Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03234686




Registration number
NCT03234686
Ethics application status
Date submitted
23/07/2017
Date registered
31/07/2017
Date last updated
3/07/2024

Titles & IDs
Public title
Deferiprone to Delay Dementia (The 3D Study)
Scientific title
Deferiprone to Delay Dementia (The 3D Study): a Clinical Proof of Concept Study
Secondary ID [1] 0 0
DEF001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mild Cognitive Impairment 0 0
Prodromal Alzheimer's Disease 0 0
Mild Alzheimer's Disease 0 0
Condition category
Condition code
Neurological 0 0 0 0
Alzheimer's disease
Neurological 0 0 0 0
Dementias
Mental Health 0 0 0 0
Other mental health disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Deferiprone 600mg delayed release tablets
Treatment: Drugs - Placebo Oral Tablet

Experimental: Deferiprone - Patients will orally receive the equivalent 15 mg/kg of 600mg delayed release Deferiprone tablets twice daily.

Placebo comparator: Placebo - Patients will receive matching placebo tablets designed to mimic the experimental treatment, twice daily


Treatment: Drugs: Deferiprone 600mg delayed release tablets
The active substance, Deferiprone, is a member of the 3-hydroxypyrid-4-one class of iron chelators, which have a high affinity for ferric iron, binding it in a 3:1 (Deferiprone:iron) molar ratio.

Treatment: Drugs: Placebo Oral Tablet
The placebo will mimic the Deferiprone arm in every way, except the placebo will not include the active ingredient
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Efficacy of Deferiprone
Timepoint [1] 0 0
12 months
Secondary outcome [1] 0 0
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Timepoint [1] 0 0
12 months
Secondary outcome [2] 0 0
Brain Iron Levels
Timepoint [2] 0 0
12 months

Eligibility
Key inclusion criteria
1. Able to provide written informed consent in accordance with federal, local and institutional guidelines. For subjects unable to provide written consent, consent will be provided by the Person Responsible per local regulations.
2. Age =65 years, or =55 years if they have been diagnosed by a psychiatrist or neurologist with dementia, or if they have a validated previous positive amyloid PET scan.
3. Weight between 40 and 120 kg
4. Have an available caregiver
5. Have = 6 years of education (any) and able to follow testing instructions.
6. Have visual and auditory acuity sufficient to perform neuropsychological testing.
7. Have prior evidence of AD pathology, by a positive amyloid assessment, or amyloid PET scan.
8. Demonstrate abnormal memory function in the last 6 months or at screening: International Shopping List Test (ISLT) >1.5 SD below the age adjusted mean
9. Subjective or clinical history of retrospective cognitive decline =6 months
10. Evidence of mild symptomatology, as defined by a screening MMSE score of = 20 points
11. Meet National Institute on Ageing/Alzheimer's Association Diagnostic Guidelines for Alzheimer's Disease (NIAAA) research criteria for mAD or pAD
12. If receiving medication for symptomatic AD, have a stable dosing regimen for 3 months prior to screening.
13. Females of Child Bearing Potential (FCBP) must have confirmed negative serum pregnancy test within the 21 days prior to randomization.
14. FCBP and male subjects who are sexually active with FCBP must agree to use highly effective contraception during the study and until 90 days after the last dose of treatment (for sexually active male participants whose partners are FCBP) or until 30 days after the last dose of treatment (for women of childbearing potential participants).
Minimum age
65 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Clinically significant haematological disorder, including moderate or severe anaemia (blood haemoglobin <110 g/L, WHO definition)
2. Iron deficiency (serum ferritin < 10 ng/mL)
3. Clinically significant abnormal haematological results (sufficiently outside the normal range to warrant further investigation). Mild anaemia (haemoglobin =110 g/L) is not an exclusion.
4. Clinically significant abnormal renal or liver function results (sufficiently outside the normal range to warrant further investigation)
5. Presence of non-AD condition that may affect cognition, such as but not limited to Parkinson's Disease (PD), normal pressure hydrocephalus, sleep apnoea requiring O2 treatment
6. Clinically evident vascular disease that could potentially affect the brain, such as but not limited to significant carotid or vertebral stenosis, aortic aneurysm, cerebral haemorrhage
7. History of any stroke in the past 2 years, or transient ischemic attack within the last 6 months
8. History of persistent neurologic deficit, intracranial tumour or structural brain damage
9. History of infection that could affect brain function (eg HIV and syphilis)
10. Autoimmune disorders that potentially cause progressive neurologic disease with associated cognitive deficits, such as but not limited to multiple sclerosis, lupus
11. Major psychiatric illness (depression is acceptable if patient has not had an episode within the past year or is considered in remission or controlled by treatment)
12. A history of relapsing neutropenia.
13. Presence of agranulocytosis or with a history of agranulocytosis
14. Known hypersensitivity to DFP or excipients.
15. Alcohol and/or substance abuse
16. MRI evidence of clinically-significant cerebrovascular pathology. Focal white matter lesions, = 2 lacunar infarcts in non-critical sites and other minor pathology assessed by the investigator to not be causing the current cognitive impairment, will not lead to exclusion.
17. Active major medical illness
18. FCBP not using adequate method of contraception or who is pregnant or nursing
19. Inability to provide informed consent
20. Participation in another clinical trial within 3 months prior to inclusion in the study
21. Subjects for whom MRI is contraindicated (severe claustrophobia, pacemaker, incompatible surgical material, unmovable electronic pump implant)
22. Negative amyloid PET scan or CSF in the last 2 years.
23. Hospital Anxiety and Depression Scale (scores > 8/21 are disqualified).
24. Subject cannot commit to regular blood tests with the interval between tests not exceeding 10 days from the scheduled visit for the duration of the study.
25. Subject has planned surgery which does not permit regular blood tests with the interval between tests not exceeding 10 days from the scheduled visit.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/01/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/02/2023
Sample size
Target
Accrual to date
Final
81
Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
Recruitment hospital [1] 0 0
KaRa Institute of Neurological Diseases - Macquarie Park
Recruitment hospital [2] 0 0
Hunter New England Local Health District - Waratah
Recruitment hospital [3] 0 0
Box Hill Hospital - Box Hill
Recruitment hospital [4] 0 0
Austin Health - Heidelberg
Recruitment hospital [5] 0 0
Alfred Hospital - Melbourne
Recruitment hospital [6] 0 0
NeuroCentrix - Noble Park
Recruitment hospital [7] 0 0
Royal Melbourne Hospital - Parkville
Recruitment hospital [8] 0 0
Australian Alzheimer's Research Foundation - Nedlands
Recruitment postcode(s) [1] 0 0
2113 - Macquarie Park
Recruitment postcode(s) [2] 0 0
2298 - Waratah
Recruitment postcode(s) [3] 0 0
3128 - Box Hill
Recruitment postcode(s) [4] 0 0
3084 - Heidelberg
Recruitment postcode(s) [5] 0 0
3004 - Melbourne
Recruitment postcode(s) [6] 0 0
3174 - Noble Park
Recruitment postcode(s) [7] 0 0
3050 - Parkville
Recruitment postcode(s) [8] 0 0
6009 - Nedlands

Funding & Sponsors
Primary sponsor type
Other
Name
Neuroscience Trials Australia
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
The Florey Institute of Neuroscience and Mental Health
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ashley I. Bush
Address 0 0
The Florey Institute of Neuroscience and Mental Health
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT03234686