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

Fujian Medical University (FJMU)

Status and phase: Unknown; Early Phase 1

Conditions: Aceruloplasminemia

Treatments: Drug: Deferiprone treated

Study type: Interventional

Funder types: Other

Identifiers

NCT04184453

MRCTA,ECFAH of FMU [2019]252

Details and patient eligibility

About

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.

Full 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.

Enrollment: 5 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

5 participants in 1 patient group

Deferiprone treated

Experimental group

Description:

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

Treatment:

Drug: Deferiprone treated