Milder stem cell transplant could tame aggressive sickle cell disease
NCT ID NCT01499888
First seen Jul 08, 2026 · Last updated Jul 09, 2026 · Updated 1 time
Summary
This clinical trial tests a stem cell transplant using a milder conditioning regimen—without standard chemotherapy—to treat people with aggressive sickle cell disease who cannot take or have failed hydroxyurea therapy. The transplant uses stem cells from a matched sibling donor, along with immune-suppressing drugs and low-dose radiation, aiming to reduce serious complications like strokes and severe pain episodes. The study focuses on whether the donor cells successfully engraft and how safe the procedure is in this high-risk group.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Allogeneic non-myeloablative stem cell transplant with alemtuzumab, sirolimus, and low-dose total body irradiation
- What this could lead to
- If successful, this approach could offer a way to control sickle cell disease without the harsh side effects of traditional chemotherapy, potentially reducing complications like strokes and pain crises.
- What could go wrong
- This is an early-phase trial (Phase 1/2) with a small number of participants. The transplant still carries risks of graft failure, infection, or graft-versus-host disease, and it may not work for everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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45 people
The number who actually took part.
- Started
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Nov 2011
- Expected to finish
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Oct 2029
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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16 to 60 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Patients with sickle cell disease, subtype Hgb SS, SC, or SB disease who are on chronic transfusion therapy for a prior stroke or those patients who were intolerant of hydroxyurea therapy or were being treated with hydroxyurea therapy and were complicated by at least one of the following: * Stroke or central nervous system event lasting longer than 24 hours * Frequent vaso-occlusive pain episodes, defined as ≥ 3 per year severe enough to interfere with the patient's normal daily function or require medical attention in the clinic, emergency room, acute care center, or hospital * Recurrent episodes of priapism, defined as ≥ 2 per year requiring emergency room visits * Acute chest syndrome with recurrent hospitalizations, defined as ≥ 2 lifetime events * Red-cell alloimmunization (≥ 2 antibodies) during longterm transfusion therapy * Bilateral proliferative retinopathy with major visual impairment in at least one eye * Osteonecrosis of 2 or more joints * Sickle cell nephropathy * Stage I or II sickle lung disease * Symptoms of pulmonary hypertension and mean pulmonary artery pressure \> 25mmHg * Age 16-60 years * Karnofsky performance status of 70 or higher * Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40% * Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥ 50% * Estimated GFR ≥ 30mL/min as calculated by the modified MDRD equation * ALT ≤ 3x upper limit of normal * No evidence of chronic active hepatitis or cirrhosis * HIV-negative * Patient is not pregnant * History of compliance with medications and medical care * Patient is able and willing to sign informed consent * Patient has an HLA-identical matched related donor
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
University of Illinois at Chicago
Chicago, Illinois, 60612, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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