Advanced CancersPhase 1NCT01266057
Sirolimus or Vorinostat and Hydroxychloroquine in Advanced Cancer
A Phase 1 study testing Hydroxychloroquine, Sirolimus and Vorinostat in people with Advanced Cancers, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to find the highest tolerable dose of sirolimus or vorinostat that can be given in combination with hydroxychloroquine to patients with advanced cancer. The safety of these drug combinations will also be studied. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Cancers
- What is being tested?
- Hydroxychloroquine, Sirolimus and Vorinostat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 143 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Apr 2011to 11 Feb 2021
- 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
- Patients with advanced or metastatic cancers that are refractory to standard therapy, relapsed after standard therapy, or who have no standard therapy available that improves survival by at least three months.
- Patients must be >/= 18 years.
- Patients must be >/= 3 weeks beyond treatment with a cytotoxic chemotherapy regimen, or therapeutic radiation, or major surgery. Patients may have received palliative localized radiation immediately before or during treatment provided that radiation is not delivered to the only site of disease being treated under this protocol. For biologic/targeted agents patients must be >/= 5 half-lives or >/= 3 weeks form the last dose (whichever comes first).
- ECOG performance status </= 2
- Patients must have adequate organ and marrow function defined as: absolute neutrophil count >/= 1,000/mL;platelets >/=50,000/mL; creatinine </= 2 X ULN; total bilirubin </= 2.0 (exceptions may apply to benign non-malignant indirect hyperbilirubinemia such as Gilbert syndrome); ALT(SGPT) </= 5 X ULN; Exception for patients with liver metastasis: total bilirubin </= 3 x ULN; ALT(SGPT) </= 8 X ULN;cholesterol </= 350 mg/dL; triglycerides </= 400 mg/dL (sirolimus and hydroxychloroquine only).
- Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 30 days after the last dose.
- Patients must be able to understand and be willing to sign a written informed consent document.
Who cannot join
- Uncontrolled intercurrent illness, including, but not limited to, uncontrolled infection, uncontrolled asthma, need for hemodialysis, need for ventilatory support.
- Pregnant or lactating women.
- History of hypersensitivity to sirolimus.
- History of hypersensitivity to vorinostat
- History of hypersensitivity to hydroxychloroquine
- History of hypersensitivity to any component of the formulation.
Show all 13Show fewer
- Patients unwilling or unable to sign informed consent document.
- Patients with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption.
- Patients with known glucose-6-phosphate dehydrogenase deficiency.
- Patients with porphyria cutanea tarda.
- Patients with psoriasis.
- Patients with pre-existing maculopathy or retinopathy of the eye.
- Patients who have a pre-existing myopathy.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: A Phase I Trial of Sirolimus (mTOR Inhibitor) or Vorinostat (HDAC Inhibitor) in Combination With Hydroxychloroquine (Autophagy Inhibitor) in Patients With Advanced Malignancies
The goal of this clinical research study is to find the highest tolerable dose of sirolimus or vorinostat that can be given in combination with hydroxychloroquine to patients with advanced cancer. The safety of these drug combinations will also be studied.
Read the detailed description
Study Drug Dose Level:
If you are found to be eligible to take part in this study, you will be assigned to a dose level of hydroxychloroquine and either sirolimus or vorinostat, based on when you joined this study, availability of spots for each drug combination, and what your doctor thinks is in your best interest. Up to 11 dose levels of the sirolimus and hydroxychloroquine combination and 7 dose levels of the vorinostat and hydroxychloroquine combination will be tested. Three (3) to 6 participants will be enrolled at each dose level. The first group of participants will receive the lowest dose level of the study drug combination. Each new group will receive a higher dose than the group before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of each drug combination is found.
Once the highest tolerated dose of each drug combination is found, up to 28 more participants (14 per combination) will be given this dose. The study doctor will decide which drug combination each participant is given, based on their tumor type.
Study Drug Administration:
Each study "cycle" is 21 days.
You will take hydroxychloroquine and either vorinostat or sirolimus by mouth, 1 time a day, every day. You should take the pills at about the same time each day with food and a cup (8 ounces) of water.
Study Visits:
At every study visit, you will be asked about any health conditions you have, drugs you may be taking, and if you have had any side effects.
Weekly During Cycle 1:
° Blood (about 2 teaspoons) will be drawn for routine tests.
At the beginning of each cycle beginning with 2:
- You will have a physical exam.
- Your medical history will be recorded.
- You will be asked if you have any muscle weakness or difficulty while moving.
Every 6 weeks, you will have an x-ray, CT scan, MRI scan, and/or PET/CT to check the status of the disease. If the study doctor thinks it is needed, they will be performed more often. If you are able to become pregnant, you will have a blood (about 1 teaspoon) or urine pregnancy test.
About every 3 months, you will have an eye exam.
Length of Study:
You may continue taking the study drugs for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drug if the disease gets worse or intolerable side effects occur.
This is an investigational study. Sirolimus is FDA approved and commercially available for the treatment of patients with a kidney transplant. Vorinostat is FDA approved and commercially available for the treatment of cutaneous T-cell lymphoma. Hydroxychloroquine is FDA approved and commercially available to treat malaria. The use of these drugs combinations is investigational.
Up to 224 patients will take part in this study. All will be enrolled at MD Anderson.
- Conditions
- Advanced Cancers
- Being tested
- Hydroxychloroquine, Sirolimus, Vorinostat
- Keywords
- Advanced Malignancies, Metastatic cancers, Sirolimus, Hydroxychloroquine, Vorinostat, Plaquenil, Rapamune, Saha, Suberoylanilide Hydroxamic Acid, MSK-390, Zolinza
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Also called Plaquenil
Starting dose of 200 mg by mouth every day for a 21 day cycle.
SirolimusDrug
Also called Rapamune
Starting dose of 2 mg by mouth every day for a 21 day cycle.
VorinostatDrug
Also called SAHA, Suberoylanilide Hydroxamic Acid, MSK-390, Zolinza
Starting dose of 200 mg by mouth per day for a 21 day cycle.
| Study group | Role | What they receive |
|---|---|---|
| Hydroxychloroquine + Sirolimus | Experimental | Hydroxychloroquine starting dose of 200 mg by mouth every day for a 21 day cycle. Sirolimus starting dose of 2 mg by mouth every day for a 21 day cycle. Receives: Drug: Hydroxychloroquine; Drug: Sirolimus |
| Hydroxychloroquine + Vorinostat | Experimental | Hydroxychloroquine starting dose of 200 mg by mouth every day for a 21 day cycle. Vorinostat starting dose of 200 mg by mouth per day for a 21 day cycle. Receives: Drug: Hydroxychloroquine; Drug: Vorinostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD) of Sirolimus or Vorinostat in Combination with Hydroxychloroquine in Participants with Advanced Cancers
Maximum tolerated dose (MTD) defined defined as the dose level below the dose at which 2 of 6 patients experience drug-related dose limiting toxicity (DLT) in the first treatment cycle.
Dose limiting toxicity (DLT) defined as: Any grade 3 or 4 non-hematologic toxicity as defined in the NCI CTC v3.0 that is possibly, probably or definitely related to any of the three study medications, even if expected. This is to include symptoms/signs of vascular leak or cytokine release syndrome; or any severe or life-threatening complication or abnormality not defined in the NCI-CTCAE that is possibly, probably or definitely related to the therapy.
Measured: 21 day cycles, approximately 4 weeks for DLT assessment
Secondary outcomes (1)
Antitumor Efficacy of Sirolimus or Vorinostat in Combination with Hydroxychloroquine in Participants with Advanced Cancers
Participants with lymphoma measured per the WHO criteria,68 and all others evaluated using RECIST 1.1 criteria.69.
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2010 (estimated)
- Study start28 Apr 2011
- Primary completion11 Feb 2021
- Study completion11 Feb 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced CancersStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 143 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Filip Janku, MD, PHD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01266057
- Sponsor's protocol ID
- 2010-0588
- Other IDs
- NCI-2011-00303
- Status last verified
- Mar 2021
- First submitted
- 22 Dec 2010
- Last updated
- 4 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Trial of Sirolimus (mTOR Inhibitor) or Vorinostat (HDAC Inhibitor) in Combination With Hydroxychloroquine (Autophagy Inhibitor) in Patients With Advanced Malignancies
- ClinicalTrials.gov ID
- NCT01266057
- Acronym / sponsor ID
- 2010-0588
- Conditions
- Advanced Cancers
- Keywords
- Advanced Malignancies, Metastatic cancers, Sirolimus, Hydroxychloroquine, Vorinostat, Plaquenil, Rapamune, Saha
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 143 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 28 Apr 2011 (actual)
- Primary completion
- 11 Feb 2021 (actual)
- Study completion
- 11 Feb 2021 (actual)
- First posted
- 24 Dec 2010 (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 goal of this clinical research study is to find the highest tolerable dose of sirolimus or vorinostat that can be given in combination with hydroxychloroquine to patients with advanced cancer. The safety of these drug combinations will also be studied.
Detailed description
Study Drug Dose Level:
If you are found to be eligible to take part in this study, you will be assigned to a dose level of hydroxychloroquine and either sirolimus or vorinostat, based on when you joined this study, availability of spots for each drug combination, and what your doctor thinks is in your best interest. Up to 11 dose levels of the sirolimus and hydroxychloroquine combination and 7 dose levels of the vorinostat and hydroxychloroquine combination will be tested. Three (3) to 6 participants will be enrolled at each dose level. The first group of participants will receive the lowest dose level of the study drug combination. Each new group will receive a higher dose than the group before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of each drug combination is found.
Once the highest tolerated dose of each drug combination is found, up to 28 more participants (14 per combination) will be given this dose. The study doctor will decide which drug combination each participant is given, based on their tumor type.
Study Drug Administration:
Each study "cycle" is 21 days.
You will take hydroxychloroquine and either vorinostat or sirolimus by mouth, 1 time a day, every day. You should take the pills at about the same time each day with food and a cup (8 ounces) of water.
Study Visits:
At every study visit, you will be asked about any health conditions you have, drugs you may be taking, and if you have had any side effects.
Weekly During Cycle 1:
° Blood (about 2 teaspoons) will be drawn for routine tests.
At the beginning of each cycle beginning with 2:
- You will have a physical exam.
- Your medical history will be recorded.
- You will be asked if you have any muscle weakness or difficulty while moving.
Every 6 weeks, you will have an x-ray, CT scan, MRI scan, and/or PET/CT to check the status of the disease. If the study doctor thinks it is needed, they will be performed more often. If you are able to become pregnant, you will have a blood (about 1 teaspoon) or urine pregnancy test.
About every 3 months, you will have an eye exam.
Length of Study:
You may continue taking the study drugs for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drug if the disease gets worse or intolerable side effects occur.
This is an investigational study. Sirolimus is FDA approved and commercially available for the treatment of patients with a kidney transplant. Vorinostat is FDA approved and commercially available for the treatment of cutaneous T-cell lymphoma. Hydroxychloroquine is FDA approved and commercially available to treat malaria. The use of these drugs combinations is investigational.
Up to 224 patients will take part in this study. All will be enrolled at MD Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hydroxychloroquine + Sirolimus | Experimental | Drug: Hydroxychloroquine, Drug: SirolimusHydroxychloroquine starting dose of 200 mg by mouth every day for a 21 day cycle. Sirolimus starting dose of 2 mg by mouth every day for a 21 day cycle. |
| Hydroxychloroquine + Vorinostat | Experimental | Drug: Hydroxychloroquine, Drug: VorinostatHydroxychloroquine starting dose of 200 mg by mouth every day for a 21 day cycle. Vorinostat starting dose of 200 mg by mouth per day for a 21 day cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients with advanced or metastatic cancers that are refractory to standard therapy, relapsed after standard therapy, or who have no standard therapy available that improves survival by at least three months.
- Patients must be >/= 18 years.
- Patients must be >/= 3 weeks beyond treatment with a cytotoxic chemotherapy regimen, or therapeutic radiation, or major surgery. Patients may have received palliative localized radiation immediately before or during treatment provided that radiation is not delivered to the only site of disease being treated under this protocol. For biologic/targeted agents patients must be >/= 5 half-lives or >/= 3 weeks form the last dose (whichever comes first).
- ECOG performance status </= 2
- Patients must have adequate organ and marrow function defined as: absolute neutrophil count >/= 1,000/mL;platelets >/=50,000/mL; creatinine </= 2 X ULN; total bilirubin </= 2.0 (exceptions may apply to benign non-malignant indirect hyperbilirubinemia such as Gilbert syndrome); ALT(SGPT) </= 5 X ULN; Exception for patients with liver metastasis: total bilirubin </= 3 x ULN; ALT(SGPT) </= 8 X ULN;cholesterol </= 350 mg/dL; triglycerides </= 400 mg/dL (sirolimus and hydroxychloroquine only).
Key exclusion criteria (13 listed)
- Uncontrolled intercurrent illness, including, but not limited to, uncontrolled infection, uncontrolled asthma, need for hemodialysis, need for ventilatory support.
- Pregnant or lactating women.
- History of hypersensitivity to sirolimus.
- History of hypersensitivity to vorinostat
- History of hypersensitivity to hydroxychloroquine
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 |
|---|---|---|
| HydroxychloroquineAlso: PlaquenilStarting dose of 200 mg by mouth every day for a 21 day cycle. | Drug | Hydroxychloroquine + Sirolimus, Hydroxychloroquine + Vorinostat |
| SirolimusAlso: RapamuneStarting dose of 2 mg by mouth every day for a 21 day cycle. | Drug | Hydroxychloroquine + Sirolimus |
| VorinostatAlso: SAHA, Suberoylanilide Hydroxamic Acid, MSK-390, ZolinzaStarting dose of 200 mg by mouth per day for a 21 day cycle. | Drug | Hydroxychloroquine + Vorinostat |
Primary outcome
Maximum Tolerated Dose (MTD) of Sirolimus or Vorinostat in Combination with Hydroxychloroquine in Participants with Advanced Cancers
Time frame: 21 day cycles, approximately 4 weeks for DLT assessment
Maximum tolerated dose (MTD) defined defined as the dose level below the dose at which 2 of 6 patients experience drug-related dose limiting toxicity (DLT) in the first treatment cycle. Dose limiting toxicity (DLT) defined as: Any grade 3 or 4 non-hematologic toxicity as defined in the NCI CTC v3.0 that is possibly, probably or definitely related to any of the three study medications, even if expected. This is to include symptoms/signs of vascular leak or cytokine release syndrome; or any severe or life-threatening complication or abnormality not defined in the NCI-CTCAE that is possibly, probably or definitely related to the therapy.
1 secondary outcome
Antitumor Efficacy of Sirolimus or Vorinostat in Combination with Hydroxychloroquine in Participants with Advanced Cancers
Time frame: 6 weeks
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 Mar 2021.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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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.
Filip Janku, MD, PHD 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 Hydroxychloroquine, Sirolimus and Vorinostat in people with Advanced Cancers, run by M.D. Anderson Cancer Center 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 7 inclusion and 13 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 Mar 2021. 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?
Maximum Tolerated Dose (MTD) of Sirolimus or Vorinostat in Combination with Hydroxychloroquine in Participants with Advanced Cancers (time frame: 21 day cycles, approximately 4 weeks for DLT assessment). 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 Mar 2021.