VISmodegib for ORbital and Periocular Basal Cell Carcinoma (VISORB)

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ClinicalTrials.gov Identifier: NCT02436408

Recruitment Status : Completed

First Posted : May 6, 2015

Results First Posted : October 28, 2021

Last Update Posted : October 28, 2021

View this study on Beta.ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

University of Michigan Rogel Cancer Center

Study Type	Interventional
Study Design	Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Condition	Carcinoma, Basal Cell
Intervention	Drug: Vismodegib
Enrollment	35

Participant Flow

Recruitment Details
Pre-assignment Details


Arm/Group Title	Vismodegib
Arm/Group Description	150mg taken orally once daily
Arm/Group Description	150mg taken orally once daily
Period Title: Overall Study
Started	35
Completed	34
Not Completed	1
Reason Not Completed
Death	1

Baseline Characteristics

Arm/Group Title		Vismodegib
Arm/Group Description		150mg taken orally once daily
Arm/Group Description		150mg taken orally once daily
Overall Number of Baseline Participants		35
Baseline Analysis Population Description		[Not Specified]
Baseline Analysis Population Description		[Not Specified]
Age, Continuous Median (Full Range) Unit of measure: Years
	Number Analyzed	35 participants
		69 (45 to 95)
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	35 participants
	Female	17 48.6%
	Male	18 51.4%
Ethnicity (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	35 participants
	Hispanic or Latino	0 0.0%
	Not Hispanic or Latino	34 97.1%
	Unknown or Not Reported	1 2.9%
Region of Enrollment Measure Type: Number Unit of measure: Participants
United States	Number Analyzed	35 participants
United States		35

Outcome Measures

1.Primary Outcome


Title	The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS).
Description	The VAWS was developed for the purpose of this study. It is made up of...
Description	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.
Time Frame	Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Vismodegib
Arm/Group Description:	150mg taken orally once daily
Arm/Group Description:	150mg taken orally once daily
Overall Number of Participants Analyzed	34
Measure Type: Count of Participants Unit of Measure: Participants
	34 100.0%

2.Secondary Outcome


Title	Number of Patients With Progressive Disease (PD)
Description	Treatment response will be determined per Response Evaluation Criteria...
Description	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).
Time Frame	Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Vismodegib
Arm/Group Description:	150mg taken orally once daily
Arm/Group Description:	150mg taken orally once daily
Overall Number of Participants Analyzed	34
Measure Type: Count of Participants Unit of Measure: Participants
	0 0.0%

3.Secondary Outcome


Title	Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Good Tolerance of Vismodegib
Description	Tolerance will be self-reported by patient. Treatment response will be...
Description	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).
Time Frame	Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Vismodegib
Arm/Group Description:	150mg taken orally once daily
Arm/Group Description:	150mg taken orally once daily
Overall Number of Participants Analyzed	34
Measure Type: Count of Participants Unit of Measure: Participants
	7 20.6%

4.Secondary Outcome


Title	Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Poor Tolerance of Vismodegib
Description	Tolerance will be self-reported by patient. Treatment response will be...
Description	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).
Time Frame	Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Vismodegib
Arm/Group Description:	150mg taken orally once daily
Arm/Group Description:	150mg taken orally once daily
Overall Number of Participants Analyzed	34
Measure Type: Count of Participants Unit of Measure: Participants
	27 79.4%

Adverse Events

Time Frame	Up to 15 months after start of study treatment; data was collected on study for 62 months total
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	Vismodegib
Arm/Group Description	150mg taken orally once daily
Arm/Group Description	150mg taken orally once daily
All-Cause Mortality
	Vismodegib
	Affected / at Risk (%)
Total	2/35 (5.71%)
Serious Adverse Events Serious Adverse Events
	Vismodegib
	Affected / at Risk (%)
Total	9/35 (25.71%)
Cardiac disorders
Acute coronary syndrome ^†	1/35 (2.86%)
Heart failure ^†	1/35 (2.86%)
Gastrointestinal disorders
Colitis ^†	1/35 (2.86%)
Gastrointestinal disorders - Other ^†	1/35 (2.86%)
Nausea ^†	2/35 (5.71%)
Vomiting ^†	2/35 (5.71%)
Infections and infestations
Infections and infestations - Other ^†	1/35 (2.86%)
Lung infection ^†	2/35 (5.71%)
Sepsis ^†	1/35 (2.86%)
Metabolism and nutrition disorders
Hypoglycemia ^†	1/35 (2.86%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other ^†	1/35 (2.86%)
Renal and urinary disorders
Acute kidney injury ^†	1/35 (2.86%)
Respiratory, thoracic and mediastinal disorders
Aspiration ^†	1/35 (2.86%)
Pulmonary edema ^†	1/35 (2.86%)
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other ^†	1/35 (2.86%)
Vascular disorders
Vascular disorders - Other ^†	1/35 (2.86%)
† Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Vismodegib
	Affected / at Risk (%)
Total	34/35 (97.14%)
Blood and lymphatic system disorders
Anemia ^†	2/35 (5.71%)
Cardiac disorders
Sinus bradycardia ^†	1/35 (2.86%)
Ear and labyrinth disorders
Hearing impaired ^†	1/35 (2.86%)
Tinnitus ^†	1/35 (2.86%)
Eye disorders
Blurred vision ^†	1/35 (2.86%)
Cataract ^†	2/35 (5.71%)
Dry eye ^†	1/35 (2.86%)
Eye disorders - Other ^†	8/35 (22.86%)
Eye pain ^†	1/35 (2.86%)
Eyelid function disorder ^†	3/35 (8.57%)
Watering eyes ^†	3/35 (8.57%)
Gastrointestinal disorders
Abdominal pain ^†	2/35 (5.71%)
Constipation ^†	6/35 (17.14%)
Diarrhea ^†	3/35 (8.57%)
Gastroesophageal reflux disease ^†	5/35 (14.29%)
Nausea ^†	11/35 (31.43%)
Vomiting ^†	4/35 (11.43%)
General disorders
Chills ^†	1/35 (2.86%)
Edema face ^†	2/35 (5.71%)
Edema limbs ^†	1/35 (2.86%)
Fatigue ^†	12/35 (34.29%)
Fever ^†	1/35 (2.86%)
General disorders and administration site conditions - Other ^†	1/35 (2.86%)
Pain ^†	4/35 (11.43%)
Infections and infestations
Papulopustular rash ^†	2/35 (5.71%)
Injury, poisoning and procedural complications
Bruising ^†	1/35 (2.86%)
Fall ^†	2/35 (5.71%)
Investigations
Alanine aminotransferase increased ^†	1/35 (2.86%)
Alkaline phosphatase increased ^†	1/35 (2.86%)
Aspartate aminotransferase increased ^†	1/35 (2.86%)
Creatinine increased ^†	1/35 (2.86%)
Weight loss ^†	12/35 (34.29%)
Metabolism and nutrition disorders
Anorexia ^†	12/35 (34.29%)
Hyperglycemia ^†	2/35 (5.71%)
Hyperkalemia ^†	1/35 (2.86%)
Musculoskeletal and connective tissue disorders
Arthralgia ^†	3/35 (8.57%)
Arthritis ^†	2/35 (5.71%)
Back pain ^†	4/35 (11.43%)
Generalized muscle weakness ^†	5/35 (14.29%)
Muscle weakness lower limb ^†	3/35 (8.57%)
Musculoskeletal and connective tissue disorder - Other ^†	2/35 (5.71%)
Myalgia ^†	23/35 (65.71%)
Pain in extremity ^†	1/35 (2.86%)
Nervous system disorders
Dizziness ^†	3/35 (8.57%)
Dysgeusia ^†	26/35 (74.29%)
Facial muscle weakness ^†	1/35 (2.86%)
Peripheral motor neuropathy ^†	1/35 (2.86%)
Spasticity ^†	7/35 (20.00%)
Psychiatric disorders
Agitation ^†	1/35 (2.86%)
Hallucinations ^†	1/35 (2.86%)
Insomnia ^†	2/35 (5.71%)
Libido decreased ^†	1/35 (2.86%)
Reproductive system and breast disorders
Erectile dysfunction ^†	1/35 (2.86%)
Respiratory, thoracic and mediastinal disorders
Dyspnea ^†	1/35 (2.86%)
Epistaxis ^†	1/35 (2.86%)
Nasal congestion ^†	4/35 (11.43%)
Productive cough ^†	1/35 (2.86%)
Sinus disorder ^†	1/35 (2.86%)
Skin and subcutaneous tissue disorders
Alopecia ^†	17/35 (48.57%)
Photosensitivity ^†	1/35 (2.86%)
Pruritus ^†	1/35 (2.86%)
Rash maculo-papular ^†	1/35 (2.86%)
Skin and subcutaneous tissue disorders - Other ^†	4/35 (11.43%)
Skin ulceration ^†	2/35 (5.71%)
Urticaria ^†	1/35 (2.86%)
Surgical and medical procedures
Surgical and medical procedures - Other ^†	1/35 (2.86%)
Vascular disorders
Hypotension ^†	1/35 (2.86%)
† Indicates events were collected by systematic assessment

Limitations and Caveats

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.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	Cagri Besirli, MD, PhD
Organization:	University of Michigan Rogel Cancer Center
Phone:	734-232-8404
EMail:	cbesirli@med.umich.edu

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

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. Erratum In: JAMA Oncol. 2016 Jul 1;2(7):966.

Layout table for additonal information

Responsible Party:	University of Michigan Rogel Cancer Center
ClinicalTrials.gov Identifier:	NCT02436408
Other Study ID Numbers:	UMCC 2014.022 HUM00082579 ( Other Identifier: University of Michigan )
First Submitted:	May 1, 2015
First Posted:	May 6, 2015
Results First Submitted:	September 29, 2021
Results First Posted:	October 28, 2021
Last Update Posted:	October 28, 2021

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