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New radiation technique aims to make stem cell transplants safer for sickle cell patients
NCT ID NCT05384756
First seen Jun 26, 2026 · Last updated Jul 31, 2026 · Updated 5 times
Summary
This early-phase trial tests a new way to prepare people with sickle cell disease for a stem cell transplant. Instead of full-body radiation, doctors use targeted radiation to the bone marrow and lymph nodes, along with drugs to prevent rejection and graft-versus-host disease. The goal is to see if this approach is safe and can reduce side effects while still allowing the new stem cells to take hold.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Alemtuzumab and sirolimus (drugs) plus total marrow and lymphoid irradiation (radiation therapy)
- What this could lead to
- If successful, this approach could make stem cell transplants safer and more effective for people with sickle cell disease, reducing organ damage from radiation.
- What could go wrong
- This is a very early phase 1 trial with only 2 participants, so results may not apply to others. There are risks of graft-versus-host disease, infection, and other serious side effects.
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
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 20 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2022
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
-
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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12 to 40 years
- Sex
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Anyone
- Healthy volunteers
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Accepted
You do not need to have the condition being studied to take part.
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: * Documented informed consent of the participant and/or legally authorized representative * Assent, when appropriate, will be obtained per institutional guidelines * Registered into Risk Evaluation and Mitigation Strategies (REMS) program * Age: 12-40 years * Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 * Have a diagnosis of sickle cell disease, be at a high risk for disease related morbidity or mortality, which must be defined by one of the following disease status criteria: * 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 red blood cell (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. * Have a related donor who is matched on at least 8/10 human leukocyte antigen (HLA)-A, B, C, and DRB1 Loci * Total bilirubin =\< 2.5 x upper limit normal (ULN( (unless has Gilbert's disease) (performed within 30 days prior to day 1 of protocol) * Aspartate aminotransferase (AST) =\< 1.5 x ULN (performed within 30 days prior to day 1 of protocol) * Alanine aminotransferase (ALT) =\< 1.5 x ULN (performed within 30 days prior to day 1 of protocol) * Creatinine clearance (CrCl) of \>= 60 mL/min per 24 hour urine test or the Cockcroft-Gault formula (performed within 30 days prior to day 1 of protocol) * If not receiving anticoagulants: International Normalized Ratio (INR) OR Prothrombin (PT) =\< 1.5 x ULN (performed within 30 days prior to day 1 of protocol) * If on anticoagulant therapy: PT must be within therapeutic range of intended use of anticoagulants * If not receiving anticoagulants: Activated Partial Thromboplastin Time (aPTT) =\<1.5 x ULN (performed within 30 days prior to day 1 of protocol) * If on anticoagulant therapy: aPTT must be within therapeutic range of intended use of anticoagulants * Left ventricular ejection fraction (LVEF) \>= 50% (performed within 30 days prior to day 1 of protocol) * Note: To be performed within 28 days prior to Day 1 of protocol therapy. * If able to perform pulmonary function tests: Forced expiratory volume in 1 second (FEV1), force vital capacity (FVC), and diffused lung capacity of carbon monoxide (DLCO) (diffusion capacity) \>= 50% of predicted (corrected for hemoglobin) * If unable to perform pulmonary function tests: Oxygen (O 2) saturation \> 92% on room air * Note: To be performed within 28 days prior to Day 1 of protocol therapy. * Seronegative for human immunodeficiency virus (HIV) antigen (Ag)/antibody (Ab) combo, hepatitis C virus (HCV), active hepatitis B virus (HBV) (surface antigen negative), and syphilis (rapid plasma regain \[RPR\]) * If positive, hepatitis C ribonucleic acid (RNA) quantitation must be performed * Meets other institutional and federal requirements for infectious disease titer requirements * Note Infectious disease testing to be performed within 28 days prior to day 1 of protocol therapy * Women of childbearing potential (WOCBP): negative urine or serum pregnancy test * If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be require. * Agreement by females and males of childbearing potential to use an effective method of birth control or abstain from heterosexual activity for the course of the study through at least Six months after the last dose of protocol therapy. * Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for \> 1 year (women only) * DONOR: Age =\< 60 years * DONOR: Medical history and physical examination confirm good health status as defined by institutional standards * DONOR: Serologies for: Hepatitis B (HBV) Core Antibody, HIV I/II Antibody, human T-lymphotropic virus (HTLV) - I/II antibody, HCV antibody, Hepatitis B surface antigen, Serologic Test for Syphilis, HIV-1/HCV/HBV nucleic acid, West Nile virus nucleic acid, Trypanosoma cruzi antibody, Cytomegalovirus (CMV) antibody, (AKA: Donor Room Serologies) * DONOR: Female donors of childbearing potential must have a negative serum or urine beta human chorionic gonadotropin (b-HCG) test within 30 days of initiation of conditioning, 30 days of patients admission for conditioning and 7 days of mobilization or bone marrow harvest. * DONOR: The donor must be informed of the investigational nature of this study and have signed a consent form in accordance with Federal Guidelines and the guidelines of the participating institution Exclusion Criteria: * Prior allogeneic or autologous stem cell transplant * Patients who are receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy. * History of allergic reactions attributable to compounds of similar chemical or biologic composition to study agent * 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. * Active infection requiring antibiotics * Females only: Pregnant or breastfeeding * Any other condition that would, in the Investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures * Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics) * DONOR: Evidence of active infection * DONOR: Medical or physical reason which makes the donor unlikely to tolerate or cooperate with growth factor therapy and leukapheresis if donating peripheral stem cells or unlikely to tolerate general anesthesia and bone marrow collection if donating a bone marrow * DONOR: Factors which place the donor at increased risk for complications from leukapheresis or granulocyte colony-stimulating Factor (G-CSF) therapy if donating peripheral stem cells or general anesthesia and bone marrow collection if donating a bone marrow * DONOR: Lactating female or, if of child-bearing potential, is unwilling to implement adequate birth control * DONOR: HIV positive * DONOR: Prior radiation therapy
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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
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City of Hope Medical Center
Duarte, California, 91010, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a softer transplant cure sickle cell disease?