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

Tracking Information
First Submitted Date ICMJE	January 14, 2012
First Posted Date ICMJE	February 8, 2012
Last Update Posted Date	April 9, 2014
Study Start Date ICMJE	December 2011
Actual Primary Completion Date	March 2014 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: February 7, 2012)	Change in laboratory values. [ Time Frame: 2 years ]; Change from baseline in laboratory values, as a measure of safety.
Original Primary Outcome Measures ICMJE	Same as current
Change History	Complete list of historical versions of study NCT01528384 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: February 7, 2012)	Results of diagnostic imaging using RECIST criteria. [ Time Frame: 2 years ]
Original Secondary Outcome Measures ICMJE	Same as current
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Ph 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient
Official Title ICMJE	A Phase 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient and Have Failed Prior Systemic Therapy
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 study hypothesis is that this drug will be safe in pediatric patients.
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.
Study Type ICMJE	Interventional
Study Phase	Phase 1
Study Design ICMJE	Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Condition ICMJE	Argininosuccinate Synthetase Deficient
Intervention ICMJE	Drug: ADI-PEG 20; 4.5, 9 and 18 mg/m2 dosage weekly dosing intramuscular until progressive disease; Other Name: pegylated arginine deiminase
Study Arms	Not Provided
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Completed
Actual Enrollment ICMJE; (submitted: April 8, 2014)	8
Original Estimated Enrollment ICMJE; (submitted: February 7, 2012)	30
Actual Study Completion Date	March 2014
Actual Primary Completion Date	March 2014 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	Inclusion Criteria: 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 Criteria: 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.
Sex/Gender	Sexes Eligible for Study: All
Ages	4 Years to 18 Years (Child, Adult)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	United States
Removed Location Countries
Administrative Information
NCT Number ICMJE	NCT01528384
Other Study ID Numbers ICMJE	POLARIS2009-002
Has Data Monitoring Committee	No
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Not Provided
Responsible Party	Polaris Group
Study Sponsor ICMJE	Polaris Group
Collaborators ICMJE	Not Provided
Investigators ICMJE	Principal Investigator: Cynthia Herzog, MD M.D. Anderson Cancer Center
PRS Account	Polaris Group
Verification Date	April 2014
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP