Patients with documented transfusional iron overload ages 6 to less than 18 years old will be studied. Patients with transfusion-dependent anemias will be eligible regardless of the cause of their anemia. MedDRA version: 14.1 Level: LLT Classification code 10065974 Term: Chronic iron overload System Organ Class: 10027433 - Metabolism and nutrition disorders
Conditions
Interventions
Product Name: FBS0701 50 mg capsule
Product Code: FBS0701
Pharmaceutical Form: Capsule, hard
CAS Number: 1173092-59-5
Current Sponsor code: FBS0701
Concentration unit: mg milligram(s)
Concentration ty
Sponsors
FERROKIN BIOSCIENCES INC.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Parents willing and able to sign the approved informed consent for their children and patients between the ages of 6 and 17 willing and able to provide their assent (based on institutional guidelines). 2. Able to swallow whole capsules. 3. Age >6 and 10 milliseconds (Note: Patients not able to have an MRI will be considered iron overloaded on the basis of serum ferritin only.) 7. Serum ferritin >500 ng/mL at Screening. 8. Mean of the previous three pre-transfusion hemoglobin concentrations =7.5 g/dL. 9. If appropriate depending on age, patient agrees to use an approved method of contraception from Screening and until 30 days after the last administration of the study drug. Agreed methods of contraception for the patient or the patient’s partner may include: condom; use of approved birth control pills, patches, implants or injections; use of diaphragm with vaginal spermicide; use of an intrauterine device (IUD); and/or surgical sterilization (vasectomy or tubal ligation at least six months prior to dosing). Patients practicing abstinence must agree to use an approved method of contraception should they become sexually active during the study. Are the trial subjects under 18? yes Number of subjects for this age range: 15 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. As a result of medical review, physical examination (including height and weight) 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 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, e.g. serum creatinine above the upper limit of normal or proteinuria greater than 1 gm per day. 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. ALT >180 IU/mL at Screening. 10. Use of any investigational agent within the 30 days prior to Baseline testing. 11. Pregnant or lactating females.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: -To evaluate the single-dose pharmacokinetics of FBS0701 in the pediatric iron overloaded population 6 years and older. -To assess the long term safety, tolerability and efficacy of FBS0701 in the pediatric iron overloaded population 6 years and older.;Secondary Objective: NA;Primary end point(s): 1-Plasma levels, half-life of FBS0701 and other pharmacokinetic parameters following a single capsule dose of FBS0701 at 16 mg/kg with 7 days of follow-up. 2- During the chronic dosing phase, safety and tolerability of FBS0701 based on clinical assessments (e.g., incidence and severity of Adverse Events, physical examinations including vital signs, clinical laboratory values, and ECGs); efficacy will be evaluated based on changes in liver and cardiac iron concentration determined by MRI in patients able to be studied using this method; serum ferritin in all patients;Timepoint(s) of evaluation of this end point: The pharmacokinetic endpoints will be evaluated during the initial PK phase (1 week) for those patients involved. 2. The safety portion of the chronic dosing endpoints will be evaluated throughout the 48 week dosing period and 4 weeks of safety follow-up. The pharmacodynamic portion of this endpoint will be evaluated at Week 24 and 48 (MRI assessments); and at every safety laboratory assessment (serum ferritin analysis). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): NA;Timepoint(s) of evaluation of this end point: NA | — |
Countries
Canada, Italy, United States
Contacts
Public ContactClinical Operation
FerroKin BioScience Ltd
Outcome results
None listed