Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload

Status: Recruiting
Phase: Phase 2
Diagnosis: Pediatric Hematology/Blood Related
NCT ID: NCT01363908 (View complete trial on ClinicalTrials.gov)
DFCI Protocol ID:

 

This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the chronic dosing phase, during which patients will receive an additional 48 weeks of SSP-004184AQ dosing. Two age groups will be studied: 6-<12, and 12-<18 years old. The study is designed to initially assess the pharmacokinetics and safety of SSP-004184AQ in older children (adolescents, 12-<18 years old) and then if deemed safe, in younger children (6-<12 years old).

 

Conducting Institutions:
Children's Hospital Boston, Dana-Farber Cancer Institute

Overall PI:
Rachael Grace, MD, Children's Hospital Boston

Site-responsible Investigators:

Contacts:
Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org

Eligibility Criteria

Inclusion Criteria 1. Able to swallow whole capsules. 2. Transfusion-dependent patients who have transfusional iron overload requiring chronic treatment with deferoxamine, deferasirox, or deferiprone. 3. Willing to discontinue all existing iron chelation therapies throughout study period. 4. For patients able to have an MRI, Baseline liver iron concentration and cardiac MRI T2* per protocol requirements. 5. Serum ferritin greater than 500 ng/mL at Screening. 6. Mean of the previous three pre-transfusion hemoglobin concentrations greater than or equal to 7.5 g/dL. 7. Agrees to use an approved method of contraception throughout the study period. Exclusion Criteria 1. As a result of medical review, physical examination or Screening investigations, the Principal Investigator considers the patient unfit for the study. 2. Iron overload from causes other than transfusional hemosiderosis. 3. Severe cardiac dysfunction. 4. Non-elective hospitalization within the past 30 days prior to Baseline testing. 5. Evidence of clinically significant oral, cardiovascular, gastrointestinal, hepatic, renal, endocrine, pulmonary, neurologic, psychiatric, or skin disorder that contra-indicates dosing with FBS0701. 6. Evidence of significant renal insufficiency. 7. Known sensitivity to any ingredient in the FBS0701 formulation. 8. Platelet count below 100,000/µL or absolute neutrophil count less than 1500/mm3 at Screening. 9. Screening ALT outside of protocol requirements. 10. Use of any investigational agent within the 30 days prior to Baseline testing. 11. Pregnant or lactating females.
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