Treatment of adult Wilson's Disease patients with gene therapy

Phase 1

Recruiting

• Conditions: Wilson's Disease; MedDRA version: 20.0Level: LLTClassification code: 10047988Term: Wilson's disease Class: 10010331; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: CTIS2023-509998-23-00
• Lead Sponsor: Vivet Therapeutics

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-04-02
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified