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

NCT04106258 | Completed Phase 4

• 1 other identifier: HMME-C1904
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Port-wine Stain

Outcome Measures

Primary Outcomes (2)

• 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

Study Arms (2)

low light dose

EXPERIMENTAL

PDT is applied to the patients at low light dose : power density of 60mW/cm2 for 20 minutes

Drug: Hemoporfin PDT

high light dose

EXPERIMENTAL

PDT is applied to the patients at high light dose : power density of 75mW/cm2 for 20 minutes

Drug: Hemoporfin PDT

Interventions

Hemoporfin PDT DRUG

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.

high light dose; low light dose

Eligibility Criteria

• Age: 2 Years - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd.: lead

Study Sites (1)

Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine

Shanghai, Shanghai Municipality, 200011, China

Location

MeSH Terms

Conditions

Port-Wine Stain

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 25, 2019
• First Posted: September 27, 2019
• Study Start: May 27, 2020
• Primary Completion: February 15, 2023
• Study Completion: February 15, 2023
• Last Updated: December 20, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)