Clinical Curative Effect Evaluation Study of Treatment of... | NCT04184453

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

NCT04184453

Sponsor

First Affiliated Hospital of Fujian Medical University

Status

Unknown status

Last Update Posted

Nov 23, 2021Actual

Enrollment

5Estimated

Phase

Early Phase 1

Conditions

Aceruloplasminemia

Interventions

Deferiprone treated

Countries

China

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Nov 2021

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Dec 23, 2019Actual

Primary Completion Date

Oct 23, 2022Estimated

Completion Date

Nov 23, 2022Estimated

First Submitted Date

Nov 29, 2019

First Submission Date that Met QC Criteria

Nov 29, 2019

First Posted Date

Dec 3, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 21, 2021

Last Update Posted Date

Nov 23, 2021Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Wan-Jin Chen, Professor, First Affiliated Hospital of Fujian Medical UniversityPrincipal Investigator

Lead Sponsor

First Affiliated Hospital of Fujian Medical UniversityOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Early Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

5Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Deferiprone treated	Experimental	Deferiprone (25 mg/kg/day) was given to the enrolled patient.	Drug: Deferiprone treated

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Deferiprone treated	Drug	Deferiprone (25 mg/kg/day) was given to the enrolled patients.	Deferiprone treated

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

80 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Jin He	Contact	13645050186		hejin@fjmu.edu.cn
Wanjin Chen	Contact	13860601359	059187982772	wanjinchen75@fjmu.edu.cn

Overall Officials

Name	Affiliation	Role
Wanjin Chen	Department of Neurology , First Affiliated Hospital Fujian Medical University	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Department of Neurology , First Affiliated Hospital Fujian Medical University	Recruiting	Fuzhou	Fujian	350004	China

Condition Browse Module

MeSH Terms

ID	Term
C536004	Familial apoceruloplasmin deficiency

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided