Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

NCT02728388 | Recruiting Phase 2 FDA Drug

• 1 other identifier: PRO00026795
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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.

Conditions

NEUROFIBROMATOSIS 1

Keywords

NF1; Neurofibroma; Neurofibromatosis Type 1

Outcome Measures

Primary Outcomes (1)

• Time to disease progression

  The time it takes for 50% growth in tumor size over baseline measurements.

  3 years

Secondary Outcomes (1)

• Tumor growth rate

  3 years

Study Arms (1)

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).

Drug: aminolevulinic acid

Interventions

aminolevulinic acid DRUG

Drug: Levulan Kerastick Drug: Topical placebo Device: Omnilux Revive Procedure: Photodynamic therapy Other: Tumor growth rate measurements

Also known as: Photodynamic therapy

PDT Treatment

Eligibility Criteria

• Age: 14 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Donald Basel: lead

Study Sites (1)

Donald G Basel

Milwaukee, Wisconsin, 53226, United States

RECRUITING

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibroma

Interventions

Aminolevulinic Acid; Photochemotherapy

Condition Hierarchy (Ancestors)

Neurofibromatoses; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms

Intervention Hierarchy (Ancestors)

Levulinic Acids; Keto Acids; Carboxylic Acids; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins; Combined Modality Therapy; Therapeutics; Drug Therapy; Phototherapy

Study Officials

• Donald G Basel, MD

  Medical College of Wisconsin

  STUDY DIRECTOR

Central Study Contacts

Donald G Basel, MD

CONTACT

414-266-4921; dbasel@mcw.edu

Paula Engelking, BS, CRC

CONTACT

414-266-3289; pengelki@mcw.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: March 25, 2016
• First Posted: April 5, 2016
• Study Start: August 1, 2016
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: May 8, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)