NCT04184453

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
5

participants targeted

Target at below P25 for early_phase_1

Timeline
Completed

Started Dec 2019

Typical duration for early_phase_1

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 29, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

December 3, 2019

Completed
20 days until next milestone

Study Start

First participant enrolled

December 23, 2019

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 23, 2022

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

November 23, 2022

Completed
Last Updated

November 23, 2021

Status Verified

November 1, 2021

Enrollment Period

2.8 years

First QC Date

November 29, 2019

Last Update Submit

November 21, 2021

Conditions

Aceruloplasminemia

Keywords

AceruloplasminemiaDeferipronePancreas MRI

Outcome Measures

Primary Outcomes (2)

  • 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

Study Arms (1)

Deferiprone treated

EXPERIMENTAL

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

Drug: Deferiprone treated

Interventions

Deferiprone treatedDRUG

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

Also known as: Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients
Deferiprone treated

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age 18-80 years
  • Diagnosed with aceruloplasminaemia
  • Genetically confirmed diagnosis of aceruloplasminaemia

You may not qualify if:

  • Contraindications to deferiprone therapy
  • Pregnancy was excluded in women of childbearing age

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Department of Neurology , First Affiliated Hospital Fujian Medical University

Fuzhou, Fujian, 350004, China

RECRUITING

MeSH Terms

Conditions

Familial apoceruloplasmin deficiency

Study Officials

  • Wanjin Chen

    Department of Neurology , First Affiliated Hospital Fujian Medical University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Jin He

CONTACT

13645050186hejin@fjmu.edu.cn

Wanjin Chen

CONTACT

13860601359wanjinchen75@fjmu.edu.cn

Study Design

Study Type
interventional
Phase
early phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: Patients who was diagnosed as aceruloplasminiaemia
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

November 29, 2019

First Posted

December 3, 2019

Study Start

December 23, 2019

Primary Completion

October 23, 2022

Study Completion

November 23, 2022

Last Updated

November 23, 2021

Record last verified: 2021-11

Locations

CN(1)