Interleukin-2 Treatment for Wiskott-Aldrich Syndrome
Completed · Phase 1
Conditions studied: Wiskott-Aldrich Syndrome (WAS), X-linked Thrombocytopenia
In brief
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.
Key facts
- Study ID
- NCT00774358
- Run by
- Soma Jyonouchi
- People needed
- 9
- Starts
- 2008-10-01
- Expected to finish
- 2016-09-01
- Last updated by the study team
- 2017-08-16
Who can join
Age: 2 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Age: Subjects age greater than 24 months
- Weight: Subjects greater than 12.5 kilograms
- Disease status: WAS classified as Grade 1-4
- Informed Consent: Written informed consent of the subject (if an adult) or parental permission, and assent of the child subject provided justification is made for the inclusion of children in the study
You may not qualify if…
- Prior or planned hematopoetic transplant
- WAS classified as currently Grade 5 (Malignancy or autoimmune disease including the following: Crohn's disease, scleroderma, thyroiditis, inflammatory arthritis, diabetes mellitus, oculo-bulbar myasthenia gravis, crescentic IgA glomerulonephritis, cholecystitis, cerebral vasculitis, Stevens-Johnson syndrome and bullous pemphigoid . Not included here are: Hepatitis C virus induced vasculitis, alopecia areata and systemic lupus erythematosus.)
- Known previous reaction to IL-2
- Subjects taking immunosuppressive medications that might alter study results
- Subjects taking nephrotoxic, cytotoxic, cardiotoxic, or hepatotoxic medications (including medications for hypertension)
- Subjects currently taking systemic corticosteroids (not included here: topical and inhaled corticosteroids)
- Subjects taking Interferon alpha
- Use of any other investigational agent in the last 30 days
- Women of childbearing potential not using contraception method(s), as well as women who are breastfeeding
- Subjects with abnormal cardiac, hepatic and CNS function
Where it is running
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Hospital of the University of Pennsylvania — Philadelphia, Pennsylvania, United States
- The Texas Children's Hospital — Houston, Texas, United States
Full record on ClinicalTrials.gov
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