Basal Cell Nevus SyndromePhase 2VismodegibNCT01556009
Trial Comparing the Effects of Intermittent Vismodegib vs. PDT in Patients With Multiple Basal Cell Carcinomas
A Phase 2 study testing Vismodegib and Aminolevulinic acid %20 topical solution in people with Basal Cell Nevus Syndrome and Gorlin's Syndrome, run by UCSF Benioff Children's Hospital Oakland at 3 sites in United States.
Trial summary
The purpose of this study is to evaluate and compare the safety and efficacy of intermittent vismodegib and of Photodynamic Therapy (PDT). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Basal Cell Nevus Syndrome, Gorlin's Syndrome
- What is being tested?
- Vismodegib and Aminolevulinic acid %20 topical solution
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 3 sitesUnited States
- Study dates
- Apr 2012to Dec 2015
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Show all 13Show fewer
- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center
New York, New York, 10032
Children's Hospital Oakland Research Institiute
Oakland, California, 94609
Children's Hospital Research Center Oakland
Oakland, California, 94609
About this study
Official title: A Phase II Randomized, Open Label Trial Comparing the Effects of Intermittent Vismodegib Versus PDT on the Maintenance of Benefit Following 7 Months of Continuous Vismodegib Treatment in Patients With Multiple Basal Cell Carcinomas
The purpose of this study is to evaluate and compare the safety and efficacy of intermittent vismodegib and of Photodynamic Therapy (PDT).
Read the detailed description
This is a Phase II, 28 month, randomized, two arm multicenter clinical study design. During the initial 7 months of the study, all 24 subjects will receive vismodegib, 150mg/day. They then will be randomized in a 1:1 ratio to receive intermittent vismodegib, 150 mg/day, during months 10-13, 16-19, and 22-25 or to receive treatment with PDT at month 10 and at three month intervals thereafter. The safety and efficacy of intermittent vismodegib and of PDT will be assessed at the time of the subjects' visits to the Study Center and at the time of telephone contacts. A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months. The DSMB review will focus on adverse events and efficacy results. Subjects will be monitored for the presence of surrogate endpoint biomarkers (SEBs) at each Study visit.
- Conditions
- Basal Cell Nevus Syndrome, Gorlin's Syndrome
- Being tested
- Vismodegib, Aminolevulinic acid %20 topical solution
- Keywords
- Basal Cell Carcinoma, Photodynamic Therapy
- Medical subject headings
- Basal Cell Nevus Syndrome, Carcinoma, Basal Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VismodegibDrug
Also called Erivedge, GDC-0449
150 mg per day for 7 continuous months then randomized for 3 month intervals to 28 months.
Aminolevulinic acid %20 topical solutionDrug
Also called Levulan
20% topical solution applied every three months from month 10, 13, 16, 19, 22. Applied and incubated for three hours.
| Study group | Role | What they receive |
|---|---|---|
| Drug (Vismodegib) | Active comparator | Vismodegib taken orally 150 mg per day for 7 continuous months then randomized for 3 month intervals to 28 months. Receives: 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. Receives: Drug: Aminolevulinic acid %20 topical solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to Recurrence to Baseline SEB Burden Following 7 Months of Continuous Vismodegib Treatment.
Primary:
- 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).
Measured: 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.
- To assess the safety of intermittent vismodegib in patients with multiple BCCs (BCNS and non-BCNS) during months 8-28. ii. To assess resistance of SEBs to treatments in patients with multiple BCCs (BCNS and non-BCNS) treated intermittently during months 8-28. iii. To assess the degree of reduction of SEBs after 7 months of continuous daily vismodegib therapy. iv. To conduct an exploratory evaluation in non-BCNS patients with multiple BCCs (high burden of disease) of the efficacy and tolerability of intermittent vismodegib vs PDT
Measured: A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2012 (estimated)
- Study startApr 2012
- Primary completionDec 2015
- Study completionDec 2015
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.
- Other trials for Basal Cell Nevus SyndromeStudies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCSF Benioff Children's Hospital Oakland
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Ervin Epstein, MD, Principal investigator, Children's Hospital Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01556009
- Sponsor's protocol ID
- 2011-077
- Other IDs
- ML28244
- Acronym
- Vismodegib
- Status last verified
- Oct 2020
- First submitted
- 14 Mar 2012
- Last updated
- 2 Nov 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Randomized, Open Label Trial Comparing the Effects of Intermittent Vismodegib Versus PDT on the Maintenance of Benefit Following 7 Months of Continuous Vismodegib Treatment in Patients With Multiple Basal Cell Carcinomas
- ClinicalTrials.gov ID
- NCT01556009
- Acronym / sponsor ID
- Vismodegib / 2011-077
- Conditions
- Basal Cell Nevus Syndrome, Gorlin's Syndrome
- Keywords
- Basal Cell Carcinoma, Photodynamic Therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- UCSF Benioff Children's Hospital OaklandUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- Apr 2012
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 16 Mar 2012 (estimated)
- Results
- Posted 2 Nov 2020
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 evaluate and compare the safety and efficacy of intermittent vismodegib and of Photodynamic Therapy (PDT).
Detailed description
This is a Phase II, 28 month, randomized, two arm multicenter clinical study design. During the initial 7 months of the study, all 24 subjects will receive vismodegib, 150mg/day. They then will be randomized in a 1:1 ratio to receive intermittent vismodegib, 150 mg/day, during months 10-13, 16-19, and 22-25 or to receive treatment with PDT at month 10 and at three month intervals thereafter. The safety and efficacy of intermittent vismodegib and of PDT will be assessed at the time of the subjects' visits to the Study Center and at the time of telephone contacts. A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months. The DSMB review will focus on adverse events and efficacy results. Subjects will be monitored for the presence of surrogate endpoint biomarkers (SEBs) at each Study visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Drug (Vismodegib) | Active comparator | Drug: VismodegibVismodegib taken orally 150 mg per day for 7 continuous months then randomized for 3 month intervals to 28 months. |
| Aminolevulinic acid %20 topical solution | Active comparator | Drug: Aminolevulinic acid %20 topical solutionAminolevulinic acid HCL 20% topical solution applied every three months from month 10, 13, 16, 19, 22. Applied and incubated for three hours. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (13 listed)
- 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.
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 |
|---|---|---|
| VismodegibAlso: Erivedge, GDC-0449150 mg per day for 7 continuous months then randomized for 3 month intervals to 28 months. | Drug | Drug (Vismodegib) |
| Aminolevulinic acid %20 topical solutionAlso: Levulan20% 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 |
Primary outcome
Time to Recurrence to Baseline SEB Burden Following 7 Months of Continuous Vismodegib Treatment.
Time frame: A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months.
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).
1 secondary outcome
The Cumulative Diameter (Burden) of SEBs in Patients Treated Intermittently With Vismodegib vs PDT During Months 8-28 Maintenance Period.
Time frame: A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Nov 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Ervin Epstein, MD principal investigator
Children's Hospital Research Institute
3 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 Vismodegib and Aminolevulinic acid %20 topical solution in people with Basal Cell Nevus Syndrome and Gorlin's Syndrome, run by UCSF Benioff Children's Hospital Oakland at 3 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 8 inclusion and 13 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 2 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 2 Nov 2020). 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?
Time to Recurrence to Baseline SEB Burden Following 7 Months of Continuous Vismodegib Treatment. (time frame: A Data Safety Monitoring Board (DSMB) will review results for an interim analysis when 12 subjects have completed 28 months.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2 Nov 2020.