Status:
COMPLETED
Ph 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient
Lead Sponsor:
Polaris Group
Conditions:
Argininosuccinate Synthetase Deficient
Eligibility:
All Genders
4-18 years
Phase:
PHASE1
Brief Summary
This is a phase 1 study of pegylated arginine deiminase (ADI-PEG 20) in pediatric patients who deficient in the expression of tumor argininosuccinate synthetase, and have failed prior treatments. The ...
Detailed Description
This phase 1 study will use a classic 3 dose levels by 3 patients per dose level design. Once the maximum tolerated dose is reached, additional patients may be enrolled at that dose level.
Eligibility Criteria
Inclusion
- Prior diagnosis of failed current standard treatment(s), or AEs associated with prior systemic agent(s) that resulted in discontinuance of that agent(s).
- Argininosuccinate synthetase deficient tumor.
- Measurable disease as assessed by \>5% blasts on bone marrow aspirate for patients with leukemia or using RECIST 1.1 criteria for solid tumors.
- Age 4 to ≤ 18 years.
- No prior systemic treatment for the tumor in the last 2 weeks for solid tumors and 1 week for leukemia, except hydroxyurea used only to control very high WBC.
- Fully recovered from any prior surgery and no major surgery within 4 weeks of initiating treatment. Surgery for placement of vascular access devices is acceptable.
- Female subjects of 10 years and older and male subjects must be asked to use appropriate contraception for both the male and female for the duration of the study.
- No concurrent investigational studies are allowed.
- For solid tumor subjects only: Absolute neutrophil count (ANC) \>750/µL.
- For solid tumor subjects only: Platelets \>50,000/µL.
- For solid tumor subjects only: Serum uric acid ≤ 8 mg/dL (with or without medication control).
- Serum creatinine ≤ 1.5 x the upper limit of normal range, or, if serum creatinine \>1.5 x the upper limit of normal range, then the creatinine clearance must be ≥ 60 mL/min.
Exclusion
- Serious infection requiring treatment with systemically administered antibiotics at the time of study entrance.
- Pregnancy or lactation.
- Expected non-compliance.
- Uncontrolled intercurrent illness.
- Subjects who have had any anticancer treatment prior to entering the study and have not recovered to baseline or ≤ Grade 1 AEs due to agents administered.
- Subjects who had been treated with ADI-PEG 20 previously.
- History of seizure disorder not related to underlying cancer.
- Known HIV positivity.
Key Trial Info
Start Date :
December 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2014
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT01528384
Start Date
December 1 2011
End Date
March 1 2014
Last Update
April 9 2014
Active Locations (1)
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1
MD Anderson Cancer Center
Houston, Texas, United States, 77030