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Sponsors and Collaborators: |
Children's Hospital of Philadelphia Department of Defense |
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Information provided by: | Children's Hospital of Philadelphia |
ClinicalTrials.gov Identifier: | NCT00716469 |
Plexiform neurofibromas (PN) represent one of the most significant complications of NF1. They are a significant cause of morbidity in 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.
Condition | Intervention | Phase |
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Neurofibroma |
Drug: LS11 |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety Study |
Official Title: | CHP-856: 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 |
Estimated Enrollment: | 24 |
Study Start Date: | July 2008 |
Estimated Study Completion Date: | July 2010 |
Estimated Primary Completion Date: | July 2010 (Final data collection date for primary outcome measure) |
LS11 is supplied as 100mg/vial. Two dosages (30 and 40 mg/m2) will be used in this study.
Escalating doses 50, 100, 150 and 200 J/cm of light will be used.
Ages Eligible for Study: | 3 Years to 21 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Tumor Size/Shape/Location:All tumors must:
Laboratory: Patients must have adequate bone marrow, renal, and hepatic function assessed within 7 days prior to start of therapy.
Hematologic:
Absolute neutrophil count ≥ 1000/ul Platelet count ≥ 100,000/ul Hemoglobin ≥ 8 g/dL PT/PTT ≤ 1.2 times institutional upper limit of normal
Bilirubin ≤ 1.5 times upper limit of normal for age ALT ≤ 2.5 times institutional upper limit of normal for age Albumin ≥ 2 g/dL
Patient must not be pregnant or breast-feeding. Patients of childbearing or child fathering potential must be willing to use a medically acceptable form of birth control, which includes abstinence, while being treated on this study.
Exclusion Criteria:
Contact: Michael J Fisher, M.D. | 215-590-2800 | fisherm@email.chop.edu |
Contact: Ratnakar Patti, Ph.D. | 267-426-5503 | pattir@email.chop.edu |
United States, Pennsylvania | |
The Children's Hospital of Philadelphia | Recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
Contact: Michael Fisher, M.D. 215-590-2800 fisherm@email.chop.edu | |
Contact: Ratnakar Patti, Ph.D. 267-426-5503 pattir@email.chop.edu |
Principal Investigator: | Michael J Fisher, M.D. | Children's Hospital of Philadelphia |
Responsible Party: | The Children's Hospital of Philadelphia ( Michael Fisher, M.D. ) |
Study ID Numbers: | CHP-856 PDT, 2006-10-5008 |
Study First Received: | July 14, 2008 |
Last Updated: | August 7, 2009 |
ClinicalTrials.gov Identifier: | NCT00716469 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Neurofibroma Plexiform NF1 Photo dynamic therapy |
Talaporfin Neurofibromatosis 1 Photosensitizing Agents Neurofibroma Neuromuscular Diseases Neurofibromatosis Type 1 Radiation-Sensitizing Agents |
Peripheral Nervous System Diseases Neurofibromatoses Peripheral Nervous System Neoplasms Neurofibroma, Plexiform Nerve Sheath Neoplasms Nervous System Neoplasms |
Neoplasms by Histologic Type Talaporfin Antineoplastic Agents Physiological Effects of Drugs Neoplasms, Nerve Tissue Nervous System Diseases Pharmacologic Actions Photosensitizing Agents Neoplasms Radiation-Sensitizing Agents |
Neurofibroma Neuromuscular Diseases Therapeutic Uses Peripheral Nervous System Diseases Peripheral Nervous System Neoplasms Neurofibroma, Plexiform Dermatologic Agents Nerve Sheath Neoplasms Nervous System Neoplasms |