Description:
The investigators wish to determine the time to disease progression for benign neurofibromas
treated with Levulan Kerastick topical photosensitizer and red light photodynamic therapy
(PDT) in patients with neurofibromatosis type 1 (NF1).
The investigators also wish to measure tumor size for control and treatment tumors in order
to gain insights into tumor growth rates.
Title
- Brief Title: Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II
- Official Title: Topical Photodynamic Therapy (PDT) With Levulan® Kerastick® for Benign Dermal Neurofibromas Phase II
Clinical Trial IDs
- ORG STUDY ID:
PRO00026795
- NCT ID:
NCT02728388
Conditions
Interventions
| Drug | Synonyms | Arms |
|---|
| aminolevulinic acid | Photodynamic therapy | PDT Treatment |
Purpose
The investigators wish to determine the time to disease progression for benign neurofibromas
treated with Levulan Kerastick topical photosensitizer and red light photodynamic therapy
(PDT) in patients with neurofibromatosis type 1 (NF1).
The investigators also wish to measure tumor size for control and treatment tumors in order
to gain insights into tumor growth rates.
Detailed Description
Neurofibromas contain a large quantity of fibrous matter, and it is not anticipated that
significant reductions in tumor size can be achieved in large, long established, tumors.
Cutaneous neurofibromas, which do not usually become apparent until puberty, continue to
increase in size and number throughout adulthood. The psychosocial burden of these
disfiguring tumors is significant, and the targeted age group (14-30) is at a life period
associated with an acceleration in tumor growth. It is for these reasons that the
investigators hope to affect the growth rate of less established tumors, in order to prevent
or lessen this burden as the patient progresses further into adulthood.
Therefore, the investigators wish to determine the time to disease progression (defined as
50% growth in size over baseline) for benign neurofibromas treated with PDT in patients with
neurofibromatosis type 1 (NF1) in subjects aged 14-30.
The treatment will consist of choosing several neurofibromas of similar size, and applying a
topical drug called Levulan, or just the topical application alone (a placebo). Within 24
hours of drug treatment, the neurofibromas will be illuminated with red light (both Levulan
and placebo). The Levulan is what is known as a photosensitizer, and will be activated by the
red light to potentially kill some of the tumor cells. Approximately every 6 months after,
for three years, the tumors will be measured by digital photography and ultrasound to see if
they are growing more slowly than the ones with the placebo application alone.
Trial Arms
| Name | Type | Description | Interventions |
|---|
| PDT Treatment | Experimental | Each subject will have either placebo or Levulan Kerastick topical application applied to matched sets of neurofibromas. Each subject will have both sets, in order to serve as his/her own control subject.
16 to 24 hours post study drug treatment, both sets of neurofibromas, Levulan and placebo treated, will be irradiated with red light (630 nm) from an Omnilux Revive light device at 100 mW/cm2 for 1000 seconds (16.7 minutes). | |
Eligibility Criteria
Inclusion Criteria:
1. Patient is 14 years or older.
2. Diagnosed NF1, determined by American Academy of Neurology Guidelines (see Diagnosis
section).
3. Tumor Location: cutaneous, trunk, or limbs only.
4. Tumor Type: superficial dermal neurofibromas ≤4mm deep.
5. Patient has provided written informed consent.
6. Patient is willing to and can comply with study follow-up requirements.
7. Absence of any other malignancy.
Exclusion Criteria:
1. Life expectancy less than 3 years.
2. Pregnancy.
3. Cutaneous photosensitivity to the wavelengths used to active PDT.
4. A diagnosis of porphyria.
5. Allergy to aminolevulinic acid or any of the topical solution vehicle components.
6. Previous chemotherapy within 6 weeks of proposed PDT.
7. Other concurrent tumor therapy.
| Maximum Eligible Age: | 30 Years |
| Minimum Eligible Age: | 14 Years |
| Eligible Gender: | All |
| Healthy Volunteers: | No |
Primary Outcome Measures
| Measure: | Time to disease progression |
| Time Frame: | 3 years |
| Safety Issue: | |
| Description: | The time it takes for 50% growth in tumor size over baseline measurements. |
Secondary Outcome Measures
| Measure: | Tumor growth rate |
| Time Frame: | 3 years |
| Safety Issue: | |
| Description: | Measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates. |
Details
| Phase: | Phase 2 |
| Primary Purpose: | Interventional |
| Overall Status: | Recruiting |
| Lead Sponsor: | Harry T Whelan, MD |
Trial Keywords
- NF1
- Neurofibroma
- Neurofibromatosis Type 1
Last Updated
November 27, 2020
