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| ID | Type | Description | Link |
|---|---|---|---|
| CHP 894 | Other Identifier | Children's Hospital of Philadelphia |
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This is a Phase II pilot study to evaluate engraftment and toxicity of patients with non-malignant diseases using a reduced intensity conditioning regimen in the setting of allogeneic transplant for non malignant diseases. Bone Marrow or cord blood will be acceptable as a stem cell source.
Recently, reduced intensity conditioning (RIC) regimens have been used for both adult patients with leukemias and pediatric patients with non-malignant diseases. These regimens are better tolerated, resulting in less transplant related morbidity and mortality. Stable mixed chimerism, while insufficient for eradication of leukemias, may be sufficient to cure patients with non-malignant diseases.
There are two conditioning regimens in this protocol for children >6 months. Alemtuzumab (Campath), Fludarabine and Melphalan are used. The regimens differ by the timing and dosing of Alemtuzumab (Campath). The two timings are distal and intermediate.
The conditioning regimen for children with immunodeficiencies <6 months omits melphalan, and substitutes two days of busulfan. This regimen is successfully used in the United Kingdom, and has been successful in a 3 month old infant at the Children's Hospital of Philadelphia (CHOP) who engrafted with a haploidentical donor.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RIC: Distal Campath | Experimental | Day Treatment Day - 22 Inpatient: Alemtuzumab (Campath) test dose IV or SQ (subcutaneously) (subcutaneously) over 2 hours Day - 21 to-19 Alemtuzumab IV/ SQ (subcutaneously) Day - 7 to -3 Readmission to hospital Fludarabine IV Day - 2 Melphalan IV Day - 1 Begin cyclosporine infusion Day 0 Transplant: Bone marrow or cord blood infusion |
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| RIC:Intermediate Campath | Experimental | Day Treatment Day - 14 to-10 Inpatient: Alemtuzumab (Campath) IV or SQ (subcutaneously) Day - 7 to -3 Fludarabine IV Day - 2 Melphalan 140 mg/m2 IV Day - 1 Cyclosporine infusion starts Day 0 Transplant: Bone marrow or cord blood infusion |
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| RIC: Mini Busulfan | Experimental | Day Treatment Day - 8 Alemtuzumab (Campath) IV or SQ (subcutaneously) Day - 7 Alemtuzumab (Campath) IV or SQ (subcutaneously) Day - 6 Alemtuzumab (Campath) IV or SQ (subcutaneously) Busulfan IV Fludarabine IV Day - 5 Alemtuzumab (Campath) IV or SQ (subcutaneously) Busulfan IV Fludarabine IV Day - 4 Alemtuzumab (Campath) IV or SQ (subcutaneously) Fludarabine IV Day - 3 Fludarabine IV Day - 2 Fludarabine IV Cyclosporine infusion Day - 1 Rest Day 0 Transplant: Bone marrow or cord blood infusion |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RIC: Distal Campath | Drug | Campath, Fludarabine, Melphalan, Cyclosporine, Cellcept (MMF) |
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| Measure | Description | Time Frame |
|---|---|---|
| Engraftment | engraftment of patients with non-malignant disorders will be evaluated using a reduced-intensity conditioning regimen | Post Transplant -100 days |
| Measure | Description | Time Frame |
|---|---|---|
| Survival | Event free survival will be evaluated by the time interval to either the primary or late graft failure, disease recurrence or death. | 1 year post transplant |
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Inclusion Criteria:
Age >6 months- 25 years
Diseases eligible for Distal Alemtuzumab:
Diseases eligible for Intermediate Alemtuzumab
Organ criteria:
No active infections
Exclusion criteria
1. Uncontrolled bacterial, fungal or viral infections
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| Name | Affiliation | Role |
|---|---|---|
| Timothy J Olson, MD | Children's Hospital of Philadelphia | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 26348869 | Derived | King AA, Kamani N, Bunin N, Sahdev I, Brochstein J, Hayashi RJ, Grimley M, Abraham A, Dioguardi J, Chan KW, Douglas D, Adams R, Andreansky M, Anderson E, Gilman A, Chaudhury S, Yu L, Dalal J, Hale G, Cuvelier G, Jain A, Krajewski J, Gillio A, Kasow KA, Delgado D, Hanson E, Murray L, Shenoy S. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol. 2015 Dec;90(12):1093-8. doi: 10.1002/ajh.24183. Epub 2015 Oct 6. |
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| RIC:Intermediate Campath | Drug | Campath, Fludarabine, Melphalan, Cyclosporine, Cellcept (MMF) |
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| RIC: Mini Busulfan | Drug | Campath, Fludarabine, Busulfan, Cyclosporine, Cellcept (MMF) |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Jun 23, 2026 |
| ID | Term |
|---|---|
| D007153 | Immunologic Deficiency Syndromes |
| D006453 | Hemoglobinopathies |
| ID | Term |
|---|---|
| D007154 | Immune System Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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