Interleukin-2 Treatment for Wiskott-Aldrich Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00774358
• Date of registration: 16/10/2008
• Prospective Registration: No
• Primary sponsor: Soma Jyonouchi
• Public title: Interleukin-2 Treatment for Wiskott-Aldrich Syndrome WAS
• Scientific title: Reinstituting Natural Killer Cell Cytotoxicity and Cytoskeletal Dynamics in Wiskott-Aldrich Syndrome With IL-2 Therapy
• Date of first enrolment: October 2008
• Target sample size: 9
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00774358
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Soma Jyonouchi, MD
• Affiliation: Children's Hospital of Philadelphia

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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



Age minimum: 24 Months
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Wiskott-Aldrich Syndrome (WAS)

X-linked Thrombocytopenia

Intervention(s)

Drug: Interleukin-2

Primary Outcome(s)

Safety and Tolerability [Time Frame: 1 year]

Secondary Outcome(s)

Number and severity of infections [Time Frame: 1 year]

Food allergies [Time Frame: 1 year]

Eczema [Time Frame: 1 year]

Requirement for treatment dose antibiotics [Time Frame: 1 year]

NK cell cytotoxicity [Time Frame: 1 year]

Evaluate effects on cytoskeletal dynamics [Time Frame: 1 year]

Secondary ID(s)

2007-6-5354

1R01FD004091-01A1

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Texas Children's Hospital