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NCT01700049

Study Evaluating the Efficacy of Oral Vismodegib in Various Histologic Subtypes

Completed Phase 2 Results posted Last updated 23 May 2019
What this trial tests

Phase 2 trial testing vismodegib (150 mg PO daily) in Basal Cell Carcinoma in 28 participants. Completed in 3 July 2018.

Timeline
14 January 2013
Primary endpoint
3 July 2017
3 July 2018

Quick facts

Lead sponsorMayo Clinic
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment28
Start date14 January 2013
Primary completion3 July 2017
Estimated completion3 July 2018
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

Mayo Clinic

Who can join

18 and older, any sex, with Basal Cell Carcinoma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Efficacy of Vismodegib Primary · Week 24

The efficacy of vismodegib was defined as the number of tumor biopsies with positive pathology after 24 weeks. Subjects had one target lesion and up to 3 additional non-target lesions. A cumulative total of 65 tumors was measured after 24 weeks of treatment. Histopathological subtypes were categorized primarily as infiltrative, nodular and superficial.

Infiltrative
GroupValue95% CI
Open Label Oral Vismodegib2
Nodular
GroupValue95% CI
Open Label Oral Vismodegib5
Superficial
GroupValue95% CI
Open Label Oral Vismodegib1
Keratotic
GroupValue95% CI
Open Label Oral Vismodegib1
Safety of Vismodegib Secondary · Up to 18 months

The safety of Vismodegib was evaluated by monitoring adverse effects. All adverse events, expected and unexpected, were recorded and categorized using the Common Terminology Criteria for Adverse Events (CTCAE) guide. This is a set of criteria from the National Cancer Institute (NCI) used to standardize classification of adverse effects of drugs. Grade 1 events are defined as mild, grade 2 as moderate, grade 3 as severe; grade 4 as life-threatening and grade 5 as death.

Grade 1
GroupValue95% CI
Open Label Oral Vismodegib133
Grade 2
GroupValue95% CI
Open Label Oral Vismodegib48
Grade 3
GroupValue95% CI
Open Label Oral Vismodegib8
Grade 4
GroupValue95% CI
Open Label Oral Vismodegib1
Grade 5
GroupValue95% CI
Open Label Oral Vismodegib2
Onset of Efficacy of Vismodegib Secondary · Up to week 24

Onset of efficacy was measured by tumor surface area reduction or increase. Subjects had one target lesion and up to 3 additional non-target lesions. Surface area of a cumulative total of 65 tumors (27 target lesions and 38 non-target lesions) was measured after 24 weeks of treatment. A complete response was defined as 100% reduction in tumor surface area. Partial response was defined as greater than 50% reduction in tumor surface area. Stable disease was defined as less than 50% reduction in tumor surface area and Progressive disease was defined as greater than 20% increase in tumor surface a

Complete response
GroupValue95% CI
Open Label Oral Vismodegib13
Partial response
GroupValue95% CI
Open Label Oral Vismodegib27
Stable Disease
GroupValue95% CI
Open Label Oral Vismodegib24
Progressive Disease
GroupValue95% CI
Open Label Oral Vismodegib1

Adverse events — posted to ClinicalTrials.gov

Time frame: The study period during which all Adverse Events (AE) and Serious Adverse Events (SAE) must be reported begins after informed consent is obtained and initiation of study treatment and ends 30 days following the last administration of study treatment or study discontinuation/termination, whichever is earlier.. Reporting threshold: 4%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Open Label Oral Vismodegib
Serious: 4/28 (14%)
Deaths: 2/28

Serious adverse events (6 terms)

ReactionSystemOpen Label Oral Vismodegib
Myocardial infarctionCardiac disorders
Duodenal ulcerGastrointestinal disorders
Neurogenic claudicationNervous system disorders
Gastrointestinal bleedGastrointestinal disorders
Exacerbation of low back painGeneral disorders
Congestive Heart FailureCardiac disorders
Other adverse events (63 terms — click to expand)

ReactionSystemOpen Label Oral Vismodegib
Dysgeusia/Loss of TasteGeneral disorders
Muscle spasmsMusculoskeletal and connective tissue disorders
AlopeciaSkin and subcutaneous tissue disorders
Decreased appetite/anorexia/weight lossMetabolism and nutrition disorders
NauseaGeneral disorders
FatigueGeneral disorders
DiarrheaGastrointestinal disorders
Joint achesMusculoskeletal and connective tissue disorders
Upper respiratory infectionInfections and infestations
Itching/pruritusSkin and subcutaneous tissue disorders
SwellingGeneral disorders
Muscle achesMusculoskeletal and connective tissue disorders
ConstipationGastrointestinal disorders
DyspepsiaGastrointestinal disorders
GastroenteritisGastrointestinal disorders
Wound infectionInfections and infestations
Abnormal hair growthGeneral disorders
InsomniaGeneral disorders
ComedonesSkin and subcutaneous tissue disorders
Scalp tendernessGeneral disorders
VertigoEar and labyrinth disorders
HeadacheGeneral disorders
Blurry visionEye disorders
Urinary Tract InfectionInfections and infestations
Actinic keratosisSkin and subcutaneous tissue disorders
FallsGeneral disorders
VomitingGeneral disorders
DehydrationGeneral disorders
Decrease in eyelashes/eyebrowsGeneral disorders
RashSkin and subcutaneous tissue disorders
Biopsy site inflammationSkin and subcutaneous tissue disorders
Viral syndromeInfections and infestations
EsophagitisGastrointestinal disorders
TinnitusEar and labyrinth disorders
Stunted nail growthGeneral disorders
Maxillary fibrous cystMusculoskeletal and connective tissue disorders
BloatingGeneral disorders
Vaginal bleedingReproductive system and breast disorders
Dry mouthGeneral disorders
Tooth painGeneral disorders

Most-reported serious reactions: Myocardial infarction, Duodenal ulcer, Neurogenic claudication, Gastrointestinal bleed, Exacerbation of low back pain, Congestive Heart Failure.

Data from ClinicalTrials.gov NCT01700049 adverse events section.

Sponsor's own description

The purpose of this study is to investigate the safety and effectiveness of oral vismodegib therapy in the treatment of different 'histologic subtypes' of basal cell skin cancer (BCC). The term 'histologic subtype' refers to how the cells and tumor tissue looks under the microscope. Three different 'histologic subtypes' of basal cell skin cancer (infiltrative/morpheaform, nodular and superficial) will be examined in this study.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Targeting cancer stem cell pathways for cancer therapy.
    Yang L, Shi P, Zhao G, Xu J, et al · · 2020 · cited 1354× · PMID 32296030 · DOI 10.1038/s41392-020-0110-5
  2. Targeting the Sonic Hedgehog Signaling Pathway: Review of Smoothened and GLI Inhibitors.
    Rimkus TK, Carpenter RL, Qasem S, Chan M, et al · · 2016 · cited 411× · PMID 26891329 · DOI 10.3390/cancers8020022
  3. Hedgehog Signaling: From Basic Biology to Cancer Therapy.
    Wu F, Zhang Y, Sun B, McMahon AP, et al · · 2017 · cited 243× · PMID 28286127 · DOI 10.1016/j.chembiol.2017.02.010
  4. Targeting the tumor stroma for cancer therapy.
    Xu M, Zhang T, Xia R, Wei Y, et al · · 2022 · cited 195× · PMID 36324128 · DOI 10.1186/s12943-022-01670-1
  5. Hedgehog signaling in tissue homeostasis, cancers, and targeted therapies.
    Jing J, Wu Z, Wang J, Luo G, et al · · 2023 · cited 160× · PMID 37596267 · DOI 10.1038/s41392-023-01559-5
  6. Hedgehog Signaling in Cancer: A Prospective Therapeutic Target for Eradicating Cancer Stem Cells.
    Sari IN, Phi LTH, Jun N, Wijaya YT, et al · · 2018 · cited 155× · PMID 30423843 · DOI 10.3390/cells7110208
  7. Hedgehog Pathway Inhibitors as Targeted Cancer Therapy and Strategies to Overcome Drug Resistance.
    Nguyen NM, Cho J. · · 2022 · cited 88× · PMID 35163655 · DOI 10.3390/ijms23031733
  8. Signaling pathways in the regulation of cancer stem cells and associated targeted therapy.
    Manni W, Min W. · · 2022 · cited 52× · PMID 36226253 · DOI 10.1002/mco2.176

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Other recruiting trials for Basal Cell Carcinoma

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01700049.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing