Carcinoma, Basal CellPhase 4VISORBNCT02436408

VISmodegib for ORbital and Periocular Basal Cell Carcinoma

A Phase 4 study testing Vismodegib in people with Carcinoma, Basal Cell, run by University of Michigan Rogel Cancer Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Basal cell carcinoma (BCCA) is the most common human cancer, and frequently affects facial structures. While rarely fatal, facial BCCA can be disfiguring and expensive to treat. Vismodegib is a small molecule inhibitor of SMO developed for the treatment of tumors in which the Hh signaling pathway appears to contribute… Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Carcinoma, Basal Cell
What is being tested?: Vismodegib
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 35 participants
Where: 1 siteUnited States
Study dates: 15 Jul 2015to Sep 2020
Phase: Phase 4
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

Adult patients over 18 years of age with locally advanced or recurrent orbital or periorbital basal cell carcinoma (BCCA), or a medial canthal BCCA that threatens the lacrimal drainage system.
Clinical assessment score obtained at baseline.
Medical Oncology screening performed at baseline.
Adequate BCCA size and location.
Adequate hematopoietic capacity, hepatic and renal function.
Male patients must agree to use condoms during treatment and for 3 months after last dose.

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Male patients must agree to not donate sperm during treatment and for 3 months after last dose.
Participant must agree not to donate blood during the study and for 7 months after last dose.
Informed consent signed.
If the patient consents to enroll, then blood will be drawn and stored for biomarker analysis.

Who cannot join

Inability or unwillingness to swallow capsules.
Inability or unwillingness to comply with study procedures.
Pregnant, lactating, or breast feeding women.
Women of childbearing potential.
Uncontrolled medical illness.
Dementia or significantly altered mental status that would prohibit the understanding or rendering of informed consent and compliance with the requirements of this protocol.
Age under 18 years.

Trial locations and contacts

1 site in 1 country.

United States1 site

University of Michigan Hospital
Ann Arbor, Michigan, 48109

Other trials in United States

About this study

Official title: VISmodegib for ORbital and Periocular Basal Cell Carcinoma (VISORB)

Basal cell carcinoma (BCCA) is the most common human cancer, and frequently affects facial structures. While rarely fatal, facial BCCA can be disfiguring and expensive to treat.

Vismodegib is a small molecule inhibitor of SMO developed for the treatment of tumors in which the Hh signaling pathway appears to contribute to the development and maintenance of tumorigenesis. Vismodegib was recently approved by the Food and Drug Administration (FDA) for treatment of metastatic and locally advanced BCCA. Recent reports have suggested that vismodegib treatment for orbital BCCA may facilitate eye preservation even if surgery is eventually required

In order to assess the potential of vismodegib to improve the ophthalmic outcomes following treatment for orbital and/or periocular BCCA, this study will follow patients with globe-threatening orbital and lacrimal-threatening periocular BCCA who are being treated with vismodegib as standard of care.

Patients with tumors that do not respond to treatment with Vismodegib, and those who have a good response but poor tolerance of Vismodegib, will be offered surgical excision of the tumor. Patients with a good response and good tolerance of Vismodegib may continue the treatment as long as clinically indicated.

Conditions: Carcinoma, Basal Cell
Being tested: Vismodegib
Medical subject headings: Carcinoma, Basal Cell

What participants receive

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

VismodegibDrug

Study groups in NCT02436408
Study group	Role	What they receive
Vismodegib	Experimental	150mg taken orally once daily Receives: Drug: Vismodegib

What is being measured

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

The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS).
The VAWS was developed for the purpose of this study. It is made up of standard ophthalmic exam points, as well as subjective assessment of tearing and overall patient satisfaction. The maximum score is 50 and a score of 21 or greater will be considered a good outcome.
Measured: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.

Secondary outcomes (3)

Number of Patients With Progressive Disease (PD)
Treatment response will be determined per Response Evaluation Criteria in Solid Tumors (RECIST) protocol, using clinical measurements and/or tumor imaging measurements (determined by treating physician).
Measured: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Good Tolerance of Vismodegib
Tolerance will be self-reported by patient. Treatment response will be determined per Response Evaluation Criteria in Solid Tumors (RECIST) protocol, using clinical measurements and/or tumor imaging measurements (determined by treating physician).
Measured: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Poor Tolerance of Vismodegib
Tolerance will be self-reported by patient. Treatment response will be determined per Response Evaluation Criteria in Solid Tumors (RECIST) protocol, using clinical measurements and/or tumor imaging measurements (determined by treating physician).
Measured: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.

Timeline

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

15 Jul 2015Sep 2020

First posted6 May 2015 (estimated)
Study start15 Jul 2015
Primary completionSep 2020
Study completionSep 2020

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 (3)Papers the sponsor has linked to this study.
Other trials for Carcinoma, Basal CellStudies of the same condition, open and under way.
More trials run by University of Michigan Rogel 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 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 35 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02436408
Sponsor's protocol ID: UMCC 2014.022
Other IDs: HUM00082579
Acronym: VISORB
Status last verified: Sep 2021
First submitted: 1 May 2015
Last updated: 28 Oct 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Unsworth SP, Tingle CF, Heisel CJ, Eton EA, Andrews CA, Chan MP, Bresler SC, Kahana A. Analysis of residual disease in periocular basal cell carcinoma following hedgehog pathway inhibition: Follow up to the VISORB trial. PLoS One. 2022 Dec 1;17(12):e0265212. doi: 10.1371/journal.pone.0265212. eCollection 2022.
PubMed 36455049
Kahana A, Unsworth SP, Andrews CA, Chan MP, Bresler SC, Bichakjian CK, Durham AB, Demirci H, Elner VM, Nelson CC, Kim DS, Joseph SS, Swiecicki PL, Worden FP. Vismodegib for Preservation of Visual Function in Patients with Advanced Periocular Basal Cell Carcinoma: The VISORB Trial. Oncologist. 2021 Jul;26(7):e1240-e1249. doi: 10.1002/onco.13820. Epub 2021 May 31.
PubMed 33988881
Rao RC, Chan MP, Andrews CA, Kahana A. EZH2, Proliferation Rate, and Aggressive Tumor Subtypes in Cutaneous Basal Cell Carcinoma. JAMA Oncol. 2016 Jul 1;2(7):962-3. doi: 10.1001/jamaoncol.2016.0021. No abstract available.
PubMed 27054919

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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All Carcinoma, Basal Cell trials

Study at a glance

From the registry record, last updated by the sponsor on 28 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: VISmodegib for ORbital and Periocular Basal Cell Carcinoma (VISORB)
ClinicalTrials.gov ID: NCT02436408
Acronym / sponsor ID: VISORB / UMCC 2014.022
Conditions: Carcinoma, Basal Cell
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 35 participants (actual)
Lead sponsor: University of Michigan Rogel Cancer CenterUniversity, hospital or other
Study start: 15 Jul 2015 (actual)
Primary completion: Sep 2020 (actual)
Study completion: Sep 2020 (actual)
First posted: 6 May 2015 (estimated)
Results: Posted 28 Oct 2021

Clinical summary

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

Basal cell carcinoma (BCCA) is the most common human cancer, and frequently affects facial structures. While rarely fatal, facial BCCA can be disfiguring and expensive to treat.

Study design

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

Arm	Type	Receives
Vismodegib	Experimental	Drug: Vismodegib150mg taken orally once daily

Who may qualify

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

Key inclusion criteria (10 listed)

Adult patients over 18 years of age with locally advanced or recurrent orbital or periorbital basal cell carcinoma (BCCA), or a medial canthal BCCA that threatens the lacrimal drainage system.
Clinical assessment score obtained at baseline.
Medical Oncology screening performed at baseline.
Adequate BCCA size and location.
Adequate hematopoietic capacity, hepatic and renal function.

Key exclusion criteria (7 listed)

Inability or unwillingness to swallow capsules.
Inability or unwillingness to comply with study procedures.
Pregnant, lactating, or breast feeding women.
Women of childbearing potential.
Uncontrolled medical illness.

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
Vismodegib	Drug	Vismodegib

Primary outcome

The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS).
Time frame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
The VAWS was developed for the purpose of this study. It is made up of standard ophthalmic exam points, as well as subjective assessment of tearing and overall patient satisfaction. The maximum score is 50 and a score of 21 or greater will be considered a good outcome.

3 secondary outcomes

Number of Patients With Progressive Disease (PD)
Time frame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Good Tolerance of Vismodegib
Time frame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Poor Tolerance of Vismodegib
Time frame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.

Results and safety

Results posted to the registry on 28 Oct 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Vismodegib	35	34

Primary outcome: The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS).

Time frame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.. Count of participants. Unit: Participants

Measure	Vismodegib n = 34
Value	34

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to 15 months after start of study treatment; data was collected on study for 62 months total. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Vismodegib	9 of 35 (25.7%)	34 of 35 (97.1%)	2 of 35

Limitations and caveats (from the sponsor): The initial study design aimed to enroll 50 patients; however, the study was terminated early as a consequence of the therapeutic and administrative challenges posed by the novel coronavirus pandemic and because the interim analysis revealed that the study at the point of termination was sufficient to achieve statistical significance.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 36455049Unsworth SP, Tingle CF, Heisel CJ, Eton EA, Andrews CA, Chan MP, Bresler SC, Kahana A. Analysis of residual disease in periocular basal cell carcinoma following hedgehog pathway inhibition: Follow up to the VISORB trial. PLoS One. 2022 Dec 1;17(12):e0265212. doi: 10.1371/journal.pone.0265212. eCollection 2022.
Derived from this study: PubMed 33988881Kahana A, Unsworth SP, Andrews CA, Chan MP, Bresler SC, Bichakjian CK, Durham AB, Demirci H, Elner VM, Nelson CC, Kim DS, Joseph SS, Swiecicki PL, Worden FP. Vismodegib for Preservation of Visual Function in Patients with Advanced Periocular Basal Cell Carcinoma: The VISORB Trial. Oncologist. 2021 Jul;26(7):e1240-e1249. doi: 10.1002/onco.13820. Epub 2021 May 31.
Derived from this study: PubMed 27054919Rao RC, Chan MP, Andrews CA, Kahana A. EZH2, Proliferation Rate, and Aggressive Tumor Subtypes in Cutaneous Basal Cell Carcinoma. JAMA Oncol. 2016 Jul 1;2(7):962-3. doi: 10.1001/jamaoncol.2016.0021. No abstract available.

Related trials for Carcinoma, Basal Cell

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Basal Cell Carcinoma Chemoprevention Trial
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Active, not recruitingPhase 1/2
A Beta-only IL-2 ImmunoTherapY Study
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A Study Comparing Cemiplimab-based Treatments for Adult Participants With Locally Advanced Basal Cell Skin Cancer (laBCC)
Locally Advanced Basal Cell Carcinoma
Enrolling by invitation
TruScore Standalone Clinical Study
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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.

Cagri Besirli, M.D., Ph.D. principal investigator

University of Michigan

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

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Vismodegib in people with Carcinoma, Basal Cell, run by University of Michigan Rogel 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 10 inclusion and 7 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 28 Oct 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

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 28 Oct 2021). 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?

The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS). (time frame: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.). 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 NCT02436408, which also lists 3 linked publications.

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 28 Oct 2021.