Condition category
Haematological Disorders
Date applied
Date assigned
Last edited
Retrospectively registered
Overall trial status
Recruitment status
No longer recruiting
Publication status

Plain English Summary

Not provided at time of registration

Trial website

Contact information



Primary contact

Dr Medical Monitor


Contact details

FerroKin BioSciences
2729 Debbie Court
San Carlos
CA 94070
United States of America

Additional identifiers

EudraCT number

2010-019645-25 number


Protocol/serial number


Study information

Scientific title

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



Study hypothesis

FBS0701 is a safe and tolerable orally available iron chelator when administered chronically daily to patients with transfusional iron overload.

FBS0701 is an oral iron chelator designed to treat iron overload associated with chronic transfusion.

Ethics approval

The Essex 1 Research Ethics Committee (REC), July 2010, ref: 10/H031/37

Study design

Multicentre phase II open-label randomized trial

Primary study design


Secondary study design

Randomised controlled trial

Trial setting


Trial type


Patient information sheet

Not available in web format, please use contact details below to request a patient information sheet


Transfusional iron overload; hereditary and acquired anemias


Patients will be assigned to recieve either 16 mg/kg/day or 32 mg/kg/day of FBS0701 capsules orally once daily. There is no comparator or placebo arm. Screening procedures are carried out over 45 days. Duration of treatment is 24 weeks and the duration of follow-up is for a further 4 weeks beyond the end of the intervention period.

Intervention type



Phase II

Drug names


Primary outcome measure

1. To evaluate the safety and tolerability based on clinical assessments of two doses of FBS0701 when administered daily to patients with transfusional iron overload, as assessed by:
1.1. Adverse event occurrence
1.2. Changes in vital signs
1.3. 12-lead ECG
1.4. Physical examination
1.5. Clinical laboratory assessments
The assessments above will be carried out at intervals throughout the screening, treatment and follow-up phase.
2. To identify a differential response between dose groups in liver iron content determined by magnetic resonance imaging (MRI). MRI assessments will be performed at baseline, week 12 and week 24.

Secondary outcome measures


Overall trial start date


Overall trial end date


Reason abandoned (if study stopped)


Participant 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, β-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

Participant type


Age group




Target number of participants

Approximately 40 patients, 20 patients in each treatment arm.

Participant 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

Recruitment start date


Recruitment end date



Countries of recruitment

Italy, Thailand, Turkey, United Kingdom, United States of America

Trial participating centre

FerroKin BioSciences, Inc.
San Carlos
CA 94070
United States of America

Sponsor information


FerroKin BioSciences Inc. (USA)

Sponsor details

2729 Debbie Court
San Carlos
CA 94070
United States of America

Sponsor type




Funder type


Funder name

FerroKin BioSciences Inc. (USA)

Alternative name(s)

Funding Body Type

Funding Body Subtype


Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Basic results (scientific)


Publication list

2012 results in:

Publication citations

  1. Results

    Neufeld EJ, Galanello R, Viprakasit V, Aydinok Y, Piga A, Harmatz P, Forni GL, Shah FT, Grace RF, Porter JB, Wood JC, Peppe J, Jones A, Rienhoff HY, A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload., Blood, 2012, 119, 14, 3263-3268, doi: 10.1182/blood-2011-10-386268.

Additional files

Editorial Notes

22/03/2016: added link to results - basic reporting.