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

NCT ID: NCT04184453

Last Updated: 2021-11-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

EARLY_PHASE1

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-12-23

Study Completion Date

2022-11-23

Brief Summary

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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

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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.

Conditions

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Aceruloplasminemia

Keywords

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Aceruloplasminemia Deferiprone Pancreas MRI

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Patients who was diagnosed as aceruloplasminiaemia
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Deferiprone treated

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

Group Type EXPERIMENTAL

Deferiprone treated

Intervention Type DRUG

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

Interventions

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Deferiprone treated

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

Intervention Type DRUG

Other Intervention Names

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

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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First Affiliated Hospital of Fujian Medical University

OTHER

Sponsor Role lead

Responsible Party

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Wan-Jin Chen

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Wanjin Chen

Role: PRINCIPAL_INVESTIGATOR

Department of Neurology , First Affiliated Hospital Fujian Medical University

Locations

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Department of Neurology , First Affiliated Hospital Fujian Medical University

Fuzhou, Fujian, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Jin He

Role: CONTACT

Phone: 13645050186

Email: [email protected]

Wanjin Chen

Role: CONTACT

Phone: 13860601359

Email: [email protected]

Facility Contacts

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HeJin

Role: primary

Other Identifiers

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MRCTA,ECFAH of FMU [2019]252

Identifier Type: -

Identifier Source: org_study_id