Hematopoietic stem cell transplant for patients with immune deficiencies
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
This study is testing if a type of stem cell transplant can help people with immune deficiencies feel better and improve their health.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | N/A to 50 Years |
| Sex | All |
| Sponsor | Masonic Cancer Center, University of Minnesota Academic / other |
| Locations | 1 site (Minneapolis, Minnesota) |
| Trial ID | NCT01652092 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the effectiveness of allogeneic hematopoietic stem cell transplantation (HSCT) as a treatment for patients with primary immune deficiencies. Participants will be assigned to one of four treatment arms based on their specific condition and clinical history, which may include fully myeloablative, reduced toxicity ablative, reduced intensity conditioning, or no preparative regimen. The study aims to determine the most appropriate regimen to improve patient outcomes in various immunodeficiency disorders.
Who should consider this trial
Good fit: Ideal candidates include individuals diagnosed with severe combined immunodeficiency, Omenn's syndrome, Wiskott-Aldrich syndrome, and other specified primary immune deficiencies.
Not a fit: Patients with mild immunodeficiencies or those who do not meet the specific eligibility criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve immune function and quality of life for patients with severe immunodeficiencies.
How similar studies have performed: Previous studies have shown success with allogeneic HSCT in treating similar conditions, indicating a promising approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria:
* Diagnosis of immunodeficiency or histiocytic disorder including the following:
* Severe combined immunodeficiency (SCID - all variants)
* Second bone marrow transplant (BMT) for SCID (after graft rejection)
* Omenn's Syndrome
* Reticular dysgenesis
* Wiskott-Aldrich syndrome
* Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte syndrome)
* Hyper IgM Syndrome (CD40 Ligand Deficiency)
* Common variable immunodeficiency (CVID) with severe phenotype
* Chronic Granulomatous Disease (CGD)
* Other severe Combined Immune Deficiencies (CID)
* Hemophagocytic Lymphohistiocytosis (HLH)
* X-linked Lymphoproliferative Disease (XLP)
* Chediak-Higashi Syndrome (CHS)
* Griscelli Syndrome
* Langerhans Cell Histiocytosis (LCH)
* Acceptable stem cell sources include:
* HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow
* HLA identical or up to a 1 antigen mismatched unrelated BM donor
* Sibling donor cord blood with acceptable HLA match and cell dose as per current institutional standards
* Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum cell dose of \>5 x 10\^7 nucleated cells/kg as per current institutional guidelines
* Double unrelated umbilical cord blood units that are:
* up to 2 antigen mismatched to the patient
* up to 2 antigen mismatched to each other
* minimum cell dose of at least one single unit must be ≥ 3.5 x 10\^7 nucleated cells/kg
* combined dose of both units must provide a total cell dose of ≥ 5 x 10\^7 nucleated cells/kg
* Age: 0 to 50 years
* Adequate organ function and performance status.
Exclusion Criteria
* pregnant or breastfeeding
* active, uncontrolled infection and/or HIV positive
* acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
Where this trial is running
Minneapolis, Minnesota
- Masonic Cancer Center, University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)
Study contacts
- Principal investigator: Christen Ebens, MD — Masonic Cancer Center, University of Minnesota
- Study coordinator: Christen Ebens, MD
- Email: ebens012@umn.edu
- Phone: 612-626-2778
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.