NCT02655315

Conservative Iron Chelation as a Disease-modifying Strategy in Parkinson's Disease

Conservative Iron Chelation as a Disease-modifying Strategy in Parkinson's Disease. European Multicentre, Parallel-group, Placebo-controlled, Randomized Clinical Trial of Deferiprone"

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 372 (actual)
• Sponsor: University Hospital, Lille · Academic / Other
• Sex: All
• Age: 80 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Parkinson Disease

Interventions

Type	Name	Description
DRUG	Deferiprone	15 mg / kg twice daily morning and evening (30mg / kg per day).The treatment lasts nine months.
DRUG	Placebo	the placebo twice daily morning and evening. The treatment lasts nine months

Timeline

• Start date: 2016-02-09
• Primary completion: 2020-09-22
• Completion: 2020-09-22
• First posted: 2016-01-14
• Last updated: 2026-02-05

Locations

25 sites across 8 countries: Austria, Czechia, France, Germany, Netherlands, Portugal, Spain, United Kingdom