Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
| Status: | Recruiting |
|---|---|
| Conditions: | Infectious Disease, HIV / AIDS, Hematology |
| Therapuetic Areas: | Hematology, Immunology / Infectious Diseases |
| Healthy: | No |
| Age Range: | Any - 25 |
| Updated: | 3/27/2019 |
| Start Date: | January 2008 |
| End Date: | December 2020 |
| Contact: | Barbara McGlynn |
| Email: | mcglynn@email.chop.edu |
| Phone: | 215-590-1303 |
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.
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.
- Distal campath is initiated 22 days prior to the allogeneic transplant.
- Intermediate campath is initiated 14 days prior to allogeneic transplant.
The conditioning regimen for children with immunodeficiencies <6 months omits melphalan, and
substitutes two days of busulfan. This regimen is successfully used in the UK, and has been
successful in a 3 month old infant at CHOP who engrafted with a haploidentical donor.
(Campath), Fludarabine and Melphalan are used. The regimens differ by the timing and dosing
of Alemtuzumab (Campath). The two timings are distal and intermediate.
- Distal campath is initiated 22 days prior to the allogeneic transplant.
- Intermediate campath is initiated 14 days prior to allogeneic transplant.
The conditioning regimen for children with immunodeficiencies <6 months omits melphalan, and
substitutes two days of busulfan. This regimen is successfully used in the UK, and has been
successful in a 3 month old infant at CHOP who engrafted with a haploidentical donor.
Inclusion Criteria:
1. Age >6 months- 25 years
2. Diseases eligible for Distal Alemtuzumab:
- Immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome
- Sickle cell disease
- Thalassemia major
- Bone marrow failure
3. Diseases eligible for Intermediate Alemtuzumab
- Hemophagocytic lymphohistiocytosis other macrophage activation syndromes, severe
Langerhans histiocytosis
- Severe combined immune deficiency, adenosine deaminase deficiency, common
variable immunodeficiency
- Wiskott-Aldrich syndrome
4. Organ criteria:
- Cardiac: Echocardiogram shortening fraction >27%
- Renal: Serum creatinine less than 1.5 times the upper limit of normal for age
- Hepatic: liver function tests must be less than 5 times the upper limit of normal
5. No active infections
Exclusion criteria
1. Uncontrolled bacterial, fungal or viral infections
We found this trial at
1
site
South 34th Street
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
215-590-1000
Principal Investigator: Nancy J Bunin, MD
Phone: 215-590-1303
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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