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A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
Bu çalışma, yetişkin Wilson hastalığı (Wilson's disease) olan hastalarda VTX-801 adlı bir gen terapisini incelemektedir. Çalışmaya, doğrulanmış Wilson hastalığı tanısı olan, en az bir yıldır durumu stabil seyreden, 18-65 yaş arası hastalar katılmaktadır. Çalışma Faz 1/Faz 2 aşamasında 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.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Main Inclusion Criteria: * Male or female aged 18 and 65 years inclusive * Confirmed diagnosis of WD * Treated for WD according to international recommendations with no current evidence for inadequate treatment * Stable WD for ≥ 1 year, defined as: (i) No significant change in neurologic examination and in status of mood disorder and (ii) Stable laboratory parameters used to assess copper metabolism Main Exclusion Criteria: * ALT level ≥ 2 ULN that is not readily explained by extrinsic factors * Total bilirubin \> 1.5 x ULN in the absence of proven Gilbert's syndrome; in case of Gilbert's syndrome, direct bilirubin \> ULN * INR \> 1.2 * Any signs of liver cirrhosis decompensation, including gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrollment visit * Patient has moderate or severe renal impairment defined as eGFR CKD-EPI \< 60 mL/min/1.73 m2, or patient has nephritis or nephrotic syndrome * Any history or current evidence of HIV-1, HIV-2, HTLV 1, or HTLV-2 infection * Any history or current evidence of hepatitis B infection * Any history of hepatitis C infection, unless previous viral RNA assays in two samples, collected at least 6 months apart, are negative * Positive QuantiFERON®-TB Gold tuberculosis test result * Any concomitant disorder/condition - including hepatic disorders - or treatment possibly interfering with the conduct or evaluation of the study * Any history of diabetes * Pregnancy or breastfeeding * Body Mass Index ≥ 35 kg/m2 Other protocol defined Inclusion/ Exclusion criteria may apply