EUCTR2012-000845-11-GBActive, not recruitingPhase 1
A randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN) - TIRCO
Conditions
Drugs
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- ApoPharma Inc
- Enrollment
- 90
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
Inclusion Criteria
- •1.Males and females 4 years of age and older at screening visit;
- •2.Patients must have PKAN, confirmed by genetic testing;
- •3.Patients having a BAD total score > = 3 at the screening visit;
- •4.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 2 months prior to the screening visit and stimulation parameters / pump settings must remain stable for the duration of the trial. Enrollment of non-DBS patients will be given priority in order to ensure the majority can undergo imaging;
- •5. Potentially sexually active female patients of childbearing potential must have a negative pregnancy test result at Screening Visit (if applicable; in cases where the Investigator determines there is no reasonable risk of pregnancy because of significant incapacity, pregnancy testing will not be performed);
- •6.Fertile potentially sexually active males must use an effective method of contraception or must confirm partner’s use of effective contraception;
- •7.Informed consent/assent obtained before any study-related activities are undertaken;
- •8.Ability and willingness to adhere to the protocol including appointments and evaluation schedule.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 72
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 18
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
Exclusion Criteria
- •1.Evidence of iron deficiency defined by Fe:TIBC ratio <15%, or serum ferritin < 12 ng/mL;
- •2.Treatment with deferiprone in the past 12 months;
- •3.Previous failure of treatment with deferiprone, or previous discontinuation of treatment with deferiprone due to adverse events;
- •4.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;
- •5.Disorders associated with neutropenia (absolute neutrophil count (ANC) < 1.5 x 109/L) or thrombocytopenia (platelet count < 50 x 109/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 ANC has fully recovered at the screening visit;
- •6.Pregnant, breastfeeding, or planning to become pregnant during the study;
- •7.Initiation or discontinuation of treatment with baclofen, trihexyphenidyl, clonazepam, tizanidine within 30 days prior to baseline; and initiation or discontinuation of treatment with tetrabenazine within 90 days prior to baseline;
- •8. Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the baseline;
- •9. Currently taking iron chelators;
- •10.Patients who, in the opinion of the physician, represent a high medical or psychological risk;
- •11. History of or active drug or alcohol use or dependence that, in the opinion of the site investigator, would interfere with adherence to study requirements;
- •12.Patients and patient's legal representative (if applicable) with a mental incapacity, unwillingness or language barriers precluding adequate understanding or co-operation.
- •13.Baclofen pump placement less than two months prior to the beginning of the study.
Investigators
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