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Photodynamic Therapy (PDT) for malignant brain tumor in children.

Photodynamic Therapy (PDT) for malignant brain tumor in children.

5

Malignant brain tumors in children and young adolescent between the ages of 6 and 20 years

This is the single-center, non-randomized, Phase I/II clinical trial. Patients who met the following inclusion and exclusion criteria were eligible for enrollment. Inclusion criteria: 1. Patients who receives treatment at either outpatient or admission in the Department of Neurosurgery, Tokyo Women's Medical University Hospital; 2. Males and females aged 6 years or older but less than 20 years at the time of consent; 3. Primary cases who are suspicious for primary malignant brain tumor based on preoperative imaging, underwent biopsy prior to resection, or recurrence cases. Exclusion criteria: 1. Those who have no indication for surgical removal; 2. Those who cannot use the Talaporfin sodium due to allergies or other reasons; 3. Patients with brain tumors other than the targeted histology; 4. Those who require visual evoked potential monitoring during operation; 5. Pregnant women; 6. Those who are expected to be unable to comply with light shading management; 7. Those who is regarded as unsuitable by the principal investigator for any reason. Those who is regarded as unsuitable by the principal investigator for any reason. The dose of Talaporfin sodium was determined by dose-escalation from 10 mg/m2 - 20 mg/m2 - 40 mg/m2 in every 3 cases starting from the first enrolled case, with the final target of 40 mg/m2, the same dose as in adults. If an adverse event of CTCAE version 4.0 grade 3 or higher was observed, the dose escalation was to be sustained. If two consecutive patients had grade 3 or higher adverse events, the dose was to be reduced. Since the assessment of photosensitivity requires at least 2 weeks, new cases were enrolled at least 2 weeks apart, and if photosensitivity appeared, the next case was not enrolled until the symptoms disappeared.

Partial seizure (CTCAE grade 2) (6, 7 POD) in case 2, fever (CTCAE grade 2) (20 POD) in case 2, fever (CTCAE grade 2) (3 POD) in case 3, palpitation (CTCAE grade 1) (3 POD) in case 6

Five patients with a mean age of 149.3 months (129-189 months) at the time of PDT received six surgeries and had a mean follow-up of 23.2 months (1-44 months) after PDT. Histopathological diagnoses included supratentorial anaplastic ependymomas in two (3 surgeries), anaplastic astrocytoma in one, diffuse midline glioma, H3K27M mutation in one, and glioblastomas in one. The outcome was survival in two patients and death in three patients. The mean number of PDT shots were calculated as 6.5 shots (1-14 shots). Photosensitivity test were performed on average 14.7 post operative days (13-15 post operative days). All cases were negative for photosensitivity test. Seizures and fevers were commonly observed, which are likely surgery-related symptoms, and there was no photosensitive skin rash or liver dysfunction, which are common adverse effects in adults.

We conducted a phase I/II clinical study of PDT for malignant brain tumors in children. No adverse events corresponding to CTCAE grade 3 or higher were observed, indicating that TS can be used safely in children. However, the efficacy of PDT, another objective of this study, could not be demonstrated due to the small number of cases and the diverse clinical backgrounds, including histological diagnosis and treatment received.

April. 01, 2023

No

N/A

https://jrct.niph.go.jp/latest-detail/jRCTs031180360

kawamata takakazu

Tokyo Women's Medical University Hospital

8-1, Kawada-cho, Shinjuku-ku, Tokyo

tkawamata@twmu.ac.jp

chiba kentaro

Tokyo Women's Medical University Hospital

8-1, Kawada-cho, Shinjuku-ku, Tokyo, Japan

+81-3-3353-8111

chibak920@gmail.com

Complete

Nov. 02, 2017

Interventional

single arm study

open(masking not used)

no treatment control/standard of care control

single assignment

treatment purpose

1. Patients treated at the department of neurosurgery, Tokyo Women's Medical University, Tokyo, Japan(in both outpatient clinic and admission).
2. Man and woman, over the age of 6 years old and below the age of 20 years old at enrollment.
3. In an initial case, patients who are highly suspicious for primary malignant brain tumor from pre-operative neuro-imaging. In a recurrent case, patient who is diagnosed as primary malignant brain tumor at previous histopathological diagnosis.

1. Patients without surgical tumor removal.
2. Unusable for Talaporfin Sodium.
3. Non intended histopathological types of brain tumor.
4. Patients necessary for VEP monitoring.
5. Pregnancy or possibility of pregnancy.
6. Patients who are expected poor compliance with light shielding protection.
7. Patients regarded as ineligible by principal investigator.

Both

Malignant brain tumor in children

Medication: Talaporfin sodium (Leserphyrin)
Dose: Dose-up from 10mg/square-20mg/square-40mg/square in sequence in every 3 cases since initial enrolled case. Finally, single drip intra-venous infusion of 40mg/square is required.
Timing: 22-26 hours bafore surgical removal.

Laser irradiation in a wave length of 664+/-2nm for tumor removal cavity 22-26 hours after drip intra-venous infusion of Talaporfin sodium.

1. Safety (including photosensitivity test)(phase I)
2. Overall survival (OS) rate at 12 months after PDT (phase II)

1. Overall survival (OS)
2. Progression-free survival (PFS) rate at 6 months after PDT.
3. Progression-free survival
4. Safety (including photosensitivity test)

+81-42-984-4122

Mar. 07, 2019

none