Assessing the safety, tolerability, and pharmacodynamics of FBS0701 in the treatment of chronic iron overload requiring chelation therapy

Phase 2

Completed

• Conditions: Transfusional iron overload; Haematological Disorders; hereditary and acquired anemias

• Registration Number: ISRCTN17052121
• Lead Sponsor: FerroKin BioSciences Inc. (USA)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-09-13
• Last Update Posted: 17 October 2016

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

1. Age: 18-60 years old at screening
2. Transfusional iron overload due to:
2.1. Hereditary anemias such as sickle cell disease, ß-thalassemia and Blackfan-Diamond anemia
2.2. Acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure
3. Patients must also be transfusion-dependent (8 or more transfusions annually) and require chronic treatment with deferoxamine, deferasirox, and/or deferiprone
4. Willing to discontinue all existing iron chelation therapies throughout the study period
5. Serum ferritin >500 ng/mL at screening
6. Baseline liver iron concentration (LIC) and cardiac T2* MRI per protocol requirements
7. Mean of the previous three pre-transfusion haemoglobin concentrations = 7.5 g/dL
8. Agrees to use an approved method of contraception througout the study

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 hospitalisation within the 30 days prior to baseline testing
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
5. Cardiac left ventricular ejection fraction outside of protocol requirements
6. Known sensitivity to magnesium stearate, croscarmellose sodium or FBS0701
7. Platelet count below 150,000/µL and/or absolute neutrophil count less than 1500/mm3 at screening
8. Alkaline phosphatase, Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) outside of protocol requirements
9. Use of any investigational agent within the 30 days prior to the baseline testing

Study & Design

• Study Type: Interventional
• Study Design: Not specified