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Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency
Bu çalışma, Alfa-1 Antitripsin Eksikliği (alpha-1 antitrypsin deficiency — SERPINA1 genindeki mutasyona bağlı kalıtsal bir hastalık) olan hastalarda bir gen terapisini (ADVM-043) inceliyor. ZZ veya Z Null genotipi olan, 18 yaş üstü hastalar dahil edilmiştir. Faz 1/Faz 2 aşamasında yürütülmüş ve tamamlanmıştır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Key Inclusion Criteria: * Capable of providing informed consent * Alpha1AT genotype of ZZ or Z Null * Males and females 18 years and older * Ongoing treatment with A1AT augmentation is not required, however any subject receiving A1AT augmentation therapy must be willing to washout. Washout is defined as at least 8 weeks between last augmentation therapy and pre-treatment plasma A1AT level * Willing to remain off PAT for at least 3 months following treatment * Body mass index 18 to 35 kg/m2 * Fertile men and women of childbearing potential must agree to use barrier contraception for 3 months after treatment Key Exclusion Criteria: * FEV1 \<35 percent of predicted value at the Screening visit * Receiving systemic corticosteroids or other immunosuppressive medications * Immunodeficiency disease or evidence of active infection of any type, including human immunodeficiency virus * Abnormal liver function tests * Organ transplant recipient or awaiting transplantation * Participation in another current or previous gene transfer study * AAVrh.10 neutralizing antibody titer ≥ 1:5 * Female who is pregnant or lactating * History of alcohol or drug abuse within the past 5 years * Any history of allergies that may prohibit study-specific investigations * Receiving an investigational medicinal product or participating in another investigational study within 3 months prior to consent * Cigarette smoking, or any other tobacco use, e-cigarettes or other recreational inhalant within 1 year of the Screening Visit