Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders (RIC)

  • STATUS
    Recruiting
  • End date
    Dec 8, 2024
  • participants needed
    50
  • sponsor
    Children's Hospital of Philadelphia
Updated on 8 April 2022
fludarabine
mycophenolate mofetil
immunodeficiency
busulfan
cyclosporine
melphalan
transplant conditioning
shortening fraction
reduced intensity conditioning
alemtuzumab
campath
bone marrow failure
wiskott-aldrich syndrome
thalassemia
histiocytosis
severe combined immunodeficiency

Summary

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.

Description

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 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.

Details
Condition Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
Treatment RIC: Distal Campath, RIC:Intermediate Campath, RIC: Mini Busulfan
Clinical Study IdentifierNCT01050855
SponsorChildren's Hospital of Philadelphia
Last Modified on8 April 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Age >6 months- 25 years
Diseases eligible for Distal Alemtuzumab
Immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome
Sickle cell disease
Thalassemia major
Bone marrow failure
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
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
No active infections

Exclusion Criteria

Uncontrolled bacterial, fungal or viral infections
Clear my responses

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