Phase 2
N=30
Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload
Iron Overload Due to Repeated Red Blood Cell Transfusions
Bottom Line
View on ClinicalTrials.gov: NCT03802916 ↗Enrolled (actual)
30
Serious AEs
0.0%
Results posted
Jun 2021
Primary outcome: Primary: The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern. — 0; 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Deferiprone DR tablets 1000 mg (Low dosage) (Drug); Deferiprone DR tablets 1000 mg (High dosage) (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- ApoPharma
- Primary completion
- Dec 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern. |
0; 0; 0; 0 | — |
| SECONDARY The Percentage of Patients in Each Treatment Group Who Report Post-dose Occurrences of Gastrointestinal (GI) Distress. |
3; 3 | — |
| SECONDARY The Percentage of Patients in Each Group Who Indicate That They Prefer the Deferiprone DR Formulation Over the Immediate-release Formulation. |
13; 13 | 0.0074 sig |
Summary
Safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload.
Eligibility Criteria
Inclusion Criteria
- Male or female aged ≥ 18 years.
- Diagnosis of thalassemia syndrome, sickle cell disease, or other disorder requiring a regular regimen of red blood cell transfusions.
- On a stable regimen (≥3 months) of Ferriprox tablets for the treatment of systemic iron overload.
- Absolute neutrophil count ≥1.5 x 10^9/L at screening.
- A record of at least 12 measured alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels.
Exclusion Criteria
- Receipt of any iron chelator other than Ferriprox (i.e., combination therapy) in the last 3 months, or planning to receive it at any time during the period of the study.
- ALT and/or AST value > 5 times the upper limit of normal (ULN) at screening
- Active case of hepatitis B or C at screening.
Data sourced from ClinicalTrials.gov (NCT03802916). 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.