Clinical trial NCT05171894: A Study to Evaluate Efficacy and Safety of Light Dose in Subjects with PWB Treated with Hemoporfin PDT
Official title: A Multi-Center, Randomized, Double-Blind, Vehicle-Controlled, Sequential Group Comparison Study to Evaluate the Safety and Efficacy of Light Dose in Subjects with Port-wine Birthmarks Treated with Hemoporfin Photodynamic Therapy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Port-wine Birthmarks
Port-Wine Stain
Nevus Flammeus
Keywords: Hemoporfin
Port-wine Birthmarks
Port-Wine Stain
Nevus Flammeus
Photodynamic therapy
Lead sponsor: Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2021-12-29
Enrollment: 84
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: Inclusion Criteria:

1. Subject must be 18 to 65 years of age inclusive, at the time of signing the informed consent.
2. Subject is Fitzpatrick skin type I-VI.
3. 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.
4. 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.
5. The subject has a clinical diagnosis of PWB located on the face and/or neck.
6. The longest diameter of the treatment area is ≥3 cm, and the short diameter is ≥2 cm.
7. 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.
8. 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:

1. Subject is pregnant, lactating, or is planning to become pregnant during the study.
2. Subject has plaque/nodular changes and severe hypertrophy within the target PWB area.
3. Subject has Sturge-Weber syndrome or PWB involving ophthalmic nerves.
4. 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.
5. Subject is immunosuppressed related to medication use and/or disease.
6. Subject has clinically significant ECG abnormalities at Screening
7. Subject has clinically significant laboratory abnormalities as assessed by the Investigator at Screening.
8. Subject has received any therapy on the treatment region that, in the Investigator's opinion, may affect the target PWB area.
9. Subject is currently enrolled in an investigational drug or device study, including clinical studies in dermatology.
10. Subject has used an investigational drug or investigational device treatment within 30 days prior to C1D1.
11. 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).
12. 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.
13. 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.

Primary outcomes:
measure: Stage One：Incidence of any local and systemic adverse events.
timeFrame: From baseline until end of study, up to approximately 44 weeks
measure: Stage Two: Port Wine Birthmark-Investigator Global Assessment (PWB-IGA) scale score reduction.
timeFrame: From baseline until end of study, up to approximately 44 weeks
Secondary outcomes:
measure: Stage One：Change from Baseline in overall PWB-IGA severity score and other scales.
timeFrame: From baseline until end of study, up to approximately 44 weeks
measure: Stage Two: Change from Baseline in overall PWB-IGA severity score and other scales.
timeFrame: From baseline until end of study, up to approximately 44 weeks
measure: Stage Two: Incidence of any local and systemic adverse events.
timeFrame: From baseline until end of study, up to approximately 44 weeks
Other outcomes:
measure: Stage One：Maximum observed plasma concentration (Cmax) of Hemoporfin
timeFrame: On the first day of the Cycle 1 (each cycle is 56 days)
measure: Stage One：Area under the concentration-time curve (AUC 0-∞) from time 0 to infinity of Hemoporfin
timeFrame: On the first day of the Cycle 1 (each cycle is 56 days)
measure: Stage One：Terminal elimination half-life (t1/2) of Hemoporfin
timeFrame: On the first day of the Cycle 1 (each cycle is 56 days)
measure: Stage One：Time to Cmax (Tmax) of Hemoporfin
timeFrame: On the first day of the Cycle 1 (each cycle is 56 days)
Locations:
city: Irvine
country: United States
facility: UCI Health Beckman Laser Institute & Medical Clinic
state: California
city: San Diego
country: United States
facility: Dermatology Cosmetic Associates of La Jolla, Inc. d/b/a West Dermatology Research Center
state: California
city: Aventura
country: United States
facility: Skin and Cancer Associates (SCA) and the Center for Cosmetic Enhancement (CCE)
state: Florida
city: Miami
country: United States
facility: Miami Dermatology and Laser Institute
state: Florida
city: Hunt Valley
country: United States
facility: Maryland Dermatology, Laser, Skin & Vein Institute
state: Maryland
city: Bethlehem
country: United States
facility: St. Luke's University Health Network
state: Pennsylvania
Study officials:
References: