A Study to Evaluate Efficacy and Safety of Light Dose in Subjects With PWB Treated With Hemoporfin + PDT

Study Overview
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Brief Summary
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This is a multi-center, randomized, double-blind, vehicle-controlled, and sequential group Phase 2 study. Eligible subjects aged 18 to 65 years old with PWB of face and/or neck area (except subjects for Stage One) will receive Hemoporfin PDT or vehicle PDT in 8-week cycles at fixed drug dose (5 mg/kg) and different light fluence.

Detailed Description
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This is a multi-center, randomized, double-blind, vehicle-controlled, and sequential group Phase 2 study. Eligible subjects aged 18 to 65 years old with PWB of face and/or neck area (except subjects for Stage One) will receive Hemoporfin PDT or vehicle PDT in cycles at fixed drug dose (5 mg/kg) and different light fluences. This study will be conducted in two sequential stages, each contains the same schedule, which includes Screening Period, Treatment Period apart; subsequent treatment is based on efficacy evaluation, and End of Study.

Official Title
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A Multi-Center, Randomized, Double-Blind, Vehicle-Controlled, Sequential Group Comparison Study to Evaluate the Safety and Efficacy of Light Dose (Fluence) in Subjects With Port-wine Birthmark of the Face and/or Neck Treated With Hemoporfin + Green Light Photodynamic Therapy

Conditions
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Port-wine Birthmark, Port-Wine Stain, Nevus Flammeus

Intervention / Treatment
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* Drug: Hemopfin+PDT
* Drug: Vehicle+PDT


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subject must be 18 to 65 years of age inclusive, at the time of signing the informed consent. Subject is Fitzpatrick skin type I-VI. A male subject must agree to use contraception during the Treatment Period and for at least 6 months after the last dose of study treatment and refrain from donating sperm during this period. A female subject is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies: Not a woman of childbearing potential (WOCBP) . OR A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 30 days after the last dose of study treatment. The subject has a clinical diagnosis of PWB located on the face and/or neck. The longest diameter of the treatment area is ≥3 cm, and the short diameter is ≥2 cm. Subject is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Subject, in the Investigator's opinion, is in good general health and free of any disease state or physical condition that may impair the evaluation of PWB or expose the subject to an unacceptable risk by study participation. Exclusion Criteria: Subject is pregnant, lactating, or is planning to become pregnant during the study. Subject has plaque/nodular changes and severe hypertrophy within the target PWB area. Subject has Sturge-Weber syndrome or PWB involving ophthalmic nerves. Subject has any skin pathology or condition that, in the Investigator's opinion, could interfere with the evaluation of the study drug or requires use of interfering topical, systemic, or surgical therapy. Subject is immunosuppressed related to medication use and/or disease. Subject has clinically significant ECG abnormalities at Screening Subject has clinically significant laboratory abnormalities as assessed by the Investigator at Screening. Subject has received any therapy on the treatment region that, in the Investigator's opinion, may affect the target PWB area. Subject is currently enrolled in an investigational drug or device study, including clinical studies in dermatology. Subject has used an investigational drug or investigational device treatment within 30 days prior to C1D1. Subject is known or in the opinion of the Investigator likely to be noncompliant with the requirements of the study protocol (eg, due to alcoholism, drug dependency, mental incapacity). Subject has a history of either significant neurological events (such as major stroke) or a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study. Subject has an unstable cardiac disease or has any medical condition that in the opinion of the Investigator may worsen from receipt of study treatment or subject participation.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 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: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Hemoporfin+A J/cm2 PDT<br>Participants will receive Hemoporfin 5 mg/kg of body weight via intravenous (IV) infusion and fixed laser irradiation for certain time. | Drug: Hemopfin+PDT<br>* All qualified subjects randomized to Hemoporfin PDT treatment to receive up to 3 cycles of treatment.<br>* Other names: Fumeida with green light photodynamic therapy;|
| Experimental: Hemoporfin+B J/cm2PDT<br>Participants will receive Hemoporfin 5 mg/kg of body weight via intravenous (IV) infusion and fixed laser irradiation for certain time. | Drug: Hemopfin+PDT<br>* All qualified subjects randomized to Hemoporfin PDT treatment to receive up to 3 cycles of treatment.<br>* Other names: Fumeida with green light photodynamic therapy;|
| Experimental: Hemoporfin+C J/cm2 PDT<br>Participants will receive Hemoporfin 5 mg/kg of body weight via intravenous (IV) infusion and fixed laser irradiation for certain time. | Drug: Hemopfin+PDT<br>* All qualified subjects randomized to Hemoporfin PDT treatment to receive up to 3 cycles of treatment.<br>* Other names: Fumeida with green light photodynamic therapy;|
| Placebo Comparator: Placebo+A J/cm2 PDT<br>Participants will receive Vehicle (Saline) via intravenous (IV) infusion and fixed laser irradiation for certain time. | Drug: Vehicle+PDT<br>* All qualified subjects randomized to vehicle PDT treatment to receive up to 3 cycles of treatment.<br>* Other names: saline with green light photodynamic therapy;|
| Placebo Comparator: Placebo+B J/cm2PDT<br>Participants will receive Vehicle (Saline) via intravenous (IV) infusion and fixed laser irradiation for certain time. | Drug: Vehicle+PDT<br>* All qualified subjects randomized to vehicle PDT treatment to receive up to 3 cycles of treatment.<br>* Other names: saline with green light photodynamic therapy;|
| Placebo Comparator: Placebo+C J/cm2 PDT<br>Participants will receive Vehicle (Saline) via intravenous (IV) infusion and fixed laser irradiation for certain time. | Drug: Vehicle+PDT<br>* All qualified subjects randomized to vehicle PDT treatment to receive up to 3 cycles of treatment.<br>* Other names: saline with green light photodynamic therapy;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Stage One：Incidence of any local and systemic adverse events. | To compare the efficacy of multiple light doses (fluence) of Hemoporfin PDT To investigate the safety of multiple light doses (fluence) of Hemoporfin/vehicle PDT in subjects with Port-wine birthmark (PWB) | From baseline until end of study, up to approximately 44 weeks | 
| Stage Two: Port Wine Birthmark-Investigator Global Assessment (PWB-IGA) scale score reduction. | To compare the efficacy of multiple light doses (fluence) of Hemoporfin PDT with vehicle PDT in subjects with PWB of face and/or neck. | From baseline until end of study, up to approximately 44 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Stage One：Change from Baseline in overall PWB-IGA severity score and other scales. | To further evaluate the efficacy of multiple light doses (fluence) of Hemoporfin/vehicle PDT in subjects with PWB. | From baseline until end of study, up to approximately 44 weeks | 
| Stage Two: Change from Baseline in overall PWB-IGA severity score and other scales. | To further evaluate the efficacy of multiple light doses (fluence) of Hemoporfin/vehicle PDT in subjects with PWB. | From baseline until end of study, up to approximately 44 weeks | 
| Stage Two: Incidence of any local and systemic adverse events. | To investigate the safety of multiple light doses (fluence) of Hemoporfin/vehicle PDT in subjects with Port-wine birthmark (PWB) | From baseline until end of study, up to approximately 44 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemoporfin, Port-wine Birthmark, Port-Wine Stain, Nevus Flammeus, Photodynamic therapy