Phase 3
N=68
Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration
Pantothenate Kinase-Associated Neurodegeneration
Bottom Line
View on ClinicalTrials.gov: NCT02174848 ↗Enrolled (actual)
68
Serious AEs
48.5%
Results posted
Jul 2019
Primary outcome: Primary: Number of Participants With Adverse Events — 22; 42; 12; 14 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Deferiprone oral solution (Drug)
- Age
- Pediatric, Adult, Older Adult · 5+ yrs
- Sex
- All
- Sponsor
- ApoPharma
- Primary completion
- Mar 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events |
22; 42; 12; 14; 2; 1 | — |
| SECONDARY Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study |
4.4; 1.9; 1.4; 1.4 | 0.0500 |
| SECONDARY Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies |
4.4; 1.4 | 0.0206 sig |
| SECONDARY Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies |
1.9; 1.4 | 0.2684 |
| SECONDARY Proportion of Patients With Improved or Unchanged BAD Score |
3; 17; 9; 17 | 0.0821 |
| SECONDARY Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies |
4.4; 4.7 | 0.3306 |
Summary
Patients with PKAN will be treated with the iron chelator deferiprone for 18 months. Only patients who have completed the earlier study TIRCON2012V1 (NCT01741532), a double-blind placebo-controlled trial in which participants were randomized to receive either deferiprone or placebo for 18 months, are eligible to enroll.
Eligibility Criteria
Inclusion Criteria
- Completed study TIRCON2012V1
Exclusion Criteria
- Withdrew from the study TIRCON2012V1 for reasons of safety
- Plan to participate in another clinical trial at any time from the day of enrolment until 30 days post-treatment in the current study
Data sourced from ClinicalTrials.gov (NCT02174848). 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.