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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT01039636




Registration number
NCT01039636
Ethics application status
Date submitted
23/12/2009
Date registered
25/12/2009
Date last updated
10/02/2012

Titles & IDs
Public title
Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload
Scientific title
A PHASE 1b OPEN LABEL, MULTI-CENTER, ESCALATING MULTIPLE DOSE STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF FBS0701 IN PATIENTS WITH TRANSFUSIONAL IRON OVERLOAD
Secondary ID [1] 0 0
FBS0701-CTP-03
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Transfusional Iron Overload 0 0
Beta-thalassemia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Diet and Nutrition 0 0 0 0
Other diet and nutrition disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - FBS0701

Experimental: FBS0701 - 5 escalating doses - 5 escalating doses of FBS0701 in 5 cohorts of 4 patients each.


Treatment: Drugs: FBS0701
Oral FBS0701 for 7 days at one of the following doses: 3 mg/kg/day or 8 mg/kg/day or 16 mg/kg/day or 32 mg/kg/day or 40 mg/kg/day
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability by AEs, Physical Exam, Clinical Laboratory assessments
Timepoint [1] 0 0
35 days (7 days treatment and 28 days follow up)
Primary outcome [2] 0 0
Steady state plasma level and half-life of FBS0701
Timepoint [2] 0 0
7 days of dosing
Secondary outcome [1] 0 0
Urinary excretion of iron
Timepoint [1] 0 0
7 days of dosing

Eligibility
Key inclusion criteria
- At least 18 years old

- Transfusion dependent (at least 8 transfusions per year) with transfusional iron
overload needing treatment with deperoxamine, deferasirox or deperiprone.

- Willing to discontinue current iron chelation therapy for 2-5 days prior to enrollment
and for a total of up to 3 weeks

- Willing to fast after midnight prior to each dose

- Serum ferritin greater than 400ng/ml

- Liver iron concentration greater than or equal to 1.5 mg iron. (determined by MRI or
SQUID within 12 months prior to enrollment)

- Cardiac iron greater than or equal to 12 milliseconds (determined by MRI t2* within 18
months prior to enrollment)

- Mean of last 3 pre-transfusion hemoglobin values must be greater than or equal to 7.5
g/dl

- Agrees to use approved contraception from Screening and until 30 days after last
administration of study drug. Abstinence OK.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Principal Investigator considers patient unfit for study after conducting a medical
review, physical examination and other screening assessments.

- Non-elective hospitalization with past 30 days

- Evidence of clinically relevant oral, cardiovascular, gastrointestinal, hepatic,
renal, endocrine, pulmonary, neurologic, psychiatric or skin disorder.

- Evidence of significant renal insufficiency; serum creatine above upper limit of
normal or proteinuria greater than 2 gm per day or calculated creatinine clearance of
less than or equal to 60ml/min

- Platelet count below 150,000,000/ml and/or absolute neutrophil count less then
1500/mm3

- Alkaline phosphatase or AST greater than 5 times the upper limit of normal or ALT
greater than 4 times the upper limit of normal

- Female patients who are pregnant or lactating

- Use of any investigational agent within the last 30 days

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
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/11/2009
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
20
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
Pennsylvania
Country [4] 0 0
Thailand
State/province [4] 0 0
Bangkok

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
FerroKin BioSciences, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this research study is to study the safety of increasing doses of FBS0701, and
to see how quickly the study medication is absorbed and how quickly it disappears from the
bloodstream. FBS0701 is a new, oral iron chelator - a medication taken by mouth that
increases the body's elimination of iron. Iron chelators are used in patients who develop
iron overload from their transfusions. Four increasing doses of FBS0701 will be tested during
this study. The study will start with the lowest dose given to 4 patients (3 mg/kg/day. The
next group of 4 patients will receive the next high dose (8mg/kg/day only after the results
of the first 4 patients are examined and it is determined safe to continue. Participating
patients will take the study medication for 7 days and be followed for 28 days after their
last dose to determine if they have any reactions to the study medication - therefore a total
of 35 days on study. Patients will need to give up to 17 blood samples over the screening
period and first 15 days of the study (a total of about 9 tablespoons). Patients will not
need to stay overnight in the clinic but will need to visit the clinic 10 times for screening
and on-study visits over the 35 days. Patients currently taking an iron chelator will need to
stop that treatment for up to 22 days (up to 5 days before they start the study and for 15
days during the study). The results of this study will be helpful in determining the safety
of the drug and the best doses of FBS0701 to be used in the next study which will assess the
effectiveness of this new iron chelator.
Trial website
https://clinicaltrials.gov/ct2/show/NCT01039636
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ellis Neufeld, MD
Address 0 0
Boston Children's Hospital
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT01039636