MedPath

Onreltea (Brimonidine) Gel In Pediatric Patients With Capillary Malformations

Phase 3
Terminated
Conditions
Capillary Malformations
Interventions
Registration Number
NCT02764411
Lead Sponsor
The Hospital for Sick Children
Brief Summary

Capillary Malformations (CM) affect a significant proportion of otherwise healthy children and may lead to psychological discomfort if left untreated. A significant proportion of untreated lesions undergo soft tissue thickening and darker discoloration later in life due to progressive ectasia of the affected vessels. While laser treatment is available, its use may be limited due to need for repeated sedation/general anesthetic use, partial response and cost.

The investigators propose to conduct an open-label, prospective, cohort study using Onreltea ( Brimonidine) gel for treatment of facial capillary malformations in children. The study medication will be applied topically on affected area of the skin daily for 12 weeks. Follow up visits will occur at at Week 1,4,8,12, and 16 to assess the efficacy and safety of the proposed treatment.

The study second aim is to explore the feasibility of conducting a multicenter placebo controlled study.

Detailed Description

The investigators are planning to enroll in the study 20 participants at SickKids.

It is a prospective, open label, cohort study. Patients enrolled in the study will be followed at the Hospital For Sick Children for 16 weeks. They will come for the study visits 6 times: in 1 week, 4,8,12, and 16 weeks after the treatment has been started. During each study visit the study investigators will assess any changes in the characteristics of CM lesion(s) captured by a Chromometer \*, Analogue Scale and Erythema Assessment tools. Participants or their parents will assess the changes at the final study visit (VAS and EA tools).

Patients will be provided with study medication for all duration of the study treatment (12 weeks).

The results of the treatment will be compared with the baseline data to evaluate the efficacy and safety of Onreltea (Brimonidine) gel in children with facial capillary malformations.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
6
Inclusion Criteria
  • diagnosis of facial capillary malformation (port-wine stain, PWS) made by a dermatologist
  • age: 12-17 years of age
  • weight > 45 kg
  • lesions with a surface area < 100 cm2
  • signed consent and assent for study participation
Exclusion Criteria
  • skin breakdown overlying the malformation due to other dermatological conditions (e.g. eczema, psoriasis)
  • current or treatment with laser the past 3 months
  • other topical treatments used within the past 4 weeks (e.g. rapamycin, corticosteroids, calcineurin inhibitors, etc)
  • known chronic renal or hepatic disorders
  • known cardiovascular disorders
  • other systemic medications that potentially interact with Brimonidine (opiates, chlorpromazine, methyphenidate, reserpine, etc)
  • mixed capillary/ venous or lymphatic malformations
  • known allergy to one of the constituents of Onreltea
  • pregnancy, or sexually active subjects of child-bearing potential (CBP), unwilling to use contraception during the study (such as barrier method, or oral contraceptives).

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Onreltea ( Brimonidine)Brimonidine 0.33% gelAll the patients enrolled in the study will apply Onreltea ( Brimonidine 0.33%) gel on the affected skin area for 12 weeks (from Day 0 to Week 12 visit).
Primary Outcome Measures
NameTimeMethod
The change in the color of the capillary malformation using Chroma meter values (Δa, ΔE) at 12 weeks.12 weeks

Measurement of erythema will be performed using Chroma Meter, CR-400, Konica, Minolta, Osaka, Japan. The meter readings will result in 3 values: L- refers to the relative light intensity, ranging from 0 (black) to +100 (white); a-captures color saturation, ranging from +60 (green) to -60 (red) and b- captures color spectrum from +60 (blue) to -60 (yellow). In most studies both, changes in a (Δa) and overall changes in the composite score (ΔE calculated as √((ΔL\*before-ΔL\*after)\^2+(Δa\*before-Δa\*after)\^2+(Δb\*before-ΔL\*after)\^2 ) are obtained.

Secondary Outcome Measures
NameTimeMethod
Changes in CEA scores at 12, 16 weeks compared to baseline12 and 16 weeks

A Clinician Erythema Assessment scale (CEA), consisting of a 0-4 numerical scale as follows:

0- clear skin, no erythema

1. almost clear skin, slight redness

2. mild erythema, definite redness

3. moderate erythema, marked redness

4. severe erythema, fiery redness

Correlation between iVAS, pVAS, CEA, pEA and Chroma Meter values1,4,8,12,16 weeks

pVAS - patient/parent's Visual Analogue Scale assessment; pEA- patient/parent Erythema Assessment

Percentage of patients achieving 75% and 100% resolution of the lesion12 weeks

by iVAS and chromo meter values

Frequency of observed and reported adverse events (AE)16 weeks

AE documented in patient diary and mentioned at each study visit

Changes in the color of the lesion (Δa, ΔE) at each follow up visit including the last visit at 16 weeks compared to baseline1,4,8,16 weeks

Same as in primary outcome measure

Changes in the iVAS at 12 and 16 weeks compared to baseline12 and 16 weeks

Investigator's assessment of changes on the Visual Analogue Scale ( iVAS)

Trial Locations

Locations (1)

The Hospital For Sick Children

🇨🇦

Toronto, Ontario, Canada

The Hospital For Sick Children
🇨🇦Toronto, Ontario, Canada
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