Sickle Cell DiseaseNCT02179359
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
A study testing Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen and Myeloablative Preparative Regimen in people with Sickle Cell Disease and Transfusion Dependent Alpha- or Beta- Thalassemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer. Read more about this study
- Who is it for?
- Ages Up to 55 years
- Conditions the study is for
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia
- What is being tested?
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen and Myeloablative Preparative Regimen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Sep 2014to 21 Nov 2024
- Phase
- Interventional
- 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 Up to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
- Acceptable stem cell source identified
- Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)
- Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
- Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase <5 times the upper limit of institutional normal
- Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%
Who cannot join
- active, uncontrolled infection
- pregnant or breastfeeding
- HIV positive
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Medical Center, Fairview
Minneapolis, Minnesota, 55455
About this study
Official title: MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia
- Being tested
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
- Keywords
- Stem Cell Transplant
- Medical subject headings
- Anemia, Sickle Cell, beta-Thalassemia, Anemia, Diamond-Blackfan, Hemoglobinuria, Paroxysmal, Thrombasthenia, Neutropenia, Severe Congenital, Autosomal Recessive 3, Shwachman-Diamond Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Reduced Toxicity Ablative RegimenDrug
- Anti-thymocyte Globulin (ATG)
- Fludarabine
- Busulfan
- Stem Cell Infusion Day 0
Reduced Intensity Preparative RegimenDrug
- Alemtuzumab
- Cyclophosphamide
- Fludarabine
- Total Body Irradiation (TBI)
- Stem Cell Infusion Day 0
Myeloablative Preparative RegimenDrug
- Alemtuzumab
- Cyclophosphamide
- Busulfan
- Stem Cell Infusion Day 0
| Study group | Role | What they receive |
|---|---|---|
| Reduced Toxicity Ablative Regimen | Experimental | For use in patients with a matched sibling donor or unrelated UCB donor and DBA patients who are <12 years and/or have mild/moderate iron exposure. Receives: Drug: Reduced Toxicity Ablative Regimen |
| Reduced Intensity Preparative Regimen | Experimental | For use in patients with unrelated donor bone marrow and for DBA patients who are >12 years and/or have significant iron exposure. Receives: Drug: Reduced Intensity Preparative Regimen |
| Myeloablative Preparative Regimen | Experimental | For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia. Receives: Drug: Myeloablative Preparative Regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
incidence of graft failure
Measured: 42 days
Secondary outcomes (2)
overall survival
Measured: 6 months, 1 and 2 years
disease free survival
patient no longer needing red blood cell transfusion and/or a hemoglobin S level at that of the donor ( sickle cell disease only)
Measured: 6 months, 1 and 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2014 (estimated)
- Study start2 Sep 2014
- Primary completion21 Nov 2024
- Study completion21 Nov 2024
Why it stopped: Replaced by another study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ashish Gupta, MBBS, MPH, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02179359
- Sponsor's protocol ID
- 2014OC034
- Other IDs
- MT2014-10C
- Status last verified
- Feb 2025
- First submitted
- 27 Jun 2014
- Last updated
- 3 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
- ClinicalTrials.gov ID
- NCT02179359
- Acronym / sponsor ID
- 2014OC034
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia
- Keywords
- Stem Cell Transplant
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Replaced by another study
- Enrolment
- 38 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 2 Sep 2014
- Primary completion
- 21 Nov 2024 (actual)
- Study completion
- 21 Nov 2024 (actual)
- First posted
- 1 Jul 2014 (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.
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Reduced Toxicity Ablative Regimen | Experimental | Drug: Reduced Toxicity Ablative RegimenFor use in patients with a matched sibling donor or unrelated UCB donor and DBA patients who are <12 years and/or have mild/moderate iron exposure. |
| Reduced Intensity Preparative Regimen | Experimental | Drug: Reduced Intensity Preparative RegimenFor use in patients with unrelated donor bone marrow and for DBA patients who are >12 years and/or have significant iron exposure. |
| Myeloablative Preparative Regimen | Experimental | Drug: Myeloablative Preparative RegimenFor use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia. |
Who may qualify
- Ages
- Up to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
- Acceptable stem cell source identified
- Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)
- Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
- Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase <5 times the upper limit of institutional normal
Key exclusion criteria (3 listed)
- active, uncontrolled infection
- pregnant or breastfeeding
- HIV positive
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 |
|---|---|---|
| Reduced Toxicity Ablative Regimen* Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan * Stem Cell Infusion Day 0 | Drug | Reduced Toxicity Ablative Regimen |
| Reduced Intensity Preparative Regimen* Alemtuzumab * Cyclophosphamide * Fludarabine * Total Body Irradiation (TBI) * Stem Cell Infusion Day 0 | Drug | Reduced Intensity Preparative Regimen |
| Myeloablative Preparative Regimen* Alemtuzumab * Cyclophosphamide * Busulfan * Stem Cell Infusion Day 0 | Drug | Myeloablative Preparative Regimen |
Primary outcome
incidence of graft failure
Time frame: 42 days
2 secondary outcomes
overall survival
Time frame: 6 months, 1 and 2 years
disease free survival
Time frame: 6 months, 1 and 2 years
Results and safety
The study was stopped early (reason given: Replaced by another study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2025.
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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.
Ashish Gupta, MBBS, MPH principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen and Myeloablative Preparative Regimen in people with Sickle Cell Disease and Transfusion Dependent Alpha- or Beta- Thalassemia, run by Masonic Cancer Center, University of Minnesota 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: stopped early, as of the sponsor's last update on 3 Mar 2025. 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.
Are results available for this study?
The study was stopped early (reason given: Replaced by another study). 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?
incidence of graft failure (time frame: 42 days). 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 3 Mar 2025.