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Haploidentical Transplant for Sickle Cell Disease

This study is looking at a new way to perform a stem cell transplant for people with sickle cell disease. It uses a special type of donor called "haploidentical," which means the donor is a partial match, like a parent or child. The treatment involves two cycles of pre-transplant immunosuppressive therapy using Fludarabine and Dexamethasone, followed by a conditioning regimen with rATG, Fludarabine, and Busulfan, and then the stem cell transplant. The main goal is to see how safe this treatment is by tracking any unacceptable side effects within the first 100 days after the transplant. You might be able to join if you are between 1 and 30 years old, have sickle cell anemia (Hgb SS or SB° Thalassemia) with high levels of Hgb S, and have experienced a significant neurological event like a stroke or have increased transcranial Doppler velocity. The study aims to expand donor options and find a safe transplant method.

Study design
This is an interventional study that plans to enroll 11 participants. It is testing a specific treatment approach for sickle cell disease.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint measures adverse events up to 100 days post-transplant, with evaluation at 190 days.

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

NCT03279094

Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

Recruiting
PHASE1Ages 1–30InterventionalTreatment
City of Hope Medical Center
~11 participants
Updated 2026-05-12 on ClinicalTrials.gov
What's tested:Hematopoietic stem cell transplantation

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Rate of unacceptable adverse events that are defined as any of the following events that occur from start of pre-transplant immunosuppressive therapy to the first 100 days post HCT:
Measured over 190 days

Conditions

Sickle Cell Disease

Explore related trials

Sickle Cell DiseaseHemoglobin S

Where it's being run

1 sites across 1 states
California1

Study leadership

  • Anna B. Pawlowska, MD · PRINCIPAL_INVESTIGATOR · City of Hope Medical Center

Who to contact

Anna B. Pawlowska, MD
[email protected]
626-218-8442
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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Diagnosis: Patients with sickle cell anemia (Hgb SS or SB° Thalassemia) with baseline Hgb S more than 60%.
Disease status:
Significant neurologic event (stroke) or any neurological deficit lasting \> 24 hours; or increased transcranial Doppler velocity (\>200 m/s).
History of one or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea).
History of one or more severe vaso-occlusive pain crises per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with hydroxyurea).
Recurrent priapism requiring medical therapy.
Osteonecrosis of two or more joints despite the institution of supportive care measures.
Prior treatment with regular RBC transfusion therapy, defined as receiving 8 or more transfusions per year for \> 1 year to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome)
Echocardiograph finding of tricuspid valve regurgitation jet (TRJ) velocity ≥ 2.5 m/sec.
Ages 1 to 30.
Child Bearing Potential- Transplantation could be teratogenic and/or lethal to the developing fetus. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control or abstinence) prior to study entry and for six months following duration of study participation. Should a woman become pregnant or suspect that she is pregnant while participating on the trial, she should inform her treating physician immediately.
Informed Consent/Assent: All subjects must have the ability to understand and the willingness to sign a written informed consent.
The recipient must have a related donor who is genotypically haploidentical on HLA-A, B, C and DRB1 loci.
No HLA matched sibling or 10/10 matched unrelated donor is available.

Exclusion

Any uncontrolled illness including ongoing or active bacterial, viral or fungal infection.
Patients may not be receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy.
History of allergic reactions attributed to compounds of similar chemical or biologic composition to any in the pre- or post-transplant regimen.
Pregnant women are excluded from this study.
Patients with any active malignancy are ineligible for this study, other than non-melanoma skin cancers.
Medical problem or neurologic/psychiatric dysfunction which would impair patient ability to be compliant with the medical regimen and to tolerate transplantation or would prolong hematologic recovery which in the opinion of the principal investigator would place the recipient at unacceptable risk.
Prior autologous or allogeneic transplant.
Fully HLA-matched related or unrelated donor is available to donate.
Non-Compliance: Subjects, who in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.

What this trial measures

  • Rate of unacceptable adverse events that are defined as any of the following events that occur from start of pre-transplant immunosuppressive therapy to the first 100 days post HCT:190 days

    * Rate of death of any causes * Rate of study discontinuation or early withdrawal * Rate of graft failure • Primary graft failure is defined as failure to achieve a neutrophil count of 0.5 x 109/L before day +42 or mixed chimerism with failure to achieve \<30% Hgb S on electrophoresis after day +180. Secondary graft failure is defined as recovery followed by a sustained loss of initial graft. * Rate of grade 4 non-hematological toxicities per NCI CTCAE v4.03 that last more than 21 days