patients with NBIA MedDRA version: 9.1 Level: SOC Classification code 10029205 Term: Nervous system disorders
Conditions
Interventions
Trade Name: FERRIPROX
Pharmaceutical Form: Oral solution
INN or Proposed INN: Deferiprone
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Sponsors
AZIENDA OSPEDALIERA OSPEDALI GALLIERA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients aged ≥ 18 years affected from a neurological de generation with brain iron accumulation documented by RMI and who do not present sistemyc iron overload escluded by RMI and/or SQUID, MID, Needle Liver Biopsy. Patients who signed and witnessed written informed consent. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients unable to undergo MRI. Patients with serum creatinine > 1.5 mg/dl. History of malignancy of any organ system. Patients with cardiovascolar, renal and liver deseases that do not allow the administration of the study drug. Hypersensitivity to deferiprone Patients unwilling or unable to comply with the protocol. Pregnant or breastfeeding women are not elegible to partecipate in the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of deferiprone in patients with NBIA;Secondary Objective: To evaluate the tolerability of deferiprone;Primary end point(s): To evaluate the efficacy of deferiprone in patients with NBIA | — |
Countries
Italy
Outcome results
None listed