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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04184453
Recruitment Status : Recruiting
First Posted : December 3, 2019
Last Update Posted : January 21, 2020
Sponsor:
Information provided by (Responsible Party):
Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University

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
Aceruloplasminemia 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Patients 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 : December 23, 2019
Estimated Primary Completion Date : June 23, 2020
Estimated Study Completion Date : November 23, 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Deferiprone

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




Primary Outcome Measures :
  1. Change of pancreas MRI [ Time Frame: Up to 3 months ]
    Pancreas MRI is related to iron accumulation of pancreas

  2. Change of glycated hemoglobin (HbA1c) [ Time Frame: Up to 3 months ]
    HbA1c is a measure of long-term blood sugar control



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04184453


Contacts
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Contact: Jin He 13645050186 hejin@fjmu.edu.cn
Contact: Wanjin Chen 13860601359 ext 059187982772 wanjinchen75@fjmu.edu.cn

Locations
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China, Fujian
Department of Neurology , First Affiliated Hospital Fujian Medical University Recruiting
Fuzhou, Fujian, China, 350004
Contact: HeJin    13645050186      
Sponsors and Collaborators
First Affiliated Hospital of Fujian Medical University
Investigators
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Principal Investigator: Wanjin Chen Department of Neurology , First Affiliated Hospital Fujian Medical University

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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: December 3, 2019    Key Record Dates
Last Update Posted: January 21, 2020
Last Verified: December 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University:
Aceruloplasminemia
Deferiprone
Pancreas MRI
Additional relevant MeSH terms:
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Neurodegenerative Diseases
Iron Metabolism Disorders
Metabolic Diseases
Nervous System Diseases
Deferiprone
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action