- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04528355
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC (PRO-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)
Study Overview
Status
Conditions
Intervention / Treatment
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 Type
Enrollment (Estimated)
Contacts and Locations
Study Contact
- Name: Shawna McIntyre, RN
- Phone Number: 412-692-5552
- Email: mcintyresm@upmc.edu
Study Contact Backup
- Name: Paul Szabolcs, MD
- Phone Number: 412-692-5427
- Email: paul.szabolcs@chp.edu
Study Locations
-
-
Pennsylvania
-
Pittsburgh, Pennsylvania, United States, 15224
- Recruiting
- UPMC Children's Hospital of Pittsburgh
-
Contact:
- Shawna McIntyre, RN
- Phone Number: 412-692-5552
- Email: mcintyresm@upmc.edu
-
Sub-Investigator:
- Jessie Barnum, MD
-
Sub-Investigator:
- Maria Escolar, MD
-
Sub-Investigator:
- Randy Windreich, MD
-
Sub-Investigator:
- Craig Byersdorfer, MD
-
Sub-Investigator:
- Elizabeth Stenger, MD
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
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.
Study Plan
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
incidence of acute graft versus host disease (GVHD)
Time Frame: up to 5 years
|
grades 3-4, chronic extensive GVHD
|
up to 5 years
|
overall survival after HSCT
Time Frame: up to 5 years
|
review of the existing medical records to check on the participant's survival status
|
up to 5 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
|
up to 5 years
|
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
|
by 6, 9, and 12 months post-HSCT
|
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
|
over the first year post transplant
|
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
|
up to 5 years
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Paul Szabolcs, MD, UPMC Children's Hospital of Pittsburgh
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
- Rheumatoid Arthritis
- Inflammatory Bowel Disease
- Crohn's Disease
- Gaucher Disease
- Osteopetrosis
- Omenn Syndrome
- Bare Lymphocyte Syndrome (BLS)
- Combined Immune Deficiency (CID) syndromes
- Wiskott-Aldrich Syndrome
- Leukocyte adhesion deficiency
- Chronic granulomatous disease (CGD)
- IPEX syndrome
- Autoimmune Lymphoproliferative Syndrome (ALPS)
- Hemophagocytic Lymphohistiocytosis (HLH) syndromes
- Lymphocyte Signaling defects
- Congenital Amegakaryocytic Thrombocytopenia (CAMT)
- Hurler syndrome (MPS I)
- Metachromatic leukodystrophy (MLD)
- X-linked adrenoleukodystrophy (ALD)
- Thalassemia major
- Sickle cell disease (SCD)
- Diamond Blackfan Anemia (DBA)
- Severe Combined Immune Deficiency (SCID) with NK cell activity
- Hyper IgM (XHIM) syndrome
- Chediak-Higashi Syndrome
- Hurler syndrome (MPS II)
- Krabbe Disease, also known as Globoid Cell Leukodystrophy
- Alpha Mannosidosis
- IPEX or IPEX-like Syndromes
Additional Relevant MeSH Terms
- Pathologic Processes
- Immunologic Deficiency Syndromes
- Immune System Diseases
- Disease
- Bone Marrow Diseases
- Hematologic Diseases
- Infant, Newborn, Diseases
- Genetic Diseases, Inborn
- Syndrome
- Primary Immunodeficiency Diseases
- Metabolic Diseases
- Bone Marrow Failure Disorders
- Pancytopenia
- Congenital Bone Marrow Failure Syndromes
Other Study ID Numbers
- STUDY20070105
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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