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T-Cell Depleted Bone Marrow Transplant for Sickle Cell Disease and Other Anemias

This study is looking at a special type of bone marrow transplant for people with severe sickle cell disease, beta-thalassemia major, or Diamond-Blackfan anemia. It uses stem cells from donors who are not a perfect match, either unrelated volunteers or family members (haploidentical). The study uses a process called "T-cell depletion" with CD3/CD19 depleted leukocytes or CD45RA depleted leukocytes, along with medicines like Hydroxyurea, Rituximab, and Alemtuzumab. This approach aims to help more patients get a transplant while reducing side effects like graft-versus-host disease (when the donor cells attack the patient's body). The study will look at whether the transplant is rejected, serious side effects within 100 days, and acute graft-versus-host disease for up to two years. You can join if you are between 5 and 40 years old and have severe sickle cell disease with frequent pain crises.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 5 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
You would be followed for graft rejection and acute graft-versus-host disease for an average of two years after the transplant.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT03653338

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Recruiting
PHASE1Ages 5–40InterventionalTreatment
Paul Szabolcs
~5 participants
Updated 2026-08-13 on ClinicalTrials.gov
What's tested:CD3/CD19 depleted leukocytesCD45RA depleted leukocytesHydroxyureaRituximabAlemtuzumabFludarabine

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Graft rejection
Measured over Day -30 through study completion, an average of 2 years
+5 more outcomes measured

Conditions

Sickle Cell Anemia
Beta-thalassemia Major
Diamond-blackfan Anemia

Explore related trials

Beta-thalassemia MajorDiamond-Blackfan AnemiaSickle Cell Disease

Where it's being run

1 sites across 1 states
Pennsylvania1

Study leadership

  • Paul Szabolcs, MD · PRINCIPAL_INVESTIGATOR · University of Pittsburgh

Who to contact

Paul Szabolcs, MD
[email protected]
412-692-6225
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Recurrent acute painful episodes (also known as vaso-occlusive crises; VOC) despite supportive care, minimum of 2 new pain events per year requiring hospitalization for parenteral pain management in the previous 2 years.
Recurrent acute chest syndrome (ACS) despite supportive care, minimum of 2 episodes in preceding 2-year period.
Stroke or neurologic event lasting \> 24 hours with an accompanying infarct on MRI in any patient for all ages; Brain MRI with silent infarct without clinical event in patients ≤ 16 years.
Chronic transfusion therapy defined as \> 8 packed red blood cell transfusions per year in the year prior to enrollment and/or evidence of red blood cell alloimmunization.
Elevated transcranial Doppler velocities - \> 200 cm/s, via the non-imaging technique or \> 185 cm/s by the imaging technique measured on 2 separate occasions ≥ 1-month apart
Elevated TRV \> 2.6m/s in patients ≥ 16 years old.
Sickle-related renal insufficiency and/or sickle hepatopathy and/or any irreversible end-organ damage in patients ≥ 16 years old.
Creatinine clearance or GFR ≥ 45 ml/min/1.73m.
Hepatic transaminases (ALT/AST) ≤ 3 x upper limit of normal.
Liver MR imaging for iron content should be performed in all patients with Ferritin \> 500 ng/mL. If hepatic iron content \> 10mg Fe/g liver should have hepatology consultation and liver biopsy to confirm absence of cirrhosis, fibrosis or hepatitis.
Adequate cardiac function as measure by echocardiogram (shortening fraction \> 26% or ejection fraction \> 40% or \>80% of age-specific normal).
Pulmonary evaluation testing demonstrating FEV1/FVC ≥ 60% of predicted for age and/or resting pulse oximeter ≥ 92% on room air.
Cardiology clearance to proceed with conditioning regimen and HSCT.
Pulmonology clearance to proceed with conditioning regimen and HSCT. 6. Subjects must be human immunodeficiency virus (HIV) negative by PCR. 7. Negative pregnancy test for females ≥10 years old or who have reached menarche, unless surgically sterilized. 8. All females of childbearing potential and sexually active males must agree to use an FDA approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause a birth defect. 9. Subject and/or parent guardian will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting (Refer to section, 10. Hydroxyurea must have been trialed and failed in patients with sickle cell disease.

What this trial measures

  • Graft rejectionDay -30 through study completion, an average of 2 years

    How frequent, if any, graft rejection occurs

  • Post Transplant treatment related mortalityBy day 100

    Number of deaths that occurred from treatment

  • Acute Graft versus host diseaseDay 0 through study completion, an average of 2 years

    The number of patients who develop acute graft versus host disease (GVHD)post transplant

  • Chronic Graft versus host diseaseDay 0 through study completion, an average of 2 years

    The number of patients who develop chronic graft versus host disease (GVHD) post transplant

  • Post Transplant treatment related mortalityDay 180

    Number of deaths that occurred from treatment

  • Post Transplant treatment related mortality1 year

    Number of deaths that occurred from treatment