Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
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 FBS0701. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of FBS0701; and the chronic dosing phase, during which patients will receive an additional 48 weeks of FBS0701 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 FBS0701 in older children (adolescents, 12-<18 years old) and then if deemed safe, in younger children (6-<12 years old).
Children's Hospital Boston, Dana-Farber Cancer Institute
Rachel Grace, MD,
Children's Hospital Boston
Dana-Farber Cancer Institute:
Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP,
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.
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
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.