Study to evaluate FBS0701 in a pediatric population with transfusioanl iron overload

• Conditions: 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: 14.1Level: LLTClassification code 10065974Term: Chronic iron overloadSystem Organ Class: 10027433 - Metabolism and nutrition disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2011-001718-32-IT
• Lead Sponsor: FERROKIN BIOSCIENCES INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-03-20
• Last Update Posted: 23 March 2015

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 30

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 <18 years. 4. Transfusion-dependent patients who have transfusional iron overload requiring chronic treatment with deferoxamine, deferasirox, or deferiprone. A transfusion dependent patient is defined in this study as one with a minimum transfusion history totaling more than 20 units of packed red blood cells OR a calculated iron load based on transfusion history of 200 mg/kg AND a transfusion requirement of 7 or more transfusions per year; or, in the case of sickle cell anemia, be iron overloaded but receiving transfusion exchange therapy in lieu of transfusions. 5. Willing to discontinue all existing iron chelation therapies for a minimum period of one to five days prior to enrollment, for the initial PK period of 8 days (if applicable), and for up to 49 weeks if entering into the chronic dosing phase. 6. Patients able to have an MRI must have: a) liver iron concentration (LIC) =2 and <30 mg/g (dry weight, liver) b) cardiac MRI T2* >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

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified