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Phase I Clinical Study of the Safety of Photodynamic Therapy (PDT) Using LS11 in Children With Plexiform Neurofibromas (PDT)

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ClinicalTrials.gov Identifier: NCT00716469
Recruitment Status : Terminated (The study is terminated due to expiration of study materials.)
First Posted : July 16, 2008
Last Update Posted : April 15, 2015
United States Department of Defense
Information provided by (Responsible Party):
Michael Fisher, Children's Hospital of Philadelphia

Brief Summary:

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.

Condition or disease Intervention/treatment Phase
Neurofibroma Drug: LS11 Phase 1

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : July 2008
Primary Completion Date : July 2012
Study Completion Date : July 2012

Arm Intervention/treatment
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).
Drug: LS11
LS11 will be given as a one-time IV infusion over 3-5 minutes.
Other Name: Talaporfin sodium

Primary Outcome Measures :
  1. To determine the safety and tolerability of photodynamic therapy (PDT) for the treatment of plexiform neurofibromas in children. [ Time Frame: Week 4 and 12 ]

Secondary Outcome Measures :
  1. To evaluate the change in quality of life of treated patients. [ Time Frame: Week 4 and 12 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age: Patients must be ≥ 3 and ≤ 21 years of age.
  • Tumor: Patients must have a debilitating, severely disfiguring, life-threatening, or progressive plexiform neurofibroma (PN), which is not surgically resectable and for which there is no other standard medical management.
  • Measurable Disease: Patients must have a measurable PN assessed by MRI within 2 weeks prior to starting therapy.
  • Tumor Size/Shape/Location:All tumors must:

    1. have a minimum dimension that is ≥ 5 cm in the plane of intended 25mm length Light Source insertion
    2. have a minimum dimension that is ≥ 3.15 cm perpendicular to the plane of intended 25mm length Light Source insertion
    3. be accessible for percutaneous CT (and ultrasound if needed) guided Light Source insertion;
    4. have a shape and location such that the minimum distance between the Light Source and any "critical structure" (defined in section 4.3.4) will be as follows:
  • Minimum radial distance = 2.5 cm
  • Minimum distance from proximal end of Light Source = 2.5 cm
  • Minimum distance from distal end of Light Source = 2 cm
  • For patients with NF1: In addition to PN, all study subjects must have at least one other diagnostic criteria for NF1.
  • Performance Status: Patients should have a life expectancy of at least 6 months and a Karnofsky (≥ 16 years of age) or Lansky (≤16 years of age) Performance Score ≥ 60.
  • Prior/Concurrent Therapy: Patients must have recovered from any specific acute toxicity associated with prior therapy. No concurrent anti-tumor therapy is allowed.
  • Laboratory: Patients must have adequate bone marrow, renal, and hepatic function assessed within 7 days prior to start of therapy.

    1. 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

    2. Renal: Serum creatinine within upper limit of institutional norm
    3. Hepatic:

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

  • Pregnancy: Female patients of childbearing potential must have negative serum or urine pregnancy test. 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.
  • Informed Consent: All patients or their legal guardians (if the patient is less than 18 years old) must sign an IRB approved document of informed consent indicating their understanding of the investigational nature and the risks of this study before beginning therapy. When appropriate, pediatric patients will be included in all discussions in order to obtain verbal assent.

Exclusion Criteria:

  • Patients < 3 or > 21 years of age.
  • Tumors that are not debilitating, severely disfiguring, life-threatening, or progressive
  • Patients with baseline pain or neuropathy related to their target lesion that is so severe that it effects activities of daily living (i.e. grade 3 or 4).
  • Tumors that would require Light Source placement such that a "critical structure" is within the minimum distance required between a "critical structure" and Light Source.
  • Tumor that is inaccessible for percutaneous implantation of light source by interventional radiology.
  • Patients with a history of porphyria.
  • Concomitant use of other drugs known to produce skin photosensitivity (e.g. tetracyclines, sulfonamides, phenothiazines, sulfonylureas, thiazide diuretics, and griseofulvin)
  • Patients with any serious medical illnesses that, in the investigator's opinion, would compromise a patient's ability to tolerate this therapy.
  • Patients receiving any other chemotherapeutics or investigational agents.
  • Patients with uncontrolled infections.
  • Patients taking NSAIDs or anti-coagulants.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00716469

United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Children's Hospital of Philadelphia
United States Department of Defense
Principal Investigator: Michael J Fisher, M.D. Children's Hospital of Philadelphia

Responsible Party: Michael Fisher, Assoc. Prof., Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT00716469     History of Changes
Other Study ID Numbers: 2006-10-5008
CHP-856 PDT ( Other Identifier: Children's Hospital of Philadelphia )
First Posted: July 16, 2008    Key Record Dates
Last Update Posted: April 15, 2015
Last Verified: April 2015

Keywords provided by Michael Fisher, Children's Hospital of Philadelphia:
Photo dynamic therapy

Additional relevant MeSH terms:
Nerve Sheath Neoplasms
Neurofibroma, Plexiform
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Nervous System Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Antineoplastic Agents
Photosensitizing Agents
Dermatologic Agents