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Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

Primary Purpose

NEUROFIBROMATOSIS 1

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
aminolevulinic acid
Sponsored by
Donald Basel
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for NEUROFIBROMATOSIS 1 focused on measuring NF1, Neurofibroma, Neurofibromatosis Type 1

Eligibility Criteria

14 Years - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Donald G BaselRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PDT Treatment

Arm Description

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).

Outcomes

Primary Outcome Measures

Time to disease progression
The time it takes for 50% growth in tumor size over baseline measurements.

Secondary Outcome Measures

Tumor growth rate
Measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.

Full Information

First Posted
March 25, 2016
Last Updated
July 17, 2023
Sponsor
Donald Basel
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1. Study Identification

Unique Protocol Identification Number
NCT02728388
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 2016 (undefined)
Primary Completion Date
December 2025 (Anticipated)
Study Completion Date
December 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Donald Basel

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
NEUROFIBROMATOSIS 1
Keywords
NF1, Neurofibroma, Neurofibromatosis Type 1

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
PDT Treatment
Arm Type
Experimental
Arm Description
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).
Intervention Type
Drug
Intervention Name(s)
aminolevulinic acid
Other Intervention Name(s)
Photodynamic therapy
Intervention Description
Drug: Levulan Kerastick Drug: Topical placebo Device: Omnilux Revive Procedure: Photodynamic therapy Other: Tumor growth rate measurements
Primary Outcome Measure Information:
Title
Time to disease progression
Description
The time it takes for 50% growth in tumor size over baseline measurements.
Time Frame
3 years
Secondary Outcome Measure Information:
Title
Tumor growth rate
Description
Measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.
Time Frame
3 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
14 Years
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patient is 14 years or older. Diagnosed NF1, determined by American Academy of Neurology Guidelines (see Diagnosis section). Tumor Location: cutaneous, trunk, or limbs only. Tumor Type: superficial dermal neurofibromas ≤4mm deep. Patient has provided written informed consent. Patient is willing to and can comply with study follow-up requirements. Absence of any other malignancy. Exclusion Criteria: Life expectancy less than 3 years. Pregnancy. Cutaneous photosensitivity to the wavelengths used to active 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Donald G Basel, MD
Phone
414-266-4921
Email
dbasel@mcw.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Paula Engelking, RN, BSN, MBA
Phone
414-266-3289
Email
pengelki@mcw.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Donald G Basel, MD
Organizational Affiliation
Medical College of Wisconsin
Official's Role
Study Director
Facility Information:
Facility Name
Donald G Basel
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Donald G Basel, MD
Phone
414-266-4921
Email
dbasel@mcw.edu
First Name & Middle Initial & Last Name & Degree
Paula Engelking
Phone
4142663289
Email
pengelki@mcw.edu

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

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