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Basal Cell CarcinomaPhase 1NCT03467789

Vitamin D as a Nutritional Neoadjuvant During Photodynamic Therapy of Basal Cell Carcinoma

A Phase 1 study testing Dietary Vitamin D3 pre-treatment, Photodynamic therapy and Serum Maintenance Vitamin D3 in people with Basal Cell Carcinoma and Basal Cell Nevus Syndrome, run by Case Comprehensive Cancer Center at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is to study 50 patients with multiple Basal Cell Carcinoma (BCC) who will be receiving Photodynamic Therapy (PDT) as treatment for their tumors. This study wants to establish the optimal conditions for treating BCC tumors with PDT. Read more about this study

Who is it for?
Any age
What is being tested?
Dietary Vitamin D3 pre-treatment, Photodynamic therapy and Serum Maintenance Vitamin D3
Could I receive a placebo?
No, everyone receives an active treatment
How many people
37 participants
Where
2 sitesUnited States
Study dates
1 Oct 2018to 6 Jan 2023
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Diagnosis of Basal Cell Nevus Syndrome (BCNS) as defined in the Consensus Statement from the first International colloquium on BCNS.
  • Major Criteria are:
  • (1) BCC prior to age 20 years, or excessive number of BCCs out of proportion to prior sun exposure and skin type;
  • (2) keratocyst of the jaw prior to age 20;
  • (3) palmar or plantar pitting;
  • (4) lamellar calcification of the falx cerebri;
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  • (5) medulloblastoma;
  • (6) first degree relative with BCNS;
  • (7) Patched-1 (PTCH1) gene mutation.
  • Minor Criteria are:
  • (1) rib anomalies, or other specific skeletal malformations including kyphoscoliosis and short 4th metacarpals;
  • (2) macrocephaly;
  • (3) cleft/lip or palate;
  • (4) fibroma of the heart or ovary;
  • (5) ocular abnormalities;
  • For diagnosis of BCNS, the participant must have either 2 major criteria, one major and two minor criteria.
  • 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

Who cannot join

  • Pregnant or nursing.
  • At risk for hypercalcemia (renal disease, sarcoidosis, etc.)
  • Taking vismodegib or a hedgehog pathway inhibitor; must stop at least 3 months prior to visit 1.
  • Taking any topical treatment on their BCC tumors; must stop at least 1 month prior.
  • Taking Vitamin D or multivitamin supplements; must stop at least 1 month prior.
  • Currently undergoing treatment for other cancers with medical or radiation therapy.
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  • Participants with a known hypersensitivity to 5-aminolevulinic acid or any component of the study material.
  • Participants with history of a photosensitivity disease, such as porphyria cutanea tarda.
  • Currently participating in another clinical trial.

Trial locations and contacts

2 sites in 1 country.

United States2 sites
  • Cleveland Clinic Taussig Cancer institute, Case Comprehensive Cancer Center

    Cleveland, Ohio, 44195

  • Medical Dermatology Specialists Phoenix

    Phoenix, Arizona, 85006

Other trials in United States

About this study

Official title: Vitamin D as a Nutritional Neoadjuvant During Photodynamic Therapy of Basal Cell Carcinoma in Basal Cell Nevus Syndrome

The purpose of this study is to study 50 patients with multiple Basal Cell Carcinoma (BCC) who will be receiving Photodynamic Therapy (PDT) as treatment for their tumors. This study wants to establish the optimal conditions for treating BCC tumors with PDT. Previous research suggests that taking Vitamin D prior to the start of PDT could help improve the effectiveness of the treatment in eliminating the BCC. Overall, this study will help establish oral Vitamin D3/PDT as a new combination therapy for skin cancer (BCC).

Photodynamic Therapy (PDT) is an investigational (experimental) technique that works by combining a photosensitizing topical agent and an intense light source to kill tumor cells. PDT is currently approved for the treatment of BCC in Europe, Canada, and Australia. However, it is experimental in the United States because it is not approved by the Food and Drug Administration (FDA).

Read the detailed description

The overall hypothesis is that PDT could provide exceptional benefit in patients with Basal Cell Nevus Syndrome (BCNS) and multiple BCC tumors because PDT is nonmutagenic, nonscarring, and can be safely repeated many times. The specific study hypothesis is that Vitamin D might be useful as a neoadjuvant to improve tumor responses to PDT. In preclinical studies, the investigators showed that epithelial tumors are more responsive to aminolevulinic acid (ALA)-based PDT when "primed" by pre-exposure to the dietary form of Vitamin D (cholecalciferol, D3). This study will test the hypothesis that oral D3 supplements, administered over a relatively short time, can boost the effectiveness of PDT for cutaneous (BCC) in this patient population. Patients with BCNS and multiple BCC, or normal patients with at least 3 BCC tumors, will be enrolled. They will receive three PDT treatments, at two-month intervals, over a 6 month period.

Primary Objective

  • To determine tumor clinical clearance rates after neoadjuvant D3/PDT, and after PDT alone. To accomplish this, the first two PDT treatments in each study patient will be randomized, i.e. one PDT session will be performed after D3 pretreatment, the other without any pretreatment.

Secondary Objective(s)

  • To assess the level of PpIX accumulation in BCC lesions at various treatment visits, in the absence or presence of neoadjuvant D3. (Fluorescence dosimetry measurements)
  • To assess tolerability of the technique. (Pain scale measurements)
  • To assess patient satisfaction with the technique. (Cosmetic result, and questionnaire)
  • To assess D3 serum levels (in serum) and VDR status (in leukocyte DNA), and correlate these results to clinical outcomes.

Study Design:

In this clinical study, each patient will serve as his or her own control with respect to BCC tumor responsiveness to neoadjuvant D3 supplementation. The first two PDT treatments will be randomized. Thus, patients in Group A will take D3 pills prior to the first PDT treatment, and placebo pills prior to the second PDT treatment. For patients in Group B, the order is reversed. Total amounts of D3 supplementation given will be adjusted, based upon serum 25-hydroxy-D3 levels found at baseline. Patients with VD deficiency will take 14 days of neoadjuvant D3, vs. only 5 days if the initial VD level is normal.

Being tested
Dietary Vitamin D3 pre-treatment, Photodynamic therapy, Serum Maintenance Vitamin D3
Keywords
Vitamin D, Photodynamic Therapy
Medical subject headings
Carcinoma, Basal Cell, Basal Cell Nevus Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Dietary Vitamin D3 pre-treatmentDrug

    The daily dose of D3 will always be 10,000 IU/day. Total amounts of D3 supplementation given will be adjusted, based upon serum 25-hydroxy-D3 levels found at baseline. Duration of pretreatment will be 14 days if the D3 level is < 31 ng/mL, and 5 days if the D3 level is > 31 ng/mL

  • Photodynamic therapyRadiation

    Also called PDT

    Photodynamic Therapy (PDT) is an experimental technique that works by combining a photosensitizing topical agent and an intense light source to kill tumor cells.

  • Serum Maintenance Vitamin D3Drug

    2,000 IU/d for adults, 1,000 IU/d for children taken after third visit.

Study groups in NCT03467789
Study groupRoleWhat they receive
Group A: D3 prior to first PDTExperimentalIn both groups, one PDT session is preceded by neoadjuvant PDT while the other PDT session has no pretreatment. Group A will take dietary D3 pills prior to the first PDT treatment (day 1), and placebo pills prior to the second PDT treatment (at 2 months). Both Group A and Group B will take continuous serum D3 prior to the third PDT visit (Month 4). A final assessment of lesion clearance will be performed at 6 months
Receives: Drug: Dietary Vitamin D3 pre-treatment; Radiation: Photodynamic therapy; Drug: Serum Maintenance Vitamin D3
Group B: D3 prior to second PDT visitExperimentalIn both groups, one PDT session is preceded by neoadjuvant PDT while the other PDT session has no pretreatment. Group B will receive placebo prior to their first PDT visit (day 1), and Vitamin D3 prior to their second PDT visit (at 2 months). Both Group A and Group B will take continuous D3 prior to the third PDT visit (Month 4). A final assessment of lesion clearance will be performed at 6 months
Receives: Drug: Dietary Vitamin D3 pre-treatment; Radiation: Photodynamic therapy; Drug: Serum Maintenance Vitamin D3

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. BCC: Rate of tumor clearance

    Change in tumor diameter per month. For each participant, 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 Delta-T after D3+PDT versus the difference in Delta-T after PDT alone will be tested using ANOVA.

    Measured: Up to 6 months after first treatment visit

Secondary outcomes (7)
  1. BCC: Level of protoporphyrin IX (PpIX) accumulation in BCC lesions

    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.

    Measured: Up to 6 months after first treatment visit

  2. Serum 25-hydroxy-vitamin D3 (25OH-D3) levels

    Using a 10mL blood sample, a 25OH-D3 assay will be used to determine D3 levels in the blood

    Measured: Up to 6 months after first treatment visit

  3. Number of patients with active form of leukocyte DNA vitamin D Receptor (VDR)

    Study team will collect patients' leukocyte DNA and examine the VDR gene sequences directly. Patients with the active VDR allele are postulated to have better PDT outcomes

    Measured: Up to 6 months after first treatment visit

  4. Pain scale measurement

    average pain scale measurements to assess tolerability of the technique to be taken in the week following each PDT treatment (3 total). Ranging from 0 to 10 where 0 indicates no pain and 10 indicates the worst pain possible

    Measured: Up to 6 months after first treatment visit

  5. Erythema score

    A measure of Patient Satisfaction Score. Score from photographs on a scale from 1-4 with higher scores indicating more erythema

    Measured: Up to 6 months after first treatment visit

  6. Satisfaction with treatment outcome from the technique

    A participant 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 to Extremely Dissatisfied)

    Measured: Up to 6 months after first treatment visit

  7. Satisfaction with cosmetic outcome from the technique

    A participant 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 to Extremely Dissatisfied)

    Measured: Up to 6 months after first treatment visit

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Mar 2018
  2. Study start1 Oct 2018
  3. Primary completion6 Jan 2023
  4. Study completion6 Jan 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
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 blind
Who is masked
Participant, Care provider, Investigator
Enrolment
37 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03467789
Sponsor's protocol ID
CASE5617
Status last verified
Oct 2024
First submitted
12 Mar 2018
Last updated
17 Oct 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Dietary Vitamin D3 pre-treatment, Photodynamic therapy and Serum Maintenance Vitamin D3 in people with Basal Cell Carcinoma and Basal Cell Nevus Syndrome, run by Case Comprehensive Cancer Center at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

BCC: Rate of tumor clearance (time frame: Up to 6 months after first treatment visit). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Oct 2024.

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