Severe ThalassemiaPhase 2URTHNCT01005576
Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
A Phase 2 study testing Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan in people with Severe Thalassemia, run by Washington University School of Medicine at 18 sites in United States.
Trial summary
This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects. Read more about this study
- Who is it for?
- Ages 1 year to 16 years
- Conditions the study is for
- Severe Thalassemia
- What is being tested?
- Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 18 sitesUnited States
- Study dates
- Jan 2010to Jul 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 1 year to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 1-16.00 years old
- Have transfusion dependent thalassemia major
- Shall not have an HLA-matched family donor
- Must have a suitably matched unrelated marrow donor or UCB product
- Lansky score >/= 70
- Adequate pulmonary, renal, liver, and other organ function as defined in protocol
Show all 9Show fewer
- Negative pregnancy test
- Adequate total nucleated cell or CD34+ dose of product as defined in protocol
- Iron chelation must be discontinued >/= 48 hours prior to conditioning regimen
Who cannot join
- Pregnant or breastfeeding
- HIV positive
- Prior allogeneic marrow or stem cell transplantation
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
United States18 sites
University of Michigan
Ann Arbor, Michigan, 48109
Emory University
Atlanta, Georgia, 30322
Dana Farber Cancer Institute
Boston, Massachusetts, 02115
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Children's Memorial Hospital
Chicago, Illinois, 60614
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, 43205
UT Southwestern Medical Center
Dallas, Texas, 75390
Duke University
Durham, North Carolina, 27710
Regents of University of California- UCLA
Los Angeles, California, 90095
University of Miami
Miami, Florida, 33136
Vanderbilt University
Nashville, Tennessee, 37232
Children's Hospital and Research Center at Oakland
Oakland, California, 94609
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Oregon Health and Science University
Portland, Oregon, 97239
Methodist Healthcare System of San Antonio
San Antonio, Texas, 78229
Washington University
St Louis, Missouri, 63110
All Children's Research Institute, Inc.
St. Petersburg, Florida, 33701
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children With Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial)
This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects.
This study includes a preparative regimen with Hydroxyurea, Alemtuzumab, Fludarabine, Thiotepa and Melphalan that provides intense host immunosuppression without myeloablation. The primary hypothesis is that this regimen will promote stable engraftment of unrelated donor hematopoietic cells, support normal erythropoiesis, and result in an event free survival of > 75% of children with thalassemia major.
- Conditions
- Severe Thalassemia
- Being tested
- Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
- Keywords
- Thalassemia, Alemtuzumab, Hematopoietic cell transplant, non-myeloablative
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalanDrug
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
| Study group | Role | What they receive |
|---|---|---|
| Conditioning regimen | Experimental | Hydroxyurea days -50 to -21 Alemtuzumab days -21 to -19 Fludarabine days -8 to -4 Thiotepa day -4 Melphalan day -3 Stem cell infusion day 0 Receives: Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Objective: Event-free Survival at 1 Year.
Measured: 1 year
Secondary outcomes (4)
Development of Graft Versus Host Disease (GVHD)
Measured: 2 years
Median Time to ANC Engraftment
Measured: 100 days
Median Time to Platelet Engraftment
Measured: 100 days
Incidence of Disease Recurrence
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2009 (estimated)
- Study startJan 2010
- Primary completionApr 2014
- Study completionJul 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Severe ThalassemiaStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Carelon Research, Pediatric Blood and Marrow Transplant Consortium
- Responsible party
- Sponsor
- Investigators
- Shalini Shenoy, MD, Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01005576
- Sponsor's protocol ID
- TCRN-NMD 0901
- Acronym
- URTH
- Status last verified
- Nov 2017
- First submitted
- 29 Oct 2009
- Last updated
- 13 Dec 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children With Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial)
- ClinicalTrials.gov ID
- NCT01005576
- Acronym / sponsor ID
- URTH / TCRN-NMD 0901
- Conditions
- Severe Thalassemia
- Keywords
- Thalassemia, Alemtuzumab, Hematopoietic cell transplant, non-myeloablative
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- Carelon Research, Pediatric Blood and Marrow Transplant Consortium
- Study start
- Jan 2010
- Primary completion
- Apr 2014 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 2 Nov 2009 (estimated)
- Results
- Posted 20 Oct 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects.
This study includes a preparative regimen with Hydroxyurea, Alemtuzumab, Fludarabine, Thiotepa and Melphalan that provides intense host immunosuppression without myeloablation. The primary hypothesis is that this regimen will promote stable engraftment of unrelated donor hematopoietic cells, support normal erythropoiesis, and result in an event free survival of > 75% of children with thalassemia major.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Conditioning regimen | Experimental | Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalanHydroxyurea days -50 to -21 Alemtuzumab days -21 to -19 Fludarabine days -8 to -4 Thiotepa day -4 Melphalan day -3 Stem cell infusion day 0 |
Who may qualify
- Ages
- 1 year to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 1-16.00 years old
- Have transfusion dependent thalassemia major
- Shall not have an HLA-matched family donor
- Must have a suitably matched unrelated marrow donor or UCB product
- Lansky score >/= 70
Key exclusion criteria (3 listed)
- Pregnant or breastfeeding
- HIV positive
- Prior allogeneic marrow or stem cell transplantation
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 |
|---|---|---|
| Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalanDays -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion | Drug | Conditioning regimen |
Primary outcome
Primary Objective: Event-free Survival at 1 Year.
Time frame: 1 year
4 secondary outcomes
Development of Graft Versus Host Disease (GVHD)
Time frame: 2 years
Median Time to ANC Engraftment
Time frame: 100 days
Median Time to Platelet Engraftment
Time frame: 100 days
Incidence of Disease Recurrence
Time frame: 2 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Dec 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Shalini Shenoy, MD principal investigator
Washington University School of Medicine
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan in people with Severe Thalassemia, run by Washington University School of Medicine at 18 sites 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 9 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 13 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites 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 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?
Yes. Results are posted in the registry (first posted 20 Oct 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Primary Objective: Event-free Survival at 1 Year. (time frame: 1 year). 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 13 Dec 2017.