Timolol Maleate Gel for the Treatment of Infantile Hemangioma

NCT06677853 | Completed Phase 2

• 1 other identifier: ASN001C002
• Study Type: interventional
• Target: 168
• Locations: 1 country
• Sites: 8

Brief Summary

The goal of this clinical trial is to evaluate the safety and efficacy of timolol maleate (TM) gel in subjects with superficial infantile hemangioma (IH) in the proliferative phase. The main question it aims to answer is:

• The primary endpoint (success or failure) assessment was a centralized and independent qualitative assessment based on blinded comparison on B-ultrasonography results and photographs of IH at W24 from baseline. Researchers will compare TM gel to a placebo (a look-alike substance that contains no drug) to see if TM gel works to treat IH. Participants will:
• Take the study drug 3 times daily (once in the morning, noon, and evening, respectively) for 24 weeks.
• The family members of patients are instructed to bring the patients to the clinic for regular follow-up visits at Week 4 (W4), Week 12 (W12), and Week 24 (W24) of the treatment period.
• Keep a diary of concomitant medications and adverse events.

Conditions

Infantile Hemangioma

Keywords

superficial; proliferating; Infantile Hemangioma; Timolol Maleate

Outcome Measures

Primary Outcomes (1)

• Success rate of cure of IH after 24-week treatment.

  Treatment success is defined as complete or almost complete resolution of the patient's IH at W24 compared with baseline. At the time of centralized independent assessment.

  24 weeks

Secondary Outcomes (6)

• Changes in the hemangioma volume

  baseline, week 4, 12 and 24

• Changes in hemangioma color

  baseline, weeks 4, 12 and 24

• Complete/almost complete regression rates of IH from baseline after 4 and 12-week treatment.

  baseline, weeks 4 and 12

• Complete/almost complete resolution rate of IH at each post-baseline visit (Weeks 4, 12, and 24) from baseline.

  baseline, Weeks 4, 12, and 24

• Evolution of IH at each post-baseline visit(Weeks 4, 12, and 24) from baseline.

  baseline, Weeks 4, 12 and 24

Study Arms (3)

Group1-Timolol Maleate Gel + Placebo

EXPERIMENTAL

56 patients will be randomized to group 1.

Drug: Timolol Maleate Gel+Placebo

Group 2-Timolol Maleate Gel

EXPERIMENTAL

56 patients will be randomized to group 2.

Drug: Timolol Maleate Gel

Group 3-Placebo

PLACEBO COMPARATOR

56 patients will be randomized to group 3.

Drug: Placebo

Interventions

Timolol Maleate Gel+Placebo DRUG

Subjects were applied with the 0.5% timolol maleate gel at the affected area twice a day, and with the placebo once a day for 24 weeks.

Group1-Timolol Maleate Gel + Placebo

Timolol Maleate Gel DRUG

Subjects were applied with the 0.5% timolol maleate gel at the affected area 3 times a day for 24 weeks.

Group 2-Timolol Maleate Gel

Placebo DRUG

Subjects were applied with placebo at the affected area 3 times a day for 24 weeks.

Group 3-Placebo

Eligibility Criteria

• Age: 35 Days - 150 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female infants at the age of 35 \~ 150 days;
• Infant subjects with definitive diagnosis of superficial hemangioma requiring treatment based on medical history, clinical manifestations, and imaging examination (B-ultrasonography, CT or MRI) results;
• Infant subjects with single hemangioma lesion;
• Infant subjects with the maximum hemangioma diameter being ≥ 1 cm but ≤ 10 cm;
• Infant subject with CEA ≥ Grade 2; The guardians of the infant subject understood the study contents and risks of study treatment, signed the informed consent form (ICF), and were willing to cooperate with the study conduct.

You may not qualify if:

• Infant subjects who were known to be allergic to or had history of severe allergy to timolol maleate or other β-receptor blockers;
• Infant subjects who had previously been treated with systemic, intralesional or topical corticosteroids, vincristine, α-interferon, imiquimod, propranolol, or other β-receptor blockers;
• Infant subjects breastfed by mother who was treated with β-receptor blockers, systemic (oral, intravenous or intramuscular) corticosteroids, vincristine or α-interferon while breastfeeding;
• Infant subjects who born more than 2 months premature and younger than 60 days old;
• Infant subjects who had previously been treated for hemangioma (including surgery, hormonal drugs, laser therapy, etc.);
• Infant subjects with more than one type of hemangioma requiring treatment;
• Infant subjects with other skin diseases on the hemangioma surface and surrounding skin areas, such as eczema, infantile eczema, etc.
• Infant subjects who had atrioventricular block ≥ second-degree, bradycardia (heart rate \< 100 bpm), sinoatrial syndrome, cardiogenic shock, or other congenital cardiac disorders;
• Infant subjects who were suffering from respiratory disorders such as bronchial asthma, bronchospasm and pneumonia;
• Infant subjects who were suffering from central nervous system disorders, or had symptoms of increased intracranial pressure, or had other underlying diseases that might cause or aggravate infantile hemangioma;
• Infant subjects who had systolic blood pressure \< 50 mmHg or diastolic blood pressure \< 30 mmHg;
• Infant subjects who received any other investigational drug within 4 weeks prior to screening; Infant subjects with other conditions not suitable for enrollment at the discretion of the investigator.

Sponsors & Collaborators

• Auson Pharmaceuticals Inc.: lead

Study Sites (8)

Beijing Children's Hospital,Capital Medical University

Beijing, Beijing Municipality, 100045, China

Location

Children's Hospital, Capital Institute of Pediatrics

Beijing, Beijing Municipality, 100056, China

Location

Children's Hospital of Soochow University

Suzhou, Jiangsu, 215025, China

Location

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

Shanghai, Shanghai Municipality, 200011, China

Location

Children's Hospital of Shanxi

Taiyuan, Shanxi, 030013, China

Location

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, 200080, China

Location

Kunming Children's Hospital

Kunming, Yunan, 650228, China

Location

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310052, China

Location

MeSH Terms

Conditions

Hemangioma, Capillary

Condition Hierarchy (Ancestors)

Hemangioma; Neoplasms, Vascular Tissue; Neoplasms by Histologic Type; Neoplasms

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: It was a multicenter, randomized, double-blind, placebo-controlled Phase II/III clinical study to evaluate the efficacy and the safety of TM gel in the treatment of superficial IH in the proliferative phase.

Study Record Dates

• First Submitted: October 29, 2024
• First Posted: November 7, 2024
• Study Start: October 28, 2020
• Primary Completion: June 21, 2022
• Study Completion: June 21, 2022
• Last Updated: November 7, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (8)