Nonmyeloablative Stem Cell Transplant in Children with Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor

Launched by UNIVERSITY OF CALGARY · Jul 10, 2017

Keywords

ClinConnect Summary

This clinical trial is looking at a new way to help children with sickle cell disease by using a type of stem cell transplant that doesn’t completely destroy their bone marrow first. The goal is to see if this method is safe and effective for kids who have a matched sibling donor but may not usually qualify because of blood type differences. The treatment involves special medications to help their immune system accept the new cells without causing serious side effects. This could open up new options for many children who currently can't get a transplant.

To be eligible for this study, children need to be between 1 and 18 years old and have a specific type of sickle cell disease. They also need to have had serious complications from their condition, like painful crises or strokes, despite receiving standard treatments. Participants will receive the new treatment and will be closely monitored for their health and safety during the study. This trial is currently recruiting participants, and it’s important for families to know that the study may help improve treatment options for sickle cell disease in the future.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Patients must be ≥ 12 months and \< 19 years of age at the time of study enrollment.
• * Patients must have sickle cell disease as defined by hemoglobin electropheresis, as follows:
• • homozygous Hb S disease (HbSS),
• • sickle-Hb C disease (HbSC),
• • sickle beta-plus-thalassemia (HbS/β+), or
• • sickle beta-null-thalassemia (HbS/βo)
• * Patients must meet standard eligibility criteria to undergo HSCT, including but not limited to one or more of the following:
• • history of repeated (more than 1) bony (vaso-occlusive) crisis
• • history of stroke
• • elevated transcranial Doppler velocity not eligible for hydroxyurea, as per TWiTCH trial (ie. severe vasculopathy)
• • history of acute chest crisis or splenic sequestration crisis
• • history of priapism in males
• • history of osteonecrosis
• • pulmonary hypertension as documented by tricuspid regurgitation jet velocity (TRV) \> 2.5 m/s on echocardiogram
• • red cell allo-immunization (≥ 2 antibodies) during long term transfusion therapy
• • Sickle complications should be present despite the use of hydroxyurea, but this is not an absolute requirement, if the treating team considers the patient to be at high risk for further crisis episodes.
• Exclusion Criteria:
• • Patients who are unable to comply with or follow the study protocol.
• • Patients with known hypersensitivity to sirolimus, its derivatives or to any of its components.