Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)

• Status: Completed
• Trial end date: 2017-01-11
• Target enrollment: 0
• Participant gender: All

Summary

A multi-center, placebo controlled, double-blind trial comparing the efficacy and safety of 18 months of treatment with deferiprone versus placebo in patients with PKAN.

This investigator-initiated trial was funded by the European Commission's Seventh Framework Programme (FP7/2007-2013, HEALTH-F2-2011, grant agreement No. 277984) to the TIRCON consortium (Treat Iron-Related Childhood-Onset Neurodegeneration) and by the FDA Office of Orphan Products Development (OOPD) (Dr. Elliott Vichinsky).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: ApoPharma
• Collaborator: Food and Drug Administration (FDA)
• Treatments: Deferiprone; Pharmaceutical Solutions

Criteria

Main Inclusion Criteria:

• Males or females 4 years of age and older at screening visit;

• Have PKAN, confirmed by genetic testing (supporting evidence required);

• Barry-Albright Dystonia (BAD) total score ≥ 3 at the screening visit;

• Patients who have Deep Brain Stimulation (DBS) systems or baclofen pumps in place will be eligible for the study, but they must have had a stable setting for at least two months prior to the screening visit and stimulation parameters / pump settings must remain stable for the duration of the trial:

Main Exclusion Criteria:

• Evidence of iron deficiency defined by Fe:TIBC ratio <15%, or serum ferritin <12 ng/mL;

• Treatment with deferiprone in the past 12 months;

• Previous failure of treatment with deferiprone, or previous discontinuation of treatment with deferiprone due to adverse events;

• Conditions known to contraindicate the use of deferiprone (history of agranulocytosis or recurrent episodes of neutropenia);

• A serious, unstable chronic illness not related to PKAN condition during the past 3 months before screening visit including but not limited to: hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease;

• Evidence of abnormal liver or renal function (serum liver enzyme level(s) > 3 times upper limit of normal at screening) or abnormal creatinine levels at screening visit;

• Disorders associated with neutropenia (ANC < 1.5 x 10^9/L) or thrombocytopenia (platelet count < 50 x 10^9/L) in the 12 months preceding the initiation of the study medication. Exception: for patients whose neutropenia was attributed by the treating physician to episodes of infection or to drugs associated with a decline in the neutrophil count and in whom the ANC has fully recovered at the screening visit;

• History of malignancy;

Other protocol inclusion or exclusion criteria may apply.