A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, and Pharmacodynamics of FBS0701 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy. - ND

• Conditions: Patients with transfusional iron overload due to hereditary anemias such as sickle cell disease, ß-thalassemia and Diamond-Blackfan anemia; acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure.

• Registration Number: EUCTR2010-019645-25-IT
• Lead Sponsor: FERROKIN BIOSCIENCES INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-07-15
• Last Update Posted: 18 March 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

1. Willing and able to sign the approved informed consent. 2. Age: 18-60 years old at Screening 3. Transfusional iron overload due to: hereditary anemias such as sickle cell disease, ß-thalassemia and Diamond-Blackfan anemia; acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure. Patients must also: a. Be transfusion-dependent (8 or more transfusions annually) and b. Require chronic treatment with deferoxamine, deferasirox, and/or deferiprone. 4. Willing to discontinue all existing iron chelation therapies for a minimum period of two to five days prior to first dose of FBS0701, the 24 week duration of the study and 1 week after last dose for a total of approximately 26 weeks. 5. Serum ferritin > 500 ng/mL at Screening. 6. Baseline (Day -14 to Day -7) liver iron concentration (LIC) between =3.5 and <30 mg iron per g (equivalent dry weight, liver) determined by FerriScan MRI. 7. Baseline (Day -14 to Day -7) cardiac MRI T2* =10 milliseconds. 8. Mean of the previous three pre-transfusion hemoglobin concentrations = 7.5 g/dL. 9. Agrees to use an approved method of contraception from Screening and until 28 days after the last administration of the study drug. Agreed methods of contraception may include: condom; use of birth control pills, patches, implants or injections, diaphragm with vaginal spermicide, 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. A monthly pregnancy test will be performed on all female patients of child-bearing potential during the Screening and study period.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
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 or Screening investigations, the Principal Investigator considers the patient unfit for the study. 2.Non-elective hospitalization within the 30 days prior to Baseline testing. (Patients with sickle cell anemia who are admitted to the hospital for management of sickle crisis pain whose uncomplicated hospital course was four days or less and who, 14 days prior to Baseline testing, have returned to their previous health status are acceptable.) 3.Evidence of clinically relevant oral, cardiovascular, gastrointestinal, hepatic, renal, endocrine, pulmonary, neurologic, psychiatric, immunologic, bone marrow or skin disorder as determined by the Investigator. 4.Evidence of significant renal insufficiency; e.g., serum creatinine above the upper limit of normal, proteinuria greater than 2 gm per day or calculated creatinine clearance of less than 60 mL/minute. 5.Cardiac left ventricular ejection fraction: a.Outside the locally determined normal range in the 12 months prior to Screening by echocardiography or MRI and b.<55% at Baseline testing by MRI 6.Known sensitivity to magnesium stearate, croscarmellose sodium or FBS0701 7.Platelet count below 150,000/?L and/or absolute neutrophil count less than 1500/mm3 at Screening 8.Alkaline phosphatase, AST or ALT a.>5 times the local upper limit of normal at any time in the previous 12 months or b.>3 times the upper limit of normal at Screening 9.Use of any investigational agent within the 30 days prior to the Baseline testing.

Study & Design

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