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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

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.

Condition or disease Intervention/treatment Phase
Argininosuccinate Synthetase Deficient Drug: ADI-PEG 20 Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : December 2011
Actual Primary Completion Date : March 2014
Actual Study Completion Date : March 2014

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

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

Secondary Outcome Measures :
  1. Results of diagnostic imaging using RECIST criteria. [ Time Frame: 2 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01528384

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United States, Texas
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Polaris Group
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Principal Investigator: Cynthia Herzog, MD M.D. Anderson Cancer Center

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Responsible Party: Polaris Group Identifier: NCT01528384     History of Changes
Other Study ID Numbers: POLARIS2009-002
First Posted: February 8, 2012    Key Record Dates
Last Update Posted: April 9, 2014
Last Verified: April 2014
Keywords provided by Polaris Group:
Arginine deiminase
Argininosuccinate synthetase
Phase 1
Failed other treatments
Pediatric patients