Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients
- Registration Number
- NCT04184453
- 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.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 5
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Deferiprone treated Deferiprone treated Deferiprone (25 mg/kg/day) was given to the enrolled patient.
- Primary Outcome Measures
Name Time Method Change of glycated hemoglobin (HbA1c) Up to 3 months HbA1c is a measure of long-term blood sugar control
Change of pancreas MRI Up to 3 months Pancreas MRI is related to iron accumulation of pancreas
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Department of Neurology , First Affiliated Hospital Fujian Medical University
🇨🇳Fuzhou, Fujian, China