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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04528355
Recruitment Status : Recruiting
First Posted : August 27, 2020
Last Update Posted : February 13, 2023
Sponsor:
Information provided by (Responsible Party):
Paul Szabolcs, University of Pittsburgh

Study Description
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
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.

Condition or disease Intervention/treatment
Primary Immunodeficiency (PID) Congenital Bone Marrow Failure Syndromes Inherited Metabolic Disorders (IMD) Hereditary Anemias Inflammatory Conditions Drug: data collection

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.

Study Design
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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Prospective
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)
Actual Study Start Date : August 20, 2020
Estimated Primary Completion Date : December 31, 2025
Estimated Study Completion Date : June 30, 2026

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Intervention Details:
  • 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.

Outcome Measures
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Primary Outcome Measures :
  1. incidence of acute graft versus host disease (GVHD) [ Time Frame: up to 5 years ]
    grades 3-4, chronic extensive GVHD

  2. overall survival after HSCT [ Time Frame: up to 5 years ]
    review of the existing medical records to check on the participant's survival status


Secondary Outcome Measures :
  1. Describe degree of engraftment, based upon chimerism data [ Time Frame: up to 5 years ]
    review of chimerism test results in the existing medical records to check on degree of donor engraftment measured by the percentage of donor-derived blood cells in the HSCT recipient

  2. Describe probability to discontinue systemic immunosuppression medications [ Time Frame: by 6, 9, and 12 months post-HSCT ]
    review of the existing medical records to check on the participant's current medications

  3. Describe the tempo of immune reconstitution [ Time Frame: over the first year post transplant ]
    review of the various test results in existing medical records to check on the participant's immune system recovery rate

  4. Describe the use of donor leukocyte infusion (DLI) [ Time Frame: up to 5 years ]
    review of the existing medical records to check on the participant's need for DLI


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   2 Months to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
patients with Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04528355


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

Locations
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United States, Pennsylvania
UPMC Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Shawna McIntyre, RN    412-692-5552    mcintyresm@upmc.edu   
Sub-Investigator: Jessie Barnum, MD         
Sub-Investigator: Craig Byersdorfer, MD         
Sub-Investigator: Maria Escolar, MD         
Sub-Investigator: Elizabeth Stenger, MD         
Sub-Investigator: Randy Windreich, MD         
Sponsors and Collaborators
Paul Szabolcs
Investigators
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Principal Investigator: Paul Szabolcs, MD UPMC Children's Hospital of Pittsburgh
More Information
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Responsible Party: Paul Szabolcs, Chief, BMT-CT at CHP of UPMC and Professor of Pediatrics and Immunology, University of Pittsburgh, University of Pittsburgh
ClinicalTrials.gov Identifier: NCT04528355    
Other Study ID Numbers: STUDY20070105
First Posted: August 27, 2020    Key Record Dates
Last Update Posted: February 13, 2023
Last Verified: February 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Paul Szabolcs, University of Pittsburgh:
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:
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Bone Marrow Failure Disorders
Pancytopenia
Congenital Bone Marrow Failure Syndromes
Primary Immunodeficiency Diseases
Metabolic Diseases
Syndrome
Disease
 Pathologic Processes
Hematologic Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Genetic Diseases, Inborn
Bone Marrow Diseases
Infant, Newborn, Diseases