Study of galactosidase as therapy for type IV galactosemia
Phase 2
- Conditions
- type IV galactosemiagalactosemia: galactose mutarotase: GALMD005693
- Registration Number
- JPRN-jRCTs021200008
- Lead Sponsor
- Kure Shigeo
- Brief Summary
This study demonstrated that the galactosidase treatment for type IV galactosemia can reduce blood galactose levels in lactose loading, suggesting the galactosidase may be a therapeutic agent for type IV galactosemia.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 3
Inclusion Criteria
Patients are diagnosed to type IV galactosemia by genetic test (pathogenic variants in GALM gene are identified by exome or direct sequencing) or GALM enzyme activity test.
Exclusion Criteria
(1) Patients with severe liver disease or liver injury (AST, ALT > 1.25 x upper limits of normal)
(2) Patients have participated to other clinical study or trial
(3) Patients with constipation which is in need of treat
(4) Patients with psychological problem
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method