Phase 4
Completed N=44
Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis
Iron Overload
Source: ClinicalTrials.gov NCT03372083 ↗
Enrolled (actual)
44
Serious AEs
4.6%
Results posted
Aug 2020
Primary outcomePrimary: Number of Participants With Selected Gastrointestinal Disorders up to 24 Weeks — 0; 0; 0; 0 Participants
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
This study employed a prospective, single-arm, global multi-center interventional open-label, non-randomized design to identify and assess safety profile of the crushed deferasirox FCT when administered up to 24 weeks in pediatric patients aged ≥2 to <6 years with transfusional hemosiderosis. The study was designed to enroll a minimum of 40 patients. Forty-four patients were treated and analyzed.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Selected Gastrointestinal Disorders up to 24 Weeks |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Adverse Events Profile |
22; 0; 0 | — |
| SECONDARY Number of Participants With Notable Changes in ECG Values From Baseline |
— | — |
| SECONDARY Absolute Change From Baseline in Serum Ferritin (SF) |
2152.7; -118.4; -17.5; -52.8; 71.9; -64.3 | — |
| SECONDARY Number of Participants With Worst Post-baseline Values in Selected Chemistry Parameters |
16; 0; 28; 0; 0; 25 | — |
| SECONDARY Number of Participants With Clinically Significant Auditory Assessments Changes From Baseline |
— | — |
| SECONDARY Number of Participants With Clinically Significant Ocular Assessments Changes From Baseline |
— | — |
| SECONDARY Absolute Change From Baseline in Systolic and Diastolic Blood Pressures (mmHg) |
100.0; -0.1; -2.3; -0.3; 0.3; -1.4 | — |
| SECONDARY Absolute Change From Baseline in Pulse Rate (Bpm) |
102.4; -1.1; -2.7; -3.1; -1.0; -0.3 | — |
| SECONDARY Absolute Change From Baseline in Body Temperature (°C) |
36.43; -0.03; -0.10; 0.00; 0.02; -0.03 | — |
| SECONDARY Absolute Change From Baseline in Body Weight (kg) |
15.26; 0.08; 0.19; 0.31; 0.25; 0.43 | — |
| SECONDARY Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Adherence |
-2.4; -2.6; -1.9; -1.4; -1.4; -1.0 | — |
| SECONDARY Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Type of Medicine Child Like Scoring |
0; 4; 0; 11; 14; 0 | — |
| SECONDARY Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring |
6; 5; 5; 0; 0; 0 | — |
| SECONDARY Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Rank Based on Child's Preference Scoring |
0; 1; 12; 7; 11; 1 | — |
| SECONDARY Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Concerns |
1.1; 1.2; 0.8; 0.4; 0.4; 0.2 | — |
| SECONDARY Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Adherence |
10.4; 9.8; 10.3; 9.0; 9.9; 10.6 | — |
| SECONDARY Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Type of Medicine Child Like Scoring |
0; 1; 0; 5; 3; 0 | — |
| SECONDARY Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring |
0; 1; 2; 1; 0; 1 | — |
| SECONDARY Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Rank Based on Child's Preference Scoring |
2; 1; 2; 4; 1; 0 | — |
| SECONDARY Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Concerns |
9.7; 9.4; 8.9; 3.7; 3.9; 3.4 | — |
| SECONDARY Palatability Score in Chelation Naive Participants |
10.4; 10.9; 10.8 | — |
| SECONDARY Number of Chelation Naive Participants With Palatability After Taste Item Scoring |
3; 4; 3; 1; 0; 2 | — |
| SECONDARY Palatability Score in Participants Pre-treated With Deferasirox |
10.7; 10.3; 10.6 | — |
| SECONDARY Number of Participants Pre-treated With Deferasirox With Palatability After Taste Item Scoring |
5; 5; 15; 7; 0; 1 | — |
| SECONDARY GI Symptom Score in Chelation Naive Participants |
6.8; 7.1; 7.0; 6.8; 6.2; 7.0 | — |
| SECONDARY Number of Participants With GI Bowel Movements Item Scoring in Chelation Naive Participants |
1; 1; 8; 2; 1; 0 | — |
| SECONDARY GI Symptom Score in Participants Pre-treated With Deferasirox |
6.3; 6.0; 6.4; 6.4; 6.5; 6.7 | — |
| SECONDARY Number of Participants With GI Bowel Movements Item Scoring in Participants Pre-treated With Deferasirox |
0; 1; 30; 1; 0; 1 | — |
Eligibility Criteria
Key Inclusion Criteria
- Patients ≥2 to 500 ng/mL, measured at screening visit 1 and requiring a DFX daily dose equivalent to FCT ≥ 7mg/kg/day.
- For patients on a prior chelator other than DFX (e.g. deferiprone or deferoxamine) or chelation naive: Serum ferritin (SF) >1000 ng/mL measured at screening visits 1 and 2.
Key Exclusion Criteria
- Patients that receive more than one iron chelator at the same time as current iron chelation treatment. (Patients who have received combination therapy in their medical history but are currently being treated with a single ICT agent are eligible.)
- Patients continuing on deferoxamine or deferiprone in addition to study treatment. (Patients switching to or continuing on deferasirox are eligible).
- Unresolved adverse events if the patient was previously treated with deferiprone or deferoxamine or deferasirox.
- Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void sample urine measured at screening visit 1.
- Serum creatinine > age adjusted ULN measured at any screening visit
- Creatinine clearance below 90 mL/minute measured at any screening visit. Creatinine clearance using the Schwartz formula will be estimated from serum creatinine measured at each respective visit.
- ALT and/or AST > 2.5 x ULN measured at screening visit 1.
- Total bilirubin (TBIL) >1.5 x ULN measured at screening visit 1.
- Patients with significant impaired GI function or GI disease that may significantly alter the absorption of oral deferasirox FCT (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
- History of and/or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive.
- Liver disease with severity of Child-Pugh Class B or C.
- History of hypersensitivity to any of the study drug or excipients.
- Patients participating in another clinical trial or receiving an investigational drug.
- Patients with a known history of HIV seropositivity.
- Patients unwilling or unable to comply with the protocol.
- History of malignancy of any organ system, treated or untreated, within the past 5 years whether or not there is evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin.
- Significant medical condition interfering with the ability to partake in this study (e.g. uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease: cardiovascular, renal, hepatic, etc.).
- Female patients who reach menarche and they or their caregivers refuse pregnancy testing and/or if there is a positive pregnancy test result.
Data sourced from ClinicalTrials.gov (NCT03372083). 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.