Transfusional Iron OverloadPhase 1NCT01039636
Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload
A Phase 1 study testing FBS0701 in people with Transfusional Iron Overload and Beta-thalassemia, run by FerroKin BioSciences, Inc. at 5 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transfusional Iron Overload, Beta-thalassemia
- What is being tested?
- FBS0701
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 5 sitesin 3 countries
- Study dates
- Nov 2009to Dec 2010
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
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- 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.
Who cannot join
- 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
Trial locations and contacts
5 sites in 3 countries.
5 sites in 3 countries
United States3 sites
Children's Hospital of Boston
Boston, Massachusetts, 02115
Children's Hospital of Oakland
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official 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
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.
- Conditions
- Transfusional Iron Overload, Beta-thalassemia
- Being tested
- FBS0701
- Keywords
- Beta-thalassemia, Sickle cell anemia, Transfusional iron overload, Iron overload, Iron chelation
- Medical subject headings
- beta-Thalassemia, Anemia, Sickle Cell, Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FBS0701Drug
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
| Study group | Role | What they receive |
|---|---|---|
| FBS0701 - 5 escalating doses | Experimental | 5 escalating doses of FBS0701 in 5 cohorts of 4 patients each. Receives: Drug: FBS0701 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability by AEs, Physical Exam, Clinical Laboratory assessments
Measured: 35 days (7 days treatment and 28 days follow up)
Steady state plasma level and half-life of FBS0701
Measured: 7 days of dosing
Secondary outcomes (1)
Urinary excretion of iron
Measured: 7 days of dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Dec 2009 (estimated)
- Study startNov 2009
- Primary completionDec 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transfusional Iron OverloadStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- FerroKin BioSciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ellis Neufeld, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01039636
- Sponsor's protocol ID
- FBS0701-CTP-03
- Status last verified
- Jan 2011
- First submitted
- 23 Dec 2009
- Last updated
- 10 Feb 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT01039636
- Acronym / sponsor ID
- FBS0701-CTP-03
- Conditions
- Transfusional Iron Overload, Beta-thalassemia
- Keywords
- Beta-thalassemia, Sickle cell anemia, Transfusional iron overload, Iron overload, Iron chelation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- FerroKin BioSciences, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2009
- Primary completion
- Dec 2010 (actual)
- First posted
- 25 Dec 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FBS0701 - 5 escalating doses | Experimental | Drug: FBS07015 escalating doses of FBS0701 in 5 cohorts of 4 patients each. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FBS0701Oral 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 | Drug | FBS0701 - 5 escalating doses |
Primary outcome
Safety and tolerability by AEs, Physical Exam, Clinical Laboratory assessments
Time frame: 35 days (7 days treatment and 28 days follow up)
Steady state plasma level and half-life of FBS0701
Time frame: 7 days of dosing
1 secondary outcome
Urinary excretion of iron
Time frame: 7 days of dosing
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Feb 2012.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ellis Neufeld, MD principal investigator
Boston Children's Hospital
5 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing FBS0701 in people with Transfusional Iron Overload and Beta-thalassemia, run by FerroKin BioSciences, Inc. at 5 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Feb 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Australia, Thailand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability by AEs, Physical Exam, Clinical Laboratory assessments (time frame: 35 days (7 days treatment and 28 days follow up)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Feb 2012.