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.

Conditions

Eligibility

Eligible Ages
All ages
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 1
Study Type
Interventional
Allocation
Randomized
Intervention Model
Crossover Assignment
Intervention Model Description
Each patient serves as his/her own control. Two PDT sessions are given, constituting Arm 1 and Arm 2. Arm 1 is placebo pill, then PDT. Arm 2 is Vit D pill, then PDT. In any given patient, the order of Arm 1 vs. Arm 2 will be randomized.
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Placebo Comparator
Placebo pill prior to PDT
BCC tumors will be treated with ALA-PDT, without any active pretreatment
  • Other: Placebo pill
    An inert placebo pill will be taken for several days prior to PDT
  • Drug: PDT
    Levulan (5-aminolevulinic acid) is applied to tumors for 4 hr, then illuminated with blue light
    Other names:
    • photodynamic therapy
Active Comparator
Vitamin D pill prior to PDT
Patients will take oral Vit D supplements (10,000 IU/day) immediately prior to PDT of their BCC tumors.
  • Dietary Supplement: Vitamin D
    10,000 units of cholecalciferol will be taken for several days prior to PDT
  • Drug: PDT
    Levulan (5-aminolevulinic acid) is applied to tumors for 4 hr, then illuminated with blue light
    Other names:
    • photodynamic therapy

Recruiting Locations

Medical Dermatology Specilists
Phoenix, Arizona 85006
Contact:
Nathalie Zeitouni, MD
602-354-5770
nathaliezeitouni@email.arizona.edu

More Details

Status
Recruiting
Sponsor
The Cleveland Clinic

Study Contact

Jeff Negrey, MA
216-636-5504
negreyj2@ccf.org

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.