Status:
COMPLETED
Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Iron Overload
Eligibility:
All Genders
2-6 years
Phase:
PHASE4
Brief 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 administer...
Detailed Description
The study included a screening period (from Day 0-14) with two visits at least 7 days apart to assess eligibility of patients that were chelation naïve or on a prior iron chelator treatment other than...
Eligibility Criteria
Inclusion
- Key
- Patients ≥2 to \<6 years old diagnosed with transfusional hemosiderosis
- Documented history of red blood cell transfusions
- Written informed consent/assent before any study-specific procedures. The consent will be obtained from caregiver(s) or patient's legal representative. Investigators will also obtain assent of patients according to local, regional, or national regulations.
- For patients on prior DFX: Serum ferritin (SF) \>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
- 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.
Key Trial Info
Start Date :
January 16 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 5 2019
Estimated Enrollment :
44 Patients enrolled
Trial Details
Trial ID
NCT03372083
Start Date
January 16 2018
End Date
December 5 2019
Last Update
August 25 2020
Active Locations (10)
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1
Novartis Investigative Site
Zagazig, Egypt, 44519
2
Novartis Investigative Site
Cona, FE, Italy, 44100
3
Novartis Investigative Site
Cagliari, ITA, Italy, 09121
4
Novartis Investigative Site
Napoli, Italy, 80138