Study in Infants With Infantile Hemangioma to Compare Propranolol Gel to Placebo

A Randomised, Double Blind, Controlled, Multicentre Study in Infants With Infantile Hemangioma to Compare Propranolol Gel to Placebo.

There is an unsatisfied medical need for a first-line treatment of localized uncomplicated proliferating Infantile Hemangioma with a good benefit/risk profile.

Pierre Fabre Dermatologie has developed a new formulation of propranolol (V0400 GL 01A) which is a topical gel adapted to paediatric use.

The objective of this study is to evaluate topical propranolol efficacy and safety in the management of localized hemangioma.

Study Overview

• Status: Completed
• Conditions: Infantile Hemangioma
• Intervention / Treatment: Drug: propranolol gel; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 82
• Phase: Phase 2

Contacts and Locations

Study Locations

• France
  • Bordeaux, France, 33076
    • CHU Bordeaux Hôpital Pellegrin-Enfants - Unité de Dermatologie pédiatrique
  • Lille, France, 59020
    • Hôpital Saint Vincent de Paul - Dermatologie Pédiatrique
  • Lyon Bron, France, 69677
    • CHU Lyon Est Hôpital mère enfant - Consultation des angiomes
  • Marseille, France, 13385
    • CHU Timone - Service de dermatologie
  • Nantes, France, 44093
    • Hôpital Mère-Enfant - Service de Néonatologie et Réanimation Pédiatrique
  • Paris, France, 75517
    • CHU Necker enfants malades - Service de dermatologie
  • Saint-Etienne, France, 42055
    • CHU Saint-Etienne Hôpital Nord - Service de dermatologie
  • Toulouse, France, 31100
    • CHU Toulouse Hôpital des enfants - Département cardio-pédiatrique
  • Tours, France, 37044
    • Hôpital de Clocheville - Centre de Pédiatrie Gatien
• Poland
  • Gdańsk, Poland, 80-803
    • Pomorskie Centrum Traumatologii im. M. Kopernika w Gdańsku Klinika Chirurgii i Urologii Dzieci i Młodzieży GUMed
  • Lodz, Poland, 91-738
    • Uniwersytet Medyczny w Łodzi Klinika Chirurgii i Onkologii Dziecięcej
  • Warszawa, Poland, 04-730
    • Instytut "Pomnik-Centrum Zdrowia Dziecka", Klinika Onkologii
• Spain
  • Barcelona, Spain, 08025
    • Hospital Sant Pau de Barcelona
  • Madrid, Spain, 28009
    • Hospital Universitario Infantil Niño Jesus
  • Madrid, Spain, 28056
    • Hospital La Paz
  • Sevilla, Spain, 41013
    • Hospital Universitario Virgen Del Rocio
  • Valencia, Spain, 46014
    • Hospital General Universitario de Valencia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 11 months (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Main Inclusion Criteria:

• only one proliferating Infantile Hemangioma present anywhere on the body except on the head, the neck, the hands and on the diaper area, with largest diameter diameter ≥ 1cm and ≤ 5 cm.

Main Exclusion Criteria:

• more than one Infantile Hemangioma with largest diameter ≥ 1cm
• medically unstable health status that may interfere with his/her ability to complete the study
• Infantile Hemangioma requires, according to Investigator's judgment, a systemic treatment
• the patient has previously been administered treatment for IH or surgical and/or medical procedures (e.g. laser therapy) have been performed to treat the IH

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Topical administration twice daily
Experimental: propranolol gel	Drug: propranolol gel; Topical administration twice daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Complete/nearly complete resolution of the Infantile Hemangioma at week 12.	Complete/nearly complete resolution of the Infantile Hemangioma at week 12 compared to baseline	week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
On-site parent(s) or guardian(s) qualitative assessments of efficacy	categorical endpoints for Infantile Hemangioma evolution (4-points scale)	week 2 , week 4, week 8, week 12 and week 24
Persistence of efficacy 12 weeks after the end of treatment	Persistence of complete/nearly complete resolution of the Infantile Hemangioma at week 24 compared to week 12.	Week 24
Safety profile (descriptive analysis of AE)		Day 0, week 2, week 4, week 8, week 12 and week 24
Local tolerance of the propranolol gel(description over time by treatment group)		week 2, week 4, week 8 and week 12

Collaborators and Investigators

• Sponsor: Pierre Fabre Dermatology

Study record dates

Study Major Dates

• Study Start: January 1, 2012
• Primary Completion (Actual): May 1, 2013
• Study Completion (Actual): May 1, 2013

Study Registration Dates

• First Submitted: January 6, 2012
• First Submitted That Met QC Criteria: January 13, 2012
• First Posted (Estimate): January 19, 2012

Study Record Updates

• Last Update Posted (Estimate): October 30, 2014
• Last Update Submitted That Met QC Criteria: October 29, 2014
• Last Verified: October 1, 2014

More Information

Terms related to this study

• Keywords: neoplasm; physiological effects of drugs; Hemangioma; therapeutic uses; Propranolol gel; capillary neoplasm; vascular tissue; neoplasm by histologic type; pharmacologic actions; adrenergic beta-antagonists; adrenergic antagonist; adrenergics agent; neurotransmitter agent; molecular mechanism of pharmacological action
• Additional Relevant MeSH Terms: Skin Diseases; Neoplasms by Histologic Type; Neoplasms; Congenital Abnormalities; Skin Abnormalities; Neoplasms, Vascular Tissue; Hemangioma, Capillary; Port-Wine Stain; Hemangioma; Physiological Effects of Drugs; Adrenergic beta-Antagonists; Adrenergic Antagonists; Adrenergic Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Anti-Arrhythmia Agents; Antihypertensive Agents; Vasodilator Agents; Propranolol
• Other Study ID Numbers: V00400 GL 2 01 1A; 2011-003144-50 (EudraCT Number)