Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: data collection.
- Who it may be relevant to
- Registry conditions: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias. Basic parameters: 2 months — 60 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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)
Overview
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.
Detailed description
Hematopoietic stem cell transplantation (HSCT) from a healthy donor can cure or alleviate a broad spectrum of non-malignant disorders (NMD). Although reduced-intensity conditioning (RIC) regimens promise decreased treatment-related morbidity and mortality, graft failure and infections are limiting the use of RIC in chemotherapy-naive patients. Dr. Szabolcs have completed several trials to evaluate a novel RIC regimen of alemtuzumab, hydroxyurea, fludarabine, melphalan, and thiotepa. The last trial at UPMC Children's Hospital of Pittsburgh of a highly effective and biologically rational chemotherapy-based RIC regimen paired with simple alemtuzumab dosing strata was tested and resulted in outstanding survival and remarkably low rates of graft failure. The favorable outcome described may serve as a toxicity and efficacy reference for emerging gene therapy strategies as well.
This prospective collection of clinical data will allow the investigators to further assess engraftment, GVHD, immunosuppressant use and overall survival in this patient population.
Interventions
- 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.
Primary outcome measures
- incidence of acute graft versus host disease (GVHD) [Time frame: up to 5 years]
- overall survival after HSCT [Time frame: up to 5 years]
Secondary outcome measures (4)
- Describe degree of engraftment, based upon chimerism data [Time frame: up to 5 years]
- Describe probability to discontinue systemic immunosuppression medications [Time frame: by 6, 9, and 12 months post-HSCT]
- Describe the tempo of immune reconstitution [Time frame: over the first year post transplant]
- Describe the use of donor leukocyte infusion (DLI) [Time frame: up to 5 years]
Eligibility criteria
Inclusion criteria
- Patient, parent, or legal guardian must have given written informed consent.
- Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.
- 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
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- UPMC Children's Hospital of Pittsburgh — Pittsburgh
Identifiers
NCT: NCT04528355 · STUDY20070105