Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients
| Status: | Completed |
|---|---|
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 12/12/2018 |
| Start Date: | November 24, 2014 |
| End Date: | November 7, 2018 |
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
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.
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.
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.
We found this trial at
7
sites
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