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

Primary Purpose

Aceruloplasminemia

Status
Recruiting
Phase
Early Phase 1
Locations
China
Study Type
Interventional
Intervention
Deferiprone treated
Sponsored by
First Affiliated Hospital of Fujian Medical University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Aceruloplasminemia focused on measuring Aceruloplasminemia, Deferiprone, Pancreas MRI

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Department of Neurology , First Affiliated Hospital Fujian Medical UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Deferiprone treated

Arm Description

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

Outcomes

Primary Outcome Measures

Change of pancreas MRI
Pancreas MRI is related to iron accumulation of pancreas
Change of glycated hemoglobin (HbA1c)
HbA1c is a measure of long-term blood sugar control

Secondary Outcome Measures

Full Information

First Posted
November 29, 2019
Last Updated
November 21, 2021
Sponsor
First Affiliated Hospital of Fujian Medical University
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1. Study Identification

Unique Protocol Identification Number
NCT04184453
Brief Title
Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients
Official Title
Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients
Study Type
Interventional

2. Study Status

Record Verification Date
November 2021
Overall Recruitment Status
Recruiting
Study Start Date
December 23, 2019 (Actual)
Primary Completion Date
October 23, 2022 (Anticipated)
Study Completion Date
November 23, 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
First Affiliated Hospital of Fujian Medical University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Aceruloplasminemia
Keywords
Aceruloplasminemia, Deferiprone, Pancreas MRI

7. Study Design

Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Model Description
Patients who was diagnosed as aceruloplasminiaemia
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Deferiprone treated
Arm Type
Experimental
Arm Description
Deferiprone (25 mg/kg/day) was given to the enrolled patient.
Intervention Type
Drug
Intervention Name(s)
Deferiprone treated
Other Intervention Name(s)
Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients
Intervention Description
Deferiprone (25 mg/kg/day) was given to the enrolled patients.
Primary Outcome Measure Information:
Title
Change of pancreas MRI
Description
Pancreas MRI is related to iron accumulation of pancreas
Time Frame
Up to 3 months
Title
Change of glycated hemoglobin (HbA1c)
Description
HbA1c is a measure of long-term blood sugar control
Time Frame
Up to 3 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jin He
Phone
13645050186
Email
hejin@fjmu.edu.cn
First Name & Middle Initial & Last Name or Official Title & Degree
Wanjin Chen
Phone
13860601359
Ext
059187982772
Email
wanjinchen75@fjmu.edu.cn
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Wanjin Chen
Organizational Affiliation
Department of Neurology , First Affiliated Hospital Fujian Medical University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Department of Neurology , First Affiliated Hospital Fujian Medical University
City
Fuzhou
State/Province
Fujian
ZIP/Postal Code
350004
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
HeJin
Phone
13645050186

12. IPD Sharing Statement

Learn more about this trial

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

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