Topical Tazarotene in Treating Patients With Basal Cell Skin Cancer and Basal Cell Nevus Syndrome on the Face

This study has been completed.
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Hospital & Research Center Oakland
ClinicalTrials.gov Identifier:
NCT00489086
First received: June 20, 2007
Last updated: January 13, 2016
Last verified: January 2016
  Purpose

RATIONALE: Drugs used in chemotherapy, such as tazarotene, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying topical tazarotene to see how well it works in treating patients with basal cell skin cancer and basal cell nevus syndrome on the face.


Condition Intervention Phase
Neoplastic Syndrome
Drug: tazarotene
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Single Arm Open-Label Clinical Trial of Chemotherapy of BCC's With Tazarotene 0.1% in Subjects With Basal Cell Nevus Syndrome

Resource links provided by NLM:


Further study details as provided by Children's Hospital & Research Center Oakland:

Primary Outcome Measures:
  • Complete Response Rate [ Time Frame: 36 months ] [ Designated as safety issue: No ]
    The primary endpoint used to evaluate tazarotene efficacy for BCC chemotherapy was the complete response (CR) rate, defined as the complete visible disappearance of a patient's "target" lesion during the the 18 months of tazarotene application and its failure to recur during the ensuing 18-months. We defined surgical removal of a target lesion as a treatment failure. The primary endpoint was assessed based on intention to treat analysis such that any subject who underwent the baseline evaluation and applied at least 1 dose of tazarotene was included in the analysis. Drop-outs were considered non-responders. A priori treatment success for tazarotene was defined as a CR rate of at least 50%, and treatment failure was defined as a CR rate of 25% or less.


Secondary Outcome Measures:
  • Time to Lesion Clearance [ Time Frame: 36 months ] [ Designated as safety issue: No ]
  • Time to Progression [ Time Frame: 36 months ] [ Designated as safety issue: No ]
  • Estimated Duration of Complete Response [ Time Frame: 36 months ] [ Designated as safety issue: No ]
  • Overall Response at Treated Lesions [ Time Frame: 36 months ] [ Designated as safety issue: No ]

Enrollment: 36
Study Start Date: July 2004
Study Completion Date: June 2012
Primary Completion Date: June 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Tazarotene Cream
Open label
Drug: tazarotene
Tazarotene is a member of the acetylenic class of retinoids.
Other Name: tazorac

Detailed Description:

OBJECTIVES:

  • Determine whether topical tazarotene, administered over a period of 18 months as a chemopreventive agent, reduces the incidence of basal cell carcinomas (BCCs) on treated skin in patients with basal cell nevus syndrome (BCNS).
  • Expand and refine chemopreventive strategies in patients with BCNS who are at high risk for the development of BCCs.

OUTLINE: This is an open-label, multicenter study.

Patients apply topical tazarotene cream to the face once daily for 18 months in the absence of unacceptable toxicity.

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

List of Inclusion Criteria:

  1. Study subjects must have at least one basal cell carcinoma ≥ 3mm in diameter (target lesion) on any area of the skin except the face, chest, and back (and not impinging on vital sites) diagnosed clinically by a Study Investigator at the baseline visit.
  2. Study subjects must meet diagnostic criteria for basal cell nevus syndrome including major criterion #1 plus one additional major criterion or two of the minor criteria outlined in Table I. A first degree relative would satisfy BCNS diagnostic criteria with any two major criteria or any single major plus two minor criteria.

Table I. BCNS Diagnostic Criteria

Major criteria

  1. More than 2 BCCs or one under the age of 20 years
  2. Odontogenic keratocysts of the jaw proven by histology
  3. Three or more palmar and/or plantar pits
  4. Bilamellar calcification of the falx cerebri (if less than 20 years old)
  5. Fused, bifid, or markedly splayed ribs.
  6. First degree relative with basal cell nevus syndrome (BCNS)
  7. PTCH1 gene mutation in normal tissue*

Minor criteria

  1. Macrocephaly determined after adjustment for height
  2. Congenital malformations: cleft lip or palate, frontal bossing, "coarse face."
  3. Skeletal abnormalities: Sprengel deformity, marked pectus deformity
  4. Radiological abnormalities: bridging of the sella turcica, vertebral anomalies such as hemivertebrae, fusion or elongation of the vertebral bodies.
  5. Ovarian fibroma
  6. Medulloblastoma

3.The subject is from 18-75 years of age, inclusive.

4. If the subject is female and of child-bearing potential (women are considered not of childbearing potential if they are at least 2 years post-menopausal and/or surgically sterile), she:

i. has been using adequate contraception (abstinence, IUD, birth control pills, or spermicidal gel with diaphragm or condom) since her last menses and will use adequate contraception during the study, and ii. is not lactating, and iii. has documented one negative serum pregnancy test within 14 days prior to study entry.

5. The subject is willing to abstain from application of non-study topical medications to the skin of the face for the duration of the study, including prescription and over the counter preparations.

6. The subject is willing not to have targeted BCCs treated by their PSCP 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.

List of Exclusion Criteria:

  1. The subject has used topical or systemic therapies that might interfere with the evaluation of the study medication during the study.
  2. The subject has a history of hypersensitivity to any of the ingredients in the study medication formulations.
  3. The subject is unable to return for follow-up tests.
  4. The subject has uncontrolled systemic disease, including known HIV positive patients.
  5. The subject has a history of other skin conditions or significant illness that would interfere with evaluation of the study medication.
  6. 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.
  7. The subject has a history of invasive cancer within the past five years excluding non-melanoma skin cancer, Stage I cervical cancer, or CLL Stage 0.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00489086

Locations
United States, California
Children's Hospital and Research Center Oakland
Oakland, California, United States, 94609
United States, New York
Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center
New York, New York, United States, 10032
Sponsors and Collaborators
Children's Hospital & Research Center Oakland
National Cancer Institute (NCI)
Investigators
Principal Investigator: Ervin Epstein, MD Children's Hospital & Research Center Oakland
Principal Investigator: David R. Bickers, MD Herbert Irving Comprehensive Cancer Center
  More Information

Responsible Party: Children's Hospital & Research Center Oakland
ClinicalTrials.gov Identifier: NCT00489086     History of Changes
Obsolete Identifiers: NCT00500643
Other Study ID Numbers: CDR0000551655  R01CA109584  P30CA082103 
Study First Received: June 20, 2007
Results First Received: July 31, 2013
Last Updated: January 13, 2016
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by Children's Hospital & Research Center Oakland:
nevoid basal cell carcinoma syndrome
basal cell carcinoma of the skin

Additional relevant MeSH terms:
Syndrome
Basal Cell Nevus Syndrome
Neoplasms
Disease
Pathologic Processes
Odontogenic Cysts
Jaw Cysts
Bone Cysts
Cysts
Carcinoma, Basal Cell
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
Genetic Diseases, Inborn
Tazarotene
Nicotinic Acids
Dermatologic Agents
Keratolytic Agents
Vitamin B Complex
Vitamins
Micronutrients

ClinicalTrials.gov processed this record on August 24, 2016