Sickle Cell DiseasePhase 2NCT05736419
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
A Phase 2 study testing Fludarabine, Cyclophosphamide and Tacrolimus in people with Sickle Cell Disease and Thalassemia, Beta, run by Memorial Sloan Kettering Cancer Center at 6 sites in United States.
Trial summary
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. Read more about this study
- Who is it for?
- Ages 2 to 50 years
- Conditions the study is for
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia
- What is being tested?
- Fludarabine, Cyclophosphamide and Tacrolimus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 6 sitesUnited States
- Study dates
- 9 Feb 2023to 9 Feb 2027
- 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 2 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age ≥ 2 and ≤ 50 years
- Suitable haploidentical donor.
- Performance score ≥ 70% by Karnofsky Performance Scale or 0 to 1 by ECOG (age > 16 years), or Lansky Play-Performance Scale ≥ 70% (age ≤ 16 years).
- Adequate major organ system function as demonstrated by:
- For patients ≥ 18 years of age:
- eGFR ≥ 50 mL/min by Cockcroft-Gault formula Formula: ((140 - Age) x Weight (kg)) / (72 x Serum Creatinine (mg/dL) Female Adjustment: Multiply result by 0.85
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- For patients < 18 years of age:
- Serum creatinine clearance: glomerular filtration rate [GFR]) must be >50 mL/min/1.73 m2 as calculated by the Schwartz formula
- Conjugated (direct) bilirubin less than 3x upper limit of normal.
- ALT or AST ≤ 3 times institutional upper limit of normal.
- Left ventricular ejection fraction ≥ 50%.
- Diffusing capacity for carbon monoxide (DLCO) ≥ 50% predicted, corrected for hemoglobin. For children < 7 years of age who are unable to perform PFT, oxygen saturation > 92% on room air by pulse oximetry.
- For SCD patients: HbSS, HbSC, HbS/β° with one or more of the following complications:
- Acute chest syndrome: 2 or more episodes in the 2 years preceding enrollment
- Vaso-occlusive episodes: 3 or more episodes in the 2 years preceding enrollment
- Recurrent priapism: 2 or more episodes in the 2 years preceding enrollment
- History of osteomyelitis or osteonecrosis
- Cerebrovascular disease:
- Imaging evidence of prior overt or silent stroke
- History of a neurologic event resulting in focal neurologic deficits lasting > 24 hours
- Abnormal transcranial Doppler: Timed average maximum mean velocity ≥ 200 cm/sec in terminal portion of the carotid or proximal portion of the middle cerebral artery or > 185 cm/sec plus evidence of intracranial vasculopathy if imaging TCD is used
- Pulmonary hypertension: Confirmed by right heart catheterization with mean pulmonary arterial pressure ≥ 25 mmHg or mean pulmonary vascular resistance > 2 Wood units
- Red blood cell alloimmunization (> 3 alloantibodies)
- For thalassemia patients: Any genotype, with all of the following:
- Onset of red blood cell transfusion dependence during the first 3 years of life
- RBC transfusion history > 225 mL/kg/year or > 15 lifetime RBC transfusions
- Pre-transfusion hemoglobin ≤ 7 g/dL
- Hepatosplenomegaly
- Patient or the patient's legal representative, parent(s) or guardian should be able to provide written informed consent. Assent of a minor if participant's age is at least seven and less than eighteen years.
- For sexually active men and women of childbearing potential, must agree to use a form of contraception considered effective and medically acceptable by the Investigator.
Who cannot join
- Prior myeloablative allogeneic HCT.
- Overt stroke or CNS instrumentation (e.g. for Moyamoya disease) within 6 months of enrollment.
- Liver cirrhosis. Mild fibrosis will be permitted, i.e. fine reticulin or grade 1 of 4, with bridging fibrosis.
- Hepatic iron content ≥ 3 mg Fe/g liver dry weight, if applicable
- Active hepatitis B or C.
- Other uncontrolled infections.
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- Other malignancy/cancer diagnosis unless in remission after definitive therapy for a minimum of 2 years. Exceptions: Ductal carcinoma in situ, basal cell carcinoma, cervical intraepithelial neoplasia.
- Positive pregnancy test in a woman with child-bearing potential, defined as not post-menopausal for 12 months or no previous surgical sterilization.
- Inability to comply with medical therapy or follow-up.
- Known history of allergic reactions to any constituents of the stem cell product, including a known history of allergic reactions to DMSO.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
Memorial Sloan Kettering at Basking Ridge (Consent only)
Basking Ridge, New Jersey, 07920
Maria Cancio, MD, 212-639-2446
- Recruiting
Memorial Sloan Kettering Suffolk - Commack (Consent only)
Commack, New York, 11725
Maria Cancio, MD, 212-639-2446
- Recruiting
Memorial Sloan Kettering Westchester (Consent only)
Harrison, New York, 10604
Maria Cancio, MD, 212-639-2446
- Recruiting
Memorial Sloan Kettering Monmouth (Consent only)
Middletown, New Jersey, 07748
Maria Cancio, MD, 212-639-2446
- Recruiting
Memorial Sloan Kettering Bergen (Consent only)
Montvale, New Jersey, 07645
Maria Cancio, MD, 212-639-2446
- Recruiting
Memorial Sloan Kettering Nassau (All protocol activities)
Rockville Centre, New York, 11553
Maria Cancio, MD, 212-639-2446
About this study
Official title: Pre-Transplant Immune Suppression With Hematopoietic Cell Transplantation From Haploidentical Donors for Adults and Children With Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia
- Being tested
- Fludarabine, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, Rabbit ATG
- Keywords
- allogeneic hematopoietic cell transplantation, Sickle Cell Disease, Beta Thalassemia, Thalassemia, 23-009, Memorial Sloan Kettering Cancer Center
- Medical subject headings
- Anemia, Sickle Cell, beta-Thalassemia, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
PK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform.
CyclophosphamideDrug
Cyclophosphamide will be administered Post-Transplant
TacrolimusDrug
Tacrolimus will be administered beginning on day +5
Mycophenolate mofetil (MMF) will be administered three times daily starting on day +5.
Rabbit ATGBiological
The dose and schedule of ATG will be determined according to the nomogram in Appendix A
DexamethasoneDrug
Standard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36.
BortezomibDrug
Bortezomib on days -71, -68, -65, -61, -43, -40, -37, and -33
RituximabDrug
Rituximab on days -71, -58, -43, and -30.
| Study group | Role | What they receive |
|---|---|---|
| Participants with Sickle Cell Disease or β-Thalassemia | Experimental | Participants will have severe sickle cell disease or transfusion-dependent β-thalassemia. Receives: Drug: Fludarabine; Drug: Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Biological: Rabbit ATG; Drug: Dexamethasone; Drug: Bortezomib; Drug: Rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment related mortality/TRM or primary graft failure
The primary outcome is to estimate treatment-related mortality (TRM) or primary graft failure at 1 year post-HCT.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2023
- Study start9 Feb 2023
- Primary completion9 Feb 2027 (estimated)
- Study completion9 Feb 2027 (estimated)
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 Memorial Sloan Kettering Cancer CenterThe 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
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Maria Cancio, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05736419
- Sponsor's protocol ID
- 23-009
- Status last verified
- Jun 2026
- First submitted
- 9 Feb 2023
- Last updated
- 3 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pre-Transplant Immune Suppression With Hematopoietic Cell Transplantation From Haploidentical Donors for Adults and Children With Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)
- ClinicalTrials.gov ID
- NCT05736419
- Acronym / sponsor ID
- 23-009
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia
- Keywords
- allogeneic hematopoietic cell transplantation, Sickle Cell Disease, Beta Thalassemia, Thalassemia, 23-009, Memorial Sloan Kettering Cancer Center
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Study start
- 9 Feb 2023 (actual)
- Primary completion
- 9 Feb 2027 (estimated)
- Study completion
- 9 Feb 2027 (estimated)
- First posted
- 21 Feb 2023 (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.
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with Sickle Cell Disease or β-Thalassemia | Experimental | Drug: Fludarabine, Drug: Cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Rabbit ATG, Drug: Dexamethasone, Drug: Bortezomib, Drug: RituximabParticipants will have severe sickle cell disease or transfusion-dependent β-thalassemia. |
Who may qualify
- Ages
- 2 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (30 listed)
- Age ≥ 2 and ≤ 50 years
- Suitable haploidentical donor.
- Performance score ≥ 70% by Karnofsky Performance Scale or 0 to 1 by ECOG (age > 16 years), or Lansky Play-Performance Scale ≥ 70% (age ≤ 16 years).
- Adequate major organ system function as demonstrated by:
- Conjugated (direct) bilirubin less than 3x upper limit of normal.
Key exclusion criteria (10 listed)
- Prior myeloablative allogeneic HCT.
- Overt stroke or CNS instrumentation (e.g. for Moyamoya disease) within 6 months of enrollment.
- Liver cirrhosis. Mild fibrosis will be permitted, i.e. fine reticulin or grade 1 of 4, with bridging fibrosis.
- Hepatic iron content ≥ 3 mg Fe/g liver dry weight, if applicable
- Active hepatitis B or C.
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 |
|---|---|---|
| FludarabinePK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform. | Drug | Participants with Sickle Cell Disease or β-Thalassemia |
| CyclophosphamideCyclophosphamide will be administered Post-Transplant | Drug | Participants with Sickle Cell Disease or β-Thalassemia |
| TacrolimusTacrolimus will be administered beginning on day +5 | Drug | Participants with Sickle Cell Disease or β-Thalassemia |
| Mycophenolate MofetilMycophenolate mofetil (MMF) will be administered three times daily starting on day +5. | Drug | Participants with Sickle Cell Disease or β-Thalassemia |
| Rabbit ATGThe dose and schedule of ATG will be determined according to the nomogram in Appendix A | Biological | Participants with Sickle Cell Disease or β-Thalassemia |
| DexamethasoneStandard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36. | Drug | Participants with Sickle Cell Disease or β-Thalassemia |
| BortezomibBortezomib on days -71, -68, -65, -61, -43, -40, -37, and -33 | Drug | Participants with Sickle Cell Disease or β-Thalassemia |
| RituximabRituximab on days -71, -58, -43, and -30. | Drug | Participants with Sickle Cell Disease or β-Thalassemia |
Primary outcome
Number of participants with treatment related mortality/TRM or primary graft failure
Time frame: 1 year
The primary outcome is to estimate treatment-related mortality (TRM) or primary graft failure at 1 year post-HCT.
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 3 Jun 2026.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
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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.
Maria Cancio, MD principal investigator
Memorial Sloan Kettering Cancer Center
6 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 Fludarabine, Cyclophosphamide and Tacrolimus in people with Sickle Cell Disease and Thalassemia, Beta, run by Memorial Sloan Kettering Cancer Center at 6 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 30 inclusion and 10 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 3 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 6 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?
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 Maria Cancio, MD (212-639-2446, canciom@mskcc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment related mortality/TRM or primary graft failure (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 3 Jun 2026.