Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodeg… (NCT01741532) | Clinical Trial Compass
CompletedPhase 3
Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
United States, Germany, Italy89 participantsStarted 2012-12-13
Plain-language 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).
Who can participate
Age range4 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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…
What they're measuring
1
Change in Score on Barry-Albright Dystonia Scale
Timeframe: Baseline to 18 Months
2
Score on Patient Global Impression of Improvement at End of Study