Clinical trial 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?
An investigational injectable medicine being studied as part of a treatment plan for sickle cell disease. is designed to lower the body's immune system so it is less likely to reject new stem cells.
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
- This study is testing whether a less intense transplant preparation can safely help children with sickle cell disease who have a sibling donor with a different blood type.
- Who it's for
- You may be able to join if you are a child with sickle cell disease and have a matched sibling donor who has a different blood type.
- What's involved
- You would receive a small amount of medicine that lowers your immune system, a low dose of whole-body radiation, and a drug that keeps your body from attacking the new cells. You will also have regular blood tests and check‑ins at the hospital.
- Time commitment
- The study lasts about 6 months, with several hospital visits for treatments and follow‑up blood tests.
- 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.
- homozygous Hb S disease (HbSS),
- sickle-Hb C disease (HbSC),
- sickle beta-plus-thalassemia (HbS/β+), or
- sickle beta-null-thalassemia (HbS/βo)
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.
- homozygous Hb S disease (HbSS),
- sickle-Hb C disease (HbSC),
- sickle beta-plus-thalassemia (HbS/β+), or
- sickle beta-null-thalassemia (HbS/βo)
- 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 is enrolling
- 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, but 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?
This study is enrolling now at Calgary, Alberta, Canada. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.