Phase I Clinical Study of the Safety of Photodynamic Therapy (PDT) Using LS11 in Children With Plexiform Neurofibromas
Plexiform neurofibromas (PN) represent one of the most significant complications of NF1. They are a significant cause of morbidity in neurofibromatosis type 1 (NF1) by causing pain, impaired function, and disfigurement. They may become life-threatening through mechanical compression of vital organs such as the trachea, great vessels, or spinal cord, and may significantly interfere with normal function when located in the extremities or orbit. The only effective therapy for PN is total surgical excision. However, due to local infiltration of normal tissue, gross total resection is usually not feasible, and often PN are completely unresectable due to their location, size, and multiplicity. To date, other therapeutic modalities, including radiotherapy and chemotherapy, have not shown efficacy in PN.
In the present study, local photodynamic therapy will be investigated. Photodynamic therapy (PDT) utilizes a drug, called a photosensitizer or photosensitizing agent, and a particular type of light. When photosensitizers are exposed to a specific wavelength of light, they produce a form of oxygen that kills nearby cells. PDT is expected to result in treatment response with shrinkage of tumor. The main purpose of the study is to determine the maximum amount of light that can be safely used with LS11 for PDT in children with plexiform neurofibromas.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase I Clinical Study of the Safety of Photodynamic Therapy (PDT) Using Intratumoral Delivery of Non-coherent Light for Photoactivation of LS11 in Children With Plexiform Neurofibromas|
- To determine the safety and tolerability of photodynamic therapy (PDT) for the treatment of plexiform neurofibromas in children. [ Time Frame: Week 4 and 12 ] [ Designated as safety issue: Yes ]
- To evaluate the change in quality of life of treated patients. [ Time Frame: Week 4 and 12 ] [ Designated as safety issue: Yes ]
|Study Start Date:||July 2008|
|Study Completion Date:||July 2012|
|Primary Completion Date:||July 2012 (Final data collection date for primary outcome measure)|
Experimental: LS11 Administration
Treatment will be given with a standard 3+3 light dose escalation. The "Treatment Period" will be 28 days. The LS11 dose will be 30mg/m2. The initial light dose will be 50 J/cm. If criteria for dose escalation are met, then the light dose will be escalated to 100J/cm, 150J/cm and 200J/cm. Once the maximum light dose is determined, up to 6 additional patients will be treated at that level to gain further experience with this modality prior to phase II testing. In particular, at least 3 subjects <12 years of age will be enrolled at the maximum tolerable dose (MTD) to allow for further evaluation of safety in younger children. If grade 3 or 4 toxicities are noted at light dose level #1, the LS11 dose will be decreased to 20mg/m2 (2/3 of the standard dose).
LS11 will be given as a one-time IV infusion over 3-5 minutes.
Other Name: Talaporfin sodium
Show Detailed Description
Please refer to this study by its ClinicalTrials.gov identifier: NCT00716469
|United States, Pennsylvania|
|The Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Michael J Fisher, M.D.||Children's Hospital of Philadelphia|