Phase 2 Sickle Cell Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• The patient must be at least 12 months old and under 19 years old at the time of joining the study.
• The patient must have a confirmed diagnosis of sickle cell disease, specifically one of these types: homozygous sickle cell disease (HbSS), sickle-Hb C disease (HbSC), sickle beta-plus-thalassemia, or sickle beta-null-thalassemia, as confirmed by a blood test called hemoglobin electrophoresis.
• The patient must meet the standard requirements for a stem cell transplant (HSCT), which may include one or more of the following disease complications: more than one episode of severe bone pain crisis; a history of stroke; dangerously high blood flow velocity in brain arteries that is not suitable for treatment with hydroxyurea; a history of acute chest crisis or a crisis involving the spleen; a history of prolonged painful erections (in males); a history of bone tissue damage (osteonecrosis); high blood pressure in the lungs, confirmed by a heart ultrasound result above a certain threshold; or the development of two or more antibodies against red blood cells during long-term transfusion treatment.
• These sickle cell complications should ideally be present even while the patient is already taking hydroxyurea, although this is not a strict requirement if the treating medical team considers the patient to be at high risk of further crises.

Who may not be able to join:

• Patients who are unable to follow or stick to the study procedures and requirements.
• Patients who are known to have had an allergic reaction to sirolimus (a medication used in the study) or any of its related compounds or ingredients.

Important: Always verify eligibility with the trial site directly before applying.