Cyclophosphamide and Sirolimus for the Treatment of Metastatic, RAI-refractory, Differentiated Thyroid Cancer
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ClinicalTrials.gov Identifier: NCT03099356 |
Recruitment Status :
Recruiting
First Posted : April 4, 2017
Last Update Posted : December 16, 2021
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Condition or disease | Intervention/treatment | Phase |
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Metastatic Thyroid Cancer | Drug: Cyclophosphamide Drug: Sirolimus | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 19 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open Label Phase II Trial Evaluating the Efficacy of Cyclophosphamide and Sirolimus for the Treatment of Metastatic, RAI-refractory, Differentiated Thyroid Cancer |
Actual Study Start Date : | April 27, 2017 |
Estimated Primary Completion Date : | May 12, 2022 |
Estimated Study Completion Date : | May 12, 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: Cyclophosphamide and Sirolimus
Sirolimus 4 mg, PO, days 1-28 as well as Cyclophosphamide 100 mg, PO, days 1-5 and 15-19
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Drug: Cyclophosphamide
Cyclophosphamide 100 mg, PO, days 1-5 and 15-19 Drug: Sirolimus Sirolimus 4 mg, PO, days 1-28 |
- 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.
- The number of patients that experience toxicity [ Time Frame: Patients are followed for toxicity up to 30 days after the last dose of study drug ]The number of patients that experience toxicity by type will be reported.
- Median overall survival time [ Time Frame: Patients will be followed until death or up to 2 years ]The median duration of time from start of treatment until death
- Median progression free survival time [ Time Frame: Patients will be followed for response until progression or up to 2 Years ]The median duration of time from start of treatment until progression. Progression is defined as at least a 20% increase in the sum of the LD (longest diameter) of target lesions, taking as reference the smallest sum LD recorded since the treatment started, or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions.

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03099356
Contact: Cancer AnswerLine | 1-800-865-1125 | canceranswerline@umich.edu | |
Contact: Paul Swiecicki, M.D. | pswiecic@med.umich.edu |
United States, Michigan | |
University of Michigan Cancer Center | Recruiting |
Ann Arbor, Michigan, United States, 48109 | |
Contact: Paul Swiecicki, M.D. pswiecic@med.umich.edu | |
Contact: Francis Worden, M.D. 734-936-0453 fworden@umich.edu | |
Principal Investigator: Paul Swieciki, M.D. |
Principal Investigator: | Paul Swiecicki, M.D. | University of Michigan Rogel Cancer Center |
Responsible Party: | University of Michigan Rogel Cancer Center |
ClinicalTrials.gov Identifier: | NCT03099356 |
Other Study ID Numbers: |
UMCC 2017.013 HUM00126559 ( Other Identifier: University of Michigan ) |
First Posted: | April 4, 2017 Key Record Dates |
Last Update Posted: | December 16, 2021 |
Last Verified: | December 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | No |
Thyroid Neoplasms Thyroid Diseases Endocrine System Diseases Endocrine Gland Neoplasms Neoplasms by Site Neoplasms Head and Neck Neoplasms Sirolimus Cyclophosphamide Immunosuppressive Agents Immunologic Factors |
Physiological Effects of Drugs Antirheumatic Agents Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Myeloablative Agonists Anti-Bacterial Agents Anti-Infective Agents Antibiotics, Antineoplastic Antifungal Agents |