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

NCT00907283 | Unknown Phase 2

• 2 other identifiers: Deferiprone08EUDRACT NUMBER 2008-005206-39
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Neurodegenerative Disease; Iron Overload

Keywords

NBIA; Iron overload AND brain; Iron chelation AND neurodegeneration; Brain iron Overload; Neurodegeneration; Brain iron Accumulation

Outcome Measures

Primary Outcomes (1)

• 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upper limit of normal or if the neutrophil counts decrease to less than 1.5 x 109/L (1500 cells/µl) the Patient will be withdrawn from the study.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)

Study Arms (1)

deferiprone

EXPERIMENTAL

15 mg/Kg/twice for 1 year

Drug: Deferiprone

Interventions

Deferiprone DRUG

15 mg/Kg/twice for 1 year

Also known as: EU/1/99/108/002 Ferriprox 100 mg/ml Oral solution, EU/1/99/108/003 Ferriprox 100 mg/ml Oral solution

deferiprone

Eligibility Criteria

• Age: 1 Year - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients who have given informed consent.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Ente Ospedaliero Ospedali Galliera: lead

Study Sites (3)

Neurological Pathology Department, Brotzu Hospital

Cagliari, 09134, Italy

Location

Centre of Microcitemia and Congenital Anemias, Galliera Hospital

Genoa, 16128, Italy

Location

Clinic of Neurology, University of Genoa

Genoa, 16132, Italy

Location

Related Publications (2)

• 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.

  PMID: 18383118 BACKGROUND

• 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. doi: 10.3324/haematol.2011.043018. Epub 2011 Jul 26.

  PMID: 21791473 RESULT

MeSH Terms

Conditions

Neurodegenerative Diseases; Iron Overload; Nerve Degeneration

Interventions

Deferiprone; Solutions

Condition Hierarchy (Ancestors)

Nervous System Diseases; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pharmaceutical Preparations

Study Officials

• Gian Luca Forni, MD

  E.O. Ospedali Galliera. Centro della Microcitemia e delle Anemie Congenite -Ematology - Genoa Italy

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: May 21, 2009
• First Posted: May 22, 2009
• Study Start: November 1, 2008
• Primary Completion: December 1, 2024
• Study Completion: December 1, 2024
• Last Updated: February 9, 2023

Record last verified: 2023-02

Locations

IT (3)