Phase 2 Gaucher 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:

• A suitable donor match is available — either a cord blood unit (from a blood bank) or a matched unrelated bone marrow or blood stem cell donor that meets specific matching and cell count requirements.
• Your kidneys are working well enough, shown by blood test results within acceptable ranges.
• Your liver enzyme levels are within an acceptable range on blood tests.
• Your heart is functioning adequately, confirmed by an ultrasound or scan of the heart.
• Your lungs are functioning well enough, confirmed by breathing tests and/or a pulse oxygen reading of 92% or above while breathing normal air.
• You (or your parent/guardian) are able to give written consent to take part in the trial.
• If you have gone through puberty and/or have periods, you must have a negative pregnancy test.
• You have tested negative for HIV.
• You have a non-cancerous condition that may be treated with a stem cell transplant, which may include conditions such as: severe immune system disorders (such as SCID, Wiskott-Aldrich Syndrome, or chronic granulomatous disease), bone marrow failure conditions (such as Dyskeratosis Congenita or Osteopetrosis), inherited metabolic disorders (such as Hurler syndrome, Krabbe Disease, or adrenoleukodystrophy), inherited blood conditions (such as sickle cell disease, thalassemia major, or Diamond Blackfan Anemia), or certain inflammatory conditions (such as Crohn's disease).
• If you have sickle cell disease, you must also have experienced at least one of the following: a stroke, two or more pain crises in the past year, acute chest syndrome, bone/joint damage, or priapism.

Who may not be able to join:

• You have had a stem cell transplant from a donor within the last 6 months.
• You currently have any active cancer or a condition called myelodysplastic syndrome (a blood/bone marrow disorder).
• You have severe aplastic anemia that was not present from birth.
• You have a bacterial, viral, or fungal infection that is not under control and is getting worse despite treatment.
• You are currently pregnant or breastfeeding.
• You have poorly controlled high blood pressure in the lungs (pulmonary hypertension).
• You have any condition that would prevent you from being able to attend regular follow-up appointments or assessments.

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