Wilson's Disease
Conditions
Brief summary
The purpose of this long-term study is to determine whether Zinc Acetate is effective and safe in the treatment of Wilson's disease among Japanese.
Detailed description
Wilson disease is an autosomal recessive disorder with copper metabolism. In Japan, the standard treatment is the use of copper chelating agents, such as D-penicillamine and trientine. In this study, we investigate efficacy on zinc acetate in Japanese patients with Wilson disease.
Interventions
DRUGNPC-02
zinc acetate
Sponsors
Nobelpharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Wilson's disease(adult, infant, pregnant woman)
Exclusion criteria
* Acute hepatitis * Malignant tumor
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and Efficacy | During study period (up to 96W ) | Number of patients who have at least one adverse events. ALT Change |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| NPC-02 zinc acetate
NPC-02: zinc acetate | 37 |
| Total | 37 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | A change of address | 1 |
| Overall Study | Adverse Event | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | NPC-02 |
|---|---|
| Age, Customized ≥16 | 20 participants |
| Age, Customized ≥1 - <6 | 2 participants |
| Age, Customized ≥6 - <16 | 15 participants |
| Sex: Female, Male Female | 24 Participants |
| Sex: Female, Male Male | 13 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 37 / 37 |
| serious Total, serious adverse events | 4 / 37 |
Outcome results
Primary
Safety and Efficacy
Number of patients who have at least one adverse events. ALT Change
Time frame: During study period (up to 96W )
Population: The analysis was performed based on FAS population
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| NPC-02 | Safety and Efficacy | 37 participants |