Status:
COMPLETED
Interleukin-2 Treatment for Wiskott-Aldrich Syndrome
Lead Sponsor:
Soma Jyonouchi
Collaborating Sponsors:
Baylor College of Medicine
Conditions:
Wiskott-Aldrich Syndrome (WAS)
X-linked Thrombocytopenia
Eligibility:
All Genders
24+ years
Phase:
PHASE1
Brief 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 ...
Detailed Description
The Wiskott-Aldrich syndrome (WAS) is a fatal genetic disease of the immune system that results from a mutation of the WAS protein (WASp) gene. Immune cells that carry this mutation have a decreased a...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
October 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2016
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT00774358
Start Date
October 1 2008
End Date
September 1 2016
Last Update
August 16 2017
Active Locations (3)
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1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
2
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
3
The Texas Children's Hospital
Houston, Texas, United States, 77030