Phase 2
Completed N=173
Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
Chronic Iron Overload Due to Transfusion-dependant Anemias
Source: ClinicalTrials.gov NCT02125877 ↗
Enrolled (actual)
173
Serious AEs
16.8%
Results posted
Oct 2016
Primary outcomePrimary: Overall Safety as Measured by Frequency of Adverse Events — 89.5; 89.7; 15.1; 18.4 Percentage of participants
Summary
Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Safety as Measured by Frequency of Adverse Events |
89.5; 89.7; 15.1; 18.4; 0; 1.1 | — |
| PRIMARY Overall Safety as Measured by Changes in Laboratory Values From Baseline |
9.3; 8.0; 3.5; 5.7; 8.1; 13.8 | — |
| SECONDARY Frequency of Selected Gastro-intestinal (GI) Adverse Events |
61.6; 58.6; 26.7; 26.4; 15.1; 8.0 | — |
| SECONDARY Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) |
10.3; 7.6; 5.2; 2.8; 12.9; 13.8 | — |
| SECONDARY Palatability Questionnaire Score |
9.0; 10.8; 8.8; 10.8; 9.3; 10.8 | — |
| SECONDARY Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary |
1.4; 1.9; 1.8; 1.1; 1.4; 1.1 | — |
| SECONDARY Number of Participants With Weekly Average Compliance of Medication Consumption |
56; 53; 64; 64; 62; 56 | — |
| SECONDARY Weekly Dose Violation Rate |
17.7; 15.8; 15.8; 6.7; 18.0; 8.4 | — |
| SECONDARY Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast) |
1110; 1040; 1590; 2110 | — |
| SECONDARY Observed Maximum Plasma Concentration Following Drug Administration (Cmax) |
74.6; 79.3; 118; 139 | — |
| SECONDARY Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax) |
3.57; 2.00; 2.85; 2.02 | — |
| SECONDARY Dererasirox Plasma Concentration |
39.6; 27.3; 80.8; 95.5; 37.1; 31.3 | — |
Eligibility Criteria
Key Inclusion Criteria
- Male and female patients aged ≥ 10 years
- Patients with transfusion-dependent thalassemia and iron overload, requiring deferasirox DT at doses of ≥ 30 mg/kg/day as per the investigator's decision OR Patients with very low, low or intermediate (int) risk myelodysplastic syndrome (MDS) and iron overload, requiring deferasirox DT at doses of ≥ 20 mg/kg/day as per the investigator's decision.
- History of transfusion of at least 20 PRBC units and anticipated to be transfused with at least 8 units of PRBCs annually during the study
- Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
Key Exclusion Criteria
- Creatinine clearance below the contraindication limit in the locally approved prescribing information. Creatinine clearance will be estimated from serum creatinine at screening Visit 1 and screening Visit 2 and the mean value will be used for eligibility criteria.
- Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
- ALT (SGPT) > 5xULN, unless LIC confirmed as >10 mg Fe/dw within 6 months prior to screening visit 1.
- Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2.
- Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
- Liver disease with severity of Child-Pugh Class B or C
Data sourced from ClinicalTrials.gov (NCT02125877). 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.