Iron Overload Clinical Trial
Official title:
Ferrochelating Treatment in Patients Affected by "Neurodegeneration With Brain Iron Accumulation" (NBIA)
Verified date | February 2023 |
Source | Ente Ospedaliero Ospedali Galliera |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Active, not recruiting |
Enrollment | 20 |
Est. completion date | December 2024 |
Est. primary completion date | December 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year to 80 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Italy | Neurological Pathology Department, Brotzu Hospital | Cagliari | |
Italy | Centre of Microcitemia and Congenital Anemias, Galliera Hospital | Genoa | |
Italy | Clinic of Neurology, University of Genoa | Genoa |
Lead Sponsor | Collaborator |
---|---|
Ente Ospedaliero Ospedali Galliera |
Italy,
Abbruzzese G, Cossu G, Balocco M, Marchese R, Murgia D, Melis M, Galanello R, Barella S, Matta G, Ruffinengo U, Bonuccelli U, Forni GL. A pilot trial of deferiprone for neurodegeneration with brain iron accumulation. Haematologica. 2011 Nov;96(11):1708-11 — View Citation
Forni GL, Balocco M, Cremonesi L, Abbruzzese G, Parodi RC, Marchese R. Regression of symptoms after selective iron chelation therapy in a case of neurodegeneration with brain iron accumulation. Mov Disord. 2008 Apr 30;23(6):904-7. doi: 10.1002/mds.22002. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | To evaluate the efficacy and safety of the chelator therapy with deferiprone on cerebral iron accumulations. | Safety:CBC including ANC will be monitored weekly.If the liver enzymes are greater than 2.5 fold the upper limit of normal, the drug will be withheld and the assessment repeated in 1 week. If the laboratory values continue to be over 2.5 times the upper limit of normal or if the neutrophil counts decrease to less than 1.5 x 109/L (1500 cells/µl) the Patient will be withdrawn from the study. Neutropenia/Agranulocytosis is confirmed as an Absolute Neutrophil Count being less than 1.5 x 109/L (1500 cells/µl) if counts on two consecutive days are both less than 1.5 x 109 (1500 cells/µl). | 6 months + 6 months (plus one year extension) |
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