This study adopted an open, single-arm, non-randomized, dose-escalation research design, aiming to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetic and immunogenicity characteristics of single and multiple intravenous infusions of DSL101 in patients with Wilson's disease.
In this study, low, medium and high dose groups were preset, the low dose group was accelerated titration group, and the medium and high dose groups used the traditional "3+3" method combined with Sentinel method for dose escalation.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Subjects will receive intravenous infusions of DSL101 once every four weeks.
Subjects will receive intravenous infusions of DSL101 once every four weeks.
Subjects will receive intravenous infusions of DSL101 once every four weeks.
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, China
RECRUITINGIncidence of adverse events and serious adverse events
Time frame: up to 56 weeks
Change in 24 hour urine copper
Time frame: up to week 20
Change in sum total cpper [ serum copper bound by ceruloplasmin (NCC) ]
Time frame: up to week 20
Change in serum free copper
Time frame: up to week 20
Change from baseline in serum ceruloplasmin
Time frame: up to week20
Change in serum iron concentration and serum ferritin concentration
Time frame: up to week 20
Clinical laboratory test: Biochemistry - alanine aminotransferase (ALT)
The physician will judge whether an abnormality is clinically significant
Time frame: up to week 20
Change in the total score of the Unified Wilson Disease Rating Scale (UWDRS )
The UWDRS scale consists of three parts: neurological, liver function and mental symptom. The higher scores mean a worse outcome.
Time frame: up to week 20
Number of subjects and percentage decrease in standard of care (SOC) medication euse within 20 weeks of administration
Time frame: up to week 20
PK: Average steady-state concentration (Cav,ss)
Time frame: up to week 6
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Change from baseline in seurm ceruloplasmin activity
Time frame: up to week20
Clinical laboratory test: Biochemistry - aspartate aminotransferase (AST)
Time frame: up to week 20
Clinical laboratory test: Biochemistry - alkaline phosphatase (ALP)
Time frame: up to week 20
Clinical laboratory test: Biochemistry - gamma-glutamyltransferase (γ-GGT)
Time frame: up to week 20
Clinical laboratory test: Biochemistry - total bilirubin (TBIL)
Time frame: up to week 20