Anal DysplasiaPhase 1NCT02698293
PDT Plus Vitamin D3 for Anal Dysplasia
A Phase 1 study testing Gliolan, Vitamin D3 cholecalciferol) and Photodynamic Therpay in people with Anal Dysplasia and Carcinoma in Situ, run by Abramson Cancer Center at Penn Medicine at 1 site in United States.
Trial summary
This is a phase I dose escalation study of photodynamic therapy (PDT) for the treatment of patients with pre-malignant tumors and superficial microinvasive disease of the anal canal and/or perianal skin. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Anal Dysplasia, Carcinoma in Situ
- What is being tested?
- Gliolan, Vitamin D3 cholecalciferol) and Photodynamic Therpay
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Dec 2019to 3 Aug 2022
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A histological or cytological diagnosis of high-grade dysplasia or carcinoma in-situ, within past 4 months.
- Premalignant lesions containing focal microinvasion are eligible when:
- Surgery is not clinically mandated.
- Subjects with medical conditions precluding surgery.
- Subjects whose lesions cannot be completely resected based on size or location, or where significant functional morbidity would be anticipated with further surgery.
- Patients refuse surgery.
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- The justification for inclusion of patients with microinvasive disease is based reports demonstrating the ability of photodynamic therapy to successfully treat both dysplasia and T1 squamous cell carcinoma of the anal canal
- HPV positive by Cobas or other cytological assays within past 4 months
- Documented HIV positivity
- Patients must be on highly active anti-retroviral therapy with a CD4 count >200 for the past 12 months
- Viral load <200 for 12 months for the past 12 months
- ECOG performance status of 0-1.
- 18 years of age or older.
- Study subjects capable of providing informed consent.
- Women of childbearing potential and men must agree to use a medically accepted method of birth control from the time they sign consent and until one month after receiving ALA
Who cannot join
- Study subjects in whom the lesion has invasive squamous cell carcinoma of the anal cavity which is clinically appreciable.
- Clinically occult microinvasive squamous cell carcinoma of the anal cavity which is not focal.
- Study subjects who are pregnant or lactating .
- Study subjects who have a platelet count of less than 100,000/cubic mm.
- Study subjects with elevated aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase, or total bilirubin levels >2X normal or a history of chronic liver disease or cirrhosis of the liver.
- Significant cardiovascular history that would put the study subject at risk from hypotension that may occur with ALA
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- Study subjects with porphyria or hypersensitivity to porphyrins.
- Administration of the following compounds: tetracyclines, sulfonamides, fluoroquinolones within 48 hours, or hypericin extracts within a week prior to light administration.
- Study subjects with abnormal baseline creatinine level or diagnosed kidney disease.
- Treatment with 5-FU, Imiquimod, trichloroacetic acid or ablative therapy within the previous month.
- Study subjects who have a medical history of immune suppression. This will include patients with a past transplantation requiring ongoing immunosuppressive medications.
- A history of sarcoidosis, hyperphosphatemia, or known kidney stones
Trial locations and contacts
1 site in 1 country.
United States1 site
Abramson Cancer Center of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Phase I Study of Photodynamic Therapy (PDT) Plus Vitamin D3 for High-grade Anal Dysplasia and Microinvasive Anal Cancer
This is a phase I dose escalation study of photodynamic therapy (PDT) for the treatment of patients with pre-malignant tumors and superficial microinvasive disease of the anal canal and/or perianal skin. All subjects (a maximum of 12) will be given the photosensitizer ALA orally followed by the administration of red light (629-635 nm) to the tumor from a laser. The dose of ALA will be 40 mg/kg administered approximately 4-6 hours before light administration. There will be two levels of light dose: 50 and 100 J/cm2, 3-6 patients in each. Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT. Patients will be observed for 30 days for the development of DLT. Patients will be followed up for 24 months for additional toxicity and efficacy data collection.
Read the detailed description
This is a phase I dose escalation study of photodynamic therapy (PDT) for the treatment of patients with pre-malignant tumors and superficial microinvasive disease of the anal canal and/or perianal skin. All subjects (a maximum of 12) will be given the photosensitizer ALA orally followed by the administration of red light (629-635 nm) to the tumor from a laser. The dose of ALA will be 40 mg/kg administered approximately 4-6 hours before light administration. There will be two levels of light dose: 50 and 100 J/cm2, 3-6 patients in each. Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT. Patients will be observed for 30 days for the development of DLT. Patients will be followed up for 24 months for additional toxicity and efficacy data collection.
- Conditions
- Anal Dysplasia, Carcinoma in Situ
- Being tested
- Gliolan, Vitamin D3 cholecalciferol), Photodynamic Therpay
- Medical subject headings
- Carcinoma in Situ
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GliolanDrug
Also called ALA
orally, 40 mg/kg 4-6 hours prior to light application
Vitamin D3 cholecalciferol)Drug
Also called Natures Made Vitamin D3
Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT.
Photodynamic TherpayOther
Also called PDT
Approximately 4-6 hours after photosensitizer administration, activating light will be applied.
The target mucosal lesion will be identified based on the initial clinical evaluation. Treatment light will be generated using a Modulight ML7710-630-6K diode laser, This laser produces up to 6W total power, with a peak wavelength of 630nm and 90% of laser power within 630-633 nm.
The light fluence (doses) will be 50 and 100 Joules per square centimeter.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 5-aminolevulinic acid hydrochloride (Gliolan), orally, 40 mg/kg administered approximately 4-6 hours before light administration. There will be two levels of light dose: 50 and 100 J/cm2. Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT.
Total duration of drug product administration (including any open-label lead-in, if applicable).
Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT.
None This is a phase I dose escalation study, with two levels of light dose. The design is cohorts of 3s. Light at 50 Joules and Light at 100 joules There are pre-defined DLTs. Receives: Drug: Gliolan; Drug: Vitamin D3 cholecalciferol); Other: Photodynamic Therpay |
| Cohort 2 | Experimental | 5-aminolevulinic acid hydrochloride (Gliolan), orally, 40 mg/kg administered approximately 4-6 hours before light administration. There will be two levels of light dose: 50 and 100 J/cm2. Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT Total duration of drug product administration (including any open-label lead-in, if applicable).
Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT.
None This is a phase I dose escalation study, with two levels of light dose. The design is cohorts of 3s. Light at 50 Joules and Light at 100 joules There are pre-defined DLTs. Receives: Drug: Gliolan; Drug: Vitamin D3 cholecalciferol); Other: Photodynamic Therpay |
| Cohort 3 | Experimental | 5-aminolevulinic acid hydrochloride (Gliolan), orally, 40 mg/kg administered approximately 4-6 hours before light administration. There will be two levels of light dose: 50 and 100 J/cm2. Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT Total duration of drug product administration (including any open-label lead-in, if applicable).
Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT.
None This is a phase I dose escalation study, with two levels of light dose. The design is cohorts of 3s. Light at 50 Joules and Light at 100 joules There are pre-defined DLTs. Receives: Drug: Gliolan; Drug: Vitamin D3 cholecalciferol); Other: Photodynamic Therpay |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events
DLTs have been defined
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2016 (estimated)
- Study start1 Dec 2019
- Primary completion3 Aug 2022
- Study completion3 Aug 2022
Why it stopped: Covid-19
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anal DysplasiaStudies of the same condition, open and under way.
- More trials run by Abramson Cancer Center at Penn MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Abramson Cancer Center at Penn Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Keith Cengel, MD, PhD, Principal investigator, Abramson Cancer Center at Penn Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02698293
- Sponsor's protocol ID
- UPCC 08216
- Status last verified
- Aug 2026
- First submitted
- 1 Mar 2016
- Last updated
- 4 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Photodynamic Therapy (PDT) Plus Vitamin D3 for High-grade Anal Dysplasia and Microinvasive Anal Cancer
- ClinicalTrials.gov ID
- NCT02698293
- Acronym / sponsor ID
- UPCC 08216
- Conditions
- Anal Dysplasia, Carcinoma in Situ
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Covid-19
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Abramson Cancer Center at Penn MedicineUniversity, hospital or other
- Study start
- 1 Dec 2019 (actual)
- Primary completion
- 3 Aug 2022 (actual)
- Study completion
- 3 Aug 2022 (actual)
- First posted
- 3 Mar 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase I dose escalation study of photodynamic therapy (PDT) for the treatment of patients with pre-malignant tumors and superficial microinvasive disease of the anal canal and/or perianal skin. All subjects (a maximum of 12) will be given the photosensitizer ALA orally followed by the administration of red light (629-635 nm) to the tumor from a laser. The dose of ALA will be 40 mg/kg administered approximately 4-6 hours before light administration. There will be two levels of light dose: 50 and 100 J/cm2, 3-6 patients in each. Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT. Patients will be observed for 30 days for the development of DLT. Patients will be followed up for 24 months for additional toxicity and efficacy data collection.
Detailed description
This is a phase I dose escalation study of photodynamic therapy (PDT) for the treatment of patients with pre-malignant tumors and superficial microinvasive disease of the anal canal and/or perianal skin. All subjects (a maximum of 12) will be given the photosensitizer ALA orally followed by the administration of red light (629-635 nm) to the tumor from a laser. The dose of ALA will be 40 mg/kg administered approximately 4-6 hours before light administration. There will be two levels of light dose: 50 and 100 J/cm2, 3-6 patients in each. Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT. Patients will be observed for 30 days for the development of DLT. Patients will be followed up for 24 months for additional toxicity and efficacy data collection.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: Gliolan, Drug: Vitamin D3 cholecalciferol), Other: Photodynamic Therpay5-aminolevulinic acid hydrochloride (Gliolan), orally, 40 mg/kg administered approximately 4-6 hours before light administration. There will be two levels of light dose: 50 and 100 J/cm2. Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT. Total duration of drug product administration (including any open-label lead-in, if applicable). Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT. None This is a phase I dose escalation study, with two levels of light dose. The design is cohorts of 3s. Light at 50 Joules and Light at 100 joules There are pre-defined DLTs. |
| Cohort 2 | Experimental | Drug: Gliolan, Drug: Vitamin D3 cholecalciferol), Other: Photodynamic Therpay5-aminolevulinic acid hydrochloride (Gliolan), orally, 40 mg/kg administered approximately 4-6 hours before light administration. There will be two levels of light dose: 50 and 100 J/cm2. Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT Total duration of drug product administration (including any open-label lead-in, if applicable). Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT. None This is a phase I dose escalation study, with two levels of light dose. The design is cohorts of 3s. Light at 50 Joules and Light at 100 joules There are pre-defined DLTs. |
| Cohort 3 | Experimental | Drug: Gliolan, Drug: Vitamin D3 cholecalciferol), Other: Photodynamic Therpay5-aminolevulinic acid hydrochloride (Gliolan), orally, 40 mg/kg administered approximately 4-6 hours before light administration. There will be two levels of light dose: 50 and 100 J/cm2. Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT Total duration of drug product administration (including any open-label lead-in, if applicable). Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT. None This is a phase I dose escalation study, with two levels of light dose. The design is cohorts of 3s. Light at 50 Joules and Light at 100 joules There are pre-defined DLTs. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- A histological or cytological diagnosis of high-grade dysplasia or carcinoma in-situ, within past 4 months.
- Premalignant lesions containing focal microinvasion are eligible when:
- The justification for inclusion of patients with microinvasive disease is based reports demonstrating the ability of photodynamic therapy to successfully treat both dysplasia and T1 squamous cell carcinoma of the anal canal
- HPV positive by Cobas or other cytological assays within past 4 months
- Documented HIV positivity
Key exclusion criteria (12 listed)
- Study subjects in whom the lesion has invasive squamous cell carcinoma of the anal cavity which is clinically appreciable.
- Clinically occult microinvasive squamous cell carcinoma of the anal cavity which is not focal.
- Study subjects who are pregnant or lactating .
- Study subjects who have a platelet count of less than 100,000/cubic mm.
- Study subjects with elevated aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase, or total bilirubin levels >2X normal or a history of chronic liver disease or cirrhosis of the liver.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GliolanAlso: ALAorally, 40 mg/kg 4-6 hours prior to light application | Drug | Cohort 1, Cohort 2, Cohort 3 |
| Vitamin D3 cholecalciferol)Also: Natures Made Vitamin D3Vitamin D3 (cholecalciferol) supplementation (10,000 IU daily) will be provided from 3 days prior through 14 weeks after light delivery for PDT. | Drug | Cohort 1, Cohort 2, Cohort 3 |
| Photodynamic TherpayAlso: PDTApproximately 4-6 hours after photosensitizer administration, activating light will be applied. The target mucosal lesion will be identified based on the initial clinical evaluation. Treatment light will be generated using a Modulight ML7710-630-6K diode laser, This laser produces up to 6W total power, with a peak wavelength of 630nm and 90% of laser power within 630-633 nm. The light fluence (doses) will be 50 and 100 Joules per square centimeter. | Other | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
Number of adverse events
Time frame: 18 months
DLTs have been defined
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Keith Cengel, MD, PhD principal investigator
Abramson Cancer Center at Penn Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Gliolan, Vitamin D3 cholecalciferol) and Photodynamic Therpay in people with Anal Dysplasia and Carcinoma in Situ, run by Abramson Cancer Center at Penn Medicine at 1 site 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 15 inclusion and 12 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: withdrawn, as of the sponsor's last update on 4 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 was withdrawn before enrolling participants, so there are no results.
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?
Number of adverse events (time frame: 18 months). 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 4 Aug 2026; AzTrials last refreshed its copy on 6 Nov 2026.