Nanoparticle Albumin-Bound Rapamycin in Treating Patients With Advanced Cancer With mTOR Mutations
A Pilot Study of a Rapid Access Platform for Investigational Drugs (RAPID) in Advanced Cancers
3 other identifiers
interventional
2
1 country
2
Brief Summary
This pilot trial studies how well nanoparticle albumin-bound rapamycin works in treating patients with cancer that as has spread to other places in the body and usually cannot be cured or controlled with treatment (advanced cancer) and that has an abnormality in a protein called mechanistic target of rapamycin (mTOR). Patients with this mutation are identified by genetic testing. Patients then receive nanoparticle albumin-bound rapamycin, which may stop the growth of cancer cells by blocking the mTOR enzyme, which is needed for cell growth and multiplication. Using treatments that target a patient's specific mutation may be a more effective treatment than the standard of care treatment.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for early_phase_1
Started Jan 2016
Typical duration for early_phase_1
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 1, 2016
CompletedFirst Submitted
Initial submission to the registry
January 4, 2016
CompletedFirst Posted
Study publicly available on registry
January 5, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 5, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
April 24, 2018
CompletedJune 6, 2019
June 1, 2018
1.7 years
January 4, 2016
June 5, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Proportion of confirmed responses, evaluated using the RECIST v1.1
The proportion of confirmed responses will be estimated by the number of confirmed responses divided by the total number of evaluable patients. An exact binomial confidence interval for the true confirmed response proportion will be calculated. Analysis will be carried out overall and within disease groups where warranted by sample size.
Up to 21 days
Secondary Outcomes (4)
Clinical benefit rate defined as the proportion of patients with a confirmed response or stable disease (complete response+partial response+stable disease) divided by the total number of evaluable patients
Up to 5 years
Incidence of adverse events, using the National Cancer Institute Common Terminology Criteria for Adverse Events v4.0
Up to 30 days after last dose of study treatment
Survival time
Time from registration to death due to any cause, assessed up to 5 years
Time to disease progression
Time from registration to the earliest date of documentation of disease progression, assessed up to 5 years
Other Outcomes (2)
Quality of life, measured using the EORTC QLQ-C30
Up to 24 weeks
Rate of individual mTOR pathway aberrations
Up to 5 years
Study Arms (1)
Treatment (nanoparticle albumin-bound rapamycin)
EXPERIMENTALPatients receive nanoparticle albumin-bound rapamycin IV over 30 minutes on days 1 and 8. Treatment repeats every 21 days for 24 weeks in the absence of disease progression or unacceptable toxicity.
Interventions
Correlative studies
Given IV
Ancillary studies
Eligibility Criteria
You may qualify if:
- Histologic proof of cancer which is now not amenable to curative standard treatment options
- Patient must have received at least 1 prior standard therapy for their disease
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0, 1 or 2
- Ability to provide informed written consent
- Willing to return to enrolling institution for follow-up (active monitoring phase of the study); Note: During the active monitoring phase of a study (i.e., active treatment), participants must be willing to return to the consenting institution for follow-up
- Life expectancy \>= 84 days (3 months)
- Identification of a drug target/targets through molecular profiling performed as a part of routine clinical care and treatment recommendation by the Mayo Clinic Genomics Tumor Board (GTB); NOTE: If profile matches more than 1 treatment arm, final decision for treatment arm assignment to be made by patients treating physician; it will be required for the genomic aberration to be identified through a test in a Clinical Laboratory Improvement Amendments (CLIA) workflow; assays used will range from single gene abnormalities (e.g. fluorescent in situ hybridization \[FISH\] for human epidermal growth factor receptor 2 \[ERBB2\] amplifications) to next generation sequencing based gene panels (Foundation One®) to more comprehensive assays such as whole exome sequencing; the Mayo Clinic GTB will serve as the centralized point of data synthesis to allow for assessment of molecular profiling accomplished through a heterogeneous array of tests
- Date of Mayo Clinic Genomics Tumor Board review =\< 3 months prior to registration
- Measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) version (v)1.1 criteria (for solid tumors) or equivalent criteria (for patients with non-solid tumor malignancies)
- Patient meets all sub-protocol specific criteria of each applicable sub-protocol
- Ability to complete questionnaire(s) by themselves or with assistance
- SUB-PROTOCOL AIM A: Histological confirmation of renal cell carcinoma, head and neck cancer, endometrial cancer, breast cancer, ovarian cancer, prostate cancer, squamous cell cervical or uterine cancer, or bladder cancer
- SUB-PROTOCOL AIM A: Confirmation of advanced cancer with mTOR pathway aberrations as determined through routine clinical care using pathway aberrations performed in a CLIA certified laboratory; cancer genomic profiling tests incorporating next generation sequencing from archival formalin-fixed paraffin-embedded tissue (FFPE) are validated with sensitivities and specificities of 99% and 99%, respectively; in the assay, hybrid-capture-selected deoxyribonucleic acid (DNA) libraries are sequenced to depths targeting \> 500 × coverage by non-polymerase chain reaction (PCR) duplicate read pairs, with \> 99% of exons at coverage \> 100 ×); multiplatform profiling may include immunohistochemistry and in situ hybridization methods with previously established negative/positive cutoffs performed in a CLIA certified lab; at least one pathway aberration must be identified; these must be confirmed in a CLIA certified lab; the potential mTOR aberrations that could be identified are listed below, please note that this list is not all inclusive; if a CLIA validated report lists an mTOR pathway inhibitor as a target drug for a genetic aberration, then it can be considered eligible for the purposes of this study; v-akt murine thymoma viral oncogene homolog 1 (AKT1), MTOR, phosphatidylinositol-4,5-bisphosphate 3-kinase, catalytic subunit alpha (PIK3CA), tuberous sclerosis (TSC)1, TSC2, retrovirus-associated DNA sequence (Ras) homolog enriched in brain (RHEB), serine/threonine kinase 11 (STK11), neurofibromin (NF)1/2
- SUB-PROTOCOL AIM A: Absolute neutrophil count (ANC) \>= 1500/mm\^3
- SUB-PROTOCOL AIM A: Platelet count \>= 100,000/mm\^3
- +11 more criteria
You may not qualify if:
- Uncontrolled intercurrent illness including, but not limited to: ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Failure to fully recover from acute, reversible effects of prior chemotherapy (other anti-neoplastic therapy) and radiation therapy to adverse event severity of =\< grade 1
- Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
- SUB-PROTOCOL AIM A: Any of the following:
- Pregnant women
- Nursing women
- Women of child-bearing potential, who are biologically able to conceive, or men who are able to father a child, not employing two forms of highly effective contraception
- Highly effective contraception (e.g., male condom with spermicide, diaphragm with spermicide, intra-uterine device, and total abstinence) must be used by both sexes during the study and must be continued for 6 months after the end of study treatment; Note: Oral, implantable, or injectable hormone contraceptives are not considered effective for this study
- SUB-PROTOCOL AIM A: Any of the following treatments:
- Chemotherapy within 4 weeks before treatment with nab-rapamycin
- Hormonal therapy within 4 weeks before treatment with nab-rapamycin (with the exception of leuprolide, degarelix, or goserelin)
- Immunotherapy within 4 weeks before treatment with nab-rapamycin
- Radiotherapy within 4 weeks before treatment with nab-rapamycin
- Treatment with nitrosoureas, mitomycin, or extensive radiotherapy within 6 weeks before treatment with nab-rapamycin
- Immunosuppressive agents within 3 weeks before treatment with nab-rapamycin (except corticosteroids used as antiemetics)
- +9 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Mayo Cliniclead
- National Cancer Institute (NCI)collaborator
Study Sites (2)
Mayo Clinic in Arizona
Scottsdale, Arizona, 85259, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Mitesh Borad
Mayo Clinic
Study Design
- Study Type
- interventional
- Phase
- early phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 4, 2016
First Posted
January 5, 2016
Study Start
January 1, 2016
Primary Completion
September 5, 2017
Study Completion
April 24, 2018
Last Updated
June 6, 2019
Record last verified: 2018-06