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

November 21, 2021 updated by: Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University
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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

5

Phase

  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Fujian
      • Fuzhou, Fujian, China, 350004
        • Recruiting
        • Department of Neurology , First Affiliated Hospital Fujian Medical University
        • Contact:
          • HeJin
          • Phone Number: 13645050186

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 78 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Deferiprone treated
Deferiprone (25 mg/kg/day) was given to the enrolled patient.
Drug: Deferiprone treated
Deferiprone (25 mg/kg/day) was given to the enrolled patients.
Other Names:
  • Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change of pancreas MRI
Time Frame: Up to 3 months
Pancreas MRI is related to iron accumulation of pancreas
Up to 3 months
Change of glycated hemoglobin (HbA1c)
Time Frame: Up to 3 months
HbA1c is a measure of long-term blood sugar control
Up to 3 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

First Affiliated Hospital of Fujian Medical University

Investigators

  • Principal Investigator: Wanjin Chen, Department of Neurology , First Affiliated Hospital Fujian Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 23, 2019

Primary Completion (Anticipated)

October 23, 2022

Study Completion (Anticipated)

November 23, 2022

Study Registration Dates

First Submitted

November 29, 2019

First Submitted That Met QC Criteria

November 29, 2019

First Posted (Actual)

December 3, 2019

Study Record Updates

Last Update Posted (Actual)

November 23, 2021

Last Update Submitted That Met QC Criteria

November 21, 2021

Last Verified

November 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MRCTA,ECFAH of FMU [2019]252

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Aceruloplasminemia

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