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Vitamin D and Photodynamic Therapy for Treatment of BCC in BCNS

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ClinicalTrials.gov Identifier: NCT03483441
Recruitment Status : Recruiting
First Posted : March 30, 2018
Last Update Posted : October 10, 2018
Sponsor:
Collaborators:
University of Arizona
Harvard Medical School
Information provided by (Responsible Party):
Edward Maytin, MD, PhD, The Cleveland Clinic

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Basal Cell Nevus Syndrome Basal Cell Carcinoma Other: Placebo pill Dietary Supplement: Vitamin D Drug: PDT Phase 1

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
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.
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: Vitamin D as a Nutritional Neoadjuvant During Photodynamic Therapy of Basal Cell Carcinoma in Basal Cell Nevus Syndrome
Actual Study Start Date : October 1, 2018
Estimated Primary Completion Date : May 1, 2022
Estimated Study Completion Date : May 1, 2023


Arm Intervention/treatment
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 Name: 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 Name: photodynamic therapy




Primary Outcome Measures :
  1. BCC: Rate of Tumor Clearance [ Time Frame: Selected Visits (6 months total) ]
    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 :
  1. BCC: level of PpIX accumulation in lesions [ Time Frame: Selected Visits (6 months total) ]
    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.

  2. ALA-PDT: Participant pain experienced from the technique [ Time Frame: Selected Visits (6 months total) ]
    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.

  3. ALA-PDT: Participant erythema experienced from the technique [ Time Frame: Selected Visits (6 months total) ]
    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.

  4. ALA-PDT: Satisfaction with treatment outcome from the technique [ Time Frame: Selected Visits (6 months total) ]
    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)

  5. ALA-PDT: Satisfaction with cosmetic outcome from the technique [ Time Frame: Selected Visits (6 months total) ]
    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)

  6. Vitamin D levels [ Time Frame: Selected Visits (6 months total) ]
    Vitamin D level (25-hydroxy-cholecalciferol) measured in serum

  7. Allele polymorphisms in the gene of the Vitamin D receptor (VDR) [ Time Frame: Selected Visits (6 months total) ]
    Identify the allele combination present at VDR locus



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03483441


Contacts
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Contact: Jeff Negrey, MA 216-636-5504 negreyj2@ccf.org
Contact: Edward Maytin, MD 216-444-5139 maytine@ccf.org

Locations
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United States, Arizona
University of Arizona College of Medicine Not yet recruiting
Phoenix, Arizona, United States, 85004
Contact: Nathalie Zeitouni, MD    602-406-8222    Nathalie.Zeitouni@dignityhealth.org   
Sub-Investigator: Nathalie Zeitouni, MD         
United States, Ohio
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
Contact: Jeff Negrey, MA    216-636-5504    negreyj2@ccf.org   
Principal Investigator: Edward Maytin, MD         
Sub-Investigator: Christine Warren, MD         
Sponsors and Collaborators
The Cleveland Clinic
University of Arizona
Harvard Medical School

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Responsible Party: Edward Maytin, MD, PhD, Staff Physician, The Cleveland Clinic
ClinicalTrials.gov Identifier: NCT03483441     History of Changes
Other Study ID Numbers: 18-310
First Posted: March 30, 2018    Key Record Dates
Last Update Posted: October 10, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: No plan to share data, needs HIPPA clearance and specific IRB approval, we did not plan on original data being shared

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Carcinoma
Carcinoma, Basal Cell
Basal Cell Nevus Syndrome
Syndrome
Disease
Pathologic Processes
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Basal Cell
Odontogenic Cysts
Jaw Cysts
Bone Cysts
Cysts
Neoplastic Syndromes, Hereditary
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Jaw Diseases
Stomatognathic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Vitamin D
Ergocalciferols
Vitamins
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs