Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients

Early Phase 1UnknownNCT04184453

First Affiliated Hospital of Fujian Medical University5 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Aceruloplasminemia

Interventions

Deferiprone treatedDRUG

Deferiprone (25 mg/kg/day) was given to the enrolled patients.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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

Locations (1)

Department of Neurology , First Affiliated Hospital Fujian Medical University

Fuzhou, Fujian, China

RECRUITING

Outcomes

Primary Outcomes

Change of pancreas MRI

Pancreas MRI is related to iron accumulation of pancreas

Time frame: Up to 3 months

Change of glycated hemoglobin (HbA1c)

HbA1c is a measure of long-term blood sugar control

Time frame: Up to 3 months

Central Contacts

Jin He

CONTACT

13645050186 hejin@fjmu.edu.cn

Wanjin Chen

CONTACT

13860601359 wanjinchen75@fjmu.edu.cn

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.