Infantile HemangiomaPhase 2/3NCT06677853
Timolol Maleate Gel for the Treatment of Infantile Hemangioma
A Phase 2/3 study testing Timolol Maleate Gel in people with Infantile Hemangioma, run by Auson Pharmaceuticals Inc. at 8 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 35 to 150 days
- Conditions the study is for
- Infantile Hemangioma
- What is being tested?
- Timolol Maleate Gel
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 168 participants
- Where
- 8 sitesChina
- Study dates
- 28 Oct 2020to 21 Jun 2022
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 150 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
China8 sites
Beijing Children's Hospital,Capital Medical University
Beijing, Beijing Municipality, 100045
Children's Hospital, Capital Institute of Pediatrics
Beijing, Beijing Municipality, 100056
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan, 200080
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310052
Kunming Children's Hospital
Kunming, Yunan, 650228
Shanghai Ninth People's Hospital, Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai Municipality, 200011
Children's Hospital of Soochow University
Suzhou, Jiangsu, 215025
Children's Hospital of Shanxi
Taiyuan, Shanxi, 030013
About this study
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
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.
Read more
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
- Being tested
- Timolol Maleate Gel+Placebo, Timolol Maleate Gel, Placebo
- Keywords
- superficial, proliferating, Infantile Hemangioma, Timolol Maleate
- Medical subject headings
- Hemangioma, Capillary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Timolol Maleate Gel+PlaceboDrug
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.
Timolol Maleate GelDrug
Subjects were applied with the 0.5% timolol maleate gel at the affected area 3 times a day for 24 weeks.
PlaceboDrug
Subjects were applied with placebo at the affected area 3 times a day for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Group1-Timolol Maleate Gel + Placebo | Experimental | 56 patients will be randomized to group 1. Receives: Drug: Timolol Maleate Gel+Placebo |
| Group 2-Timolol Maleate Gel | Experimental | 56 patients will be randomized to group 2. Receives: Drug: Timolol Maleate Gel |
| Group 3-Placebo | Placebo comparator | 56 patients will be randomized to group 3. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 24 weeks
Secondary outcomes (6)
Changes in the hemangioma volume
Changes in the hemangioma volume from baseline after treatment for 4, 12 and 24 weeks, adopting the estimation formula of IH volume: V = 0.07×(the average of the hemangioma's longest length and width)^3.
Measured: baseline, week 4, 12 and 24
Changes in hemangioma color
Change in the color of hemangioma after 4, 12, and 24 weeks of treatment, which is assessed by the CEA (Clinician Erythema Assessment) criteria.The color intensity (e.g., no signs of erythema;slight redness;defined redness;marked redness;fiery redness.) of the IH is assessed on a 5 point scale.
Measured: baseline, weeks 4, 12 and 24
Complete/almost complete regression rates of IH from baseline after 4 and 12-week treatment.
At the time of centralized independent assessment.
Measured: baseline, weeks 4 and 12
Complete/almost complete resolution rate of IH at each post-baseline visit (Weeks 4, 12, and 24) from baseline.
On-site qualitative assessment by the investigator of efficacy.
Measured: baseline, Weeks 4, 12, and 24
Evolution of IH at each post-baseline visit(Weeks 4, 12, and 24) from baseline.
Classified into 5 categories: completely/almost completely resolved, improved, stable, aggravated, and not assessable.
On-site qualitative assessment by the investigator of efficacy.
Measured: baseline, Weeks 4, 12 and 24
Changes in IH at each post-baseline visit(Weeks 4, 12, and 24) compared to the previous visit.
Classified into 5 categories: complete/almost complete regression, relief, stabilization, exacerbation, and unable to determine.
On-site qualitative assessment by the investigator of efficacy.
Measured: Baseline, Weeks 4, 12, and 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2024
- Study start28 Oct 2020
- Primary completion21 Jun 2022
- Study completion21 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile HemangiomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 168 participants (actual)
Sponsor and investigators
- Lead sponsor
- Auson Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06677853
- Sponsor's protocol ID
- ASN001C002
- Other IDs
- ASN001C002
- Status last verified
- Nov 2024
- First submitted
- 29 Oct 2024
- Last updated
- 7 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06677853
- Acronym / sponsor ID
- ASN001C002
- Conditions
- Infantile Hemangioma
- Keywords
- superficial, proliferating, Infantile Hemangioma, Timolol Maleate
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 168 participants (actual)
- Lead sponsor
- Auson Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Oct 2020 (actual)
- Primary completion
- 21 Jun 2022 (actual)
- Study completion
- 21 Jun 2022 (actual)
- First posted
- 7 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- 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.
| Arm | Type | Receives |
|---|---|---|
| Group1-Timolol Maleate Gel + Placebo | Experimental | Drug: Timolol Maleate Gel+Placebo56 patients will be randomized to group 1. |
| Group 2-Timolol Maleate Gel | Experimental | Drug: Timolol Maleate Gel56 patients will be randomized to group 2. |
| Group 3-Placebo | Placebo comparator | Drug: Placebo56 patients will be randomized to group 3. |
Who may qualify
- Ages
- 35 to 150 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Timolol Maleate Gel+PlaceboSubjects 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. | Drug | Group1-Timolol Maleate Gel + Placebo |
| Timolol Maleate GelSubjects were applied with the 0.5% timolol maleate gel at the affected area 3 times a day for 24 weeks. | Drug | Group 2-Timolol Maleate Gel |
| PlaceboSubjects were applied with placebo at the affected area 3 times a day for 24 weeks. | Drug | Group 3-Placebo |
Primary outcome
Success rate of cure of IH after 24-week treatment.
Time frame: 24 weeks
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.
6 secondary outcomes
Changes in the hemangioma volume
Time frame: baseline, week 4, 12 and 24
Changes in hemangioma color
Time frame: baseline, weeks 4, 12 and 24
Complete/almost complete regression rates of IH from baseline after 4 and 12-week treatment.
Time frame: baseline, weeks 4 and 12
Complete/almost complete resolution rate of IH at each post-baseline visit (Weeks 4, 12, and 24) from baseline.
Time frame: baseline, Weeks 4, 12, and 24
Evolution of IH at each post-baseline visit(Weeks 4, 12, and 24) from baseline.
Time frame: baseline, Weeks 4, 12 and 24
Changes in IH at each post-baseline visit(Weeks 4, 12, and 24) compared to the previous visit.
Time frame: Baseline, Weeks 4, 12, and 24
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Timolol Maleate Gel in people with Infantile Hemangioma, run by Auson Pharmaceuticals Inc. at 8 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Success rate of cure of IH after 24-week treatment. (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Nov 2024.