Stem Cell TransplantationPhase 1NCT00597441
Phase I Study of Umbilical Cord Blood Transplantation Followed by Third Party Thymus Transplantation
A Phase 1 study testing Thymic Transplantation for Recipients of UCB transplant in people with Stem Cell Transplantation, run by Sumitomo Pharma Switzerland GmbH at 1 site in United States.
Trial summary
The objective of this trial is to assess the toxicity of thymus transplantation following unrelated umbilical cord blood transplantation. Emphasis will be placed on adverse events that are not typically associated with umbilical cord blood transplantation. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Stem Cell Transplantation
- What is being tested?
- Thymic Transplantation for Recipients of UCB transplant
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2005to May 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 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Subjects who received a dual cord blood transplant are eligible and must have achieved donor cord blood chimerism of > 75% in both T-cell and myeloid lineages
- At the time of thymus transplantation, subjects cannot have active grade II-IV acute graft versus host disease.
- At the time of thymus transplantation, the subject must have an absolute neutrophil count of > 500/ul and a platelet count of 50,000/ul (even if achieved via transfusion).
- At the time of thymus transplantation, the subject must have < or equal to 50 circulating CD 34+ cells/ul (as measured within 3 weeks of transplant date).
- At the time of thymus transplantation, subjects cannot be on greater than 2 mg/kg/day methylprednisolone or its equivalent of steroids.
- At the time of thymus transplantation, PT and PTT must be less that 1.5 times the ULN or must be correctable to this level.
- At the time of thymus transplantation, subjects cannot require oxygen or have pulmonary disease that would be thought to make general anesthesia a high risk.
- Subjects must be between the ages of 18 and 60 years of age.
Who cannot join
- Subjects cannot have bone marrow flow cytometry demonstrating residual leukemia
- Subjects cannot have insufficient subcutaneous tissue and muscle mass to be able to accept and heal a thymus transplant, as assessed by a surgeon.
- Prior to thymus transplantation, subjects cannot have evidence of active disease with HHV6, West Nile Virus, EBV, and CMV.
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Health System
Durham, North Carolina, 27710
About this study
The objective of this trial is to assess the toxicity of thymus transplantation following unrelated umbilical cord blood transplantation. Emphasis will be placed on adverse events that are not typically associated with umbilical cord blood transplantation. Also, to determine whether engraftment of a third party thymus allograft is feasible in patients who have undergone unrelated umbilical cord blood transplantation.
Read the detailed description
The objective of this trial is to assess the toxicity of thymus transplantation following unrelated umbilical cord blood transplantation. Emphasis will be placed on adverse events that are not typically associated with umbilical cord blood transplantation. Also, to determine whether engraftment of a third party thymus allograft is feasible in patients who have undergone unrelated umbilical cord blood transplantation. Thymic engraftment will be determined by biopsy of the thymus allograft and immunohistochemical evidence of thymopoiesis. In addition, we would like to assess thymic function by measuring the number of naive T cells and the number of T cell receptor rearrangement excision circles (TRECS), and by assessing the diversity of the T cell receptor beta chain. These data will be compared to age, stem-cell donor source and disease matched historical controls whose stem cell transplants were not followed by a thymus transplant. This will be done in descriptive fashion. Other immune parameters will be followed including T, B, and NK cell numbers, T cell responses to mitogens, antigens, and immunoglobulin production.
- Conditions
- Stem Cell Transplantation
- Being tested
- Thymic Transplantation for Recipients of UCB transplant
- Keywords
- unrelated, umbilical cord blood transplantation, thymus transplantation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thymic Transplantation for Recipients of UCB transplantBiological
Subjects with hematologic malignancies or bone marrow failure disorders will undergo umbilical cord blood transplantation and two months post transplant will have thymic tissue from a third party donor transplanted into the thigh muscle.
| Study group | Role | What they receive |
|---|---|---|
| I | Experimental | Thymic tissue from third party donor Receives: Biological: Thymic Transplantation for Recipients of UCB transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Toxicity of thymus transplantation following unrelated umbilical cord blood transplantation. Emphasis will be placed on adverse events that are not typically associated with umbilical cord blood transplantation.
Measured: 5 years
Secondary outcomes (2)
To determine whether engraftment of a third party thymus allograft is feasible in patients who have undergone unrelated UCBT. Thymic engraftment will be determined by biopsy of the thymus allograft and immunohistochemical evidence of thymopoiesis.
Measured: 5 years
To assess thymic function by measuring the number of naive T cells and the number of T cell receptor rearrangement excision circles (TRECS), and by assessing the diversity of the T cell receptor beta chain.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2008 (estimated)
- Study startNov 2005
- Primary completionMay 2010
- Study completionMay 2010
Why it stopped: accrual of evaluable subjects too low
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stem Cell TransplantationStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mitchell Horwitz, MD, Principal investigator, Duke HealthMary L Markert, MD, Principal investigator, Duke Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00597441
- Sponsor's protocol ID
- Pro00007995
- Status last verified
- Mar 2022
- First submitted
- 27 Dec 2007
- Last updated
- 1 Apr 2022
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Umbilical Cord Blood Transplantation Followed by Third Party Thymus Transplantation
- ClinicalTrials.gov ID
- NCT00597441
- Acronym / sponsor ID
- Pro00007995
- Conditions
- Stem Cell Transplantation
- Keywords
- unrelated, umbilical cord blood transplantation, thymus transplantation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: accrual of evaluable subjects too low
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Sumitomo Pharma Switzerland GmbHIndustry (pharma, biotech, devices)
- Study start
- Nov 2005
- Primary completion
- May 2010 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 18 Jan 2008 (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 objective of this trial is to assess the toxicity of thymus transplantation following unrelated umbilical cord blood transplantation. Emphasis will be placed on adverse events that are not typically associated with umbilical cord blood transplantation. Also, to determine whether engraftment of a third party thymus allograft is feasible in patients who have undergone unrelated umbilical cord blood transplantation.
Detailed description
The objective of this trial is to assess the toxicity of thymus transplantation following unrelated umbilical cord blood transplantation. Emphasis will be placed on adverse events that are not typically associated with umbilical cord blood transplantation. Also, to determine whether engraftment of a third party thymus allograft is feasible in patients who have undergone unrelated umbilical cord blood transplantation. Thymic engraftment will be determined by biopsy of the thymus allograft and immunohistochemical evidence of thymopoiesis. In addition, we would like to assess thymic function by measuring the number of naive T cells and the number of T cell receptor rearrangement excision circles (TRECS), and by assessing the diversity of the T cell receptor beta chain. These data will be compared to age, stem-cell donor source and disease matched historical controls whose stem cell transplants were not followed by a thymus transplant. This will be done in descriptive fashion. Other immune parameters will be followed including T, B, and NK cell numbers, T cell responses to mitogens, antigens, and immunoglobulin production.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| I | Experimental | Biological: Thymic Transplantation for Recipients of UCB transplantThymic tissue from third party donor |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- Subjects who received a dual cord blood transplant are eligible and must have achieved donor cord blood chimerism of > 75% in both T-cell and myeloid lineages
- At the time of thymus transplantation, subjects cannot have active grade II-IV acute graft versus host disease.
- At the time of thymus transplantation, the subject must have an absolute neutrophil count of > 500/ul and a platelet count of 50,000/ul (even if achieved via transfusion).
- At the time of thymus transplantation, the subject must have < or equal to 50 circulating CD 34+ cells/ul (as measured within 3 weeks of transplant date).
- At the time of thymus transplantation, subjects cannot be on greater than 2 mg/kg/day methylprednisolone or its equivalent of steroids.
Key exclusion criteria (3 listed)
- Subjects cannot have bone marrow flow cytometry demonstrating residual leukemia
- Subjects cannot have insufficient subcutaneous tissue and muscle mass to be able to accept and heal a thymus transplant, as assessed by a surgeon.
- Prior to thymus transplantation, subjects cannot have evidence of active disease with HHV6, West Nile Virus, EBV, and CMV.
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 |
|---|---|---|
| Thymic Transplantation for Recipients of UCB transplantSubjects with hematologic malignancies or bone marrow failure disorders will undergo umbilical cord blood transplantation and two months post transplant will have thymic tissue from a third party donor transplanted into the thigh muscle. | Biological | I |
Primary outcome
Toxicity of thymus transplantation following unrelated umbilical cord blood transplantation. Emphasis will be placed on adverse events that are not typically associated with umbilical cord blood transplantation.
Time frame: 5 years
2 secondary outcomes
To determine whether engraftment of a third party thymus allograft is feasible in patients who have undergone unrelated UCBT. Thymic engraftment will be determined by biopsy of the thymus allograft and immunohistochemical evidence of thymopoiesis.
Time frame: 5 years
To assess thymic function by measuring the number of naive T cells and the number of T cell receptor rearrangement excision circles (TRECS), and by assessing the diversity of the T cell receptor beta chain.
Time frame: 5 years
Results and safety
The study was stopped early (reason given: accrual of evaluable subjects too low). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2022.
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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.
Mitchell Horwitz, MD principal investigator
Duke Health
Mary L Markert, MD principal investigator
Duke Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Thymic Transplantation for Recipients of UCB transplant in people with Stem Cell Transplantation, run by Sumitomo Pharma Switzerland GmbH 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 8 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: stopped early, as of the sponsor's last update on 1 Apr 2022. 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 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 was stopped early (reason given: accrual of evaluable subjects too low). Results are not available in the source record.
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?
Toxicity of thymus transplantation following unrelated umbilical cord blood transplantation. Emphasis will be placed on adverse events that are not typically associated with umbilical cord blood transplantation. (time frame: 5 years). 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 1 Apr 2022.