NeoplasmsPhase 1NCT00100672
Study to Determine the Maximum Tolerated Dose of LErafAON in Patients With Advanced Cancer
A Phase 1 study testing LErafAON-ETU in people with Neoplasms, run by INSYS Therapeutics Inc at 2 sites in United States.
Trial summary
The primary purpose of this study is to identify a dose of Liposome Entrapped c-raf Antisense Oligonucleotide Easy-to-Use (LErafAON-ETU) which maximizes potential benefits of the compound to patients with advanced cancer, without compromising their safety. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neoplasms
- What is being tested?
- LErafAON-ETU
- How many people
- 40 participants
- Where
- 2 sitesUnited States
- Study dates
- Dec 2004to Nov 2010
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- To be included in this study, patients must meet the following criteria:
- Be ≥18 years of age.
- Have advanced (local and/or metastatic) histologically documented cancer not considered responsive to available conventional modalities or treatment (i.e., no life prolonging therapy or therapy with a greater potential for patient benefit is available).
- Have an ECOG Performance status of 0-1.
- Have a life expectancy of >12 weeks.
- Have recovered from acute toxicities of prior treatment: *No treatment with radiotherapy or with cytotoxic or biologic agents within 3 weeks prior to study entry. At least 2 weeks must have elapsed since any prior surgery or granulocyte-stimulating growth factor therapy. Chronic treatment with non-investigational gonadotropin-releasing hormone agonists or other hormonal or supportive care is permitted. Concurrent bisphosphonate treatment is permitted if initiated ≥90 days prior to study entry. *Chronic Grade 1 toxicities due to prior treatment or other causes are permitted.
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- Be in adequate condition as evidenced by the following clinical laboratory values:
- Absolute neutrophil count (ANC) ≥1,500/mm³,
- Hemoglobin ≥9.0 g/dL,
- Platelets ≥125,000/mm³,
- PT, aPTT, creatinine, calcium, and total bilirubin ≤the institutional upper limit of normal (ULN),
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5 x ULN,
- Alkaline phosphatase ≤2.5 x ULN
- Patients (male and female) must be willing to practice an effective method of birth control during the study.
- Patient must understand the investigational nature of this study and sign an Institutional Review Board (IRB) approved informed consent form prior to the performance of any study specific procedure.
Who cannot join
- Patients are excluded from this study for the following:
- Active uncontrolled bleeding or bleeding diathesis (e.g., active peptic ulcer disease).
- Any active infection requiring parenteral or oral antibiotic treatment.
- Known infection with human immunodeficiency virus or hepatitis virus.
- Active heart disease including myocardial infarction within the previous 6 months, symptomatic coronary artery disease, arrhythmias currently requiring medication, or congestive heart failure.
- Known or suspected active central nervous system metastasis (patients stable 8 weeks after completion of treatment for central nervous system metastasis are eligible).
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- Requiring immediate palliative treatment of any kind, including surgery and/or radiotherapy
- Concurrent anti-tumor therapy (except for chronic hormonal anti-tumor therapy).
- Treatment with any investigational drug within the 30-day period prior to enrollment in the study.
- Known hypersensitivity to any of the components of LErafAON-ETU.
- Prior treatment with LErafAON (previous sonicated formulation).
- Female patients who are pregnant or breast-feeding.
- Unwilling or unable to follow protocol requirements.
- Any consideration which in the Investigator's opinion deems the patient an unsuitable candidate to receive study drug.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Premiere Oncology-Santa Monica
Santa Monica, California, 90404
premiere Oncology-Arizona
Scottsdale, Arizona, 85260
About this study
Official title: Phase I Study of an Easy-to-Use Intravenous Formulation of Liposome Entrapped C-raf Antisense Oligonucleotide (LErafAON-ETU) Administered on a Weekly Schedule in Patients With Advanced Cancer
The primary purpose of this study is to identify a dose of Liposome Entrapped c-raf Antisense Oligonucleotide Easy-to-Use (LErafAON-ETU) which maximizes potential benefits of the compound to patients with advanced cancer, without compromising their safety. This study will also assess the processing of LErafAON-ETU by the body over time. Patients will receive an intravenous infusion of LErafAON-ETU each week. Multiple blood samples will be taken for pharmacokinetic analysis during the first treatment; two samples will be taken during both the second and third treatments. Patients will be eligible to continue treatment until the occurrence of unacceptable toxicity or disease progression.
In LErafAON-ETU, antisense oligonucleotides specific to c-raf, are associated with liposomes, which are microscopic membrane-like structures created from lipids (fats). Raf-1 is a protein which plays a critical role in many aspects of cellular activation and growth. Therefore, it is thought to be an important factor that may support tumor development. LErafAON-ETU potentially limits the ability of a cell to produce the Raf-1 protein.
Read the detailed description
This Phase I, open-label, dose-escalation study is designed to determine the maximum tolerated dose of LErafAON-ETU in patients who have advanced cancer considered unresponsive to available, conventional modalities or treatments. LErafAON-ETU will be administered as an IV infusion once weekly for 3 consecutive weeks (a Treatment Cycle). A complete pharmacokinetic profile of raf-1 antisense oligonucleotide will be assessed in week 1 only; limited pharmacokinetic sampling will be done prior to and at the end of infusion in weeks 2 and 3 only. Tumor/disease evaluation will be performed upon completion of 6 infusions (2 Cycles). Dose escalation will not occur until the safety and tolerability at a given dose level has been confirmed for 1 Cycle.
- Conditions
- Neoplasms
- Being tested
- LErafAON-ETU
- Keywords
- LErafAON-ETU, Signal transduction, Neoplasm, Cancer, NeoPharm, Liposomes, Antisense, Anti-cancer, Advanced neoplasm, Advanced cancer, Chemotherapy, Refractory cancer, Metastatic cancer
- Medical subject headings
- Neoplasms, Neoplasm Metastasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LErafAON-ETUDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the maximum tolerated dose (MTD) and dose-limiting toxicity (DLT) of LErafAON-ETU.
Secondary outcomes (2)
To determine the pharmacokinetics of raf antisense oligonucleotide after intravenous administration of LErafAON-ETU
To determine any anti-tumor effects of LErafAON-ETU.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2005 (estimated)
- Study startDec 2004
- Primary completionOct 2009
- Study completionNov 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeoplasmsStudies 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
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants
Sponsor and investigators
- Lead sponsor
- INSYS Therapeutics Inc
- Sponsor type
- Industry (pharma, biotech, devices)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00100672
- Sponsor's protocol ID
- LErafAON-ETU-104-R02
- Status last verified
- Jun 2011
- First submitted
- 4 Jan 2005
- Last updated
- 4 Jul 2011
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jul 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of an Easy-to-Use Intravenous Formulation of Liposome Entrapped C-raf Antisense Oligonucleotide (LErafAON-ETU) Administered on a Weekly Schedule in Patients With Advanced Cancer
- ClinicalTrials.gov ID
- NCT00100672
- Acronym / sponsor ID
- LErafAON-ETU-104-R02
- Conditions
- Neoplasms
- Keywords
- LErafAON-ETU, Signal transduction, Neoplasm, Cancer, NeoPharm, Liposomes, Antisense, Anti-cancer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants
- Lead sponsor
- INSYS Therapeutics IncIndustry (pharma, biotech, devices)
- Study start
- Dec 2004
- Primary completion
- Oct 2009 (actual)
- Study completion
- Nov 2010 (actual)
- First posted
- 5 Jan 2005 (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.
The primary purpose of this study is to identify a dose of Liposome Entrapped c-raf Antisense Oligonucleotide Easy-to-Use (LErafAON-ETU) which maximizes potential benefits of the compound to patients with advanced cancer, without compromising their safety. This study will also assess the processing of LErafAON-ETU by the body over time. Patients will receive an intravenous infusion of LErafAON-ETU each week. Multiple blood samples will be taken for pharmacokinetic analysis during the first treatment; two samples will be taken during both the second and third treatments. Patients will be eligible to continue treatment until the occurrence of unacceptable toxicity or disease progression.
In LErafAON-ETU, antisense oligonucleotides specific to c-raf, are associated with liposomes, which are microscopic membrane-like structures created from lipids (fats). Raf-1 is a protein which plays a critical role in many aspects of cellular activation and growth. Therefore, it is thought to be an important factor that may support tumor development. LErafAON-ETU potentially limits the ability of a cell to produce the Raf-1 protein.
Detailed description
This Phase I, open-label, dose-escalation study is designed to determine the maximum tolerated dose of LErafAON-ETU in patients who have advanced cancer considered unresponsive to available, conventional modalities or treatments. LErafAON-ETU will be administered as an IV infusion once weekly for 3 consecutive weeks (a Treatment Cycle). A complete pharmacokinetic profile of raf-1 antisense oligonucleotide will be assessed in week 1 only; limited pharmacokinetic sampling will be done prior to and at the end of infusion in weeks 2 and 3 only. Tumor/disease evaluation will be performed upon completion of 6 infusions (2 Cycles). Dose escalation will not occur until the safety and tolerability at a given dose level has been confirmed for 1 Cycle.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Be ≥18 years of age.
- Have advanced (local and/or metastatic) histologically documented cancer not considered responsive to available conventional modalities or treatment (i.e., no life prolonging therapy or therapy with a greater potential for patient benefit is available).
- Have an ECOG Performance status of 0-1.
- Have a life expectancy of >12 weeks.
- Have recovered from acute toxicities of prior treatment: *No treatment with radiotherapy or with cytotoxic or biologic agents within 3 weeks prior to study entry. At least 2 weeks must have elapsed since any prior surgery or granulocyte-stimulating growth factor therapy. Chronic treatment with non-investigational gonadotropin-releasing hormone agonists or other hormonal or supportive care is permitted. Concurrent bisphosphonate treatment is permitted if initiated ≥90 days prior to study entry. *Chronic Grade 1 toxicities due to prior treatment or other causes are permitted.
Key exclusion criteria (14 listed)
- Active uncontrolled bleeding or bleeding diathesis (e.g., active peptic ulcer disease).
- Any active infection requiring parenteral or oral antibiotic treatment.
- Known infection with human immunodeficiency virus or hepatitis virus.
- Active heart disease including myocardial infarction within the previous 6 months, symptomatic coronary artery disease, arrhythmias currently requiring medication, or congestive heart failure.
- Known or suspected active central nervous system metastasis (patients stable 8 weeks after completion of treatment for central nervous system metastasis are eligible).
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 |
|---|---|---|
| LErafAON-ETU | Drug | – |
Primary outcome
To determine the maximum tolerated dose (MTD) and dose-limiting toxicity (DLT) of LErafAON-ETU.
2 secondary outcomes
To determine the pharmacokinetics of raf antisense oligonucleotide after intravenous administration of LErafAON-ETU
To determine any anti-tumor effects of LErafAON-ETU.
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 4 Jul 2011.
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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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing LErafAON-ETU in people with Neoplasms, run by INSYS Therapeutics Inc at 2 sites 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 15 inclusion and 14 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 4 Jul 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
To determine the maximum tolerated dose (MTD) and dose-limiting toxicity (DLT) of LErafAON-ETU.. 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 4 Jul 2011.