Clinical trial NCT06677853: Timolol Maleate Gel for the Treatment of Infantile Hemangioma
Official title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase II/III Clinical Study to Evaluate the Efficacy and Safety of Timolol Maleate Gel in the Treatment of Proliferating Superficial Infantile Hemangioma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Infantile Hemangioma
Keywords: superficial
proliferating
Infantile Hemangioma
Timolol Maleate
Lead sponsor: Auson Pharmaceuticals Inc.
Sponsor class: INDUSTRY
First posted: 2024-11-07
Enrollment: 168
Sex: ALL
Minimum age (years): 35
Maximum age (years): 150
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 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.
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

1. Male or female infants at the age of 35 \~ 150 days;
2. 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;
3. Infant subjects with single hemangioma lesion;
4. Infant subjects with the maximum hemangioma diameter being ≥ 1 cm but ≤ 10 cm;
5. 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.

Exclusion Criteria:

1. Infant subjects who were known to be allergic to or had history of severe allergy to timolol maleate or other β-receptor blockers;
2. Infant subjects who had previously been treated with systemic, intralesional or topical corticosteroids, vincristine, α-interferon, imiquimod, propranolol, or other β-receptor blockers;
3. Infant subjects breastfed by mother who was treated with β-receptor blockers, systemic (oral, intravenous or intramuscular) corticosteroids, vincristine or α-interferon while breastfeeding;
4. Infant subjects who born more than 2 months premature and younger than 60 days old;
5. Infant subjects who had previously been treated for hemangioma (including surgery, hormonal drugs, laser therapy, etc.);
6. Infant subjects with more than one type of hemangioma requiring treatment;
7. Infant subjects with other skin diseases on the hemangioma surface and surrounding skin areas, such as eczema, infantile eczema, etc.
8. Infant subjects who had atrioventricular block ≥ second-degree, bradycardia (heart rate \< 100 bpm), sinoatrial syndrome, cardiogenic shock, or other congenital cardiac disorders;
9. Infant subjects who were suffering from respiratory disorders such as bronchial asthma, bronchospasm and pneumonia;
10. 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;
11. Infant subjects who had systolic blood pressure \< 50 mmHg or diastolic blood pressure \< 30 mmHg;
12. 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.

Primary outcomes:
measure: Success rate of cure of IH after 24-week treatment.
timeFrame: 24 weeks
Secondary outcomes:
measure: Changes in the hemangioma volume
timeFrame: baseline, week 4, 12 and 24
measure: Changes in hemangioma color
timeFrame: baseline, weeks 4, 12 and 24
measure: Complete/almost complete regression rates of IH from baseline after 4 and 12-week treatment.
timeFrame: baseline, weeks 4 and 12
measure: Complete/almost complete resolution rate of IH at each post-baseline visit (Weeks 4, 12, and 24) from baseline.
timeFrame: baseline, Weeks 4, 12, and 24
measure: Evolution of IH at each post-baseline visit(Weeks 4, 12, and 24) from baseline.
timeFrame: baseline, Weeks 4, 12 and 24
measure: Changes in IH at each post-baseline visit(Weeks 4, 12, and 24) compared to the previous visit.
timeFrame: Baseline, Weeks 4, 12, and 24
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Children's Hospital,Capital Medical University
state: Beijing
city: Beijing
country: China
facility: Children's Hospital, Capital Institute of Pediatrics
state: Beijing
city: Suzhou
country: China
facility: Children's Hospital of Soochow University
state: Jiangsu
city: Shanghai
country: China
facility: Shanghai Ninth People's Hospital, Shanghai Jiaotong University School of Medicine
state: Shanghai
city: Taiyuan
country: China
facility: Children's Hospital of Shanxi
state: Shanxi
city: Chengdu
country: China
facility: Chengdu Women's and Children's Central Hospital
state: Sichuan
city: Kunming
country: China
facility: Kunming Children's Hospital
state: Yunan
city: Hangzhou
country: China
facility: Children's Hospital, Zhejiang University School of Medicine
state: Zhejiang
Study officials:
References: