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

ISRCTN	ISRCTN17052121
DOI	https://doi.org/10.1186/ISRCTN17052121
EudraCT/CTIS number	2010-019645-25
ClinicalTrials.gov number	NCT01186419
Secondary identifying numbers	FBS0701-CTP-04

Submission date: 02/09/2010
Registration date: 13/09/2010
Last edited: 22/03/2016

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Haematological Disorders

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English Summary

Not provided at time of registration

Contact information

Dr Medical Monitor
Scientific

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

Study information

Study design	Multicentre phase II open-label randomized trial
Primary study design	Interventional
Secondary study design	Randomised controlled trial
Study setting(s)	Hospital
Study type	Treatment
Participant information sheet	Not available in web format, please use contact details below to request a patient information sheet
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 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(s)	The Essex 1 Research Ethics Committee (REC), July 2010, ref: 10/H031/37
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
Pharmaceutical study type(s)
Phase	Phase II
Drug / device / biological / vaccine name(s)	FBS0701
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	N/A
Overall study start date	01/09/2010
Overall study end date	31/07/2011

Eligibility

Participant type(s)	Patient
Age group	Adult
Lower age limit	18 Years
Upper age limit	60 Years
Sex	Both
Target number of participants	Approximately 40 patients, 20 patients in each treatment arm.
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 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
Türkiye
United Kingdom
United States of America

Study participating centre

FerroKin BioSciences, Inc.

San Carlos
CA 94070
United States of America

Sponsor information

FerroKin BioSciences Inc. (USA)
Industry

2729 Debbie Court
San Carlos
CA 94070
United States of America

Website	http://www.ferrokin.com
"ROR"	https://ror.org/03bygaq51

Funders

Funder type

Industry

FerroKin BioSciences Inc. (USA)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Basic results				No	No
Results article	results	05/04/2012		Yes	No

Editorial Notes

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