Graft FailurePhase 2NCT06872333
Allo HSCT for High Risk Hemoglobinopathies
A Phase 2 study testing Alemtuzumab, Total Body Irradiation and Cell Infusion in people with Graft Failure and Sickle Cell Disease, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens. Read more about this study
- Who is it for?
- Ages Up to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Graft Failure, Sickle Cell Disease, Hemoglobinopathies
- What is being tested?
- Alemtuzumab, Total Body Irradiation and Cell Infusion
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 62 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Nov 2024to 1 Jun 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Sickle Cell Disease (SCD)
- SCD Patients with a fully matched sibling donor (MSD) irrespective of the frequency or severity of symptoms MSD transplant can be considered. Parents/patient must be counseled as to the risks and benefits and provide their voluntary informed consent
- Transfusion Dependent Alpha- or Beta- Thalassemia
- Diamond Blackfan Anemia
- Other Non-Malignant Hematologic Disorders
- Karnofsky ≥ 60%, Lansky play score ≥ 60. Patients with lower performance score can be considered based on study team's evaluation.
- Sexually active persons of childbearing potential or persons with partners of childbearing potential must agree to use a highly effective form of contraception during study treatment and for at least 4 months after the transplant.
Who cannot join
- Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 30 days of the start of treatment
- HIV infection with a detectable viral load. All HIV+ patients must be evaluated by infectious disease (ID) and an HIV management plan established prior to transplantation.
- Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted
- Known allergy to any of the study components
- Psychiatric illness/social situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements
- Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Masonic Cancer Center
Minneapolis, Minnesota, 55455
Ashish Gupta, MBBS, MPH
About this study
Official title: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.
- Conditions
- Graft Failure, Sickle Cell Disease, Hemoglobinopathies
- Being tested
- Alemtuzumab, Total Body Irradiation, Cell Infusion, Thymoglobulin, Fludarabine
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobinopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlemtuzumabDrug
Also called Campath
Alemtuzumab (Campath) will be administered IV over 2 hours on day -8 to day -4.
Total Body IrradiationRadiation
Also called TBI
400 cGy in 2 split fractions will be administered per Department of RadiationOncology SOPs.
Cell InfusionBiological
On day 0 the cells will be infused per cell source specific institutional guidelines
ThymoglobulinDrug
Also called Rabbit ATG
ATG will be administered IV every 24 hours beginning on day -8 for all patients.
Dosing will be model-based using Bayesian methodology13,14,15. Total doses and total number of doses (1-4 doses) will be determined based on absolute lymphocyte count and weight.
FludarabineDrug
Fludarabine will be administered IV over 1 hour every 24 hours on day -5 to day - 2. The daily dose of fludarabine will be determined by model-based dosing utilizing Bayesian methodology with a cumulative area under the curve (cAUC) of 20 mg*hr/L (range 18-22 mg*hr/L).
BusulfanDrug
Busulfan dosing and administration and therapeutic drug monitoring (TDM) per institutional guidelines. Initial busulfan dosing will be determined by model-based dosing utilizing Bayesian methods with a cumulative area under the curve (cAUC) of 75 mg*hr/L.
CyclophosphamideDrug
Also called Cyclophosphamide with MESNA
Cyclophosphamide will be administered at a dose of 14.5 mg/kg over 2 hours IV daily on days -6 and -5. Cyclophosphamide dosing is calculated based on actual body weight (ABW).
For Arm D - Cyclophosphamide 50 mg/kg IV will be administered over 2 hours on days +3 and
+4. Cyclophosphamide dosing for post-transplant is calculated based on ideal body weight (IBW) unless patient weighs less than IBW, in which case actual body weight (ABW) will be used.
SirolimusDrug
Patients on Arm A and Arm D will receive sirolimus; beginning on day -3 and continuing until day +180 for patients on Arm A or beginning on day +5 and continuing until 1 year post transplant for patients on Arm D.
TacrolimusDrug
Patients on Arm B and Arm C will receive tacrolimus, beginning on day -3 and continuing until day +180. Tacrolimus dosing and monitoring will be per institutional guidelines.
Also called (MMF)
MMF will begin on day -3 (Arm A, B \& C) or day +5 (Arm D). Patients treated on adult service will receive 15 mg/kg (max 1500 mg/dose) given every 12 hours, rounded to nearest 250 mg. Patients on pediatric service will receive 15 mg/kg (max 1000 mg/dose) given every 8 hours. MMF dosing will be monitored and altered as clinically appropriate based on institutional guidelines. MMF will be stopped at day +30 (Arms A, B \& C) or day +35 (Arm D) or 7 days after engraftment, whichever day is later, if no acute GVHD.
Plerixafor (mozobil)Drug
Plerixafor will beused to significantly increase stem cell yields on a second collection day compared to donors who continued mobilization on G-CSF only.
| Study group | Role | What they receive |
|---|---|---|
| Arm A - Closed | Experimental | Arm A Matched sib regimen - Age 6 -55 (per physician preference for patients over 6) Campath/TBI Receives: Drug: Alemtuzumab; Radiation: Total Body Irradiation; Biological: Cell Infusion; Drug: Sirolimus; Drug: Mycophenolate Mofetil |
| Arm B | Experimental | Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu Receives: Biological: Cell Infusion; Drug: Thymoglobulin; Drug: Fludarabine; Drug: Busulfan; Drug: Tacrolimus; Drug: Mycophenolate Mofetil |
| Arm C | Experimental | Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu Receives: Biological: Cell Infusion; Drug: Thymoglobulin; Drug: Fludarabine; Drug: Busulfan; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Drug: Plerixafor (mozobil) |
| Arm D | Experimental | Arm D: Haploindentical or mismatched unrelated donors (MMUD) - 0-55 years; ATG/Thiotepa/Cyclophosphamide/MESNA/Flu/TBI Receives: Drug: Sirolimus; Drug: Tacrolimus; Drug: Plerixafor (mozobil) |
| Arm E | Experimental | Matched sib regimen - Age 6-55 (per physician preference for patients over 6) Campath/TBI with peripheral blood stem cell graft Receives: Radiation: Total Body Irradiation; Biological: Cell Infusion; Drug: Thymoglobulin; Drug: Fludarabine; Drug: Cyclophosphamide; Drug: Sirolimus; Drug: Tacrolimus; Drug: Mycophenolate Mofetil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Graft versus Host Disease (GvHD)
Measured: 1 year
Secondary outcomes (4)
Overall Survival
Overall survival post HCT
Measured: 1 and 2 years
Grade 3-4 Acute GvHD
Grade 3-4 acute Graft versus Host Disease (GvHD) at 2 years post HCT.
Measured: 2 years
Chronic Graft versus Host Disease (GvHD) Free
Chronic Graft versus Host Disease (GvHD) Free at 2 years post HCT.
Measured: 2 years
Failure Free Survival
Failure Free Survival 2 years post HCT.
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 posted12 Mar 2025
- Study start19 Nov 2024
- Primary completion1 Jun 2030 (estimated)
- Study completion1 Jun 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft FailureStudies 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 2
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 62 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06872333
- Sponsor's protocol ID
- 2024LS140
- Status last verified
- Jun 2026
- First submitted
- 5 Mar 2025
- Last updated
- 4 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
- ClinicalTrials.gov ID
- NCT06872333
- Acronym / sponsor ID
- 2024LS140
- Conditions
- Graft Failure, Sickle Cell Disease, Hemoglobinopathies
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 62 participants (estimated)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 19 Nov 2024 (actual)
- Primary completion
- 1 Jun 2030 (estimated)
- Study completion
- 1 Jun 2032 (estimated)
- First posted
- 12 Mar 2025 (actual)
- 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 single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Arm A - Closed | Experimental | Drug: Alemtuzumab, Radiation: Total Body Irradiation, Biological: Cell Infusion, Drug: Sirolimus, Drug: Mycophenolate MofetilArm A Matched sib regimen - Age 6 -55 (per physician preference for patients over 6) Campath/TBI |
| Arm B | Experimental | Biological: Cell Infusion, Drug: Thymoglobulin, Drug: Fludarabine, Drug: Busulfan, Drug: Tacrolimus, Drug: Mycophenolate MofetilArm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu |
| Arm C | Experimental | Biological: Cell Infusion, Drug: Thymoglobulin, Drug: Fludarabine, Drug: Busulfan, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Drug: Plerixafor (mozobil)Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu |
| Arm D | Experimental | Drug: Sirolimus, Drug: Tacrolimus, Drug: Plerixafor (mozobil)Arm D: Haploindentical or mismatched unrelated donors (MMUD) - 0-55 years; ATG/Thiotepa/Cyclophosphamide/MESNA/Flu/TBI |
| Arm E | Experimental | Radiation: Total Body Irradiation, Biological: Cell Infusion, Drug: Thymoglobulin, Drug: Fludarabine, Drug: Cyclophosphamide, Drug: Sirolimus, Drug: Tacrolimus, Drug: Mycophenolate MofetilMatched sib regimen - Age 6-55 (per physician preference for patients over 6) Campath/TBI with peripheral blood stem cell graft |
Who may qualify
- Ages
- Up to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Sickle Cell Disease (SCD)
- SCD Patients with a fully matched sibling donor (MSD) irrespective of the frequency or severity of symptoms MSD transplant can be considered. Parents/patient must be counseled as to the risks and benefits and provide their voluntary informed consent
- Transfusion Dependent Alpha- or Beta- Thalassemia
- Diamond Blackfan Anemia
- Other Non-Malignant Hematologic Disorders
Key exclusion criteria (6 listed)
- Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 30 days of the start of treatment
- HIV infection with a detectable viral load. All HIV+ patients must be evaluated by infectious disease (ID) and an HIV management plan established prior to transplantation.
- Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted
- Known allergy to any of the study components
- Psychiatric illness/social situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements
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 |
|---|---|---|
| AlemtuzumabAlso: CampathAlemtuzumab (Campath) will be administered IV over 2 hours on day -8 to day -4. | Drug | Arm A - Closed |
| Total Body IrradiationAlso: TBI400 cGy in 2 split fractions will be administered per Department of RadiationOncology SOPs. | Radiation | Arm A - Closed, Arm E |
| Cell InfusionOn day 0 the cells will be infused per cell source specific institutional guidelines | Biological | Arm A - Closed, Arm B, Arm C, Arm E |
| ThymoglobulinAlso: Rabbit ATGATG will be administered IV every 24 hours beginning on day -8 for all patients. Dosing will be model-based using Bayesian methodology13,14,15. Total doses and total number of doses (1-4 doses) will be determined based on absolute lymphocyte count and weight. | Drug | Arm B, Arm C, Arm E |
| FludarabineFludarabine will be administered IV over 1 hour every 24 hours on day -5 to day - 2. The daily dose of fludarabine will be determined by model-based dosing utilizing Bayesian methodology with a cumulative area under the curve (cAUC) of 20 mg*hr/L (range 18-22 mg*hr/L). | Drug | Arm B, Arm C, Arm E |
| BusulfanBusulfan dosing and administration and therapeutic drug monitoring (TDM) per institutional guidelines. Initial busulfan dosing will be determined by model-based dosing utilizing Bayesian methods with a cumulative area under the curve (cAUC) of 75 mg*hr/L. | Drug | Arm B, Arm C |
| CyclophosphamideAlso: Cyclophosphamide with MESNACyclophosphamide will be administered at a dose of 14.5 mg/kg over 2 hours IV daily on days -6 and -5. Cyclophosphamide dosing is calculated based on actual body weight (ABW). For Arm D - Cyclophosphamide 50 mg/kg IV will be administered over 2 hours on days +3 and +4. Cyclophosphamide dosing for post-transplant is calculated based on ideal body weight (IBW) unless patient weighs less than IBW, in which case actual body weight (ABW) will be used. | Drug | Arm E |
| SirolimusPatients on Arm A and Arm D will receive sirolimus; beginning on day -3 and continuing until day +180 for patients on Arm A or beginning on day +5 and continuing until 1 year post transplant for patients on Arm D. | Drug | Arm A - Closed, Arm D, Arm E |
| TacrolimusPatients on Arm B and Arm C will receive tacrolimus, beginning on day -3 and continuing until day +180. Tacrolimus dosing and monitoring will be per institutional guidelines. | Drug | Arm B, Arm C, Arm D, Arm E |
| Mycophenolate MofetilAlso: (MMF)MMF will begin on day -3 (Arm A, B \& C) or day +5 (Arm D). Patients treated on adult service will receive 15 mg/kg (max 1500 mg/dose) given every 12 hours, rounded to nearest 250 mg. Patients on pediatric service will receive 15 mg/kg (max 1000 mg/dose) given every 8 hours. MMF dosing will be monitored and altered as clinically appropriate based on institutional guidelines. MMF will be stopped at day +30 (Arms A, B \& C) or day +35 (Arm D) or 7 days after engraftment, whichever day is later, if no acute GVHD. | Drug | Arm A - Closed, Arm B, Arm C, Arm E |
| Plerixafor (mozobil)Plerixafor will beused to significantly increase stem cell yields on a second collection day compared to donors who continued mobilization on G-CSF only. | Drug | Arm C, Arm D |
Primary outcome
Incidence of Graft versus Host Disease (GvHD)
Time frame: 1 year
4 secondary outcomes
Overall Survival
Time frame: 1 and 2 years
Grade 3-4 Acute GvHD
Time frame: 2 years
Chronic Graft versus Host Disease (GvHD) Free
Time frame: 2 years
Failure Free Survival
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2026.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Alemtuzumab, Total Body Irradiation and Cell Infusion in people with Graft Failure and Sickle Cell Disease, 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 7 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 4 Jun 2026. 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 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ashish Gupta, MBBS, MPH (612-626-2961, gupta461@umn.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Graft versus Host Disease (GvHD) (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 4 Jun 2026.