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New treatment option for Sickle Cell Anemia
Official title T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
ClinicalTrials.gov ID: NCT03653338
What this study is testing
What is CD3/CD19 depleted leukocytes?
CD3/CD19 depleted leukocytes is an investigational medicine, being studied as a potential treatment for sickle cell anemia.
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 purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease.
- 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 5 to 40
You may be able to join if
- Patient, parent, or legal guardian must have given written informed consent and/or assent according to FDA guidelines.
- Ages 5 years to 40 years, at time of consent.
- Diagnosis of Sickle Cell Disease (Hemoglobin SS, Sβ0-thalassemia) complicated by any of the following:
- Recurrent acute painful episodes (also known as vaso-occlusive crises; VOC) despite supportive care, minimum of 2 new pain events per year requiring...
- Recurrent acute chest syndrome (ACS) despite supportive care, minimum of 2 episodes in preceding 2-year period.
You likely can't join if
- Patients with alternate, superior donor options (matched sibling donor or matched unrelated donor).
- Patients who have undergone stem cell transplantation in the 6 months prior to anticipated conditioning.
- Patients with history of a central nervous system (CNS) event within six months prior to start of conditioning (patient will be delayed until...
- Patients who are pregnant or lactating
- Patients with uncontrolled bacterial, viral or fungal infection
- Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the...
See the full eligibility criteria
- Patient, parent, or legal guardian must have given written informed consent and/or assent according to FDA guidelines.
- Ages 5 years to 40 years, at time of consent.
- Diagnosis of Sickle Cell Disease (Hemoglobin SS, Sβ0-thalassemia) complicated by any of the following:
- Recurrent acute painful episodes (also known as vaso-occlusive crises; VOC) despite supportive care, minimum of 2 new pain events per year requiring hospitalization for parenteral pain management in the previous 2 years.
- Recurrent acute chest syndrome (ACS) despite supportive care, minimum of 2 episodes in preceding 2-year period.
- Stroke or neurologic event lasting \> 24 hours with an accompanying infarct on MRI in any patient for all ages; Brain MRI with silent infarct without clinical event in patients ≤ 16 years.
- Chronic transfusion therapy defined as \> 8 packed red blood cell transfusions per year in the year prior to enrollment and/or evidence of red blood cell alloimmunization.
- Elevated transcranial Doppler velocities - \> 200 cm/s, via the non-imaging technique or \> 185 cm/s by the imaging technique measured on 2 separate occasions ≥ 1-month apart
- Elevated TRV \> 2.6m/s in patients ≥ 16 years old.
- Sickle-related renal insufficiency and/or sickle hepatopathy and/or any irreversible end-organ damage in patients ≥ 16 years old. OR Diagnosis of beta-thalassemia or Diamond-Blackfan anemia complicated by transfusion...
- A minimum donor match of 4/8 via high resolution HLA typing at HLA-A, -B, -C, -DRB1 loci in the related setting or minimum donor match of 6/8 via high resolution HLA typing at HLA-A, -B, -C, -DRB1 loci (with the DRB1...
- Adequate function of other organ systems as measured by:
- Creatinine clearance or GFR ≥ 45 ml/min/1.73m.
- Hepatic transaminases (ALT/AST) ≤ 3 x upper limit of normal.
- Liver MR imaging for iron content should be performed in all patients with Ferritin \> 500 ng/mL. If hepatic iron content \> 10mg Fe/g liver should have hepatology consultation and liver biopsy to confirm absence of...
- Adequate cardiac function as measure by echocardiogram (shortening fraction \> 26% or ejection fraction \> 40% or \>80% of age-specific normal).
- Pulmonary evaluation testing demonstrating FEV1/FVC ≥ 60% of predicted for age and/or resting pulse oximeter ≥ 92% on room air.
- Cardiology clearance to proceed with conditioning regimen and HSCT.
- Pulmonology clearance to proceed with conditioning regimen and HSCT.
- people must be human immunodeficiency virus (HIV) negative by PCR.
- Negative pregnancy test for females ≥10 years old or who have reached menarche, unless surgically sterilized.
- All females of childbearing potential and sexually active males must agree to use an FDA approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a...
- Subject and/or parent guardian will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte harvesting (Refer to section,
- Hydroxyurea must have been trialed and failed in patients with sickle cell disease. Patient
- Patients with alternate, superior donor options (matched sibling donor or matched unrelated donor).
- Patients who have undergone stem cell transplantation in the 6 months prior to anticipated conditioning.
- Patients with history of a central nervous system (CNS) event within six months prior to start of conditioning (patient will be delayed until eligible).
- Patients who are pregnant or lactating
- Patients with uncontrolled bacterial, viral or fungal infection
- Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the...
The study team makes the final eligibility decision.
Where it's taking place
- Pittsburgh, Pennsylvania, 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 - 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, 5 years to 40 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Pittsburgh, Pennsylvania, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
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.