Refractory MalignancyPhase 1NCT00496600
Phase I Study of Patupilone and RAD001
A Phase 1 study testing Patupilone and RAD001 in people with Refractory Malignancy, run by University of Medicine and Dentistry of New Jersey at 1 site in United States.
Trial summary
This is an unblinded, dose escalation study of patupilone in combination with RAD001 in subjects with advanced cancer to find the maximum tolerated dose of each drug. The purpose of this study is to see what doses of RAD001 and patupilone are safe to use when the two drugs are used at the same time. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Refractory Malignancy
- What is being tested?
- Patupilone and RAD001
- Type of treatment
- Pill or oral medicine, Injection or infusion
- How many people
- 42 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2007to Feb 2011
- 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
- All patients must have malignancy refractory to standard therapy or for whom no standard, effective therapy is available.
- Patient must have performance status 0-2 on the ECOG Performance Status (see Appendix B)
- Patient's disease must be measurable (RECIST) or evaluable (e.g., cytologically or radiologically detectable disease such as ascites, peritoneal deposits, or lesions which do not fulfill RECIST criteria for measurable disease).
- Patients must have intact intestinal absorption
- Patients must have adequate organ function
- Patient must have recovered from toxicity of prior chemotherapy and/or radiotherapy. Patient may not have received chemotherapy in the prior 4 weeks. Patients may have not received radiotherapy in the prior 3 weeks.
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- Patient (male or female) must be ≥ 18 years old.
- Women must not be pregnant or lactating. Female patients of childbearing potential must have a negative pregnancy test within 7 days before initiation of study drug dosing. Post menopausal women must be amenorrheic for at least 12 months to be considered of non-childbearing potential. Male and female patients of reproductive potential must agree to employ an effective barrier method of birth control throughout the study, and for three months following discontinuation of study drug.
- Signed written informed consent.
Who cannot join
- Patient has received greater than 3 prior cytotoxic regimens for metastatic disease.
- Prior therapy with epothilones, rapamycin, rapamycin analogs, or known sensitivity to these agents
- Prior treatment with any investigational drug within the preceding 4 weeks.
- Patient has history of bone marrow or stem cell transplant.
- Patient has received prior radiation therapy to greater than 25% of the bone marrow.
- Patients has newly diagnosed, not yet treated or uncontrolled brain metastases or leptomeningeal disease.
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- Patient has known hypersensitivity to the components of study drugs or its analogs.
- Patient with known Grade 3 or 4 hypersensitivity to macrolide antibiotics (eg. clarithromycin, erythromycin, azithromycin).
- Patient with any active (acute or chronic) or uncontrolled infection requiring systemic therapy.
- Patients with chronic treatment with systemic steroids or another immunosuppressive agent
- Patient with known HIV infection.
- Patients with unresolved diarrhea within the last 7 days prior to start of treatment.
- Concomitant treatment with medications that are listed as clinically relevant inducers or inhibitors of cytochrome P450 (CYP3A).
- Patients taking Coumadin® or other agents containing warfarin, with the exception of low dose Coumadin® (1 mg or less) administered prophylactically for maintenance of in-dwelling lines or ports
- Herbal preparations or related over-the-counter preparations containing herbal ingredients (eg. St. John's Wort)
- Any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as: unstable angina pectoris, symptomatic congestive heart failure, myocardial infarction ≤ 6 months prior to first study treatment, serious uncontrolled cardiac arrhythmia, or severely impaired lung function
- Any patient with uncontrolled diabetes as defined by fasting serum glucose >1.5 x ULN
- Any patient with nonmalignant medical illnesses that are uncontrolled or whose control may be jeopardized by the treatment with the study therapy
- Patients with liver disease such as cirrhosis, chronic active hepatitis, or chronic persistent hepatitis
- Patients receiving immunization with attenuated live vaccines during study participation.
Trial locations and contacts
1 site in 1 country.
United States1 site
The Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
About this study
Official title: Phase I Study of Patupilone and RAD001 in Patients With Refractory Solid Tumor Malignancy
This is an unblinded, dose escalation study of patupilone in combination with RAD001 in subjects with advanced cancer to find the maximum tolerated dose of each drug. The purpose of this study is to see what doses of RAD001 and patupilone are safe to use when the two drugs are used at the same time. Other goals in this study are to learn about the effect of RAD 001 and patupilone on tumor growth; to find out what amount of RAD001 is present in the blood when it is combined with patupilone; to learn about proteins in the blood that may predict or show an effect of RAD001 or patupilone and to learn if any changes are seen in the tumor with the type of test called a PET scan.Subjects will be assigned to a dosing group. The dose of patupilone and RAD001 a subject gets depends on when they enter onto this study. The initial subjects in the study will take the lowest doses of RAD001 and patupilone. At least 3 subjects will be treated in each dosing group starting with the smallest dose. If there are few or easy to handle side effects, the next group of at least 3 people to enter in the study will get the next higher dose. This continues until the highest dose of the study drug is found that does not cause serious or hard to treat side effects. Both the subjects and the study doctor will know which dose is assigned. In this study, RAD001 will be given as tablets to take with water. RAD001 will be taken for either 7, 14, or 21 days of each 21 day cycle depending on which dosing group is assigned. If taking RAD 001 and patupilone on the same day, then RAD 001 will be taken with water however prior to receiving patupilone.Patupilone will be given by an intravenous injection (through a vein) for 20 minutes on Days 1 or day 6 of the 21-day cycle. The patupilone dose will be based on height and weight. Blood tests (approximately 1-3 teaspoons) will be done every week. Every six weeks a CT scan or MRI will be done to assess the tumor size. Subjects will continue to receive RAD001 and patupilone as long as the tumor is stable or shrinking, and not having too many side effects from treatment.
Read the detailed description
Primary Endpoint To identify the maximum tolerated doses (MTD) of the combination of patupilone and RAD001
Secondary Endpoints To assess the toxicity of patupilone and RAD001 To determine if concentrations of RAD001 are elevated in the presence of patupilone To evaluate tumor response using standard imaging modalities (CT scan, x-ray, bone scan) To develop pharmacodynamic markers that may predict or indicate response to mTOR inhibition or patupilone treatment.
- Conditions
- Refractory Malignancy
- Being tested
- Patupilone, RAD001
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PatupiloneDrug
RAD001 will be taken orally starting at a dose of 30 mg every week for the first cohort of patients, one hour prior to administration of patupilone. Patupilone will then be administered as an infusion, starting at 5 mg/m2 for the first cohort of patients. The dosage and scheduling of RAD001 and patupilone during each 21-day cycle will vary according to cohort assignment
RAD001Drug
RAD001 will be taken orally starting at a dose of 30 mg every week for the first cohort of patients, one hour prior to administration of patupilone. Patupilone will then be administered as an infusion, starting at 5 mg/m2 for the first cohort of patients. The dosage and scheduling of RAD001 and patupilone during each 21-day cycle will vary according to cohort assignment
What is being measured
The primary outcome is the main question the study is designed to answer.
To identify the maximum tolerated doses (MTD) of the combination of patupilone and RAD001
Measured: 3 years
Secondary outcomes (1)
find toxicity of patupilone and RAD001, see if concentrations of RAD001 are elevated with patupilone, evaluate tumor response using standard imaging, develop pharmacodynamic markers that may predict/indicate response to mTOR inhibition or patupilone trt
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jul 2007 (estimated)
- Study startJul 2007
- Primary completionFeb 2011
- Study completionFeb 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory MalignancyStudies 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
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Medicine and Dentistry of New Jersey
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis Pharmaceuticals, National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Mark Stein, MD, Principal investigator, Rutgers, The State University of New Jersey
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00496600
- Sponsor's protocol ID
- 050612
- Other IDs
- CRAD001US16, 0220060307
- Status last verified
- Jun 2012
- First submitted
- 3 Jul 2007
- Last updated
- 14 Jun 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jun 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Patupilone and RAD001 in Patients With Refractory Solid Tumor Malignancy
- ClinicalTrials.gov ID
- NCT00496600
- Acronym / sponsor ID
- 050612
- Conditions
- Refractory Malignancy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- University of Medicine and Dentistry of New JerseyUniversity, hospital or other
- Collaborators
- Novartis Pharmaceuticals, National Cancer Institute (NCI)
- Study start
- Jul 2007
- Primary completion
- Feb 2011 (actual)
- Study completion
- Feb 2011 (actual)
- First posted
- 4 Jul 2007 (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 an unblinded, dose escalation study of patupilone in combination with RAD001 in subjects with advanced cancer to find the maximum tolerated dose of each drug. The purpose of this study is to see what doses of RAD001 and patupilone are safe to use when the two drugs are used at the same time. Other goals in this study are to learn about the effect of RAD 001 and patupilone on tumor growth; to find out what amount of RAD001 is present in the blood when it is combined with patupilone; to learn about proteins in the blood that may predict or show an effect of RAD001 or patupilone and to learn if any changes are seen in the tumor with the type of test called a PET scan.Subjects will be assigned to a dosing group. The dose of patupilone and RAD001 a subject gets depends on when they enter onto this study. The initial subjects in the study will take the lowest doses of RAD001 and patupilone. At least 3 subjects will be treated in each dosing group starting with the smallest dose. If there are few or easy to handle side effects, the next group of at least 3 people to enter in the study will get the next higher dose. This continues until the highest dose of the study drug is found that does not cause serious or hard to treat side effects. Both the subjects and the study doctor will know which dose is assigned. In this study, RAD001 will be given as tablets to take with water. RAD001 will be taken for either 7, 14, or 21 days of each 21 day cycle depending on which dosing group is assigned. If taking RAD 001 and patupilone on the same day, then RAD 001 will be taken with water however prior to receiving patupilone.Patupilone will be given by an intravenous injection (through a vein) for 20 minutes on Days 1 or day 6 of the 21-day cycle. The patupilone dose will be based on height and weight. Blood tests (approximately 1-3 teaspoons) will be done every week. Every six weeks a CT scan or MRI will be done to assess the tumor size. Subjects will continue to receive RAD001 and patupilone as long as the tumor is stable or shrinking, and not having too many side effects from treatment.
Detailed description
Primary Endpoint To identify the maximum tolerated doses (MTD) of the combination of patupilone and RAD001
Secondary Endpoints To assess the toxicity of patupilone and RAD001 To determine if concentrations of RAD001 are elevated in the presence of patupilone To evaluate tumor response using standard imaging modalities (CT scan, x-ray, bone scan) To develop pharmacodynamic markers that may predict or indicate response to mTOR inhibition or patupilone treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- 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 (9 listed)
- All patients must have malignancy refractory to standard therapy or for whom no standard, effective therapy is available.
- Patient must have performance status 0-2 on the ECOG Performance Status (see Appendix B)
- Patient's disease must be measurable (RECIST) or evaluable (e.g., cytologically or radiologically detectable disease such as ascites, peritoneal deposits, or lesions which do not fulfill RECIST criteria for measurable disease).
- Patients must have intact intestinal absorption
- Patients must have adequate organ function
Key exclusion criteria (20 listed)
- Patient has received greater than 3 prior cytotoxic regimens for metastatic disease.
- Prior therapy with epothilones, rapamycin, rapamycin analogs, or known sensitivity to these agents
- Prior treatment with any investigational drug within the preceding 4 weeks.
- Patient has history of bone marrow or stem cell transplant.
- Patient has received prior radiation therapy to greater than 25% of the bone marrow.
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 |
|---|---|---|
| PatupiloneRAD001 will be taken orally starting at a dose of 30 mg every week for the first cohort of patients, one hour prior to administration of patupilone. Patupilone will then be administered as an infusion, starting at 5 mg/m2 for the first cohort of patients. The dosage and scheduling of RAD001 and patupilone during each 21-day cycle will vary according to cohort assignment | Drug | – |
| RAD001RAD001 will be taken orally starting at a dose of 30 mg every week for the first cohort of patients, one hour prior to administration of patupilone. Patupilone will then be administered as an infusion, starting at 5 mg/m2 for the first cohort of patients. The dosage and scheduling of RAD001 and patupilone during each 21-day cycle will vary according to cohort assignment | Drug | – |
Primary outcome
To identify the maximum tolerated doses (MTD) of the combination of patupilone and RAD001
Time frame: 3 years
1 secondary outcome
find toxicity of patupilone and RAD001, see if concentrations of RAD001 are elevated with patupilone, evaluate tumor response using standard imaging, develop pharmacodynamic markers that may predict/indicate response to mTOR inhibition or patupilone trt
Time frame: 3 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 14 Jun 2012.
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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.
Mark Stein, MD principal investigator
Rutgers, The State University of New Jersey
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 Patupilone and RAD001 in people with Refractory Malignancy, run by University of Medicine and Dentistry of New Jersey 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 9 inclusion and 20 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 14 Jun 2012. 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?
To identify the maximum tolerated doses (MTD) of the combination of patupilone and RAD001 (time frame: 3 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 14 Jun 2012.