Geç Başlangıçlı Pompe Hastalığı Olan Kişilerde Kandaki Antikor Düzeyleri ve Biyobelirteçler Üzerine Bir Çalışma

Sade dilde özet

Bu çalışma, geç başlangıçlı Pompe hastalığı (late-onset Pompe disease) olan kişilerin kanındaki antikor düzeylerini ve biyobelirteçleri (biomarkers) incelemektedir. Herhangi bir tedavi uygulanmayan bu araştırmaya, tanısı belgelenmiş hastalar katılmaktadır. Çalışma faz uygulanmayan bir gözlem çalışması olarak yürütülmekte; şu an katılımcı almamakta ancak devam etmektedir (active, not recruiting).

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

Deney bilgileri

Faz

Faz dışı

Durum

Devam ediyor, katılım kapalı

Sponsor

Astellas Gene Therapies

Müdahale

No Intervention

Başlangıç

01.02.2024

Tahmini bitiş

30.06.2027

Ülkeler

United States, Australia, Brazil, Canada, France, Germany, Italy, Japan, Spain, Taiwan, United Kingdom

İlgili genler

Merkezler

• Atlanta, United States
• Kansas City, United States
• Ann Arbor, United States
• Minneapolis, United States
• Hackensack, United States
• Cincinnati, United States
• Philadelphia, United States
• Pittsburgh, United States
• Salt Lake City, United States
• Fairfax, United States
• Adelaide, Australia
• Herston, Australia
• Flamengo, Brazil
• Porto Alegre, Brazil
• São Paulo, Brazil
• Edmonton, Canada
• Montreal, Canada
• Angers, France
• Garches, France
• Lille, France
• Limoges, France
• Marseille, France
• Nantes, France
• Nice, France
• Strasbourg, France
• Bonn, Germany
• Essen, Germany
• Höchheim, Germany
• Münster, Germany
• Florence, Italy
• Gussago, Italy
• Messina, Italy
• Milan, Italy
• Milan, Italy
• Pavia, Italy
• Pisa, Italy
• Roma, Italy
• Udine, Italy
• Kodaira-Shi, Japan
• Shinjuku-Ku, Japan

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Uygunluk kriterleri

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

Inclusion Criteria:

* Participant has a documented clinical diagnosis of LOPD.
* Participant is enzyme replacement therapy ERT-naïve (ERT-N) or if the participant is currently taking an approved ERT treatment or is participating in an ERT-interventional study, the ERT must have been received for at least 6 months or more (ERT-experienced \[ERT-E\]).
* Participant is willing and able to comply with study visits and procedures.
* Participant agrees to not start participating in any other clinical study involving an investigational study treatment, including ERT, while participating in this study.

Exclusion Criteria:

* Participant previously received an AAV-related product (any serotype).
* Participant is currently participating in a Pompe-related interventional study (other than ERT-interventional studies) or has received gene or cell therapy.
* Participant requires any invasive or noninvasive ventilation support while awake and upright (non-invasive support while sleeping with either continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) is acceptable for eligibility).
* Participant is unable to ambulate (assistive devices \[e.g., cane or walker\] are acceptable for eligibility).
* Participants who have received any ERT for less than 6 months as of the Baseline visit are not eligible.