Deferiprone to Delay Dementia (The 3D Study)
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|ClinicalTrials.gov Identifier: NCT03234686|
Recruitment Status : Recruiting
First Posted : July 31, 2017
Last Update Posted : January 24, 2018
|Condition or disease||Intervention/treatment||Phase|
|Mild Cognitive Impairment Prodromal Alzheimer's Disease Mild Alzheimer's Disease||Drug: Deferiprone 600mg delayed release tablets Drug: Placebo Oral Tablet||Phase 2|
This Phase II study is designed as a randomised, double-blinded, placebo controlled, multi-centre study for subjects with evidence of amyloid positive pAD or mAD.
Participants will be assigned randomly to two groups (Group 1 Deferiprone (15mg/kg BID orally), Group 2: Placebo). Participants will have a 2 in 3 chance to be placed in the Deferiprone group.
The study will enrol approximately 171 participants over 4 sites in Australia. The overall duration for patients will be 54 weeks. This includes a 55-day screening period, and visits on Day 1, weeks 13, 26, 38,52, and a two-week follow-up visit.
Participants will be screened for the study after signing the approved informed consent form. As part of the 55-day screening phase, subjects will undertake an extensive medical and neurological assessments as well as a PET scan.
At the baseline visit, following the screening phase, blood and urine will be taken for safety monitoring and for measuring APOE-4 gene status. Baseline signs and symptoms will be collected. An MRI will be performed All patients will start with study medication at the Baseline visit.
Participants will return to the centre on Weeks 13, 26, 38, 52 (or early termination) to undertake a neurological examination as well as an assessment of blood samples taken at the visit.
Participants must also attend weekly blood tests.
SAE's, AE's and changes to concomitant medications will be observed and evaluated throughout the study. Each study visit will have a 7-day window after the due date to account for scheduling conflicts/holidays/weekends.
Participants will be given additional study product to account for the 7-day window.
Participants must attend the weekly pathology visits with a 3-day window of the scheduled date or risk termination from the trial.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||171 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Care Provider)|
|Official Title:||Deferiprone to Delay Dementia (The 3D Study): a Clinical Proof of Concept Study|
|Actual Study Start Date :||January 19, 2018|
|Estimated Primary Completion Date :||July 2021|
|Estimated Study Completion Date :||December 2021|
Patients will orally receive the equivalent 15 mg/kg of 600mg delayed release Deferiprone tablets twice daily.
Drug: 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.
Placebo Comparator: Placebo
Patients will receive matching placebo tablets designed to mimic the experimental treatment, twice daily
Drug: Placebo Oral Tablet
The placebo will mimic the Deferiprone arm in every way, except the placebo will not include the active ingredient
- Efficacy of Deferiprone [ Time Frame: 12 months ]Comparison of the efficacy of Deferiprone (15 mg/kg) administered orally twice a day with a matching placebo in subjects with pAD (MCI with brain amyloid pathology) or mAD at 12 months relative to baseline. This will be measured by a series of paper and electronic assessments called the NTB
- Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) [ Time Frame: 12 months ]Safety and tolerability will be assessed by the incidence and severity of AEs and changes from baseline of all relevant parameters, including clinical laboratory values, vital signs, ECG and other safety biomarkers. Severity of AEs will be assessed according to the National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE), Version 4.03. All subjects will be monitored for AEs until resolution.
- Brain Iron Levels [ Time Frame: 12 months ]Using MRI to compare iron levels in various brain regions of the Deferiprone and placebo treatment groups at baseline and 12 months.
- The effect of Deferiprone on the episodic memory, executive function and attention composites [ Time Frame: 12 months ]Measured by scores on the NTB at 12 months relative to baseline
- The Association with Iron levels in the Brain and Cognitive Decline [ Time Frame: 12 months ]Using MRI to evaluate if cognitive performance is associated with a change in iron levels over a 12-month period relative to baseline.
- The Potential for Brain Iron Load to be Used to Stratify Responsiveness to Deferiprone [ Time Frame: 12 months ]Measured by baseline iron MRI and change in cognitive ability from baseline at 12 months.
- The Potential for APOE Genotype to be Used to Stratify Responsiveness to Deferiprone [ Time Frame: 12 months ]Measured by APOE genotype and changes in cognitive ability from baseline at 12 months
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03234686
|Contact: Tina Soulis||(03) 9035 email@example.com|
|Box Hill Hospital||Not yet recruiting|
|Box Hill, Victoria, Australia, 3128|
|Principal Investigator: A/Prof Amy Brodtmann|
|Heidelberg, Victoria, Australia, 3084|
|Principal Investigator: A/Prof. Michael Woodward|
|Alfred Hospital||Not yet recruiting|
|Melbourne, Victoria, Australia, 3004|
|Principal Investigator: Prof. Terry O'Brien|
|Royal Melbourne Hospital||Recruiting|
|Parkville, Victoria, Australia, 3050|
|Principal Investigator: Prof. Terence O'Brien|