NCT03099356 · University of Michigan Rogel Cancer Center
Cyclophosphamide and Sirolimus for the Treatment of Metastatic, RAI-refractory, Differentiated Thyroid Cancer
What this study is about
This study will be a non-randomly assigned pilot trial using Cyclophosphamide and Sirolimus for the treatment of metastatic differentiated thyroid cancer. Patients will be treated with Sirolimus 4 mg, PO, days 1-28 as well as Cyclophosphamide 100 mg, PO, days 1-5 and 15-19. Cycle length will be 28 days.
View original scientific description
This study will be a non-randomized pilot trial using Cyclophosphamide and Sirolimus for the treatment of metastatic differentiated thyroid cancer. Patients will be treated with Sirolimus 4 mg, PO, days 1-28 as well as Cyclophosphamide 100 mg, PO, days 1-5 and 15-19. Cycle length will be 28 days. Patients will be monitored closely for toxicity and undergo imaging to evaluate efficacy once every 2 cycles.
Interventions
DRUG
Cyclophosphamide
Cyclophosphamide 100 mg, PO, days 1-5 and 15-19
DRUG
Sirolimus
Sirolimus 4 mg, PO, days 1-28
Primary outcome measures
Percentage of patients that respond to treatment
Time frame: Patients will be followed for response until progression or up to 2 Years
The primary measure of efficacy will be the overall response rate (ORR) which is defined as those achieving either complete response (CR) or partial response (PR). Partial response is defined as at least a 30% decrease in the sum of the longest diameter (LD) of target lesions. Complete response is defined as Disappearance of all target lesions, determined by two separate observations conducted not less than 4 weeks apart (there can be no appearance of new lesions) and the disappearance of all non-target lesions and normalization of tumor marker level.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Histologically documented differentiated thyroid cancer with or without metastases, not amenable to curative treatment; or the patient has documented refusal of curative treatment
- Measurable disease (\>10 mm) and have progression of disease based on RECIST criteria. Previously irradiated tumor lesions are not considered measurable unless they have progressed since radiation.
- Previous failure of Iodine-131 (131I) therapy or not candidates to receive 131I as assessed by treating physician.
- Age ≥ 18 years
- ECOG (Eastern Cooperative Oncology Group) performance status 0-2
- Life expectance of ≥ 12 weeks
- 131I therapy not allowed within 24 weeks before entry (4 weeks if negative post-treatment scan)
- Adequate organ and marrow function
- Women of childbearing potential must have a negative serum or urine pregnancy test within 3 days prior to treatment
- Signed and dated informed consent document indicating that the patient (or legally acceptable representative) has been informed of all pertinent aspects of the trial prior to enrollment
- Willingness and ability to comply with scheduled visits, treatment plans, including willingness to take study medication, laboratory tests, and other study procedures
Exclusion criteria
- Inability to obtain Foundation One testing on archival tissue, or, lack of previous Next Generation Sequencing
- Chemotherapy, tyrosine kinase inhibitor, or radiation therapy within 4 weeks
- Prior experimental therapy within 4 weeks of planned start of this trial
- 131I therapy within 24 weeks before entry (4 weeks if negative post-treatment scan)
- Previous treatment with an mTOR inhibitor
- Patients who are currently receiving treatment with strong inhibitors or inducers of CYP3A4 or P-glycoprotein that cannot be discontinued at least one week prior to the start of treatment with Cyclophosphamide and Sirolimus
- Impairment of GI (gastrointestinal) function or GI disease that may significantly alter the absorption of study medications (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection) including dependence on a G-Tube for administration of medications.
- A serious uncontrolled medical disorder or active infection that would impair their ability to receive study treatment
- Patients with known sensitivities to either cyclophosphamide and/or sirolimus
- Patients with known urinary outflow obstruction
- Dementia or significantly altered mental status that would prohibit the understanding or rendering of informed consent and compliance with the requirements of this protocol
- Patients (male and female) having procreative potential who are not willing or not able to use adequate contraception or practicing abstinence
- Women who are pregnant or breast-feeding
- Patients residing in prison
Where
- Ann Arbor, Michigan
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Aug 1, 2025 · Source of record for eligibility and locations