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New treatment option for Sickle Cell Disease
Official title Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
ClinicalTrials.gov ID: NCT03587272
What this study is testing
What is Alemtuzumab, low dose total body irradiation, Sirolimus?
Alemtuzumab, low dose total body irradiation, Sirolimus is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for sickle cell disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 25
You may be able to join if
- Patients with genotypes hemoglobin SS and Sβ0 thalassemia must have at least one of the following:
- History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm/sec by the non-imaging technique (or ≥185 cm/sec by the...
- History of cerebral infarction on brain MRI (overt stroke, or silent stroke if ≥3 mm in one dimension, visible in two planes on fluid-attenuated...
- History of two or more episodes of acute chest syndrome (ACS) in lifetime.
- History of three or more SCD pain events requiring treatment with an opiate or IV pain medication (inpatient or outpatient) in lifetime.
You likely can't join if
- General: Life expectancy less than 6 months. Pregnant or breastfeeding patients.
- Infection Disease: Uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms)...
- Liver: Direct (conjugated) bilirubin \> 1.5 mg/dL, transaminases \>5x upper limit of normal for age.
- Cardiac: Left ventricular shortening fraction \<25% or ejection fraction \<50% by ECHO.
- Kidney: Estimated creatinine clearance less than 60 mL/min/1.73m2.
- Pulmonary function: Diffusion capacity of carbon monoxide (DLCO) \<35% (adjusted for hemoglobin). Baseline oxygen saturation \<85% or PaO2 \<70.
See the full eligibility criteria
- Patients with genotypes hemoglobin SS and Sβ0 thalassemia must have at least one of the following:
- History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm/sec by the non-imaging technique (or ≥185 cm/sec by the imaging technique) measured at a minimum of two separate occasions.
- History of cerebral infarction on brain MRI (overt stroke, or silent stroke if ≥3 mm in one dimension, visible in two planes on fluid-attenuated inversion recovery T2-weighted images).
- History of two or more episodes of acute chest syndrome (ACS) in lifetime.
- History of three or more SCD pain events requiring treatment with an opiate or IV pain medication (inpatient or outpatient) in lifetime.
- History of any hospitalization for SCD pain or ACS while receiving hydroxyurea treatment.
- History of two or more episodes of priapism (erection lasting ≥4 hours or requiring emergent medical care).
- Administration of regular RBC transfusions (≥8 transfusions in the previous 12 months).
- At least two episodes of splenic sequestration requiring red blood cell transfusion or splenectomy after at least one episode of splenic sequestration. Patients with all other sickle genotypes (hemoglobin SC, Sβ+...
- Clinically significant neurologic event (overt stroke).
- History of two or more episodes of ACS in the 2-years period preceding enrollment.
- History of three or more SCD pain events requiring treatment with an opiate or IV pain medication (inpatient or outpatient) in the 1-year period preceding enrollment.
- History of any hospitalization for SCD pain or ACS while receiving hydroxyurea treatment.
- History of two or more episodes of priapism (erection lasting ≥4 hours or requiring emergent medical care).
- Administration of regular RBC transfusions (≥8 transfusions in the previous 12 months).
- At least two episodes of splenic sequestration requiring red blood cell transfusion or splenectomy after at least one episode of splenic sequestration.
- General: Life expectancy less than 6 months. Pregnant or breastfeeding patients.
- Infection Disease: Uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms) within 1 month prior to conditioning. Patients with febrile illness or...
- Liver: Direct (conjugated) bilirubin \> 1.5 mg/dL, transaminases \>5x upper limit of normal for age.
- Cardiac: Left ventricular shortening fraction \<25% or ejection fraction \<50% by ECHO.
- Kidney: Estimated creatinine clearance less than 60 mL/min/1.73m2.
- Pulmonary function: Diffusion capacity of carbon monoxide (DLCO) \<35% (adjusted for hemoglobin). Baseline oxygen saturation \<85% or PaO2 \<70.
- Heme: History of RBC alloantibodies against donor RBC antigens (even if current antibody screen is negative). Major ABO incompatibility with donor.
The study team makes the final eligibility decision.
Where it's taking place
- Washington D.C., District of Columbia, United States
- Chicago, Illinois, United States
- New York, New York, United States
- Charlotte, North Carolina, United States
- Columbus, Ohio, United States
- Calgary, Alberta, Canada
- Toronto, Ontario, Canada
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 25 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Washington D.C., District of Columbia, United States; Chicago, Illinois, United States; New York, New York, United States; Charlotte, North Carolina, United States; Columbus, Ohio, United States; Calgary, Alberta, Canada and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.