Argininosuccinate Synthetase DeficientPhase 1NCT01528384
Ph 1 Study of ADI PEG 20 in Pediatric Subjects Who Are Argininosuccinate Synthetase (ASS) Deficient
A Phase 1 study testing ADI-PEG 20 in people with Argininosuccinate Synthetase Deficient, run by Polaris Group at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 18 years
- Conditions the study is for
- Argininosuccinate Synthetase Deficient
- What is being tested?
- ADI-PEG 20
- Type of treatment
- Injection or infusion
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2011to Mar 2014
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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.
- Conditions
- Argininosuccinate Synthetase Deficient
- Being tested
- ADI-PEG 20
- Keywords
- Arginine, Arginine deiminase, Argininosuccinate synthetase, Phase 1, Pediatric, Failed other treatments, Pediatric patients
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ADI-PEG 20Drug
Also called pegylated arginine deiminase
4.5, 9 and 18 mg/m2 dosage weekly dosing intramuscular until progressive disease
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in laboratory values.
Change from baseline in laboratory values, as a measure of safety.
Measured: 2 years
Secondary outcomes (1)
Results of diagnostic imaging using RECIST criteria.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2012 (estimated)
- Study startDec 2011
- Primary completionMar 2014
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Argininosuccinate Synthetase DeficientStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Polaris Group
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Cynthia Herzog, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01528384
- Sponsor's protocol ID
- POLARIS2009-002
- Status last verified
- Apr 2014
- First submitted
- 14 Jan 2012
- Last updated
- 9 Apr 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01528384
- Acronym / sponsor ID
- POLARIS2009-002
- Conditions
- Argininosuccinate Synthetase Deficient
- Keywords
- Arginine, Arginine deiminase, Argininosuccinate synthetase, Phase 1, Pediatric, Failed other treatments, Pediatric patients
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Polaris GroupIndustry (pharma, biotech, devices)
- Study start
- Dec 2011
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 8 Feb 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 4 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ADI-PEG 20Also: pegylated arginine deiminase4.5, 9 and 18 mg/m2 dosage weekly dosing intramuscular until progressive disease | Drug | – |
Primary outcome
Change in laboratory values.
Time frame: 2 years
Change from baseline in laboratory values, as a measure of safety.
1 secondary outcome
Results of diagnostic imaging using RECIST criteria.
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2014.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Cynthia Herzog, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ADI-PEG 20 in people with Argininosuccinate Synthetase Deficient, run by Polaris Group at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Apr 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in laboratory values. (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Apr 2014.