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

This study is currently recruiting participants.
Verified August 2012 by Polaris Group
Sponsor:
Information provided by (Responsible Party):
Polaris Group
ClinicalTrials.gov Identifier:
NCT01528384
First received: January 14, 2012
Last updated: August 8, 2012
Last verified: August 2012
History of Changes
  Purpose

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.


Condition Intervention Phase
Argininosuccinate Synthetase Deficient
 Drug: ADI-PEG 20
 Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient and Have Failed Prior Systemic Therapy

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Polaris Group:

Primary Outcome Measures:
  • Change in laboratory values. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
    Change from baseline in laboratory values, as a measure of safety.


Secondary Outcome Measures:
  • Results of diagnostic imaging using RECIST criteria. [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 30
Study Start Date: December 2011
Estimated Study Completion Date: July 2014
Estimated Primary Completion Date: January 2014 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: ADI-PEG 20
    4.5, 9 and 18 mg/m2 dosage weekly dosing intramuscular until progressive disease
    Other Name: pegylated arginine deiminase
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

Ages Eligible for Study:   4 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01528384

Contacts
Contact: John Bomalaski, MD 858-452-6688 ext 114 johnsbomalaski@polarispharma.com

Locations
United States, Texas
MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Beena Abraham     713-792-2539     BAbraham1@mdanderson.org    
Principal Investigator: Cynthia Herzog, MD            
Sub-Investigator: Robert Wells, MD            
Sub-Investigator: Cesar Nunez, MD            
Sponsors and Collaborators
Polaris Group
Investigators
Principal Investigator: Cynthia Herzog, MD M.D. Anderson Cancer Center
  More Information

No publications provided

Responsible Party: Polaris Group
ClinicalTrials.gov Identifier: NCT01528384     History of Changes
Other Study ID Numbers: POLARIS2009-002
Study First Received: January 14, 2012
Last Updated: August 8, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Polaris Group:
Arginine
Arginine deiminase
Argininosuccinate synthetase
Phase 1
 Pediatric
Failed other treatments
Pediatric patients

ClinicalTrials.gov processed this record on May 19, 2013