Mucopolysaccharidosis (including types: I, II, IIIa, IIIb, IIIc, IIId, IVa, IVb, VI, VII, IX, etc.)
Conditions
Interventions
the group treated with bromhexine hydrochloride followed by ezetimibe (Group 1):first consume Ambroxol Hydrochloride Oral Solution and then Ezetimibe tablets orally
The group treated with ezetimibe followed by bromhexine hydrochloride(Group 2):first consume Ezetimibe tablets orally and then Ambroxol Hydrochloride Oral Solution
Sponsors
Children's Hospital,Zhejiang University School of Medicine
Eligibility
Sex/Gender
All
Age
5 Years to No maximum
Inclusion criteria
Inclusion criteria: 1.Patients with mucopolysaccharidosis diagnosed clinically through enzymatic and/or genetic testing; 2.Age above 5 years old (mainly considering the age indication for ezetimibe); 3.Informed consent.
Exclusion criteria
Exclusion criteria: 1.Transplantation or stem cell therapy; 2.Liver dysfunction (ALT more than twice the normal level), renal insufficiency (serum creatinine increase >=26.5 µmol/L within 48 hours; serum creatinine increase more than 1.5 times the baseline value and above within 7 days; urine output reduction (<0.5 ml·kg^-1·h^-1) lasting more than 6 hours); 3.Hereditary fructose intolerance; 4.Allergy to ezetimibe, ambroxol, etc.; 5.Pregnant and lactating women; 6.The subject or guardian does not consent.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Urinary Glycosaminoglycan (GAG) Levels;Enzyme Activity Testing;improvement in hepatosplenomegaly;improvement in motor ability;improvement in growth rate;improvement in joints;improvement in facial appearance;improvement in skeletal deformities;improvement in cardiac issues;Liver and kidney function;blood lipids;muscle enzymes;blood sugar; | — |
Secondary
| Measure | Time frame |
|---|---|
| improvement in respiratory function;improvement in vision;improvement in hearing;improvement in speech;improvement in intelligence;Adverse reaction; | — |
Countries
China
Contacts
Public ContactZou Chaochun
Children's Hospital,Zhejiang University School of Medicine
Outcome results
None listed