BridgeMD
New treatment option for Sickle Cell Disease
Official title Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
ClinicalTrials.gov ID: NCT03214354
What this study is testing
What is Alemtuzumab?
Alemtuzumab is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for sickle cell disease.
Also referred to as Campath.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 19
You may be able to join if
- 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
You likely can't join if
- 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.
See the full eligibility 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.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Calgary, Alberta, Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 19 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Calgary, Alberta, Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.