Condition category
Haematological Disorders
Date applied
02/09/2010
Date assigned
13/09/2010
Last edited
22/03/2016
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr Medical Monitor

ORCID ID

Contact details

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

Additional identifiers

EudraCT number

2010-019645-25

ClinicalTrials.gov number

NCT01186419

Protocol/serial number

FBS0701-CTP-04

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

Acronym

NAV

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

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

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

Condition

Transfusional iron overload; hereditary and acquired anemias

Intervention

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

Drug

Phase

Phase II

Drug names

FBS0701

Primary outcome measures

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

N/A

Overall trial start date

01/09/2010

Overall trial end date

31/07/2011

Reason abandoned

Eligibility

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

Patient

Age group

Adult

Gender

Both

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

01/09/2010

Recruitment end date

31/07/2011

Locations

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

Organisation

FerroKin BioSciences Inc. (USA)

Sponsor details

2729 Debbie Court
San Carlos
CA 94070
United States of America

Sponsor type

Industry

Website

http://www.ferrokin.com

Funders

Funder type

Industry

Funder name

FerroKin BioSciences Inc. (USA)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

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

Results - basic reporting

See https://clinicaltrials.gov/ct2/show/results/NCT01186419

Publication summary

2012 results in: http://www.ncbi.nlm.nih.gov/pubmed/22251482

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.