Trials / Active Not Recruiting
Active Not RecruitingNCT01821781
Immune Disorder HSCT Protocol
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime
- Status
- Active Not Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 20 (estimated)
- Sponsor
- Washington University School of Medicine · Academic / Other
- Sex
- All
- Age
- 21 Years
- Healthy volunteers
- Not accepted
Summary
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.
Conditions
- Immune Deficiency Disorders
- Severe Combined Immunodeficiency
- Chronic Granulomatous Disease
- X-linked Agammaglobulinemia
- Wiskott-Aldrich Syndrome
- Hyper-IgM
- DiGeorge Syndrome
- Chediak-Higashi Syndrome
- Common Variable Immune Deficiency
- Immune Dysregulatory Disorders
- Hemophagocytic Lymphohistiocytosis
- IPEX
- Autoimmune Lymphoproliferative Syndrome
- X-linked Lymphoproliferative Syndrome
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan | Between days -23 and -15: alemtuzumab test dose, 3mg IV or SQ Day -14: alemtuzumab, 10mg IV or SQ Day -13: alemtuzumab, 15mg IV or SQ Day -12: alemtuzumab, 20mg IV or SQ Days -8 to -4: fludarabine, 30mg/m2 IV Day -4: thiotepa 4mg/kg IV q 12 hours Day -3: melphalan, 140mg/m2 IV Day 0: stem cell infusion Day +7: G-CSF |
Timeline
- Start date
- 2013-04-29
- Primary completion
- 2025-04-25
- Completion
- 2026-04-01
- First posted
- 2013-04-01
- Last updated
- 2026-02-19
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT01821781. Inclusion in this directory is not an endorsement.