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

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Polaris Group

ClinicalTrials.gov Identifier:

NCT01528384

First received: January 14, 2012

Last updated: April 8, 2014

Last verified: April 2014

History of Changes

Tracking Information

First Received Date ^ICMJE

January 14, 2012

Last Updated Date

April 8, 2014

Start Date ^ICMJE

December 2011

Primary Completion Date

March 2014 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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

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: 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

Enrollment ^ICMJE

Completion Date

March 2014

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

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

U.S. FDA-regulated Product

Not Provided

Plan to Share Data

Not Provided

IPD Description

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

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