Aceruloplasminemia
Conditions
Keywords
Aceruloplasminemia, Deferiprone, Pancreas MRI
Brief summary
Aceruloplasminemia is an autosomal recessive disorder characterized by iron deposition in the brain and visceral organs. Deferiprone was used to treat aceruloplasminemia in previous study. In this study, we will assess the clinical curative effect evaluation of oral deferiprone in Chinese aceruloplasminemia patients.
Detailed description
Diabetes is one of the most severe symptom in aceruloplasminemia. In this study, we use R2\* of pancreas MRI to evaluate the effect of deferiprone treatment.
Interventions
DRUGDeferiprone treated
Deferiprone (25 mg/kg/day) was given to the enrolled patients.
Sponsors
First Affiliated Hospital of Fujian Medical University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Patients who was diagnosed as aceruloplasminiaemia
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Age 18-80 years * Diagnosed with aceruloplasminaemia * Genetically confirmed diagnosis of aceruloplasminaemia
Exclusion criteria
* Contraindications to deferiprone therapy * Pregnancy was excluded in women of childbearing age
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change of pancreas MRI | Up to 3 months | Pancreas MRI is related to iron accumulation of pancreas |
| Change of glycated hemoglobin (HbA1c) | Up to 3 months | HbA1c is a measure of long-term blood sugar control |
Countries
China
Contacts
Primary ContactJin He
Backup ContactWanjin Chen
Outcome results
None listed