Phase 1
N=1
Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)
Neurofibromatoses
Bottom Line
View on ClinicalTrials.gov: NCT01682811 ↗Enrolled (actual)
1
Serious AEs
0.0%
Results posted
Jun 2021
Primary outcome: Primary: Part 1: Photosensitizer Uptake and Conversion to Protoporphyrin IX — 304 Densitometry units/micrometer squared — p=0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Part 1 Levulan injection (Drug); Part 1 Levulan surface application (Drug); Part 1 Levulan surface application twice (Drug); Part 1 Levulan surface application twice with microneedling (Drug); Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 1 (Drug); Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 2 (Drug); Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 3 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Harry T Whelan, MD
- Primary completion
- Oct 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part 1: Photosensitizer Uptake and Conversion to Protoporphyrin IX |
304 | 0.0001 sig |
| PRIMARY Part 2: Maximum Tolerated Dose (MTD) of 633 nm Red Light |
100 | — |
| SECONDARY Part 1: Optimal Occlusion Time |
NA; NA; NA; NA; NA; NA | — |
| SECONDARY Part 2: Efficacy - Lesion Area Growth Rate |
0.212; 0.081 | 0.24 |
| SECONDARY Part 2: Cosmetic Improvement |
— | — |
| SECONDARY Part 2: Pain Reduction |
— | — |
Summary
GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) tumors in the skin.
SPECIFIC OBJECTIVE This is a light dose escalation pilot study to determine the safety and efficacy of PDT using 5-aminolevulinic acid (ALA) and 633 nm light in the treatment of benign dermal neurofibromas.
Specifically, the primary goal of the current study is to determine the maximum tolerable light doses that can be administered to subjects undergoing topical photoillumination photodynamic therapy with standard application of Levulan Kerastick (ALA) for Topical Solution.
Eligibility Criteria
Inclusion Criteria
Subjects with NF1 will be selected for photodynamic therapy on the following criteria.
- Age: 18 years or older.
- NF1 will be diagnosed by American Academy of Neurology guidelines.
- Location of tumor: cutaneous, trunk or limbs only.
- Tumor type: superficial dermal neurofibromas, less than or equal to 4 mm deep.
- Growth confirmation: direct measurement for the dermal neurofibromas, ruler and photo-volumetric method.
- Informed consent of subject.
- Absence of any other malignancy.
- Only failures to meet criteria 1-6 due to the primary disease will be disqualifying
Exclusion Criteria
Subjects will be excluded from participation in the study on the basis of the following:
- Life expectancy less than 1 year.
- Pregnancy.
- Inability to consent.
- Cutaneous photosensitivity to the wavelengths used to activate PDT.
- A diagnosis of porphyria.
- Allergy to aminolevulinic acid or any of the Topical Solution Vehicle components.
- Previous chemotherapy within 6 weeks of proposed PDT.
- Other concurrent tumor therapy. -
Data sourced from ClinicalTrials.gov (NCT01682811). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.