AceruloplasminemiaEarly Phase 1NCT04184453
Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients
A Early Phase 1 study testing Deferiprone treated in people with Aceruloplasminemia, run by First Affiliated Hospital of Fujian Medical University at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Aceruloplasminemia
- What is being tested?
- Deferiprone treated
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteChina
- Study dates
- 23 Dec 2019to 23 Nov 2022
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18-80 years
- Diagnosed with aceruloplasminaemia
- Genetically confirmed diagnosis of aceruloplasminaemia
Who cannot join
- Contraindications to deferiprone therapy
- Pregnancy was excluded in women of childbearing age
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Neurology , First Affiliated Hospital Fujian Medical University
Fuzhou, Fujian, 350004
HeJin, 13645050186
About this study
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.
Read the 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.
- Conditions
- Aceruloplasminemia
- Being tested
- Deferiprone treated
- Keywords
- Aceruloplasminemia, Deferiprone, Pancreas MRI
- Medical subject headings
- Familial apoceruloplasmin deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferiprone treatedDrug
Also called Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients
Deferiprone (25 mg/kg/day) was given to the enrolled patients.
| Study group | Role | What they receive |
|---|---|---|
| Deferiprone treated | Experimental | Deferiprone (25 mg/kg/day) was given to the enrolled patient. Receives: Drug: Deferiprone treated |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of pancreas MRI
Pancreas MRI is related to iron accumulation of pancreas
Measured: Up to 3 months
Change of glycated hemoglobin (HbA1c)
HbA1c is a measure of long-term blood sugar control
Measured: Up to 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2019
- Study start23 Dec 2019
- Primary completion23 Oct 2022 (estimated)
- Study completion23 Nov 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AceruloplasminemiaStudies of the same condition, open and under way.
- More trials run by First Affiliated Hospital of Fujian Medical UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Patients who was diagnosed as aceruloplasminiaemia
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University
- Investigators
- Wanjin Chen, Principal investigator, Department of Neurology , First Affiliated Hospital Fujian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04184453
- Sponsor's protocol ID
- MRCTA,ECFAH of FMU [2019]252
- Status last verified
- Nov 2021
- First submitted
- 29 Nov 2019
- Last updated
- 23 Nov 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients
- ClinicalTrials.gov ID
- NCT04184453
- Acronym / sponsor ID
- MRCTA,ECFAH of FMU [2019]252
- Conditions
- Aceruloplasminemia
- Keywords
- Aceruloplasminemia, Deferiprone, Pancreas MRI
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- First Affiliated Hospital of Fujian Medical UniversityUniversity, hospital or other
- Study start
- 23 Dec 2019 (actual)
- Primary completion
- 23 Oct 2022 (estimated)
- Study completion
- 23 Nov 2022 (estimated)
- First posted
- 3 Dec 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Patients who was diagnosed as aceruloplasminiaemia
| Arm | Type | Receives |
|---|---|---|
| Deferiprone treated | Experimental | Drug: Deferiprone treatedDeferiprone (25 mg/kg/day) was given to the enrolled patient. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Age 18-80 years
- Diagnosed with aceruloplasminaemia
- Genetically confirmed diagnosis of aceruloplasminaemia
Key exclusion criteria (2 listed)
- Contraindications to deferiprone therapy
- Pregnancy was excluded in women of childbearing age
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Deferiprone treatedAlso: Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia PatientsDeferiprone (25 mg/kg/day) was given to the enrolled patients. | Drug | Deferiprone treated |
Primary outcome
Change of pancreas MRI
Time frame: Up to 3 months
Pancreas MRI is related to iron accumulation of pancreas
Change of glycated hemoglobin (HbA1c)
Time frame: Up to 3 months
HbA1c is a measure of long-term blood sugar control
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2021.
Related trials for Aceruloplasminemia
RecruitingObservational study
TIRCON International NBIA Registry
Neurodegeneration With Brain Iron Accumulation (NBIA), Pantothenate Kinase-associated Neurodegeneration (PKAN)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wanjin Chen principal investigator
Department of Neurology , First Affiliated Hospital Fujian Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Deferiprone treated in people with Aceruloplasminemia, run by First Affiliated Hospital of Fujian Medical University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jin He (13645050186, hejin@fjmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of pancreas MRI (time frame: Up to 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Nov 2021.