Clinical trial for Sickle Cell Disease
Official title Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody
ClinicalTrials.gov ID: NCT06358638
What this study is testing
What is Daratumumab?
Daratumumab is an investigational medicine, given as an IV infusion (through a vein), being studied as a potential treatment for sickle cell disease.
Also referred to as Darzalex.
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
- The study aims to find out if giving daratumumab before a transplant can prevent a condition where the body stops making red blood cells. It also checks if this approach is safe and works as well as transplants for patients without these antibodies.
- Who it's for
- People with sickle cell disease who have specific antibodies against their donor's red blood cells and are planning a transplant from a matching sibling.
- What's involved
- Participants will receive daratumumab before their transplant. The transplant process includes a drug called alemtuzumab, low-dose radiation, and a drug called sirolimus.
- Time commitment
- The study involves a bone marrow transplant and follow-up visits to monitor health and check for side effects.
- 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 SCD age 2-24.99 years who have a healthy HLA-identical sibling donor with major ABO incompatibility OR patients with RBC alloantibodies...
- Patients must have an absolute neutrophil count of 1 x 109/L and a platelet count of 100 x 109/L.
- History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm/sec by the non-imaging technique measured at a minimum of two...
- Progression of CNS vasculopathy on MRA determined to be secondary to SCD.
- 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...
You likely can't join if
- Life expectancy less than 6 month
- Pregnant or breastfeeding patients.
- Infectious 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. Uncontrolled cardiac arrhythmia.
- Kidney: Estimated creatinine clearance less than 60 mL/min/1.73m2.
See the full eligibility criteria
- Patients with SCD age 2-24.99 years who have a healthy HLA-identical sibling donor with major ABO incompatibility OR patients with RBC alloantibodies against other donor RBC antigens.
- Patients must have an absolute neutrophil count of 1 x 109/L and a platelet count of 100 x 109/L.
- History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm/sec by the non-imaging technique measured at a minimum of two separate occasions.
- Progression of CNS vasculopathy on MRA determined to be secondary to SCD.
- 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 in lifetime.
- History of any hospitalization for a complication secondary to SCD (does NOT include empiric hospitalizations for fever only).
- History of two or more episodes of priapism.
- Administration of regular RBC transfusions (≥8 transfusions episodes in the previous 12 months).
- Clinically significant neurologic event (overt stroke).
- History of two or more episodes of ACS in the 2-year 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.
- Life expectancy less than 6 month
- Pregnant or breastfeeding patients.
- Infectious Disease: Uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms) within 1 month prior to conditioning.
- 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. Uncontrolled cardiac arrhythmia.
- 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 \<94% at rest or PaO2 \<70.
- Heme: Available, medically suitable, and equivalent HLA-matched sibling donor, who does not have major ABO incompatibility or express RBC antigens against which the patient is alloimmunized.
The study team makes the final eligibility decision.
Where it is enrolling
- Washington D.C., District of Columbia, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
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, but 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?
This study is enrolling now at Washington D.C., District of Columbia, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.