Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507) (NCT03263559) | Clinical Trial Compass
CompletedPhase 2
Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
United States95 participantsStarted 2017-10-03
Plain-language summary
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with severe SCD; and (2) adults with severe SCD.
Who can participate
Age range5 Years – 45 Years
SexALL
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Inclusion criteria
✓. A Karnofsky/Lansky performance score of ≥ 60.
✓. Cardiac function: Left ventricular ejection fraction (LVEF) \> 40%; or LV shortening fraction \> 26% by cardiac echocardiogram or by Multi Gated Acquisition Scan (MUGA) scan.
✓. Pulmonary function: Pulse oximetry with a baseline O2 saturation of ≥ 85% and Diffusing capacity of the lung for carbon monoxide (DLCO) \> 40% (corrected for hemoglobin).
✓. Renal function: Serum creatinine ≤ 1.5 x upper limit of normal for age and estimated or measured creatinine clearance ≥ 70 mL/min/1.73 m²
✓. Hepatic function:
✓. Serum conjugated (direct) bilirubin \< 2x upper limit of normal for age as per local laboratory. Participants with hyperbilirubinemia as the result of hyperhemolysis, or a severe drop in hemoglobin post blood transfusion, are not excluded.
✓. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \< 5x upper limit of normal as per local laboratory.
✓. Liver MRI using a validated methodology per institutional preference (T2\* or R2\* or by ferriscan \[R2 MRI\]) for estimation of hepatic iron content is required for participants who are currently receiving ≥8 packed red blood cell transfusions per year for ≥1 year or have received ≥20 packed red blood cell transfusions (lifetime cumulative). Participants who have hepatic iron content ≥ 10 mg Fe/g liver dry weight by liver MRI must have a Gastroenterology/hepatology consultation with liver biopsy and histological examination including documentation of the absence of cirrhosis, bridging fibrosis, and active hepatitis.
Exclusion criteria
What they're measuring
1
Percentage of Participants With Two-Year Post-Transplant Event Free Survival (EFS)
. Participants who have an HLA-matched sibling who is able and willing to donate bone marrow. Patients with a HLA-matched unrelated donor are not excluded.
✕. Uncontrolled bacterial, viral or fungal infection in the 6 weeks before enrollment (currently taking medication with evidence of progression of clinical symptoms or radiologic findings).
✕. Evidence of HIV infection or known HIV positive serology.
✕. Participants who have received a previous hematopoetic cell transplant (HCT).
✕. Participants who have had an Encephaloduroarteriosynangiosis (EDAS) procedure in the 6 months prior to enrollment
✕. Participants who have received a prior solid organ transplant
✕. Participants who have participated in another clinical trial in which the patient received an investigational or off-label use of a drug or device within 3 months of enrollment.