Transplant protocol for immune deficiency disorders
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime
This study is testing a new way to help young patients with immune deficiency disorders successfully receive a donor stem cell transplant while keeping side effects low.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | N/A to 21 Years |
| Sex | All |
| Sponsor | Washington University School of Medicine Academic / other |
| Drugs / interventions | alemtuzumab |
| Locations | 1 site (Saint Louis, Missouri) |
| Trial ID | NCT01821781 on ClinicalTrials.gov |
What this trial studies
This study investigates a reduced intensity immunosuppressive regimen aimed at facilitating the engraftment of donor hematopoietic cells in patients with various immune deficiency disorders. The approach utilizes a combination of alemtuzumab, fludarabine, thiotepa, and melphalan to maximize host immune suppression, thereby reducing the risk of graft rejection. The trial focuses on patients aged 28 years or younger who meet specific health criteria and have a suitably matched donor. The goal is to achieve successful engraftment with manageable toxicity levels.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 28 years or younger with specific immune deficiency disorders and suitable matched donors.
Not a fit: Patients with known HIV infections, uncontrolled infections, or those who are pregnant or breastfeeding may not benefit from this study.
Why it matters
Potential benefit: If successful, this protocol could significantly improve the outcomes for patients with severe immune deficiencies by enhancing donor cell engraftment.
How similar studies have performed: Other studies have shown promise with similar immunosuppressive approaches, indicating potential for success in this area.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * \</= 28 years of age * Performance status \>/= 40 * DLCO \>/= 40% * LVEF \>/=40% or LVSF \>/=26% * Serum creatinine \< 2x ULN * Liver enzymes \</= 5x ULN * Negative pregnancy test * Suitably matched donor (6/6 matched sib UCB, 8/8 matched sib BM or PBSC, 5-6/6 matched unrelated UCB, 7-8/8 matched unrelated BM, double cord) Exclusion Criteria: * Known diagnosis of HIV I/II * Pregnant or breastfeeding * Uncontrolled invasive fungal or bacterial infections within 1 month prior to starting alemtuzumab * Uncontrolled viral infection within 1 week prior to starting alemtuzumab
Where this trial is running
Saint Louis, Missouri
- Washington University — Saint Louis, Missouri, United States (Recruiting)
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.