Trial Comparing the Effects of Intermittent Vismodegib vs. PDT in Patients With Multiple Basal Cell Carcinomas

NCT01556009 | Completed Phase 2 Results DMC

• 2 other identifiers: 2011-077ML28244
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 3

Brief Summary

The purpose of this study is to evaluate and compare the safety and efficacy of intermittent vismodegib and of Photodynamic Therapy (PDT).

Conditions

Basal Cell Nevus Syndrome; Gorlin's Syndrome

Keywords

Basal Cell Carcinoma; Photodynamic Therapy

Outcome Measures

Primary Outcomes (1)

• Time to Recurrence to Baseline SEB Burden Following 7 Months of Continuous Vismodegib Treatment.

  Primary: i. To compare the efficacy of (i) intermittent vismodegib vs. (ii) the efficacy of photodynamic therapy (PDT) in preventing the return of the burden of surgically eligible BCCs (SEBs) to baseline level following 7 months of continuous vismodegib therapy. ii. To compare the cumulative diameter (burden) of SEBs in patients treated intermittently with vismodegib vs. with photodynamic therapy (PDT).

  A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months.

Secondary Outcomes (1)

• The Cumulative Diameter (Burden) of SEBs in Patients Treated Intermittently With Vismodegib vs PDT During Months 8-28 Maintenance Period.

  A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months.

Study Arms (2)

Drug (Vismodegib)

ACTIVE COMPARATOR

Vismodegib taken orally 150 mg per day for 7 continuous months then randomized for 3 month intervals to 28 months.

Drug: Vismodegib

Aminolevulinic acid %20 topical solution

ACTIVE COMPARATOR

Aminolevulinic acid HCL 20% topical solution applied every three months from month 10, 13, 16, 19, 22. Applied and incubated for three hours.

Drug: Aminolevulinic acid %20 topical solution

Interventions

Vismodegib DRUG

150 mg per day for 7 continuous months then randomized for 3 month intervals to 28 months.

Also known as: Erivedge, GDC-0449

Drug (Vismodegib)

Aminolevulinic acid %20 topical solution DRUG

20% topical solution applied every three months from month 10, 13, 16, 19, 22. Applied and incubated for three hours.

Also known as: Levulan

Aminolevulinic acid %20 topical solution

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The subject:
• has had diagnosed at least 10 SEB (of diameter 3 mm diameter or greater on the nose or periorbital skin, 5 mm or greater elsewhere on the face, or 9 mm or greater on non-facial areas excluding the skin below the knees) during the two years before study entry.
• meet diagnostic criteria for basal cell nevus syndrome
• is willing to abstain from application of non-study topical medications to the skin for the duration of the study. For example, topical preparations containing corticosteroids (other than Triamcinolone applied no more than 6x/month).
• is willing to forego treatment of BCCs unless the BCCs are documented by Study Investigators, preferably on two separate visits, except when the PSCP believes that delay in treatment potentially might compromise the health of the subject.
• has normal laboratory tests as defined by the following: Normal hematopoietic capacity, Normal hepatic function: AST and ALT greater than or equal to 2x the upper limit of normal (ULN) Total bilirubin within normal range 0.20 mg/dl to 1.50 mg/dl or within 3x ULN for patients with Gilbert's disease Normal renal function: normal serum creatinine or measured creatinine clearance less than 50 mL/minute. Fasting cholesterol greater than or equal to 220 untreated
• be willing to not donate blood or semen for three months following discontinuation of Study medications.
• is willing to avoid pregnancy in his partner as defined by the following: Male subject is willing to use a latex condom during the study and for 3 months after the last dose during sexual contact with a female of childbearing potential, even if he has had a successful vasectomy. His partner must also use a form of birth control

You may not qualify if:

• The subject:
• has used topical or systemic therapies that might interfere with the evaluation of the study medication during the study. Specifically these include the use of: (i) glucocorticoids (other than Triamcinolone on no more than 36 days during the six months prior to study entry) to more than 5% of the skin (ii) retinoids systemically or topically to more than 5% of the skin during the six months prior to study entry; (iii) alpha-hydroxy acids to more than 5% of the skin during the six months prior to study entry (iv) 5-fluorouracil or imiquimod systemically or topically to the skin above the knees during the six months prior to study entry. (v) treatment with systemic chemotherapy within one year prior to starting study medication.
• has a history of hypersensitivity to any of the ingredients in the study medication formulations.
• is unable to return for follow-up visits and tests.
• has uncontrolled systemic disease, including known HIV positive patients.
• has history of congestive heart failure.
• has uncontrolled hypocalcemia, hypomagnesemia, or hypokalemia
• has clinically important history of liver disease, including viral or hepatitis, current alcohol abuse, or cirrhosis.
• has any condition or situation which in the Investigator's opinion may put the subject at significant risk, could confound the study results, or could interfere significantly with the subject's participation in the study.
• has a history of invasive cancer within the past five years excluding non-melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of the breast, or CLL Stage 0.
• has current, recent (within 4 weeks of Day 1), or planned participation in an experimental drug study while enrolled in this study.
• is a female who is pregnant, plans to ever to become pregnant, capable of becoming pregnant or is breast feeding.
• is a male who is unwilling or unable to comply with pregnancy prevention measures.

Sponsors & Collaborators

• UCSF Benioff Children's Hospital Oakland: lead
• Genentech, Inc.: collaborator

Study Sites (3)

Children's Hospital Oakland Research Institiute

Oakland, California, 94609, United States

Location

Children's Hospital Research Center Oakland

Oakland, California, 94609, United States

Location

Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

Basal Cell Nevus Syndrome; Carcinoma, Basal Cell

Interventions

HhAntag691; Aminolevulinic Acid

Condition Hierarchy (Ancestors)

Odontogenic Cysts; Jaw Cysts; Bone Cysts; Cysts; Neoplasms; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms, Basal Cell; Neoplastic Syndromes, Hereditary; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Jaw Diseases; Stomatognathic Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Levulinic Acids; Keto Acids; Carboxylic Acids; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Ervin H Epstein Jr., MD
• Organization: Children's Hospital of Oakland Research Institute, Oakland

eepstein@chori.org

510-450-5688

Study Officials

• Ervin Epstein, MD

  Children's Hospital Research Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 14, 2012
• First Posted: March 16, 2012
• Study Start: April 1, 2012
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: November 2, 2020
• Results First Posted: November 2, 2020

Record last verified: 2020-10

Locations

US (3)