Status:
RECRUITING
Data Collection Study of Patients with Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT with RIC
Lead Sponsor:
Paul Szabolcs
Conditions:
Primary Immunodeficiency (PID)
Congenital Bone Marrow Failure Syndromes
Eligibility:
All Genders
2-60 years
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 strat...
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 prom...
Eligibility Criteria
Inclusion
- 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.
Exclusion
Key Trial Info
Start Date :
August 20 2020
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
June 30 2026
Estimated Enrollment :
50 Patients enrolled
Trial Details
Trial ID
NCT04528355
Start Date
August 20 2020
End Date
June 30 2026
Last Update
January 9 2025
Active Locations (1)
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1
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224