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| ID | Type | Description | Link |
|---|---|---|---|
| 2015-003679-31 | EudraCT Number | ||
| Grant agreement No 633190 | Other Grant/Funding Number | European Union's Horizon 2020 | |
| HP751 | Other Identifier | CTFG VHP |
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| Name | Class |
|---|---|
| European Commission | OTHER |
| ApoPharma | INDUSTRY |
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This study evaluates the effect of iron chelation as a therapeutic strategy to slow the progression of Parkinson's disease. Half of participants will receive the deferiprone to 15 mg / kg twice daily morning and evening (30mg / kg per day), while the other half will receive a placebo. The treatment lasts nine months.
This is the new concept of "conservative iron chelation". We recently demonstrated (for the first time) the feasibility, efficacy and acceptability of the conservative iron chelation approach in pilot translational studies in Parkinson's disease with a prototype drug: deferiprone (1,2-dimethyl-3-hydroxypyridin-4-one) (in the FAIR-PARK-I project led by the applicant and funded by French Ministry of Health). The only available blood-brain-barrier-permeable iron chelator deferiprone is approved for treating systemic iron overload in transfused patients with thalassemia. Deferiprone has been on the European Union market since 1999, with a favourable risk/benefit balance at dose of 75 to 100 mg/kg/day. The investigators shall adopt a repositioning strategy by using deferiprone at a lower dose of 30 mg/kg/day in this new indication for local iron overload in Parkinson's disease. Deferiprone will be the first-in-class drug for this novel therapeutic strategy. On the basis of the preclinical and clinical data from (FAIR-PARK-I), the present (FAIR-PARK-II) project should constitute a model for future cytoprotection strategies in neurodegenerative diseases; if deferiprone treatment is associated with significant slower disease progression, it would be the first non-dopaminergic drug to have a proven disease-modifying effect in Parkinson's disease.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DEFERIPRONE | Active Comparator | Half of participants will receive the deferiprone (DFP) to 15 mg / kg twice daily morning and evening (30mg / kg per day).The treatment lasts nine months. |
|
| PLACEBO | Placebo Comparator | Half of participants will receive the placebo twice daily morning and evening. The treatment lasts nine months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deferiprone | Drug | 15 mg / kg twice daily morning and evening (30mg / kg per day).The treatment lasts nine months. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Global effect (symptomatic and disease modifying effects) on motor and non motor handicap | the change in the total Movement Disorders Society-Unified Parkinson Disease Rating Scale score between baseline and 36 weeks (i.e. the end of the placebo-controlled phase for analysis of both disease-modifying and symptomatic effects) | at 36 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Disease-modifying effect on motor and non motor handicap | It will be measured as the changes in the overall Movement Disorders Society-Unified Parkinson Disease Rating Scale score between baseline and week 40 (i.e. the end of the one-month post-treatment monitoring period), to analyse the disease-modifying effect without bias from the symptomatic effect of ongoing deferiprone treatment) on the study population |
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Inclusion Criteria:
Exclusion Criteria:
Exclusion criteria for the biomarker study and the ancillary study (i) Magnetic Resonance Imaging:
(ii) Lumbar puncture:
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| Name | Affiliation | Role |
|---|---|---|
| David Devos, MD, PhD | University Hospital, Lille | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Medizinische Universitat Innsbruck | Innsbruck | Austria | ||||
| Charles University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36449420 | Result | Devos D, Labreuche J, Rascol O, Corvol JC, Duhamel A, Guyon Delannoy P, Poewe W, Compta Y, Pavese N, Ruzicka E, Dusek P, Post B, Bloem BR, Berg D, Maetzler W, Otto M, Habert MO, Lehericy S, Ferreira J, Dodel R, Tranchant C, Eusebio A, Thobois S, Marques AR, Meissner WG, Ory-Magne F, Walter U, de Bie RMA, Gago M, Vilas D, Kulisevsky J, Januario C, Coelho MVS, Behnke S, Worth P, Seppi K, Ouk T, Potey C, Leclercq C, Viard R, Kuchcinski G, Lopes R, Pruvo JP, Pigny P, Garcon G, Simonin O, Carpentier J, Rolland AS, Nyholm D, Scherfler C, Mangin JF, Chupin M, Bordet R, Dexter DT, Fradette C, Spino M, Tricta F, Ayton S, Bush AI, Devedjian JC, Duce JA, Cabantchik I, Defebvre L, Deplanque D, Moreau C; FAIRPARK-II Study Group. Trial of Deferiprone in Parkinson's Disease. N Engl J Med. 2022 Dec 1;387(22):2045-2055. doi: 10.1056/NEJMoa2209254. | |
| 39952690 |
| Label | URL |
|---|---|
| Website for informations on the Fairpark2 study | View source |
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| Placebo | Drug | the placebo twice daily morning and evening. The treatment lasts nine months |
|
|
| baseline, at 40 weeks |
| Effect of the motor symptoms | The effect of the motor symptoms will be analysed as the change in the subscale part III of the Movement Disorders Society-Unified Parkinson Disease Rating Scale score | baseline, at 12, 36 and 40 weeks |
| Quality of life and autonomy by PDQ-39 score | It will be analyzed as the change in the Parkinson's Disease Quality of Life (PDQ-39, via a 39-item self-questionnaire) | baseline, at 36 and 40 weeks |
| Quality of life and autonomy by Clinical Global Impression score | the Clinical Global Impression scored by the examiner and the patient | baseline, at 36 and 40 weeks |
| Health economics assessment | will be performed via a specific questionnaire provides a simple descriptive profile and a single index value for health status | baseline, at 36 and 40 weeks |
| EQ-5D questionnaire | the questionnaire provides a simple descriptive profile and a single index value for health status. | baseline, at 36 and 40 weeks |
| Safety criteria | All the safety concerns will be listed in a table with the number of patients, the type the severity and the time of occurrence for
| 40 weeks |
| Effect on overall cognitive status | Measured by the score in the Montreal Cognitive Assessment | baseline, at 12, 36 and 40 weeks |
| Effect on gait disorders | Measured by the Stand Walk Sit test | baseline, at 12, 36 and 40 weeks |
| Effect on daily living | The effect on daily living will be analysed as the change in the subscale part II (activities of daily living) of the Movement Disorders Society-Unified Parkinson Disease Rating Scale score | baseline, at 12, 36 and 40 weeks |
| Effect on non-motor symptoms | The effect on non motor symptoms will be analysed as the change in the subscale part I (cognition and behavior) of the Movement Disorders Society-Unified Parkinson Disease Rating Scale score | baseline, at 12, 36 and 40 weeks |
| Lack of occurrence of motor fluctuations | The lack of occurrence of motor fluctuations will be analysed on the subscale part IV of the Movement Disorders Society-Unified Parkinson Disease Rating Scale score | baseline, at 12, 36 and 40 weeks |
| Prague |
| Czechia |
| Univerzita Karlova V Praze | Prague | Czechia |
| CHU Pellegrin | Bordeaux | France |
| Hôpital Wertheimer | Bron | France |
| Hôpital Montpied | Clermont-Ferrand | France |
| Hôpital Salengro, CHRU | Lille | France |
| CHU la TIMONE | Marseille | France |
| AP-HP, Hôpital Pitié-Salpêtrière | Paris | France |
| CHU de Strasbourg, Hôpital de Hautepierre | Strasbourg | France |
| Chu Purpan | Toulouse | France |
| University Hospital, Saarland University | Homburg | Germany |
| Christian-albrechts universität zu kiel | Kiel | Germany |
| Klinik und Poliklinik für Neurologie der Universitätsmedizin Rostock | Rostock | Germany |
| Acadamic central center, Amsterdam | Amsterdam | Netherlands |
| Radboud university medical center | Nijmegen | Netherlands |
| Centro Hospitalar e universitario de Coimbra | Coimbra | Portugal |
| Centro Hospitalar do Alto Ave | Guimarães | Portugal |
| Centro Hospitalar Lisboa Norte | Lisbon | Portugal |
| Hospital Clinic Universitari de Barcelona | Barcelona | Spain |
| Hospital de Bellvitge | Barcelona | Spain |
| Hospital de la Santa Creu i Sant Pau | Barcelona | Spain |
| Cambridge University Hospital | Cambridge | United Kingdom |
| University of Glasgow | Glasgow | United Kingdom |
| Newcastle University | Newcastle | United Kingdom |
| Derived |
| Streit WJ, Phan L, Bechmann I. Ferroptosis and pathogenesis of neuritic plaques in Alzheimer disease. Pharmacol Rev. 2025 Jan;77(1):100005. doi: 10.1124/pharmrev.123.000823. Epub 2024 Nov 22. |
| 32726602 | Derived | Mahoney-Sanchez L, Bouchaoui H, Ayton S, Devos D, Duce JA, Devedjian JC. Ferroptosis and its potential role in the physiopathology of Parkinson's Disease. Prog Neurobiol. 2021 Jan;196:101890. doi: 10.1016/j.pneurobio.2020.101890. Epub 2020 Jul 26. |
| 29380903 | Derived | Moreau C, Duce JA, Rascol O, Devedjian JC, Berg D, Dexter D, Cabantchik ZI, Bush AI, Devos D; FAIRPARK-II study group. Iron as a therapeutic target for Parkinson's disease. Mov Disord. 2018 Apr;33(4):568-574. doi: 10.1002/mds.27275. Epub 2018 Jan 30. No abstract available. |
| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |
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| ID | Term |
|---|---|
| D000077543 | Deferiprone |
| D000095485 | Bulk Drugs |
| ID | Term |
|---|---|
| D011728 | Pyridones |
| D011725 | Pyridines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D004364 | Pharmaceutical Preparations |
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