Ph 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient
This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
First received: January 14, 2012
Last updated: November 27, 2013
Last verified: November 2013
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.
Argininosuccinate Synthetase Deficient
Drug: ADI-PEG 20
||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
||A Phase 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient and Have Failed Prior Systemic Therapy
Primary Outcome Measures:
Secondary Outcome Measures:
- Results of diagnostic imaging using RECIST criteria. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
| Estimated Enrollment:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||January 2014 (Final data collection date for primary outcome measure)
Drug: ADI-PEG 20
4.5, 9 and 18 mg/m2 dosage weekly dosing intramuscular until progressive disease
Other Name: pegylated arginine deiminase
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.
|Ages Eligible for Study:
||4 Years to 18 Years
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01528384
|MD Anderson Cancer Center
|Houston, Texas, United States, 77030 |
||Cynthia Herzog, MD
||M.D. Anderson Cancer Center
No publications provided
History of Changes
|Other Study ID Numbers:
|Study First Received:
||January 14, 2012
||November 27, 2013
||United States: Food and Drug Administration
Keywords provided by Polaris Group:
ClinicalTrials.gov processed this record on March 09, 2014
Failed other treatments