Phase 3
Completed N=89
Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
Pantothenate Kinase-Associated Neurodegeneration
Source: ClinicalTrials.gov NCT01741532 ↗
Enrolled (actual)
89
Serious AEs
31.8%
Results posted
May 2019
Primary outcomePrimary: Change in Score on Barry-Albright Dystonia Scale — 2.48; 3.99 score on a scale — p=0.0761
◆ Published Evidence
Highly cited
146citations · ~21 / year
Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study.
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).
Linked Publications
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Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Score on Barry-Albright Dystonia Scale |
2.48; 3.99 | 0.0761 |
| PRIMARY Score on Patient Global Impression of Improvement at End of Study |
4.55; 4.66 | 0.7279 |
| SECONDARY Change in Score on Unified Parkinson's Disease Rating Scale |
-0.25; -0.07; 1.09; 2.36; 5.38; 2.06 | 0.7228 |
| SECONDARY Change in Score on Functional Independence Measure |
5.40; 0.69 | 0.1524 |
| SECONDARY Change in Score on WeeFIM |
4.91; -2.40 | 0.2026 |
| SECONDARY Change in Score on Pediatric Quality of Life |
1.21; 1.34; -4.90; -2.37 | 0.9759 |
| SECONDARY Change in Score on Pittsburgh Sleep Quality Index |
0.48; 0.14 | 0.6323 |
| SECONDARY Change in Level of Brain Iron |
-36.1; -0.50 | 0.0000 sig |
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 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.
Data sourced from ClinicalTrials.gov (NCT01741532) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.