A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients with Wilson's Disease - VTX-801_CLN_001

Vivet Therapeutics0 个研究点目标入组 16 人开始时间: 2024年4月2日最近更新: 2024年7月22日

适应症

试验速览

暂无简介。

•Male or female aged 18-65 years old (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 including 24- hour urinary copper, free serum copper such as NCC or CuEXC (when available), as well as liver enzymes, hemoglobin, and white blood cell count

•ALT level = 2 x ULN that is not readily explained by extrinsic factors (e.g., strenuous exercise, medication use), Any concomitant disorder/condition - including hepatic disorder - or treatment possibly interfering with the conduct or evaluation of the study, according to the Investigator., Pregnancy or breastfeeding., Body Mass Index = 35 kg/m2., Total bilirubin > 1.5x ULN in the absence of proven Gilbert’s syndrome; in case of Gilbert’s syndrome, direct bilirubin > ULN., Platelet count < 120,000/µL., 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 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

发起方

Vivet Therapeutics