Ph 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01528384
Recruitment Status : Completed
First Posted : February 8, 2012
Last Update Posted : April 9, 2014
Information provided by (Responsible Party):
Polaris Group

January 14, 2012
February 8, 2012
April 9, 2014
December 2011
March 2014   (Final data collection date for primary outcome measure)
Change in laboratory values. [ Time Frame: 2 years ]
Change from baseline in laboratory values, as a measure of safety.
Same as current
Complete list of historical versions of study NCT01528384 on Archive Site
Results of diagnostic imaging using RECIST criteria. [ Time Frame: 2 years ]
Same as current
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Ph 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient
A Phase 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient and Have Failed Prior Systemic Therapy
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 study hypothesis is that this drug will be safe in pediatric patients.
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.
Phase 1
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Argininosuccinate Synthetase Deficient
Drug: ADI-PEG 20
4.5, 9 and 18 mg/m2 dosage weekly dosing intramuscular until progressive disease
Other Name: pegylated arginine deiminase
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
March 2014
March 2014   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Prior diagnosis of failed current standard treatment(s), or AEs associated with prior systemic agent(s) that resulted in discontinuance of that agent(s).
  2. Argininosuccinate synthetase deficient tumor.
  3. Measurable disease as assessed by >5% blasts on bone marrow aspirate for patients with leukemia or using RECIST 1.1 criteria for solid tumors.
  4. Age 4 to ≤ 18 years.
  5. 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.
  6. 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.
  7. 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.
  8. No concurrent investigational studies are allowed.
  9. For solid tumor subjects only: Absolute neutrophil count (ANC) >750/µL.
  10. For solid tumor subjects only: Platelets >50,000/µL.
  11. For solid tumor subjects only: Serum uric acid ≤ 8 mg/dL (with or without medication control).
  12. 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 Criteria:

  1. Serious infection requiring treatment with systemically administered antibiotics at the time of study entrance.
  2. Pregnancy or lactation.
  3. Expected non-compliance.
  4. Uncontrolled intercurrent illness.
  5. 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.
  6. Subjects who had been treated with ADI-PEG 20 previously.
  7. History of seizure disorder not related to underlying cancer.
  8. Known HIV positivity.
Sexes Eligible for Study: All
4 Years to 18 Years   (Child, Adult)
Contact information is only displayed when the study is recruiting subjects
United States
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Polaris Group
Polaris Group
Not Provided
Principal Investigator: Cynthia Herzog, MD M.D. Anderson Cancer Center
Polaris Group
April 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP