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NCT00907283

Ferrochelating Treatment in Patients Affected by "Neurodegeneration With Brain Iron Accumulation" (NBIA)

Status unknown Phase 2 Last updated 7 February 2023
What this trial tests

Phase 2 trial testing Deferiprone in Neurodegenerative Disease in 20 participants. Status unknown.

Timeline
1 November 2008
Primary endpoint
1 December 2024
1 December 2024

Quick facts

Lead sponsorEnte Ospedaliero Ospedali Galliera
PhasePhase 2
StatusStatus unknown
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment20
Start date1 November 2008
Primary completion1 December 2024
Estimated completion1 December 2024
Sites3 locations across Italy

Drugs / interventions tested

Conditions studied

Sponsor

Ente Ospedaliero Ospedali Galliera — full company profile →

Who can join

Adults 1 to 80, any sex, with Neurodegenerative Disease or Iron Overload. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

Sponsor's own description

This trial is a multicenter, unblinded, single-arm pilot study, lasting one year (plus one year extension Amendment n.3 25 August 2009, plus two years follow-up Amendment n.7) , to evaluate the efficacy and safety of the chelator therapy with deferiprone on cerebral iron accumulations. The drug will be administered in the dosage of 15 mg/kg twice daily. The safety and tolerability of the drug will be evaluated by measuring hemochrome every seven days with leukocyte formula count. At 3, 6 and 12 months from the start of treatment, a neurological evaluation will be performed using several specific evaluation scales (International Cooperative Ataxia Rating Scale (ICARS); Unified Parkinson's Disease Rating Scale (UPDRS); Burke-Fahn-Marsden (BFM)). Every 6 months of treatment, a brain magnetic resonance image (MRI) aimed at measuring iron overload quantitatively, if possible.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Targeting ferroptosis opens new avenues for the development of novel therapeutics.
    Sun S, Shen J, Jiang J, Wang F, et al · · 2023 · cited 380× · PMID 37735472 · DOI 10.1038/s41392-023-01606-1
  2. Advances in non-dopaminergic treatments for Parkinson's disease.
    Stayte S, Vissel B. · · 2014 · cited 42× · PMID 24904259 · DOI 10.3389/fnins.2014.00113
  3. Ferroptosis: mechanisms and therapeutic targets.
    Zhou Q, Meng Y, Le J, Sun Y, et al · · 2024 · cited 32× · PMID 39568772 · DOI 10.1002/mco2.70010
  4. Towards Precision Therapies for Inherited Disorders of Neurodegeneration with Brain Iron Accumulation.
    Spaull RVV, Soo AKS, Hogarth P, Hayflick SJ, et al · · 2021 · cited 14× · PMID 34909266 · DOI 10.5334/tohm.661
  5. Long-Term Neuroradiological and Clinical Evaluation of NBIA Patients Treated with a Deferiprone Based Iron-Chelation Therapy.
    Romano N, Baiardi G, Pinto VM, Quintino S, et al · · 2022 · cited 11× · PMID 35956138 · DOI 10.3390/jcm11154524
  6. Eye-of-the-tiger Sign in Neurodegeneration with Brain Iron Accumulation.
    Goldberg M, Malik K, Jiswant R, Khaku AS. · · 2019 · cited 3× · PMID 31431841 · DOI 10.7759/cureus.4936
  7. When Pathways Converge: Iron, Lipid Peroxidation, and α-Synuclein in Ferroptosis-Driven Dopaminergic Neurodegeneration.
    Sperlich CL, Stockwell BR, Farina M. · · 2026 · PMID 41968973 · DOI 10.1111/jnc.70438

Verify or expand the search:

Other trials of Deferiprone

Trials testing the same drug.

Other recruiting trials for Neurodegenerative Disease

Currently open trials in the same condition.

Other Ente Ospedaliero Ospedali Galliera trials

Trials by the same sponsor.

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Data sources for this page

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