Gaucher Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- The patient, their parent, or legal guardian must be willing and able to sign a written consent form.
- The patient must be between 2 months and 60 years old at the time of signing consent.
- The patient must have a non-cancerous condition that can be treated with a stem cell transplant, such as:
- An immune system disorder (for example: Severe Combined Immune Deficiency/SCID, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, or similar conditions)
- A bone marrow disorder present from birth (for example: Congenital Amegakaryocytic Thrombocytopenia or Osteopetrosis)
- An inherited metabolic disorder (for example: Hurler syndrome, Krabbe Disease, X-linked adrenoleukodystrophy, Gaucher Disease, or similar conditions)
- An inherited blood disorder (for example: Thalassemia major, Sickle Cell Disease, or Diamond Blackfan Anemia)
- An inflammatory condition (for example: Crohn's Disease, Rheumatoid Arthritis, or similar conditions)
- The patient must be receiving a stem cell transplant using cord blood, bone marrow, or blood stem cells, along with a specific reduced-intensity preparation treatment (using a combination of medicines including alemtuzumab, melphalan, thiotepa, fludarabine, and hydroxyurea), as practiced at UPMC Children's Hospital of Pittsburgh.
Who may not be able to join:
- There are no listed exclusion criteria for this trial.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Paul Szabolcs, MD, UPMC Children's Hospital of Pittsburgh
Phone: 412-692-5427
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
incidence of acute graft versus host disease (GVHD); overall survival after HSCT
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.