Overview

This trial is active, not recruiting.

Condition wilson disease
Treatment wtx101
Phase phase 2
Sponsor Wilson Therapeutics AB
Start date November 2014
End date August 2017
Trial size 30 participants
Trial identifier NCT02273596, WTX101-201

Summary

The purpose of the study is to evaluate the efficacy and safety of WTX101 administered for 24 weeks in newly diagnosed Wilson Disease (WD) patients aged 18 and older with Nonceruloplasmin-bound copper (NCC) levels within or above the normal reference range at the time of enrollment. Subjects with Wilson Disease who have received either no prior Wilson Disease treatments, or have been treated for up to and including 24 months prior to study enrollment with chelation therapy (e.g. trientine, penicillamine), zinc therapy or combination therapy are eligible to participate, if all other inclusion and no exclusion criteria are met. The purpose of the 12 month Extension Phase is to evaluate the durability, and establish long-term safety and efficacy of WTX101.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
WTX101 Dosage Form: Enteric Coated Tablet WTX101 Dose: 15 - 60 mg, individualized dosing, WTX101 Frequency: QOD, QD or BID, individualized dosing WTX101 Duration: 76 weeks
wtx101
Dosage Form: 15 mg Tablets

Primary Outcomes

Measure
Nonceruloplasmin-bound copper (NCC) levels adjusted for molybdenum (Mo) plasma concentration
time frame: 24 weeks

Secondary Outcomes

Measure
Change in and time to normalisation of NCC levels adjusted for Mo plasma concentration
time frame: 24 weeks
Neurological status using the Unified Wilson's Disease Rating Scale (UWDRS)
time frame: 24 weeks
Psychiatric status using Mini International Neuropsychiatric Interview (M.I.N.I.) Tracking
time frame: 24 weeks
Clinical symptoms as assessed by the Investigators on the Clinician Global Impression (CGI) scale items 1 (severity of illness) and 2 (global improvement)
time frame: 24 weeks
Quality of Life / Patient Reported Outcome endpoint measures EQ5D
time frame: 24 weeks
Quality of Life / Patient Reported Outcome endpoint measure MMAS-8
time frame: 24 weeks
Quality of Life / Patient Reported Outcome endpoint measure TSQM
time frame: 24 weeks
Hepatic measure ALT
time frame: 24 weeks
Hepatic measure AST
time frame: 24 weeks
Hepatic measure INR
time frame: 24 weeks
Hepatic measure bilirubin)
time frame: 24 weeks
Copper endpoint: Exchangeable copper
time frame: 24 weeks
Copper endpoint: Speciation profiling
time frame: 24 weeks
Copper endpoint: 24-hour urinary copper
time frame: 24 weeks
Plasma PK parameters. Estimates of individual molybdenum PK parameters, including AUC and Cmax
time frame: 24 weeks
Incidence and severity of adverse events (AEs)
time frame: Throughout the study (screening up to follow-up)
Copper endpoint: Total copper
time frame: 24 weeks

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Willing and able to give informed consent for participation in the study. - Male or female patients, aged 18 years or older as of signing the ICF. - Able to understand and willing to comply with study procedures, restrictions and requirements, as judged by the Investigator. - Established diagnosis of Wilson Disease by Leipzig-Score ≥ 4 (Ferenci et al 2003) documented by testing as outlined in 2012 EASL WD Clinical Practice Guidelines. - NCC levels within or above the normal reference range (0.8 - 2.3 μM). - Willing to undergo 48 hour washout from current Wilson Disease treatment Exclusion Criteria: - Treatment for greater than 24 months for Wilson Disease with chelation therapy (i.e. penicillamine, trientine hydrochloride) or zinc therapy. - Decompensated hepatic cirrhosis. - Model for End-Stage Liver Disease (MELD) score > 11. - Modified Nazer score > 6 (Dhawan et al. Liver Transplant 2005). - GI bleed within past 6 months. - ALT > 5x upper limit of normal (ULN). - Marked neurological disease requiring either nasogastric (NG) feeding or intensive in-patient medical care. - Severe anaemia with a haemoglobin < 9 mg/dL.

Additional Information

Official title A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older With an Extension Phase of 12 Months
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Wilson Therapeutics AB.