Beta-ThalassemiaPhase 1/2NCT00069862
Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
A Phase 1/2 study testing Deferitrin (GT56-252) and desferoxamine (DFO) in people with Beta-Thalassemia, run by Genzyme, a Sanofi Company at 1 site in United States.
Trial summary
A clinical trial designed to compare the safety and iron excretion properties of desferoxamine (DFO) and deferitrin (GT56-252), an experimental oral iron chelator. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- Deferitrin (GT56-252) and desferoxamine (DFO)
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2003to Nov 2005
- Phase
- Phase 1/2
- 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
- Beta-thalassemia patients, 18 years of age or older, currently undergoing chronic blood transfusion therapy and iron chelation therapy who weigh more than 40 kg.
- No clinically significant findings on physical exam, medical history, or screening laboratories.
- Serum ferritin greater than 500 ng/mL, serum creatine creatinine within the normal range and platelet count greater than 100,000/mm3.
- Willing and able to discontinue DFO or L1 for the period of study.
- Woman of child-bearing potential must have a negative serum pregnancy test at screening and use a medically acceptable form of birth control during the study and for 1 month afterward. Male patients must also use barrier contraceptives during the study and for 1 month afterward.
- Have a level of understanding and willingness to cooperate with the confinement and all procedures. Able to provide voluntary signed/dated written informed consent.
Who cannot join
- Serious medical condition unrelated to Beta-Thalassemia.
- Participation in a previous investigational drug study within 30 days preceding screening.
- Patients with a known allergy to DFO that prevents chronic administration.
Trial locations and contacts
1 site in 1 country.
United States1 site
The New York Presbyterian Hospital-Weill Medical College of Cornell University
New York, New York, 10021
About this study
A clinical trial designed to compare the safety and iron excretion properties of desferoxamine (DFO) and deferitrin (GT56-252), an experimental oral iron chelator.
- Conditions
- Beta-Thalassemia
- Being tested
- Deferitrin (GT56-252), desferoxamine (DFO)
- Keywords
- Beta Thalassemia
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferitrin (GT56-252)Drug
desferoxamine (DFO)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
safety and tolerability
iron excretion in urine and stool
pharmacokinetic measurements
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2003 (estimated)
- Study startSep 2003
- Study completionNov 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-ThalassemiaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 25 participants
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00069862
- Sponsor's protocol ID
- GTC-134-102
- Status last verified
- Mar 2015
- First submitted
- 2 Oct 2003
- Last updated
- 5 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia
- ClinicalTrials.gov ID
- NCT00069862
- Acronym / sponsor ID
- GTC-134-102
- Conditions
- Beta-Thalassemia
- Keywords
- Beta Thalassemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Sep 2003
- Study completion
- Nov 2005 (actual)
- First posted
- 3 Oct 2003 (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.
A clinical trial designed to compare the safety and iron excretion properties of desferoxamine (DFO) and deferitrin (GT56-252), an experimental oral iron chelator.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Beta-thalassemia patients, 18 years of age or older, currently undergoing chronic blood transfusion therapy and iron chelation therapy who weigh more than 40 kg.
- No clinically significant findings on physical exam, medical history, or screening laboratories.
- Serum ferritin greater than 500 ng/mL, serum creatine creatinine within the normal range and platelet count greater than 100,000/mm3.
- Willing and able to discontinue DFO or L1 for the period of study.
- Woman of child-bearing potential must have a negative serum pregnancy test at screening and use a medically acceptable form of birth control during the study and for 1 month afterward. Male patients must also use barrier contraceptives during the study and for 1 month afterward.
Key exclusion criteria (3 listed)
- Serious medical condition unrelated to Beta-Thalassemia.
- Participation in a previous investigational drug study within 30 days preceding screening.
- Patients with a known allergy to DFO that prevents chronic administration.
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 |
|---|---|---|
| Deferitrin (GT56-252) | Drug | – |
| desferoxamine (DFO) | Drug | – |
Primary outcome
safety and tolerability
iron excretion in urine and stool
pharmacokinetic measurements
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 5 Mar 2015.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Deferitrin (GT56-252) and desferoxamine (DFO) in people with Beta-Thalassemia, run by Genzyme, a Sanofi Company at 1 site in United States.
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 6 inclusion and 3 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 5 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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. The study lists 3 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 5 Mar 2015.