A Pilot Study of Hemoporfin PDT in Children(2-7 Years Old) With Port-wine Stain

Study Overview
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Brief Summary
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This pilot study aims to evaluate the safety and efficacy of hemoporfin photodynamic therapy (PDT) with different light doses for port-wine stain (PWS）in 2-7 years old children. The pharmacokinetic behavior and pharmacokinetic parameters of hemoporfin in children will be investigated as well.

Official Title
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A Pilot Study of Hemoporfin Photodynamic Therapy in Children (2-7 Years Old) With Port-wine Stain

Conditions
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Port-wine Stain

Intervention / Treatment
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* Drug: Hemoporfin PDT


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Children with clinical diagnosis of PWS； ≥2 years old and <7 years old； The guardians agreed to voluntarily participate in this study and signed the informed consent agreement Exclusion Criteria: Therapy area located outside of head and neck; Other skin diseases that might interfere with the efficacy evaluation; Patients with respiratory disease, severe pulmonary dysfunction, history of airway hyperresponsiveness, or family history of suspected malignant hyperthermia; Preexist scars in the treatment area caused by previous treatment, which might interfere with the efficacy and safety evaluation; with allergic diseases; known to be allergic to eggs, milk or soy protein; known to have skin photoallergies, porphyria or known allergic history of experimental drugs (porphyrins) and chemically structure similar drugs; known allergic history of anesthetics; allergic constitution; Cicatricial constitution; Immunocompromised conditions or need long-term use of glucocorticoids and immunosuppressive agents; Electrocardiographic abnormalities or organic heart diseases; Hepatic or renal functions abnormal (alanine aminotransferase or aspartate transaminase or total bilirubin > 1.5 upper limit of normal [ULN], or serum creatinine or blood urea nitrogen > 1.5 ULN); Coagulation disorders; Patients with severe neurological, psychiatric, endocrine and cardiovascular diseases; with epilepsy history or recent epileptic seizures; Be evaluated not suitable for anaesthesia by risk assessment before anaesthesia； Previous therapy of PWS within the last 4 weeks; Participation in any clinical studies within the last 4 weeks; Be judged not suitable to participate the study by the investigators

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: low light dose<br>PDT is applied to the patients at low light dose : power density of 60mW/cm2 for 20 minutes | Drug: Hemoporfin PDT<br>* Photodynamic therapy is performed using hemoporfin under general anesthesia. Hemoporfin（5mg/kg）is infused for 20 minutes, followed by light illumination at 10 minutes from the start of infusion. Different light dose of PDT is applied to the patients.<br>|
| Experimental: high light dose<br>PDT is applied to the patients at high light dose : power density of 75mW/cm2 for 20 minutes | Drug: Hemoporfin PDT<br>* Photodynamic therapy is performed using hemoporfin under general anesthesia. Hemoporfin（5mg/kg）is infused for 20 minutes, followed by light illumination at 10 minutes from the start of infusion. Different light dose of PDT is applied to the patients.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Response rate | proportion of patients achieving at least some improvement (color blanching from the baseline >= 20%) | week 8 | 
| Incidence of adverse events and adverse reactions |  | up to 24 weeks after the treatment |