Data Collection Study of Patients with Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT with RIC

Launched by PAUL SZABOLCS · Aug 25, 2020

Keywords

ClinConnect Summary

This clinical trial is focused on collecting information about patients with certain non-cancerous disorders who are receiving stem cell transplants. Specifically, the study is looking at how a gentler type of chemotherapy, combined with a medication called alemtuzumab, can help prevent complications and support the immune system recovery after a transplant. The conditions being studied include primary immunodeficiencies, inherited blood disorders, metabolic disorders, and some inflammatory conditions.

To participate in this study, patients need to be between 2 months and 60 years old and have a disorder that can be treated with a stem cell transplant. Parents or guardians must provide written consent for younger patients. Participants will receive either umbilical cord blood, bone marrow, or stem cells from their own blood, along with the specified chemotherapy regimen. There are no specific exclusion criteria, meaning most patients with the eligible conditions may have the opportunity to join. This study is currently recruiting participants, and it aims to gather valuable data that can help improve treatment options for these disorders in the future.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Patient, parent, or legal guardian must have given written informed consent.
• • 2. Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.
• 3. Patients should have a non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to the following:
• • A. Primary Immunodeficiency Syndromes
• • Severe Combined Immune Deficiency (SCID) with NK cell activity
• • Omenn Syndrome
• • Bare Lymphocyte Syndrome (BLS)
• • Combined Immune Deficiency (CID) syndromes
• • Combined Variable Immune Deficiency (CVID) syndrome
• • Wiskott-Aldrich Syndrome
• • Leukocyte adhesion deficiency
• • Chronic granulomatous disease (CGD)
• • Hyper IgM (XHIM) syndrome
• • IPEX syndrome
• • Chediak-Higashi Syndrome
• • Autoimmune Lymphoproliferative Syndrome (ALPS)
• • Hemophagocytic Lymphohistiocytosis (HLH) syndromes
• • Lymphocyte Signaling defects
• • B. Congenital Bone Marrow Failure Syndromes
• • Congenital Amegakaryocytic Thrombocytopenia (CAMT)
• • Osteopetrosis
• • C. Inherited Metabolic Disorders (IMD)
• • Mucopolysaccharidoses
• • Hurler syndrome (MPS I)
• • Hunter syndrome (MPS II)
• • Leukodystrophies
• • Krabbe Disease, also known as globoid cell leukodystrophy
• • Metachromatic leukodystrophy (MLD)
• • X-linked adrenoleukodystrophy (ALD)
• • Other inherited metabolic disorders
• • Alpha Mannosidosis
• • Gaucher Disease
• • Other inheritable metabolic diseases where HSCT may be beneficial
• • D. Hereditary Anemias
• • Thalassemia major
• • Sickle cell disease (SCD)
• • Diamond Blackfan Anemia (DBA)
• • E. Inflammatory Conditions
• • Crohn's Disease or Inflammatory Bowel Disease
• • IPEX or IPEX-like Syndromes
• • Rheumatoid Arthritis
• • Other inflammatory conditions where HSCT may be beneficial
• • 4. Subjects receive either umbilical cord blood, bone marrow, or peripheral blood stem cell transplant with an alemtuzumab, melphalan, thiotepa, fludarabine and hydroxyurea-based, reduced-intensity conditioning regimen, according to clinical practice at UPMC Children's Hospital of Pittsburgh.
• • There are no exclusion criteria.