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

Aceruloplasminemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04184453
• Other study ID #: MRCTA,ECFAH of FMU [2019]252
• Status: Recruiting
• Phase: Early Phase 1
• Start date: December 23, 2019
• Est. completion date: November 23, 2022

Study information

• Verified date: November 2021
• Source: First Affiliated Hospital of Fujian Medical University
• Contact: Jin He
• Phone: 13645050186
• Email: hejin[@]fjmu.edu.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 5
• Est. completion date: November 23, 2022
• Est. primary completion date: October 23, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Aceruloplasminemia
• Iron Metabolism Disorders
• Neurodegenerative Diseases

Intervention

Drug:
• Deferiprone treated
Deferiprone (25 mg/kg/day) was given to the enrolled patients.

Locations

Country	Name	City	State
China	Department of Neurology , First Affiliated Hospital Fujian Medical University	Fuzhou	Fujian

Sponsors (1)

Lead Sponsor	Collaborator
First Affiliated Hospital of Fujian Medical University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change of pancreas MRI	Pancreas MRI is related to iron accumulation of pancreas	Up to 3 months
Primary	Change of glycated hemoglobin (HbA1c)	HbA1c is a measure of long-term blood sugar control	Up to 3 months