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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial registered on ANZCTR


Registration number
ACTRN12609000213246
Ethics application status
Approved
Date submitted
19/03/2009
Date registered
27/04/2009
Date last updated
5/07/2012
Type of registration
Prospectively registered

Titles & IDs
Public title
Safety study of a test oral iron chelator in male iron-overloaded patients
Scientific title
A Dose-Escalation, Phase I Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of FBS0701 in Iron-Overloaded Male Patients
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Iron overload in transfusion-dependent patients 4501 0
Condition category
Condition code
Blood 4782 4782 0 0
Other blood disorders
Blood 4976 4976 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Single dose (6 mg/kg, 10 mg/kg, 16 mg/kg or 32 mg/kg) of FBS0701 administered orally
Intervention code [1] 4250 0
Treatment: Drugs
Comparator / control treatment
Patient 1: 6 mg/kg FBS0701
Patient 2: 10 mg/kg FBS0701
Patient 3: 16 mg/kg FBS0701
Patient 4: 32 mg/kg FBS0701
Control group
Dose comparison

Outcomes
Primary outcome [1] 5642 0
To evaluate the safety and pharmacokinetics of FBS0701
Timepoint [1] 5642 0
Safety assessments and blood sampling for pharmacokinetic analysis will be preformed whilst patients are confined in the clinical facility for 24 hours, followed by visits at 36, 48 and 72 hours following dosing.
Secondary outcome [1] 241526 0
To determine dose-proportionality of ascending doses of FBS0701 by frequent blood sampling and urine collection, and pharmacokinetic analyses. Thirteeen blood samples will be taken during the first 12 hours following dosing on Day 1 of the study. Blood samples will then be taken every 12 hours to 48 hours following dosing, and then a final sample on Day 4 of the study. A continuous urine collection for 24 hours will occur following dosing.
Timepoint [1] 241526 0
For 72 hours post-dose
Secondary outcome [2] 241527 0
To characterise pharmacodynamics of FBS0701 by measuring urinary iron and magnesium.
Timepoint [2] 241527 0
For 72 hours post-dose

Eligibility
Key inclusion criteria
Trasnfusion-dependent patients wo have transfusional overload, requiring chronic treatment with deferoxamine, deferasirox or deferiprone. Patients must be willing to discontinue all existing iron chelation therapies for a minimum of seven days prior to admission to clinic.
Minimum age
18 Years
Maximum age
50 Years
Gender
Males
Can healthy volunteers participate?
No
Key exclusion criteria
Received or is anticipated to receive a new prescription systemic or topical medication within 14 days of study start, or over-the-counter medications within 48 hours. Any condition that would interfere with drug absorption (ie chronic diarrhoea).

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint(s)
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 4688 0
Commercial sector/Industry
Name [1] 4688 0
FerroKin BioSciences Inc.
Address [1] 4688 0
2729 Debbie Court
San Carlos
CA 94070
Country [1] 4688 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
FerroKin BioSciences Inc.
Address
2729 Debbie Court
San Carlos
CA 94070
Country
United States of America
Secondary sponsor category [1] 4233 0
None
Name [1] 4233 0
Address [1] 4233 0
Country [1] 4233 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 6727 0
Royal Adelaide Hospital Research Ethics Committee
Ethics committee address [1] 6727 0
North Terrace
Adelaide SA 5000
Ethics committee country [1] 6727 0
Australia
Date submitted for ethics approval [1] 6727 0
18/02/2009
Approval date [1] 6727 0
Ethics approval number [1] 6727 0

Summary
Brief summary
This aim of this study is to assess the safety of different doses of anovel orally-administered iron chelator in patients with iron-overload.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 29415 0
Address 29415 0
Country 29415 0
Phone 29415 0
Fax 29415 0
Email 29415 0
Contact person for public queries
Name 12662 0
CAMERON JOHNSON
Address 12662 0
CMAX, Level 5 East Wing
Royal Adelaide Hospital
North Terrace
Adelaide, SA, 5000
Country 12662 0
Australia
Phone 12662 0
+61 8 8222-3931
Fax 12662 0
+61 8 8223-3475
Email 12662 0
cameron.johnson@cmax.com.au
Contact person for scientific queries
Name 3590 0
DR HUGH YOUNG RIENHOFF, JR
Address 3590 0
2729 Debbie Court
San Carlos
CA 94070
Country 3590 0
United States of America
Phone 3590 0
+1-408-677-0682
Fax 3590 0
+1-508-848-3058
Email 3590 0
hrienhoff@datahooks.com

No information has been provided regarding IPD availability
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary