Skip to main content
TrialFinder
TrialFinder is for informational purposes only and does not provide medical advice. Always talk to your doctor about whether a trial is right for you.
RECRUITINGOBSERVATIONAL

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

A Prospective Outcomes Study of Pediatric and Adult Patients With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation With a Reduced-Intensity Conditioning Regimen (PRO-RIC)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Patient, parent, or legal guardian must have given written willing to sign a consent form. 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 weakened immune system 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 ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

data collection

Study subjects will receive alemtuzumab, melphalan, thiotepa, fludarabine and hydroxyurea-based, reduced-intensity conditioning regimen in accordance with clinical practice at UPMC Children's Hospital of Pittsburgh at the discretion of the treating physician. Medical data will be abstracted from subject's medical charts once the patient signs the informed consent.

Locations (1)

UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States