Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
Completed · Phase 2
Conditions studied: Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
In brief
This Phase II pilot study evaluates the safety and effectiveness of reduced-intensity conditioning (RIC) regimens prior to allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric and young adult patients with non-malignant disorders, including immunodeficiencies and hemoglobinopathies. The study examines different conditioning approaches that vary in the timing and dosing of alemtuzumab (Campath), as well as disease-specific modifications for beta thalassemia major and infants. The primary objective is to assess donor engraftment and event-free survival at one year following transplant. The study aims to determine whether RIC regimens can reduce toxicity while maintaining successful engraftment and disease control.
Key facts
- Study ID
- NCT01050855
- Run by
- Children's Hospital of Philadelphia
- People needed
- 56
- Starts
- 2008-01-01
- Expected to finish
- 2025-06-01
- Last updated by the study team
- 2026-07-21
Who can join
Age: 1 and older, up to 25. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Age >6 months- 25 years
- Diseases eligible for Distal Alemtuzumab:
- Immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome
- Sickle cell disease (Neurologic: history of stroke or any neurologic defect lasting >24 hours accompanied by infarct on MRI; or abnormal transcranial Doppler, or abnormal MRI with cerebral vasculature stenosis. OR minimum 2 episodes of acute chest syndrome in preceding 2 year period OR history 3 or more severe pain events/year in the 2 years prior to transplant.)
- Thalassemia major
- Bone marrow failure, including Kostmann's, amegakaryocytic thrombocytopenia, Blackfan-Diamond syndrome
- 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%
- Pulmonary: for those with pulmonary function tests: DLCO/VA >40% If DLCO/VA is not reported this is considered inability to perform the test. In this case Pulse Ox and Respiratory Exam will be used as evaluation criteria.
- Renal: Serum creatinine <1.5 x upper limit of normal for age
- Hepatic:; ALT and AST <5 x upper limit of normal
- Infection: No active infections.
You may not qualify if…
- Uncontrolled bacterial, fungal or viral infections.
- Bare lymphocyte syndrome (MHC class II deficiency)
Where it is running
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Full record on ClinicalTrials.gov
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