Clinical trial NCT02764411: Onreltea (Brimonidine) Gel In Pediatric Patients With Capillary Malformations
Official title: Onreltea (Brimonidine) Gel In Pediatric Patients With Capillary Malformations: A Prospective, Open-label, Cohort Study
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Capillary Malformations
Keywords: Capillary malformations
Onrealtea
port-wine stain
treatment
topical
Lead sponsor: The Hospital for Sick Children
Sponsor class: OTHER
Collaborators: name: Galderma R&D
First posted: 2016-05-06
Enrollment: 6
Sex: ALL
Minimum age (years): 12
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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).

Primary outcomes:
measure: The change in the color of the capillary malformation using Chroma meter values (Δa, ΔE) at 12 weeks.
timeFrame: 12 weeks
Secondary outcomes:
measure: Changes in the color of the lesion (Δa, ΔE) at each follow up visit including the last visit at 16 weeks compared to baseline
timeFrame: 1,4,8,16 weeks
measure: Changes in CEA scores at 12, 16 weeks compared to baseline
timeFrame: 12 and 16 weeks
measure: Changes in the iVAS at 12 and 16 weeks compared to baseline
timeFrame: 12 and 16 weeks
measure: Correlation between iVAS, pVAS, CEA, pEA and Chroma Meter values
timeFrame: 1,4,8,12,16 weeks
measure: Percentage of patients achieving 75% and 100% resolution of the lesion
timeFrame: 12 weeks
measure: Frequency of observed and reported adverse events (AE)
timeFrame: 16 weeks
Other outcomes:
measure: Predictors for a good response, defined as at least 50 % change in the erythema measured using Chroma Meter (a* after compared to a* before) at 12 weeks mark
timeFrame: 12 weeks
measure: Percentage difference in Chroma Meter values (L -relative light intensity, a - color saturation, b - color spectrum) between treated and untreated lesions (control)
timeFrame: 1,4,8,12 and 16 weeks
measure: Percentage of patients experiencing flare-up (defined as reoccurrence of the red discoloration) at the end of the study (16 weeks)
timeFrame: 16 weeks
Locations:
city: Toronto
country: Canada
facility: The Hospital For Sick Children
state: Ontario
Study officials:
affiliation: The Hospital for Sick Children
name: Elena Pope, MD
role: PRINCIPAL_INVESTIGATOR
References: