ANZCTR - Registration

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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers

Trial registered on ANZCTR

Registration number

ACTRN12619001370190

Ethics application status

Approved

Date submitted

27/08/2019

Date registered

8/10/2019

Date last updated

17/08/2022

Date data sharing statement initially provided

8/10/2019

Type of registration

Retrospectively registered

Titles & IDs

Public title

Treatment of Superficial Strawberry Birthmarks with Topical Timolol

Scientific title

Topical Timolol Treatment of Superficial Proliferating Infantile Haemangioma assessing the effectiveness of 0.5% topical timolol gel in the treatment of
superficial IH.

Secondary ID [1]

None

Universal Trial Number (UTN)

U1111-12113953

Trial acronym

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Infantile Haemangioma

Condition category

Condition code

Skin

Dermatological conditions

Intervention/exposure

Study type

Interventional

Description of intervention(s) / exposure

This is an open labelled phase II study assessing the effectiveness of 0.5% topical timolol gel in the treatment of superficial infantile haemangiomas (IHs) .
Most IHs do not require active treatment as they involute spontaneously over time. However, up to 10-15% of IH require intervention during infancy because they cause complications, such as visual and airway obstruction, ulceration, bleeding, and permanent disfigurement. Following spontaneous involution, approximately 50% of the lesions will leave a blemish, and half of these will require treatment. Currently propanolol is the mainstay treatment of problematic IHs. We have demonstrated that oral propranolol at 1.5–2 mg/kg/day is an effective and safe treatment for problematic proliferating IH. However, many of the untreated IH can cause permanent cosmectic blemish, affecting the skin and/or leaving subcutaneous fatty residuum following spontaneous involution. Earlier treatment (during the early proliferative phase) has been shown to result in better outcomes and reduced short and long-term complications.
Parents/caregivers will be shown how to apply this gel to the IH.
1 drop morning and night applied topically to the IH.
Treatment will be increased to 1 drop three times a day, morning,noon and night, after 2 months if there is no improvement and there are no adverse effects.
Treatment is continued until complete resolution of the lesion occurs, there is no change in lesion size, colour or consistency after 3 months of continued use, until the patient reaches 12 months of age, or if any criteria for removal from the study are met.

Intervention code [1]

Treatment: Drugs

Comparator / control treatment

No control group

Control group

Uncontrolled

Outcomes

Primary outcome [1]

Composite primary outcome To assess the effectiveness of 0.5% topical timolol gel in the treatment of infantile haemangioma using an Analogue scale 0-10. Before and after treatment photographs will be evaluated by an independent panel using an analogue scale of 0 (no improvement and/or deterioration) to 10 (excellent improvement). Similarly, the parents/caregivers will be asked to evaluate the results of the treatment using this analogue scale. Treatment failure will be defined as: • A score of “0” on the analogue scale above • Cessation of the growth of the lesion without regression in size, or improvement in consistency or colour • Continued growth of the lesion • Development of ulceration of the lesion or worsening of ulceration

Timepoint [1]

Baseline at first clinic appointment. 4 weeks after commencement of treatment 12 weeks after commencement of treatment 24 weeks after commencement of treatment 52 weeks primary endpoint

Primary outcome [2]

Possible adverse effect. decreased heart rate and blood pressure. Assessed at clinic appointments using digital sphygmomanometer.

Timepoint [2]

Heart rate and blood pressure measured pre-commencement of treatment. 1 hour post treatment commencement 1 month after commencement of treatment 3 m0nths after commencement of treatment 6 months after commencement of treatment 12 months primary endpoint

Secondary outcome [1]

Nil

Timepoint [1]

Nil

Eligibility

Key inclusion criteria

Infants referred with a superficial IH(s) in the cosmetic sensitive areas (face and neck), perineum, buttock and axilla, who are not suitable for oral propranolol treatment.

Minimum age

1 Months

Maximum age

10 Months

Sex

Both males and females

Can healthy volunteers participate?

Key exclusion criteria

• Premature infants (less than 6 weeks corrected gestational age)
• IH on or near mucosal surface such as the eye, mouth and anus
• Any single lesion larger than 90mm² (e.g., 3x3cm)
• Multifocal IHs with a total surface area of more than 90mm²
• IH with ulceration >1x1cm
• IH with thickness >3mm
• Subcutaneous IH
• Any infants with contraindication to ß-blocker use, e.g.:
o Cardiac disease including: 2nd or 3rd degree atrioventricular heart block, bradycardia, cardiac failure, cardiogenic shock and congenital heart disease
o Congenital hyperthyroidism
o Hypersensitivity to any components of the medication
• Weight <3.5kg
• >10 months old
• Premature infants with a history of apnoea or chronic lung disease

Study design

Purpose of the study

Treatment

Allocation to intervention

Non-randomised trial

Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)

Methods used to generate the sequence in which subjects will be randomised (sequence generation)

Masking / blinding

Open (masking not used)

Who is / are masked / blinded?

Intervention assignment

Single group

Other design features

Phase

Phase 2

Type of endpoint/s

Safety/efficacy

Statistical methods / analysis

Recruitment

Recruitment status

Recruiting

Date of first participant enrolment

Anticipated

Actual

31/08/2018

Date of last participant enrolment

Anticipated

5/04/2023

Actual

Date of last data collection

Anticipated

7/06/2023

Actual

Sample size

Target

Accrual to date

Final

Recruitment outside Australia

Country [1]

New Zealand

State/province [1]

Funding & Sponsors

Funding source category [1]

Other Collaborative groups

Name [1]

Gillies McIndoe Research Institute

Address [1]

Gillies McIndoe Research Institute PO Box 7184 Newtown Wellington 6242

Country [1]

New Zealand

Primary sponsor type

Other Collaborative groups

Name

Gillies McIndoe Research Institute

Address

Gillies McIndoe Research Institute
PO Box 7184
Newtown
Wellington
6242
New Zealand

Country

New Zealand

Secondary sponsor category [1]

None

Name [1]

Address [1]

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

Central Health and Ethics Disability Committee

Ethics committee address [1]

Ministry of Health 
PO box 5013
Wellington
6011

Ethics committee country [1]

New Zealand

Date submitted for ethics approval [1]

11/04/2018

Approval date [1]

07/06/2018

Ethics approval number [1]

18/CEN/67

Summary

Brief summary

Most IHs do not require active treatment as they involute spontaneously over time. Up to 10-15% of IHs are problematic and require intervention during infancy- with the mainstay treatment being oral propanolol. Complications of propranolol treatment of IH have been reported in up to 31% of patients in a large meta-analysis, and therefore its use is restricted to problematic IHs. However, following spontaneous involution, many of the IH lesions can cause permanent cosmetic blemish, Timolol maleate is a topical ß-blocker that has been used for the treatment of glaucoma in the paediatric population for over 30 years. Research is yet to define the optimal duration of topical treatment of IH, with studies treating patients from 24 weeks up to 10 months, or until satisfactory improvement was noted. The best outcomes for topical timolol appears to be in patients with superficial IH who used the 0.5% timolol solution for a period of at least three months.This study investigates the efficacy of 0.5% topical timolol gel for the treatment of superficial IHs.

Trial website

Trial related presentations / publications

Public notes

Contacts

Principal investigator

Name

Dr Swee Tan

Address

Gillies McIndoe Research Institute PO Box 7184 Newtown Wellington 6242

Country

New Zealand

Phone

+64 216 31000

Fax

[email protected]

Contact person for public queries

Name

Ruth Watson-Black

Address

Gillies McIndoe Research Institute PO Box 7184 Newtown Wellington 6242

Country

New Zealand

Phone

+64277460776

Fax

[email protected]

Contact person for scientific queries

Name

Swee Tan

Address

Gillies McIndoe Research Institute PO Box 7184 Newtown Wellington 6242

Country

New Zealand

Phone

+64 216 31000

Fax

[email protected]

Data sharing statement

Will the study consider sharing individual participant data?

Yes

Will there be any conditions when requesting access to individual participant data?

Persons/groups eligible to request access:
• Researchers in Infantile Haemangioma (IH)

Conditions for requesting access:
• -

What individual participant data might be shared?

• All de-identified data collected during the trial will be shared.

What types of analyses could be done with individual participant data?

• No restrictions on analyses are predicted.

When can requests for individual participant data be made (start and end dates)?

From:
Data will be available after publication of the outcomes (and any protection of IP). No specific end date of when data will be shared has been decided as yet.

To:
-

Where can requests to access individual participant data be made, or data be obtained directly?

• Requests are made to the Principal Investigator via email or website
https://www.gmri.org/ or [email protected]
Dr Swee Tan
Gillies McIndoe Research Institute
PO Box 7184
Newtown
Wellington
6242
New Zealand

Are there extra considerations when requesting access to individual participant data?

What supporting documents are/will be available?

Doc. No.	Type	Citation	Link	Email	Other Details	Attachment
3988	Study protocol			[email protected]		Study-related document.docx
3989	Ethical approval			[email protected]		Study-related document.pdf

Results publications and other study-related documents

Documents added manually

No documents have been uploaded by study researchers.

Documents added automatically

No additional documents have been identified.

Trial Review

Documents added manually

Documents added automatically