What this study is
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.
What's being tested
- data collection
Intervention · DRUG
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.
Study details
- 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)
- Sponsor
- Paul Szabolcs
- Conditions
- Primary Immunodeficiency (PID) · Congenital Bone Marrow Failure Syndromes · Inherited Metabolic Disorders (IMD) · Hereditary Anemias · Inflammatory Conditions
- Enrolling
- 50 people
- Starts
- 2020-08-20
- Est. completion
- 2028-06-30
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 2 Months – 60 YearsSex ALLHealthy volunteers Not acceptedYou may qualify if
- 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.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (1)
1 openUPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
- Shawna McIntyre, RN · Contact412-692-5552mcintyresm@upmc.edu
- Jessie Barnum, MD · Sub-investigator
- Craig Byersdorfer, MD · Sub-investigator
- Maria Escolar, MD · Sub-investigator
- Elizabeth Stenger, MD · Sub-investigator
- Randy Windreich, MD · Sub-investigator
Recruiting
Study team
Sponsor
Paul Szabolcs
Study ID STUDY20070105
Principal investigator
Paul Szabolcs, MD
UPMC Children's Hospital of Pittsburgh
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.