ambulatory and from 4 to under 8 years of age at time of randomization.
* Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
* 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).
* rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
* A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45.

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.