RECRUITING

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

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Conditions

Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)Hereditary AnemiasInflammatory ConditionsSevere Combined Immune Deficiency (SCID) with NK cell activityOmenn SyndromeBare Lymphocyte Syndrome (BLS)Combined Immune Deficiency (CID) syndromesWiskott-Aldrich SyndromeLeukocyte adhesion deficiencyChronic granulomatous disease (CGD)Hyper IgM (XHIM) syndromeIPEX syndromeChediak-Higashi SyndromeAutoimmune Lymphoproliferative Syndrome (ALPS)Hemophagocytic Lymphohistiocytosis (HLH) syndromesLymphocyte Signaling defectsCongenital Amegakaryocytic Thrombocytopenia (CAMT)OsteopetrosisHurler syndrome (MPS I)Hurler syndrome (MPS II)Krabbe Disease, also known as Globoid Cell LeukodystrophyMetachromatic leukodystrophy (MLD)X-linked adrenoleukodystrophy (ALD)Alpha MannosidosisGaucher DiseaseThalassemia majorSickle cell disease (SCD)Diamond Blackfan Anemia (DBA)Crohn's DiseaseInflammatory Bowel DiseaseIPEX or IPEX-like SyndromesRheumatoid Arthritis

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

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)

Quick Facts

Study Start:2020-08-20
Study Completion:2026-06-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04528355

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Months to 60 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Patient, parent, or legal guardian must have given written informed consent.
  2. 2. Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.
  3. 3. Patients should have a non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to the following:
  4. * Severe Combined Immune Deficiency (SCID) with NK cell activity
  5. * Omenn Syndrome
  6. * Bare Lymphocyte Syndrome (BLS)
  7. * Combined Immune Deficiency (CID) syndromes
  8. * Combined Variable Immune Deficiency (CVID) syndrome
  9. * Wiskott-Aldrich Syndrome
  10. * Leukocyte adhesion deficiency
  11. * Chronic granulomatous disease (CGD)
  12. * Hyper IgM (XHIM) syndrome
  13. * IPEX syndrome
  14. * Chediak-Higashi Syndrome
  15. * Autoimmune Lymphoproliferative Syndrome (ALPS)
  16. * Hemophagocytic Lymphohistiocytosis (HLH) syndromes
  17. * Lymphocyte Signaling defects
  18. * Congenital Amegakaryocytic Thrombocytopenia (CAMT)
  19. * Osteopetrosis
  20. * Mucopolysaccharidoses
  21. * Hurler syndrome (MPS I)
  22. * Hunter syndrome (MPS II)
  23. * Leukodystrophies
  24. * Krabbe Disease, also known as globoid cell leukodystrophy
  25. * Metachromatic leukodystrophy (MLD)
  26. * X-linked adrenoleukodystrophy (ALD)
  27. * Other inherited metabolic disorders
  28. * Alpha Mannosidosis
  29. * Gaucher Disease
  30. * Other inheritable metabolic diseases where HSCT may be beneficial
  31. * Thalassemia major
  32. * Sickle cell disease (SCD)
  33. * Diamond Blackfan Anemia (DBA)
  34. * Crohn's Disease or Inflammatory Bowel Disease
  35. * IPEX or IPEX-like Syndromes
  36. * Rheumatoid Arthritis
  37. * Other inflammatory conditions where HSCT may be beneficial
  38. 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.
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Paul Szabolcs, MD
CONTACT
412-692-5427
paul.szabolcs@chp.edu
Shawna McIntyre, RN
CONTACT
412-692-5552
mcintyresm@upmc.edu

Principal Investigator

Paul Szabolcs, MD
PRINCIPAL_INVESTIGATOR
UPMC Children's Hospital of Pittsburgh

Study Locations (Sites)

UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
United States

Collaborators and Investigators

Sponsor: Paul Szabolcs

  • Paul Szabolcs, MD, PRINCIPAL_INVESTIGATOR, UPMC Children's Hospital of Pittsburgh

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2020-08-20
Study Completion Date2026-06-30

Study Record Updates

Study Start Date2020-08-20
Study Completion Date2026-06-30

Terms related to this study

Keywords Provided by Researchers

  • Severe Combined Immune Deficiency (SCID) with NK cell activity
  • Omenn Syndrome
  • Bare Lymphocyte Syndrome (BLS)
  • Combined Immune Deficiency (CID) syndromes
  • 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
  • Congenital Amegakaryocytic Thrombocytopenia (CAMT)
  • Osteopetrosis
  • Hurler syndrome (MPS I)
  • Hurler syndrome (MPS II)
  • Krabbe Disease, also known as Globoid Cell Leukodystrophy
  • Metachromatic leukodystrophy (MLD)
  • X-linked adrenoleukodystrophy (ALD)
  • Alpha Mannosidosis
  • Gaucher Disease
  • Thalassemia major
  • Sickle cell disease (SCD)
  • Diamond Blackfan Anemia (DBA)
  • Crohn's Disease
  • Inflammatory Bowel Disease
  • IPEX or IPEX-like Syndromes
  • Rheumatoid Arthritis

Additional Relevant MeSH Terms

  • Primary Immunodeficiency (PID)
  • Congenital Bone Marrow Failure Syndromes
  • Inherited Metabolic Disorders (IMD)
  • Hereditary Anemias
  • Inflammatory Conditions