1. Name of Study: Nonmyeloablative StemCellTransplant in Children WithSickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor (Sickle-MAID)
Brief Summary: 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.
Intervention- Phase 2 trial: Drug: Alemtuzumab, Radiation: Total Body Irradiation, Drug: Sirolimus
Age range: 1 – 19yr
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 electrophoresis, 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.
Sites:
Canada, Alberta
Alberta Children’s Hospital, Calgary, Alberta, Canada, T3B 6A8
Principal Investigator: Tony Truong, MD, MPH Contact: 403-955-7272 tony.truong@ahs.ca
- Sub-Investigator: Greg Guilcher, MD Contact: 403-955-7272
- Sub-Investigator: Victor Lewis, MD
- Sub-Investigator: Michael Leaker, MD
- Sub-Investigator: Aisha Bruce, MD
- Sub-Investigator: Aru Narendran, MD, PhD
Status: Recruiting
Click here for more information about this study
Source: clinical trials.gov Identifier #: NCT03214354
Last updated: May 31st, 2018
Trials also available in Ontario: See here
2. Name of Study: Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects with Sickle Cell Disease
Sites outside of Ontario
Canada, Alberta
- Royal Alexandra Hospital, Edmonton, Alberta, Canada, T5H 3V9
- Miseracordia Community Hospital, Edmonton, Alberta, Canada, T5R 4H5
- Kaye Edmonton Clinic 3 C, Edmonton, Alberta, Canada, T6G 1Z1
- University of Alberta Hospital, Pharmacy Services, Edmonton, Alberta, Canada, T6G 1Z1
- Stollery Children’s Hospital, Edmonton, Alberta, Canada, T6G 2B7
- University of Alberta Hospital, Edmonton, Alberta, Canada, T6G 2B7
- Research Transition Facility, Edmonton, Alberta, Canada, T6G 2V2
- Grey Nuns Community Hospital, Edmonton, Alberta, Canada, T6L 5X8
- Centre Hospitalier Universitaire Sainte-Justine, Montreal, Quebec, Canada, H3T 1C5
3. Name of Study: Safety Of Rivipansel (GMI-1070) In The Treatment Of One or More Vaso-occlusive Crises In Hospitalized Subjects With Sickle Cell Disease
Sites outside on Ontario
Canada, Alberta
- University of Alberta Hospital, Pharmacy Services, Edmonton, Alberta, Canada, T6G 1Z1
- Stollery Children’s Hospital, Edmonton, Alberta, Canada, T6G 2B7
Canada, Quebec
- Centre Hospitalier Universitaire Sainte-Justine, Montreal, Quebec, Canada, H3T 1C5