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

• Status: Active, not recruiting
• Trial end date: 2020-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ente Ospedaliero Ospedali Galliera
• Treatments: Deferiprone; Iron; Pharmaceutical Solutions

Criteria

Inclusion Criteria:

• Patients over > 1 years of age who have neurological symptoms that can be correlated with cerebral-level iron overload as documented with MRI.This inclusion criteria has been amended by Amendment 6, 24 march 2011)

• Patients who have given informed consent.

Exclusion criteria:

• Inability to be subjected to MRI exam.

• Renal insufficiency (creatinine > 1.5 mg/dl).

• Neoplasias.

• Patients with average levels of ALT > 300 and patients with variations of ALT or AST of 300% during the year prior to enrolling. (At least 4 measurements in 12 months).

• Systemic cardiovascular, renal, hepatic etc., diseases that could counter-indicate the therapeutic options specified.

• Known hypersensitivity to deferiprone.

• Patient judged potentially unreliable and/or uncooperative with regard to study procedures.

• Pregnancy and breastfeeding.