Sickle Cell Disease Clinical Trial
— Sickle-AIDOfficial title:
A Phase II Pilot Study of Nonmyeloablative Conditioning Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease Who Have a Matched Related Major ABO-Incompatible Donor (Sickle-AID)
NCT number | NCT03214354 |
Other study ID # | TRU-17-001 |
Secondary ID | |
Status | Recruiting |
Phase | Phase 2 |
First received | |
Last updated | |
Start date | July 5, 2017 |
Est. completion date | July 2028 |
The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression. This study will expand the access of HSCT for patients with SCD who are currently not eligible because of donor restrictions.
Status | Recruiting |
Enrollment | 12 |
Est. completion date | July 2028 |
Est. primary completion date | July 2028 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year to 19 Years |
Eligibility | 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/ß+), or - sickle beta-null-thalassemia (HbS/ßo) - 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. |
Country | Name | City | State |
---|---|---|---|
Canada | Alberta Children's Hospital | Calgary | Alberta |
Lead Sponsor | Collaborator |
---|---|
University of Calgary |
Canada,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Incidence of pure red cell aplasia (PRCA) | Clinical definition: reticulocytopenia < 10x109/L (< 1%) lasting more than 60 days after HSCT, or Pathological definition: the absence of erythroid precursors in the marrow in the setting of adequate myeloid, lymphoid and megakaryocytic precursors | 6 months from enrollment | |
Secondary | RBC chimerism measured by peripheral blood flow cytometry | Peripheral blood for RBC chimerism on flow sorted erythroid precursor cells | 12 months | |
Secondary | RBC chimerism measured by bone marrow BFU-erythroid forming colonies | Bone marrow will be performed between Day +45 and +60 | 2 months | |
Secondary | Primary graft failure | Measured by donor chimerism from peripheral blood and bone marrow | 6 weeks | |
Secondary | Secondary graft failure | Measured by donor chimerism in peripheral blood and bone marrow | 24 months | |
Secondary | Disease recurrence | Measured by peripheral blood Hb S level | 24 months | |
Secondary | Incidence and severity of acute GVHD | Acute GVHD grade will be accessed using modified CIBMTR criteria | 100 days | |
Secondary | Incidence and severity of chronic GVHD | Chronic GVHD will be accessed using the NIH consensus criteria | 24 months |
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