Interleukin-2 Treatment for Wiskott-Aldrich Syndrome
Status:
Completed
Trial end date:
2016-09-01
Target enrollment:
Participant gender:
Summary
Funding Source--FDA OOPD.
Orphan Product Grant Number--1R01FD004091-01A1
Context: Wiskott-Aldrich syndrome (WAS) is a fatal, devastating disease with ill-defined
treatment modalities, which affects young boys. Classic WAS is characterized by a clinical
triad of thrombocytopenia, eczema and severe, recurrent infections. Despite diagnostic and
therapeutic advances most WAS patients die at less than 12 years of age due to infections,
hemorrhage, malignancy or complications from treatments. WAS patients suffer from herpesvirus
infections as a result of poor Natural Killer (NK) cell function (cytotoxicity). In the
laboratory, the investigators have seen correction of WAS Natural Killer Cell (NK) function
after treatment with Interleukin-2 (IL-2).
Objectives: Initiate a prospective clinical trial by treating WAS subjects with IL-2 and
using safety as the primary endpoint. Restoration of NK cell cytotoxicity and effects on
cytoskeletal dynamics are secondary endpoints. The investigators will also observe patient
clinical status (eczema, infections, use of treatment dose antibiotics, food allergies, etc).
Study Design/Setting/Participants: This is a prospective clinical trial treating 9 WAS
subjects in the Clinical Translational Research Center (CTRC) with IL-2.
Intervention: The investigators propose to subcutaneously administer 0.5 Million Units
(MU)/m2 of IL-2 daily to WAS subjects for 5 days. Research treatment will be repeated 2 and 4
months later. Inter-patient dose escalation will be employed to 1 MU/m2 and/or 2 MU/m2 based
on safety as the primary endpoint.
Study Measures: The investigators will observe safety and tolerability measures and perform
assays on subject blood samples prior to and after research treatment to observe improvement
in NK cell function.
Phase:
Phase 1
Details
Lead Sponsor:
Soma Jyonouchi
Collaborators:
Baylor College of Medicine Texas Children's Hospital