Hemoporfin + PDT for Port-Wine Stain

Recruiting at 5 trial locations

Overseen By Xuejing Cheng

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd.

Must not be taking: Immunosuppressants

Disqualifiers: Pregnancy, Sturge-Weber syndrome, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 56 weeks

84 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you are using medications that suppress the immune system or have skin conditions requiring treatment, you may not be eligible to participate.

What data supports the effectiveness of the treatment Hemoporfin + PDT for Port-Wine Stain?

Research shows that Hemoporfin-mediated photodynamic therapy (HMME-PDT) is effective in treating port-wine stains, with studies involving thousands of cases demonstrating its clinical efficacy. ^{1 2 3 4 5}

Is Hemoporfin + PDT safe for treating port-wine stains?

Hemoporfin, also known as hematoporphyrin monomethyl ether (HMME), has been used in photodynamic therapy (PDT) for port-wine stains and is generally considered safe. Common side effects include short-term pain, swelling, itching, and scabbing, but no severe adverse events have been reported. ^{4 5 6 7 8}

How is Hemoporfin-PDT treatment for port-wine stains different from other treatments?

Hemoporfin-PDT is unique because it uses a special light-activated drug (photosensitizer) called Hemoporfin to target and reduce the growth of blood vessel cells in port-wine stains, with a low chance of recurrence. This treatment works by inhibiting specific pathways (VEGF/Akt/mTOR) that help these cells grow, making it different from other treatments that might not target these pathways. ^{5 6 7 9 10}

Eligibility Criteria

This trial is for adults aged 18-65 with Port-Wine Stain (PWB) on the face/neck. Participants must be in good health, not planning to conceive, and willing to use contraception. It's not suitable for those outside the age range or with conditions that could affect PWB evaluation or increase risk.

Inclusion Criteria

I agree to use birth control and not donate sperm for 6 months after my last treatment dose.

My treatment area is at least 3 cm long and 2 cm wide.

I am between 18 and 65 years old.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Hemoporfin or vehicle PDT in 8-week cycles with fixed drug dose and different light fluences

56 weeks

Multiple visits per cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Hemoporfin
• Vehicle+PDF

Trial Overview The study tests Hemoporfin combined with Photodynamic Therapy (PDT) against a placebo version of PDT in treating PWB. Patients are randomly assigned to receive either treatment at fixed doses over 8-week cycles, assessing efficacy and safety.

Participant Groups

Group I: Hemoporfin+C J/cm2 Green Light Experimental Treatment 1 Intervention

Participants will receive Hemoporfin 5 mg/kg of body weight via intravenous (IV) infusion and fixed laser irradiation for certain time.

Group II: Hemoporfin+B J/cm2 Green Light Experimental Treatment 1 Intervention

Participants will receive Hemoporfin 5 mg/kg of body weight via intravenous (IV) infusion and fixed laser irradiation for certain time.

Group III: Hemoporfin+A J/cm2 Green Light Experimental Treatment 1 Intervention

Participants will receive Hemoporfin 5 mg/kg of body weight via intravenous (IV) infusion and fixed laser irradiation for certain time.

Group IV: Placebo+A J/cm2 Green Light Placebo Group 1 Intervention

Participants will receive Vehicle (Saline) via intravenous (IV) infusion and fixed laser irradiation for certain time.

Group V: Placebo+B J/cm2 Green Light Placebo Group 1 Intervention

Participants will receive Vehicle (Saline) via intravenous (IV) infusion and fixed laser irradiation for certain time.

Group VI: Placebo+C J/cm2 Green Light Placebo Group 1 Intervention

Participants will receive Vehicle (Saline) via intravenous (IV) infusion and fixed laser irradiation for certain time.

Hemoporfin is already approved in China for the following indications:

Approved in China as Hemoporfin for:

• Port-wine stains

Who Is Running the Clinical Trial?

Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd.

Lead Sponsor

Trials

24

Recruited

2,400+

Findings from Research

Photodynamic therapy using hemoporfin (HMME-PDT) was highly effective for treating port-wine stains on extremities, with three out of four patients achieving complete cure after 2 to 3 treatment sessions.

The treatment was safe, showing no significant adverse reactions or recurrence, although all patients experienced some degree of edema during follow-up.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Photodynamic therapy for port-wine stains in extremities: Report of 4 cases. Wang, S., Lee, LY., Liu, SX.[2021]

Hematoporphyrin monomethyl ether photodynamic therapy (HMME-PDT) demonstrated a high overall response rate of 87.5% in treating port-wine stains, with 12.5% of cases achieving complete cure after just one treatment.

The treatment was generally safe, with no reported allergies or serious adverse reactions, although patients did experience pain during the procedure and significant post-treatment swelling.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical study on clinical operation and post-treatment reactions of HMME-PDT in treatment of PWS. Zhang, Y., Zou, X., Chen, H., et al.[2018]

Hemoporfin photodynamic therapy (HMME-PDT) is an effective treatment for port-wine stains (PWS), showing increased cure and effective rates with more treatment sessions, particularly for the pink type of PWS.

The treatment is associated with mild local adverse reactions, such as edema and itching, but no significant systemic side effects were reported, making it a safe option for patients of various ages.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of hemoporfin-PDT on port-wine stains: A retrospective analysis of 2952 cases. Sun, J., Zhang, Y., Ma, T., et al.[2023]

In a study of 551 patients with port-wine stains (PWS), hemoporfin-mediated photodynamic therapy (HMME-PDT) was found to be effective, with treatment efficacy increasing with the number of sessions (1-3 sessions) and showing better results in patients with no prior treatments.

Factors such as the location of the lesions (with better outcomes on the maxillary and mandibular areas) and the absence of previous treatments significantly influenced the effectiveness of HMME-PDT, highlighting the importance of treatment history and lesion characteristics.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Influential Factors in the Efficacy of Hemoporfin-Mediated Photodynamic Therapy for Port-wine Stains. Min, Z., Jing, L., Jun, Z., et al.[2023]

Hematoporphyrin monomethyl ether photodynamic therapy (HMME-PDT) effectively treats port wine stains (PWS), with a 100% response rate observed in lesions showing vascular rupture after treatment, indicating a strong correlation between vascular injury and treatment efficacy.

A variety of noninvasive diagnostic techniques, including the VISIA-CR™ system and dermoscopy, were utilized to objectively evaluate treatment response, revealing significant changes in skin color, vascular morphology, and dermal structure post-HMME-PDT.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Application of different noninvasive diagnostic techniques used in HMME-PDT in the treatment of port wine stains. Wen, L., Zhang, Y., Zhang, L., et al.[2019]

Hemoporfin is a second-generation photosensitizer with a stable structure and high efficiency in producing singlet oxygen, making it effective for use in photodynamic therapy (PDT).

It has been shown to have low toxicity in the dark and a fast clearance rate, which enhances its safety profile for patients undergoing PDT.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Research progress of Hemoporfin--part one: preclinical study. Pu, Y., Chen, W., Yu, Z.[2022]

Hemoporfin (HMME) effectively induces phototoxicity and apoptosis in human umbilical vein endothelial cells (HUVECs) in a dose-dependent manner, demonstrating its potential as a photosensitizer in photodynamic therapy (PDT).

The mechanism of action involves the inhibition of endothelial cell proliferation through the reduction of VEGF-A expression and the suppression of the Akt/mTOR signaling pathway, which may explain the low recurrence rates observed in clinical applications for treating port wine stains.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

In vitro photodynamic therapy of endothelial cells using hematoporphyrin monomethyl ether (Hemoporfin): Relevance to treatment of port wine stains. Mei, Y., Xiao, X., Fan, L., et al.[2021]

Hematoporphyrin monomethyl ether (HMME) photodynamic therapy (PDT) was found to be effective in treating port-wine stains (PWS) and Sturge-Weber syndrome (SWS), with over 52% of SWS patients and 69% of PWS patients showing at least a 25% improvement in their lesions.

The treatment was deemed safe, with common side effects including short-term pain and swelling, but no severe adverse events were reported, indicating a favorable safety profile for HMME-PDT.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hematoporphyrin monomethyl ether photodynamic therapy for the treatment of Sturge-Weber syndrome and large segmental facial port-wine stain. Li, X., Diao, P., Liu, L., et al.[2022]

Hemoporfin, a novel photosensitizer, effectively induces cell death in ovarian cancer cells (SKOV3) through photodynamic therapy (PDT), primarily via direct necrosis, and its effectiveness is dose-dependent.

In a xenograft model using nude mice, Hemoporfin-based PDT significantly controlled tumor growth, suggesting its potential as a promising treatment for ovarian cancer that warrants further clinical evaluation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phototoxicity of Hemoporfin to ovarian cancer. Song, K., Kong, B., Qu, X., et al.[2006]

In a study of 321 patients treated with Hemoporfin-mediated photodynamic therapy (PDT) for port-wine stains, it was found that more treatment sessions significantly improved the response to therapy, with patients receiving two or more sessions showing better outcomes.

Smaller lesion sizes (25 cm² or less) were associated with better treatment efficacy compared to larger lesions (over 64 cm²), and the treatment was more effective on the central and peripheral face compared to the extremities and trunk.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Retrospective analysis of Hemoporfin-mediated photodynamic therapy in the treatment of naïve port-wine stains. Liu, J., Zhou, J., Hu, D., et al.[2022]

References

1. Netherlands pubmed.ncbi.nlm.nih.gov

Photodynamic therapy for port-wine stains in extremities: Report of 4 cases. [2021]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Clinical study on clinical operation and post-treatment reactions of HMME-PDT in treatment of PWS. [2018]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Efficacy of hemoporfin-PDT on port-wine stains: A retrospective analysis of 2952 cases. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Influential Factors in the Efficacy of Hemoporfin-Mediated Photodynamic Therapy for Port-wine Stains. [2023]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Application of different noninvasive diagnostic techniques used in HMME-PDT in the treatment of port wine stains. [2019]

6. Netherlands pubmed.ncbi.nlm.nih.gov

Research progress of Hemoporfin--part one: preclinical study. [2022]

7. Netherlands pubmed.ncbi.nlm.nih.gov

In vitro photodynamic therapy of endothelial cells using hematoporphyrin monomethyl ether (Hemoporfin): Relevance to treatment of port wine stains. [2021]

8. United States pubmed.ncbi.nlm.nih.gov

Hematoporphyrin monomethyl ether photodynamic therapy for the treatment of Sturge-Weber syndrome and large segmental facial port-wine stain. [2022]

9. United States pubmed.ncbi.nlm.nih.gov

Phototoxicity of Hemoporfin to ovarian cancer. [2006]

10. Netherlands pubmed.ncbi.nlm.nih.gov

Retrospective analysis of Hemoporfin-mediated photodynamic therapy in the treatment of naïve port-wine stains. [2022]