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

NCT01528384 | Completed Phase 1

• 1 other identifier: POLARIS2009-002
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

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.

Conditions

Argininosuccinate Synthetase Deficient

Keywords

Arginine; Arginine deiminase; Argininosuccinate synthetase; Phase 1; Pediatric; Failed other treatments; Pediatric patients

Outcome Measures

Primary Outcomes (1)

• Change in laboratory values.

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

  2 years

Secondary Outcomes (1)

• Results of diagnostic imaging using RECIST criteria.

  2 years

Interventions

ADI-PEG 20 DRUG

4.5, 9 and 18 mg/m2 dosage weekly dosing intramuscular until progressive disease

Also known as: pegylated arginine deiminase

Eligibility Criteria

• Age: 4 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Polaris Group: lead

Study Sites (1)

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Interventions

ADI PEG20

Study Officials

• Cynthia Herzog, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 14, 2012
• First Posted: February 8, 2012
• Study Start: December 1, 2011
• Primary Completion: March 1, 2014
• Study Completion: March 1, 2014
• Last Updated: April 9, 2014

Record last verified: 2014-04

Locations

US (1)