Clinical Trials /

Vitamin D and Photodynamic Therapy for Treatment of BCC in BCNS

NCT03483441

Description:

The purpose of this research is to evaluate the use of Photodynamic Therapy (PDT) to treat the multiple basal cell carcinoma (BCC) skin cancers in children and adults with Basal Cell Nevus Syndrome (or who have at least 3 BCC tumors present). In addition, the investigators will test a hypothesis (based upon our extensive preclinical studies in mice) that induction of a transient spike in serum Vitamin D levels through administration of dietary Vitamin D (cholecalciferol; D3) can significantly increase PDT efficacy and improve the clearance of cutaneous BCC tumors.

Related Conditions:
  • Basal Cell Carcinoma
Recruiting Status:

Recruiting

Phase:

Phase 1

Trial Eligibility

Document

Title

  • Brief Title: Vitamin D and Photodynamic Therapy for Treatment of BCC in BCNS
  • Official Title: Vitamin D as a Nutritional Neoadjuvant During Photodynamic Therapy of Basal Cell Carcinoma in Basal Cell Nevus Syndrome

Clinical Trial IDs

  • ORG STUDY ID: 18-310
  • NCT ID: NCT03483441

Conditions

  • Basal Cell Nevus Syndrome
  • Basal Cell Carcinoma

Interventions

DrugSynonymsArms
PDTphotodynamic therapyPlacebo pill prior to PDT

Purpose

The purpose of this research is to evaluate the use of Photodynamic Therapy (PDT) to treat the multiple basal cell carcinoma (BCC) skin cancers in children and adults with Basal Cell Nevus Syndrome (or who have at least 3 BCC tumors present). In addition, the investigators will test a hypothesis (based upon our extensive preclinical studies in mice) that induction of a transient spike in serum Vitamin D levels through administration of dietary Vitamin D (cholecalciferol; D3) can significantly increase PDT efficacy and improve the clearance of cutaneous BCC tumors.

Detailed Description

      Patients with a rare genetic syndrome, called Basal Cell Nevus Syndrome or Gorlin-Goltz
      Syndrome (caused by a mutation in the PTCH1 gene) will be recruited to participate in this
      study. Potential patients will be referred by their local physicians or by the BCCNS Life
      Support Network to contact a study coordinator about enrollment.

      The study will examine a combination regimen of Vitamin D3/PDT and compare its efficacy in
      treating BCC tumors to PDT alone. The hypothesis is that oral D3 supplements, administered
      over a relatively short time, can boost the effectiveness of PDT for cutaneous (BCC) in this
      patient population. Participants will receive three PDT treatments, at two-month intervals,
      over a 6 month period.

      Tumor clinical clearance rates after neoadjuvant Vitamin D3/PDT, versus PDT alone, will be
      compared within the same patient. Measurement endpoints will include tumor dimensions by
      clinical examination, tumor measurements in photographs, PpIX fluorescence, serum 25OH-D3
      levels, and characterization of VDR gene alleles. In addition, assessments of the
      tolerability (pain scale measurements) and patient satisfaction with the technique will be
      measured.
    

Trial Arms

NameTypeDescriptionInterventions
Placebo pill prior to PDTPlacebo ComparatorBCC tumors will be treated with ALA-PDT, without any active pretreatment
  • PDT
Vitamin D pill prior to PDTActive ComparatorPatients will take oral Vit D supplements (10,000 IU/day) immediately prior to PDT of their BCC tumors.
  • PDT

Eligibility Criteria

        Inclusion Criteria:

          -  A diagnosis of Basal Cell Nevus Syndrome (BCNS)

          -  At least three BCC tumors (two of which are biopsy-proven)

          -  Female subjects must not become pregnant during the study

          -  Subjects must be able to understand and willing to sign a written informed consent
             document

        Exclusion Criteria:

          -  Pregnant or nursing

          -  At risk for hypercalcemia (renal disease, sarcoidosis, etc.)

          -  Taking vismodegib or a hedgehog pathway inhibitor; must stop at least 1 month prior

          -  Taking any topical treatment on their BCC tumors; must stop at least one month prior

          -  Taking Vitamin D or multivitamin supplements; must stop at least one month prior

          -  Currently undergoing treatment for other cancers with medical or radiation therapy

          -  Patients with a known hypersensitivity to 5-aminolevulinic acid or any component of
             the study material

          -  Patients with history of a photosensitivity disease, such as porphyria cutanea tarda

          -  Currently participating in another clinical trial
      
Maximum Eligible Age:N/A
Minimum Eligible Age:N/A
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:BCC: Rate of Tumor Clearance
Time Frame:Selected Visits (6 months total)
Safety Issue:
Description:For each patient, the investigators will analyze the difference in tumor clearance between treatments, one with neoadjuvant D3+PDT, the other with PDT alone, and the other with or without D3 in order to establish a D3 replete state. The order of the first two treatments is randomized in case the assumption of a linear tumor clearance rate is incorrect. The statistical significance of the difference in tumor clearance after D3+PDT versus the difference in tumor clearance after PDT alone will be tested using ANOVA.

Secondary Outcome Measures

Measure:BCC: level of PpIX accumulation in lesions
Time Frame:Selected Visits (6 months total)
Safety Issue:
Description:For each patient, whether in the absence or presence of neoadjuvant Vitamin D3, assessments of PpIX accumulation in BCC lesions using fluorescence dosimetry measurements will be made at selected treatment visits.
Measure:ALA-PDT: Participant pain experienced from the technique
Time Frame:Selected Visits (6 months total)
Safety Issue:
Description:For each patient, pain scale measurements will be taken in the week following each PDT treatment. The absence/presence of pain will be recorded on a 0-to-10 visual/analog scale.
Measure:ALA-PDT: Participant erythema experienced from the technique
Time Frame:Selected Visits (6 months total)
Safety Issue:
Description:The investigators will record erythema (on a scale of 1 to 4+), from clinical exam and from the photographs, in the treated areas just prior to the second treatment in each PDT cycle.
Measure:ALA-PDT: Satisfaction with treatment outcome from the technique
Time Frame:Selected Visits (6 months total)
Safety Issue:
Description:A patient satisfaction questionnaire will be administered at the final study visit measuring the participant's satisfaction with the treatment outcome on a 5 point scale (Extremely Satisfied -> Extremely Dissatisfied)
Measure:ALA-PDT: Satisfaction with cosmetic outcome from the technique
Time Frame:Selected Visits (6 months total)
Safety Issue:
Description:A patient satisfaction questionnaire will be administered at the final study visit measuring the participant's satisfaction with the cosmetic outcome on a 5 point scale (Extremely Satisfied -> Extremely Dissatisfied)
Measure:Vitamin D levels
Time Frame:Selected Visits (6 months total)
Safety Issue:
Description:Vitamin D level (25-hydroxy-cholecalciferol) measured in serum
Measure:Allele polymorphisms in the gene of the Vitamin D receptor (VDR)
Time Frame:Selected Visits (6 months total)
Safety Issue:
Description:Identify the allele combination present at VDR locus

Details

Phase:Phase 1
Primary Purpose:Interventional
Overall Status:Not yet recruiting
Lead Sponsor:The Cleveland Clinic

Last Updated

March 23, 2018