Head and Neck Squamous Cell CarcinomaPhase 2NCT03153982

Ruxolitinib in Operable Head and Neck Cancer

A Phase 2 study testing Ruxolitinib in people with Head and Neck Squamous Cell Carcinoma, run by University of California, San Francisco at 2 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

The purpose of this study is to assess the safety and efficacy of ruxolitinib in patients with operable Head and neck squamous cell carcinoma (HNSCC) who are planned for definitive surgery. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Head and Neck Squamous Cell Carcinoma
What is being tested?: Ruxolitinib
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 16 participants
Where: 2 sitesUnited States
Study dates: 8 Jun 2018to 18 Oct 2023
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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

Histologically or cytologically confirmed, primary or recurrent, head and neck squamous cell carcinoma, including variants. Patients must have at least one measureable lesion in accordance with RECIST 1.1 (tumor diameter ≥ 1 cm; short-axis lymph node diameter ≥ 1.5 cm) OR by caliper measurement (tumor diameter ≥ 1 cm). Any diagnostic pretreatment biopsy sample is acceptable including fine needle aspiration (FNA).
Primary tumors of any head and neck (oral cavity, oropharynx, hypopharynx, or larynx) site will be included.
Surgical resection of head and neck must be planned, either as primary treatment or salvage. Patients must have submitted adequate pretreatment archival or fresh tissue.
Age ≥ 18 years.
Eastern Cooperative Oncology Group (ECOG) performance status 0-2 (See Appendix 1).
Women of childbearing potential (WOCBP) must have a negative serum pregnancy test (sensitivity ≤ 25 human chorionic gonadotropin (HCG) IU/L) within 4 weeks prior to registration and will be repeated within 72 hours prior to the start of study drug administration.

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Persons of reproductive potential must agree to use and utilize an adequate method of contraception throughout treatment and for at least 12 weeks after study drug is stopped. Prior to study enrollment, women of childbearing potential must be advised of the importance of avoiding pregnancy during trial participation and the potential risk factors for an unintentional pregnancy.
Adequate hematologic, renal and hepatic function, as defined by:
Absolute neutrophil count (ANC) ≥ 1,500/ul, platelets ≥ 150,000/ul.
Creatinine ≤ 1.5 x institutional upper limit of normal (ULN).
Bilirubin ≤ 1.5 x ULN, aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≤ 2.5 x ULN.
Have signed written informed consent

Who cannot join

Subjects who fail to meet the above criteria.
Prior therapy for head and neck cancer is allowed, and the number of treatments is not limited. However, any systemic therapy should have been completed at least 30 days prior to study enrollment. Any radiation to the head and neck should have been completed at least 30 days prior to study enrollment. Palliative radiation outside of the head and neck does not require a washout.
Pregnancy or breastfeeding. Women (patients or partners of male patients) of childbearing potential (WOCBP) must practice acceptable methods of birth control to prevent pregnancy. All WOCBP must have a negative pregnancy test within 4 weeks prior to registration, and this must be repeated within 72 hours prior to first receiving ruxolitinib. If the pregnancy test is positive, the patient must not receive ruxolitinib and must not be enrolled in the study.
Any unresolved chronic toxicity ≥ grade 2 from previous anticancer therapy (except alopecia and anemia), according to Common Terminology Criteria for Adverse Events v4.0 (CTCAE).
Current active infection requiring systemic antibiotic or antifungal therapy.
Acute hepatitis or known HIV.

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Treatment with a non-approved or investigational drug within 30 days prior to Day 1 of study treatment.
New York Heart Association (NYHA) Class III or IV heart disease.
History of thromboembolic event or other condition currently requiring anticoagulation with warfarin (coumadin). Patients who are treated with low molecular weight heparin or fondaparinux are eligible.
History of significant bleeding disorder unrelated to cancer, including: diagnosed congenital bleeding disorders (e.g., von Willebrand's disease, diagnosed acquired bleeding disorder within one year (e.g., acquired anti-factor VIII antibodies, or ongoing or recent (≤ 3 months) significant gastrointestinal bleeding
Concomitant Medications, any of the following should be considered for exclusion: Strong CYP3A4 inhibitors: (Patients must discontinue drug 7 days prior to starting ruxolitinib), including but not limited to boceprevir, clarithromycin, conivaptan, indinavir, itraconazole, ketoconazole, lopinavir, mibefradil, nefazodone, nelfinavir, posaconazole, ritonavir, saquinavir, telaprevir, telithromycin, or voriconazole. In addition, patients will be instructed to avoid grapefruit or grapefruit juice, starfruit, or seville oranges.
Prisoners or subjects who are compulsorily detained (involuntarily incarcerated) for treatment of either a psychiatric or physical (e.g., infectious) illness.

Trial locations and contacts

2 sites in 1 country.

United States2 sites

University of California, San Francisco
San Francisco, California, 94143
University of Arizona Cancer Center
Tucson, Arizona, 85724

Other trials in United States

About this study

Official title: Pharmacodynamic Effects and Predictive Biomarkers of Janus Kinases (JAK)/Signal Transducer and Activator of Transcription (STAT) Inhibition With Ruxolitinib in Operable Head and Neck Cancer: a Window Trial

The purpose of this study is to assess the safety and efficacy of ruxolitinib in patients with operable Head and neck squamous cell carcinoma (HNSCC) who are planned for definitive surgery.

Read the detailed description

PRIMARY OBJECTIVES:

To identify baseline and/or pharmacodynamic biomarkers of response to ruxolitinib, based upon association with quantitative change in tumor size following 14-21 days of neoadjuvant ruxolitinib in patients with operable HNSCC as determined by quantitative change in Tumor size.

SECONDARY OBJECTIVES:

To describe the tolerability of brief neoadjuvant exposure to ruxolitinib II. To assess the effect of ruxolitinib on the tumoral Ki-67 proliferation index III. To evaluate additional candidate biomarkers of ruxolitinib response or resistance in HNSCC patients as determined by quantitative change in Tumor size,

OUTLINE:

Participants will be assigned neoadjuvant ruxolitinib based on participant platelet counts at baseline. Participants with a platelet count of 200,000 or greater will take 20 mg twice daily and participants with a platelet count between 150,000 and 200,000 will take 15 mg twice daily. Participants may continue treatment for up to 4 weeks from the time of study entry to time of planned standard of care surgery for cancer. Participants will be followed up for 12-weeks post-operation.

Conditions: Head and Neck Squamous Cell Carcinoma
Being tested: Ruxolitinib
Keywords: JAK/STAT inhibition, Biomarkers
Medical subject headings: Squamous Cell Carcinoma of Head and Neck

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

RuxolitinibDrug
Also called Jakafi
Given orally

Study groups in NCT03153982
Study group	Role	What they receive
Neoadjuvant Ruxolitinib	Experimental	Participants will take 15 mg or 20 mg of ruxolitinib by mouth twice daily for up to 4 weeks during the pre-operative window for 14-21 days, or up to 28 days for delays in planned surgery. Dose will be assigned based on participant platelet count at baseline. The last dose will be taken the morning of planned surgery. Ruxolitinib will be dispensed in 5 mg tablets. Participants will either take three tables (15 mg) in the morning and evening, or four tablets in the morning and evening (20 mg). Participants will be asked to fill out a drug diary indicating when doses of study drug are taken and any side effects they experience. Receives: Drug: Ruxolitinib

What is being measured

The primary outcome is the main question the study is designed to answer.

Proportional Percent Change in Tumor Size by Group
Measured clinical ruxolitinib response of quantitative change in tumor size measured as a proportional percent (range -100% to +100%) from baseline to day 14-21 by group.
Measured: Up to 4 weeks

Secondary outcomes (4)

Number of Participants With Treatment-related Adverse Events
The number of participants with treatment-related adverse events, defined as definite, probable or possibly related to the study intervention and classified according to NCI CTCAE version 4 will be reported.
Measured: Up to 12 weeks
Number of Participants With Documented Surgical Complications
The number of participants with documented surgical complications will be reported.
Measured: Up to 12 weeks.
Median Length of Hospital Stay
The median length of hospital stay following the standard of care, surgical procedure will be reported.
Measured: Up to 12 weeks
Median Change in Ki-67 Proliferative Index Value
A high Ki-67 proliferation index means many cells are dividing quickly and that the cancer is likely to grow and spread. A Ki-67 proliferation index over 30% is typically considered high. The Ki-67 proliferative index will be measured at baseline and post-treatment tumor tissue.
Measured: Up to 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

8 Jun 201818 Oct 2023

First posted15 May 2017
Study start8 Jun 2018
Primary completion18 Oct 2023
Study completion18 Oct 2023

Why it stopped: Low accrual

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Head and Neck Squamous Cell CarcinomaStudies of the same condition, open and under way.
More trials run by University of California, San FranciscoThe 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 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 16 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03153982
Sponsor's protocol ID: 16201
Other IDs: NCI-2017-02304
Status last verified: Aug 2024
First submitted: 9 May 2017
Last updated: 30 Oct 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Qureshy Z, Li H, Zeng Y, Rivera J, Cheng N, Peterson CN, Kim MO, Ryan WR, Ha PK, Bauman JE, Wang SJ, Long SR, Johnson DE, Grandis JR. STAT3 Activation as a Predictive Biomarker for Ruxolitinib Response in Head and Neck Cancer. Clin Cancer Res. 2022 Nov 1;28(21):4737-4746. doi: 10.1158/1078-0432.CCR-22-0744.
PubMed 35929989

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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RecruitingPhase 1/2
Docetaxel and SX-682 in Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma, Salivary Gland Carcinoma, and Advanced Prostate Cancer
Head and Neck Cancer, Head and Neck Squamous Cell Carcinoma
Not yet recruiting
Tumoral Versus Luminal Microbiome in Head and Neck Squamous Cell Carcinoma
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All Head and Neck Squamous Cell Carcinoma trials

Study at a glance

From the registry record, last updated by the sponsor on 30 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Pharmacodynamic Effects and Predictive Biomarkers of Janus Kinases (JAK)/Signal Transducer and Activator of Transcription (STAT) Inhibition With Ruxolitinib in Operable Head and Neck Cancer: a Window Trial
ClinicalTrials.gov ID: NCT03153982
Acronym / sponsor ID: 16201
Conditions: Head and Neck Squamous Cell Carcinoma
Keywords: JAK/STAT inhibition, Biomarkers
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Low accrual
Enrolment: 16 participants (actual)
Lead sponsor: University of California, San FranciscoUniversity, hospital or other
Collaborators: Incyte Corporation
Study start: 8 Jun 2018 (actual)
Primary completion: 18 Oct 2023 (actual)
Study completion: 18 Oct 2023 (actual)
First posted: 15 May 2017 (actual)
Results: Posted 30 Oct 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purpose of this study is to assess the safety and efficacy of ruxolitinib in patients with operable Head and neck squamous cell carcinoma (HNSCC) who are planned for definitive surgery.

Detailed description

PRIMARY OBJECTIVES:

To identify baseline and/or pharmacodynamic biomarkers of response to ruxolitinib, based upon association with quantitative change in tumor size following 14-21 days of neoadjuvant ruxolitinib in patients with operable HNSCC as determined by quantitative change in Tumor size.

SECONDARY OBJECTIVES:

To describe the tolerability of brief neoadjuvant exposure to ruxolitinib II. To assess the effect of ruxolitinib on the tumoral Ki-67 proliferation index III. To evaluate additional candidate biomarkers of ruxolitinib response or resistance in HNSCC patients as determined by quantitative change in Tumor size,

OUTLINE:

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Neoadjuvant Ruxolitinib	Experimental	Drug: RuxolitinibParticipants will take 15 mg or 20 mg of ruxolitinib by mouth twice daily for up to 4 weeks during the pre-operative window for 14-21 days, or up to 28 days for delays in planned surgery. Dose will be assigned based on participant platelet count at baseline. The last dose will be taken the morning of planned surgery. Ruxolitinib will be dispensed in 5 mg tablets. Participants will either take three tables (15 mg) in the morning and evening, or four tablets in the morning and evening (20 mg). Participants will be asked to fill out a drug diary indicating when doses of study drug are taken and any side effects they experience.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (12 listed)

Histologically or cytologically confirmed, primary or recurrent, head and neck squamous cell carcinoma, including variants. Patients must have at least one measureable lesion in accordance with RECIST 1.1 (tumor diameter ≥ 1 cm; short-axis lymph node diameter ≥ 1.5 cm) OR by caliper measurement (tumor diameter ≥ 1 cm). Any diagnostic pretreatment biopsy sample is acceptable including fine needle aspiration (FNA).
Primary tumors of any head and neck (oral cavity, oropharynx, hypopharynx, or larynx) site will be included.
Surgical resection of head and neck must be planned, either as primary treatment or salvage. Patients must have submitted adequate pretreatment archival or fresh tissue.
Age ≥ 18 years.
Eastern Cooperative Oncology Group (ECOG) performance status 0-2 (See Appendix 1).

Key exclusion criteria (12 listed)

Subjects who fail to meet the above criteria.
Prior therapy for head and neck cancer is allowed, and the number of treatments is not limited. However, any systemic therapy should have been completed at least 30 days prior to study enrollment. Any radiation to the head and neck should have been completed at least 30 days prior to study enrollment. Palliative radiation outside of the head and neck does not require a washout.
Pregnancy or breastfeeding. Women (patients or partners of male patients) of childbearing potential (WOCBP) must practice acceptable methods of birth control to prevent pregnancy. All WOCBP must have a negative pregnancy test within 4 weeks prior to registration, and this must be repeated within 72 hours prior to first receiving ruxolitinib. If the pregnancy test is positive, the patient must not receive ruxolitinib and must not be enrolled in the study.
Any unresolved chronic toxicity ≥ grade 2 from previous anticancer therapy (except alopecia and anemia), according to Common Terminology Criteria for Adverse Events v4.0 (CTCAE).
Current active infection requiring systemic antibiotic or antifungal therapy.

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
RuxolitinibAlso: JakafiGiven orally	Drug	Neoadjuvant Ruxolitinib

Primary outcome

Proportional Percent Change in Tumor Size by Group
Time frame: Up to 4 weeks
Measured clinical ruxolitinib response of quantitative change in tumor size measured as a proportional percent (range -100% to +100%) from baseline to day 14-21 by group.

4 secondary outcomes

Number of Participants With Treatment-related Adverse Events
Time frame: Up to 12 weeks
Number of Participants With Documented Surgical Complications
Time frame: Up to 12 weeks.
Median Length of Hospital Stay
Time frame: Up to 12 weeks
Median Change in Ki-67 Proliferative Index Value
Time frame: Up to 12 weeks

Results and safety

Results posted to the registry on 30 Oct 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Neoadjuvant Ruxolitinib	16	16

Primary outcome: Proportional Percent Change in Tumor Size by Group

Time frame: Up to 4 weeks

Population analysed: Data not collected

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to 12 weeks. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Neoadjuvant Ruxolitinib	2 of 16 (12.5%)	6 of 16 (37.5%)	2 of 16

Limitations and caveats (from the sponsor): Study closed earlier than expected due to slow accrual

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 35929989Qureshy Z, Li H, Zeng Y, Rivera J, Cheng N, Peterson CN, Kim MO, Ryan WR, Ha PK, Bauman JE, Wang SJ, Long SR, Johnson DE, Grandis JR. STAT3 Activation as a Predictive Biomarker for Ruxolitinib Response in Head and Neck Cancer. Clin Cancer Res. 2022 Nov 1;28(21):4737-4746. doi: 10.1158/1078-0432.CCR-22-0744.
Individual participant dataSharing planned: No

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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.

William Ryan, MD principal investigator

University of California, San Francisco

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ruxolitinib in people with Head and Neck Squamous Cell Carcinoma, run by University of California, San Francisco at 2 sites 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 12 inclusion and 12 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: stopped early, as of the sponsor's last update on 30 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Proportional Percent Change in Tumor Size by Group (time frame: Up to 4 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03153982, which also lists 1 linked publication.

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 30 Oct 2024.