A clinical trial: treatment of adult Wilson's Disease patients with gene therapy

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 16

Inclusion Criteria

1. Male or female; 18 to 65 years old (inclusive)
2. Confirmed diagnosis of WD
3. Treated for WD according to international recommendations with no current evidence for inadequate treatment (EASL, 2012; Roberts et al,
2008)
4. 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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 16
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. ALT level = 2 x ULN that is not readily explained by extrinsic factors (e.g., strenuous exercise, medication use)
2. Total bilirubin > 1.5 x ULN in the absence of proven Gilbert's syndrome; in case of Gilbert's syndrome, direct bilirubin > ULN
3. Platelet count <120.000 /µL
4. Any signs of liver cirrhosis decompensation, including gastrointestinal
bleed within 6 months (24 weeks) prior to screening/enrolment visit
5. Patient has moderate or severe renal impairment or patient has nephritis or nephrotic syndrome
6. Any history or current evidence of HIV-1, HIV-2, HTLV 1, or HTLV-2 infection
7. Any history or current evidence of hepatitis B infection
8. Any history of hepatitis C infection, unless previous viral RNA assays in two samples, collected at least 6 months apart, are negative
9. Positive QuantiFERON®-TB Gold tuberculosis test result
10. Any concomitant disorder/condition - including hepatic disorders - or treatment possibly interfering with the conduct or evaluation of the study, according to the Investigator
11. Pregnancy or breastfeeding
12. Body Mass Index = 35 kg/m2

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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