Thalassemia MajorNCT00480506
Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thalassemia Major
An observational study of Thalassemia Major, run by Dana-Farber Cancer Institute at 2 sites in 2 countries.
Trial summary
The purpose of this study is to collect peripheral blood and bone marrow aspirate samples from thalassemia patients in Tehran, in a collaborative effort to develop an erythroid lineage specific chimerism assay applicable to patients with thalassemia. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Thalassemia Major
- How many people
- 40 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Apr 2004to Oct 2005
- Phase
- Observational
- 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
- Healthy volunteers accepted
- Adult, Older adult
Study population: Thalassemia patients being treated at Shariati Hospital in Tehran.
Who may be able to join
- Thalassemia patients being treated at Shariati Hospital in Tehran.
Who cannot join
None listed.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
About this study
The purpose of this study is to collect peripheral blood and bone marrow aspirate samples from thalassemia patients in Tehran, in a collaborative effort to develop an erythroid lineage specific chimerism assay applicable to patients with thalassemia. Development of such an assay would be useful both for identification of the exact mutation causing the disease, as well as for providing a direct method to measure and monitor the kinetics of donor erythropoiesis in this patient population following transplant.
Read the detailed description
- In the first phase of the study we will develop a panel of reagents designed to identify predominant thalassemia mutations in the Iranian population, as well as alternative highly variable erythroid specific polymorphisms. To determine how informative this panel is, these reagents will be applied to samples collected from patients homozygous for the disorder, and compared to patients with thalassemia trait and normal Iranian donors.
- In the second phase, we propose to serially measure and compare erythroid lineage chimerism with overall genomic chimerism following transplant. Samples will be collected from participants before and at 1, 2, 3, 6 and 12 months following transplant and cryopreserved. Peripheral blood from the stem cell donor will also be collected and cryopreserved. Participants will undergo myeloablative or nonmyeloablative transplant.
- Conditions
- Thalassemia Major
- Keywords
- chimerism, allogenic hematopoietic transplantation, assay
- Medical subject headings
- beta-Thalassemia
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2007 (estimated)
- Study startApr 2004
- Primary completionOct 2005
- Study completionOct 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia MajorStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe sponsor's other registered studies.
- 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
- Observational
- Time perspective
- Retrospective
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Tehran University of Medical Sciences
- Responsible party
- Principal investigator, Catherine Wu, MD, Dana-Farber Cancer Institute
- Investigators
- Catherine Wu, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00480506
- Sponsor's protocol ID
- 04-078
- Status last verified
- Feb 2012
- First submitted
- 30 May 2007
- Last updated
- 23 Feb 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thalassemia Major
- ClinicalTrials.gov ID
- NCT00480506
- Acronym / sponsor ID
- 04-078
- Conditions
- Thalassemia Major
- Keywords
- chimerism, allogenic hematopoietic transplantation, assay
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Tehran University of Medical Sciences
- Study start
- Apr 2004
- Primary completion
- Oct 2005 (actual)
- Study completion
- Oct 2005 (actual)
- First posted
- 31 May 2007 (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 study is to collect peripheral blood and bone marrow aspirate samples from thalassemia patients in Tehran, in a collaborative effort to develop an erythroid lineage specific chimerism assay applicable to patients with thalassemia. Development of such an assay would be useful both for identification of the exact mutation causing the disease, as well as for providing a direct method to measure and monitor the kinetics of donor erythropoiesis in this patient population following transplant.
Detailed description
- In the first phase of the study we will develop a panel of reagents designed to identify predominant thalassemia mutations in the Iranian population, as well as alternative highly variable erythroid specific polymorphisms. To determine how informative this panel is, these reagents will be applied to samples collected from patients homozygous for the disorder, and compared to patients with thalassemia trait and normal Iranian donors.
- In the second phase, we propose to serially measure and compare erythroid lineage chimerism with overall genomic chimerism following transplant. Samples will be collected from participants before and at 1, 2, 3, 6 and 12 months following transplant and cryopreserved. Peripheral blood from the stem cell donor will also be collected and cryopreserved. Participants will undergo myeloablative or nonmyeloablative transplant.
Study design
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Thalassemia patients being treated at Shariati Hospital in Tehran.
Key inclusion criteria (1 listed)
- Thalassemia patients being treated at Shariati Hospital in Tehran.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
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 23 Feb 2012.
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
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Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
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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.
Catherine Wu, MD principal investigator
Dana-Farber Cancer Institute
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thalassemia Major, run by Dana-Farber Cancer Institute at 2 sites in 2 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 1 inclusion and 0 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 23 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 2 sites in United States, Iran. 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.
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.
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 23 Feb 2012.