SCD-Haplo: Phase II Study of HLA-Haploidentical SCT for Aggressive SCD

• Status: Terminated
• Trial end date: 2018-09-07
• Target enrollment: 0
• Participant gender: All

Summary

Related donor stem cell transplantation using the alemtuzumab/ TBI platform has been shown to be a safe strategy to cure severe sickle cell disease. However, due to a lack of suitable donors, many patients cannot benefit from this strategy. Alternative donor sources are desperately needed to fill this gap. Nearly all patients will have a haploidentical family member who would be able to donate. The use of post transplantation cyclophosphamide has greatly improved the outcome of haploidentical stem cell transplantation. The investigators propose to combine this with alemtuzumab/TBI conditioning.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Damiano Rondelli, MD
• Treatments: Alemtuzumab; Cyclophosphamide; Everolimus; Sirolimus

Criteria

Patient Eligibility:

• Patients with sickle cell disease are eligible if they have any of the following complications:

• Stroke or central nervous system event lasting longer than 24 hours

• Frequent vaso-occlusive pain episodes, defined as ≥ 3 per year requiring emergency room, acute care center, or hospital admissions.

• Recurrent episodes of priapism, defined as ≥ 2 per year requiring emergency room visits

• Acute chest syndrome with recurrent hospitalizations, defined as ≥ 2 lifetime events

• Red-cell alloimmunization (≥ 2 antibodies) during long-term transfusion therapy

• Bilateral proliferative retinopathy with major visual impairment in at least one eye

• Osteonecrosis of 2 or more joints

• Sickle cell nephropathy, defined by a GFR < 90mL/min/1.73m2 or the presence of macroalbuminuria (urine albumin > 300 mg/g creatinine)

• Pulmonary hypertension, defined by a mean pulmonary artery pressure > 25mmHg

• Age 16-60 years

• Karnofsky performance status of 60 or higher

• Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40%

• Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥ 50% predicted (after adjustment for hemoglobin concentration)

• Estimated GFR ≥ 50mL/min as calculated by the modified MDRD equation

• ALT ≤ 3x upper limit of normal

• HIV-negative

• Patient is pregnant

• Patient is able and willing to sign informed consent

• Patient has an HLA-haploidentical relative