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A Phase 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient and Have Failed Prior Systemic Therapy


Phase 1
4 Years
18 Years
Open (Enrolling)
Both
Argininosuccinate Synthetase Deficient

Thank you

Trial Information

A Phase 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient and Have Failed Prior Systemic Therapy


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.


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.

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Change in laboratory values.

Outcome Description:

Change from baseline in laboratory values, as a measure of safety.

Outcome Time Frame:

2 years

Safety Issue:

Yes

Principal Investigator

Cynthia Herzog, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center

Authority:

United States: Food and Drug Administration

Study ID:

POLARIS2009-002

NCT ID:

NCT01528384

Start Date:

December 2011

Completion Date:

July 2014

Related Keywords:

  • Argininosuccinate Synthetase Deficient
  • Arginine
  • Arginine deiminase
  • Argininosuccinate synthetase
  • Phase 1
  • Pediatric
  • Failed other treatments
  • Pediatric patients

Name

Location

MD Anderson Cancer CenterHouston, Texas  77030-4096