Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 9:39 PM
Ignite Modification Date:
2025-12-24 @ 9:39 PM
NCT ID:
NCT01741532
Eligibility 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.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
4 Years
Study:
NCT01741532
Study Brief:
Protocol Section:
NCT01741532