ANZCTR - Registration

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Trial registered on ANZCTR

Registration number

ACTRN12622000684729

Ethics application status

Approved

Date submitted

13/04/2022

Date registered

11/05/2022

Date last updated

3/10/2023

Date data sharing statement initially provided

11/05/2022

Type of registration

Prospectively registered

Titles & IDs

Public title

ECMT-154™ for the Topical Treatment of Eczema in Children

Scientific title

Randomised Controlled Trial of ECMT-154™ vs Vehicle Control for the Topical Treatment of Eczema in Children aged 2-12 YO

Secondary ID [1]

MBS03

Universal Trial Number (UTN)

u1111-1275-6008

Trial acronym

Linked study record

Health condition

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

Eczema

Condition category

Condition code

Skin

Dermatological conditions

Intervention/exposure

Study type

Interventional

Description of intervention(s) / exposure

Manuka-oil based 2% ECMT-154™ cream is applied topically, twice daily (morning and night) for 6 weeks. The amount required will be dependent on the individual child's eczema spread, with the participants' parent/guardian recommended to apply the treatment liberally to their eczema areas. A weekly participant diary will used by the participants' parent/guardian to monitor adherence, adverse events and to capture photographs of the representative eczema lesion.
Composition Ingredients: Propylene Glycol, Sodium lauryl sulfate, Stearyl alcohol, water, White soft paraffin, PEG 3350, PEG 300, 2% ECMT-154

Intervention code [1]

Treatment: Drugs

Comparator / control treatment

Vehicle Control
Contains the same ingredients as the active intervention without the 2% ECMT-154™.
Participants' parents and guardians will be advised to follow the same treatment application as the 2% ECMT-154™ study treatment cream. A weekly participant diary will used by the participants' parent/guardian to monitor adherence, adverse events and to capture photographs of the representative eczema lesion.
Composition Ingredients include: Propylene Glycol, Sodium lauryl sulfate, Stearyl alcohol, water, White soft paraffin, PEG 3350, PEG 300

Control group

Placebo

Outcomes

Primary outcome [1]

Difference in Patient Orientated Eczema Measure (POEM for proxy completion) scores

Timepoint [1]

Baseline. Week 6 post-intervention commencement

Secondary outcome [1]

Proportion of participants with a >=4 change in POEM score compared to baseline.

Timepoint [1]

Baseline, Week 3 and 6 post-intervention commencement.

Secondary outcome [2]

Difference in POEM scores at Week Six analysed per protocol

Timepoint [2]

Week 3 and Week 6 post-intervention commencement.

Secondary outcome [3]

Difference in PO-SCORAD score, assessing patient reported severity of eczema.

Timepoint [3]

Week 3 and Week 6 post-intervention commencement.

Secondary outcome [4]

Difference in dermatologist rated SCORAD score, clinical assessment of severity of eczema.

Timepoint [4]

Week 6 post-intervention commencement.

Secondary outcome [5]

The proportion of withdrawals will be assessed by audit of study records and through statistical analysis of estimation of relative risk.

Timepoint [5]

Weekly up to Week 6 post-intervention commencement.

Secondary outcome [6]

Proportion of treatment escalation between groups. Treatment escalation will be assessed initially by patient-reported via diary entry and then will be remotely assessed and documented by the MRINZ study doctor.

Timepoint [6]

Weekly up to Week 6 post-intervention commencement.

Secondary outcome [7]

Change in Children's Dermatology Life Quality Index (CDLQI) score for participants aged 4-12 years compared to baseline.

Timepoint [7]

Baseline, Week 3 and Week 6 post-intervention commencement.

Secondary outcome [8]

Acceptability of ECMT-154 in the treatment of eczema, rated in Numerical Rating Scale score (1 unacceptable to 10 acceptable)

Timepoint [8]

Week 6 post-intervention commencement.

Secondary outcome [9]

Proportions of cutaneous and systemic events between treatment groups. Will be classified as adverse events (AEs), these will initially be patient reported via diary entry and will then be remotely assessed and documented by the study doctor and if required the study dermatologist. All AE clinical outcomes will be recorded in the study CDMA and will be analysed and presented as an estimation of relative risk.

Timepoint [9]

Week 6 post-intervention commencement.

Secondary outcome [10]

Comparison of eczema intensity SCORAD scores (Part B) between blinded pharmacists to assess inter-rater variability of scoring.

Timepoint [10]

Baseline and Week 6 post-intervention commencement.

Secondary outcome [11]

Comparison of eczema intensity SCORAD scores (Part B) between blinded pharmacists and a blinded dermatologist to assess inter-rater variability of scoring.

Timepoint [11]

Baseline and Week 6 post-intervention commencement.

Secondary outcome [12]

Ability to use the participant taken photographs for AE review as assessed by a MRINZ Investigator using a numerical rating scale (1 unacceptable - 10 acceptable).

Timepoint [12]

Week 3 post-intervention commencement

Secondary outcome [13]

Ability to use participant taken photographs for clinical assessment scoring by teledermatology on numerical rating scale (1 unacceptable to 10 acceptable).

Timepoint [13]

Week 3 post-intervention commencement only

Secondary outcome [14]

Change in Infants' Dermatitis Quality of Life (IDQOL) score for participants aged 2-4 years compared to baseline.

Timepoint [14]

Baseline, week 3, and week 6 post-intervention commencement

Eligibility

Key inclusion criteria

- Willing and able to provide written informed consent (parent/guardian) and assent (for participants aged 5-12 years)
- Participant is aged between 2 and 12 years of age, inclusive
- Parent/guardian reported, doctor diagnosis of eczema
- Patient has a representative eczema lesion, located below the clavicle that is in an area they are comfortable to have photographed.
- Patient has a POEM score of ‘moderate to severe eczema’ (8 to 24)
- Participant and parent/guardian are willing to stop all moisturisers and other skin barrier cream or emulsion used on participant during the treatment period and replace it with the investigational
product assigned in this trial. Usual facial regimens and application of sunscreen is permitted.
- Participant and parent/guardian are willing to replace participants body wash/soap with aqueous cream as supplied at enrolment.
- Participant and their parent/guardian are able to attend a follow up visit 6 weeks after they enroll in the study. This will take place at a participating pharmacy or via telephone call if required due to COVID restrictions or unanticipated inability to attend in person.
- Participant and their parent/guardian are willing and able to complete the study and comply with all study instructions.
- Parent/guardian must have internet access and ability to take a once weekly photograph of the representative eczema lesion for completing the online study diaries.

Minimum age

2 Years

Maximum age

12 Years

Sex

Both males and females

Can healthy volunteers participate?

Key exclusion criteria

- Current requirement for prescription of antibiotics or corticosteroids for the treatment of any condition (with the exception of in and intranasal corticosteroids)
- Use of antihistamines, antibiotics or corticosteroids within the last two weeks (with the exception of inhaled and intranasal corticosteroids)
- Use of immunomodulatory medications taken for eczema within the last four weeks
- Use of bleach baths within the past seven days.
- Cutaneous mycotic or bacterial disease requiring a topical or systemic therapy
- Other skin condition which may affect the assessment of eczema
- History of allergy or hypersensitivity to study treatment ingredients
- Participation in a clinical study involving an investigational product during the last three months.
- Current cold/flu like symptoms, fever, or unexplained shortness of breath..
- Any other condition which, at the investigators’ discretion, is believed may present a safety risk or impact upon the ability of the
participant to complete the study.

Study design

Purpose of the study

Treatment

Allocation to intervention

Randomised controlled trial

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

Randomisation of participants takes place at the pharmacy, electronically within REDCap, during visit one. Participants will first be randomised 1:1 to receive either the investigational treatment or vehicle control. Investigators will not have access to the randomisation schedule. Investigational product and active control will be dispensed in matching plain packaging

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

A statistician generated block randomisation schedule, using a block size of four, will be uploaded by an unblinded member of the MRINZ informatics team to the REDCap randomisation module.

Masking / blinding

Blinded (masking used)

Who is / are masked / blinded?

The people analysing the results/data

Intervention assignment

Parallel

Other design features

This study is considered to be single-blind, with central investigators unaware of treatment allocation. Participants are not advised of their treatment allocation. However, they are considered potentially unblinded due to the distinctive smell of the IMP.

Phase

Phase 2

Type of endpoint/s

Safety/efficacy

Statistical methods / analysis

No power calculation for sample size is required for this pilot. However, enrolling 50 patients total (25 in each arm) will provide reasonable precision based on between 20 and 25 degrees of freedom for chi-squared based estimation of standard deviation within each arm.

Recruitment

Recruitment status

Completed

Date of first participant enrolment

Anticipated

18/07/2022

Actual

2/08/2022

Date of last participant enrolment

Anticipated

1/04/2023

Actual

18/07/2023

Date of last data collection

Anticipated

1/06/2023

Actual

13/09/2023

Sample size

Target

Accrual to date

Final

Recruitment outside Australia

Country [1]

New Zealand

State/province [1]

Wellington

Funding & Sponsors

Funding source category [1]

Commercial sector/Industry

Name [1]

Manuka Biosciences

Address [1]

Manuka Biosciences
300 Richmond Road,
Grey Lynn, Auckland, 1021

Country [1]

New Zealand

Primary sponsor type

Commercial sector/Industry

Name

Manuka Biosciences

Address

Manuka Biosciences
300 Richmond Road,
Grey Lynn, Auckland, 1021

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 Disability Ethics Committee (HDEC)

Ethics committee address [1]

Ministry of Health
133 Molesworth Street
PO Box 5013
Wellington
6011

Ethics committee country [1]

New Zealand

Date submitted for ethics approval [1]

13/04/2022

Approval date [1]

30/06/2022

Ethics approval number [1]

HDEC Review Reference: 2022 FULL 12496

Summary

Brief summary

This study is a randomised vehicle-controlled trial of 50 pediatric participants with moderate to severe eczema.  In this study investigators who obtain consent are not aware of the randomised treatment. 

Participants and their parent/guardian will be recruited from community pharmacies by pharmacy staff, social media advertising, or those referred from a local GP. Pharmacy staff will be trained as investigators by a MRINZ investigator. The baseline visit will consist of screening, consent, demographics, eczema scoring, photo capture of a representative eczema lesion, randomisation and supply of study medication. Participants' parent/guardian will be instructed to apply their treatment twice daily for six weeks. 

In this study central  investigators are not aware of the randomised treatment. Participants and their parent/guardian will not be informed of their treatment allocation, however the odour of ECMT-154™ will not be an exact match to vehicle control. Participants and their parent/guardian are therefore considered unblinded. Pharmacy investigators will be blind to treatment allocation, with the IMP delivered in masked and non-descript dispensing systems labelled with details on the product type and with application instructions only. 
 
Participants' parent/guardian will complete a weekly electronic diary for six weeks. The electronic diaries will capture symptom scores, treatment compliance, and adverse events. The participants with their parent/guardian will return to the pharmacy at the end of week six for a final study visit. The final visit will consist of eczema scoring, photo capture of a representative eczema lesion, treatment acceptability, adverse event collection, and concomitant medication collection. A survey completed by the parent/guardian, two weeks after the final visit, will capture information about adverse events post intervention period. 
 
The primary outcome for this study is improvement in patient-reported symptoms at six weeks. Secondary outcomes include improvement in patient-reported clinical signs, improvement in patient reported quality of life, and inter-rater validity of SCORAD (intensity section) between a blinded dermatologist and pharmacy investigators.

Trial website

Trial related presentations / publications

Public notes

Contacts

Principal investigator

Name

Dr Gabby Shortt

Address

Medical Research Institute of New Zealand,
Level 7 Clinical Services Building,
Wellington Hospital,
Riddiford Street, Newtown
Wellington, 6021

Country

New Zealand

Phone

+64 4 805 0261

Fax

gabby.shortt@mrinz.ac.nz

Contact person for public queries

Name

Gabby Shortt

Address

Medical Research Institute of New Zealand,
Level 7 Clinical Services Building,
Wellington Hospital,
Riddiford Street, Newtown
Wellington, 6021

Country

New Zealand

Phone

+64 4 805 0261

Fax

gabby.shortt@mrinz.ac.nz

Contact person for scientific queries

Name

Gabby Shortt

Address

Medical Research Institute of New Zealand,
Level 7 Clinical Services Building,
Wellington Hospital,
Riddiford Street, Newtown
Wellington, 6021

Country

New Zealand

Phone

+64 4 805 0261

Fax

gabby.shortt@mrinz.ac.nz

Data sharing statement

Will individual participant data (IPD) for this trial be available (including data dictionaries)?

No/undecided IPD sharing reason/comment

IPD sharing will not occur due to the nature of the pediatric assent procedures and pilot study status.

What supporting documents are/will be available?

No Supporting Document Provided

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

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