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

Sponsor
First Affiliated Hospital of Fujian Medical University (Other)
Overall Status
Recruiting
CT.gov ID
NCT04184453
Collaborator
(none)
5
Enrollment
1
Location
1
Arm
35
Anticipated Duration (Months)
0.1
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Deferiprone treated
 Early Phase 1

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 Design

Study Type:
Interventional
Anticipated Enrollment :
5 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Intervention Model Description:
Patients who was diagnosed as aceruloplasminiaemiaPatients who was diagnosed as aceruloplasminiaemia
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients
Actual Study Start Date :
Dec 23, 2019
Anticipated Primary Completion Date :
Oct 23, 2022
Anticipated Study Completion Date :
Nov 23, 2022

Arms and Interventions

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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Change of pancreas MRI [Up to 3 months]

      Pancreas MRI is related to iron accumulation of pancreas

    2. Change of glycated hemoglobin (HbA1c) [Up to 3 months]

      HbA1c is a measure of long-term blood sugar control

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 80 Years
    Sexes Eligible for Study:
    All
    Accepts 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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Department of Neurology , First Affiliated Hospital Fujian Medical University Fuzhou Fujian China 350004

    Sponsors and Collaborators

    • First Affiliated Hospital of Fujian Medical University

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Wan-Jin Chen, Professor, First Affiliated Hospital of Fujian Medical University
    ClinicalTrials.gov Identifier:
    NCT04184453
    Other Study ID Numbers:
    • MRCTA,ECFAH of FMU [2019]252
    First Posted:
    Dec 3, 2019
    Last Update Posted:
    Nov 23, 2021
    Last Verified:
    Nov 1, 2021
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Wan-Jin Chen, Professor, First Affiliated Hospital of Fujian Medical University
    Aceruloplasminemia
    Deferiprone
    Pancreas MRI
    Additional relevant MeSH terms:
    Neurodegenerative Diseases
    Iron Metabolism Disorders
    Deferiprone

    Study Results

    No Results Posted as of Nov 23, 2021