Study in infants with infantile hemangioma to compare propranolol gel to placebo.

• Register: EUCTR
• Last refreshed on: 8 October 2021
• Main ID: EUCTR2011-003144-50-ES
• Date of registration: 14/10/2011
• Prospective Registration: Yes
• Primary sponsor: Pierre Fabre Dermatologie, Represented by Institut de Recherche Pierre Fabre
• Public title: Study in infants with infantile hemangioma to compare propranolol gel to placebo.
• Scientific title: A randomised, double blind, controlled, multicentre study in infants with infantile hemangioma to compare propranolol gel to placebo
• Date of first enrolment: 19/12/2011
• Target sample size: 80
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-003144-50
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

France, Poland, Spain

Contacts

• Name: Clinical Study Manager
• Affiliation: Institut de Recherche Pierre Fabre

• Name: Clinical Study Manager
• Affiliation: Institut de Recherche Pierre Fabre

Key inclusion & exclusion criteria

Inclusion criteria:
A patient will be eligible if he/she meets all of the following criteria:
? Written informed consent(s) for study participation and the use of the patient?s images are obtained according to national regulations from the patient?s parent(s) or legal guardian(s) prior to performing any study procedures.
? The patient is 35 to 150 days old inclusive, at inclusion.
? Only one proliferating IH is present anywhere on the body except on the head, the neck, the hands and on the diaper area, with largest diameter ? 1cm and ? 3 cm.
? If required by national regulations, patient registered with a social security or health insurance system and/or whose parent(s) or legal guardian(s) is (are) registered with a social security or health insurance system.
Are the trial subjects under 18? yes
Number of subjects for this age range: 80
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
A patient will be ineligible if he/she meets any of the following criteria:
? The patient has got more than one IH with largest diameter ? 1cm
? The patient has a medically unstable health status that may interfere with his/her ability to complete the study.
? IH requires, according to Investigator?s judgment, a systemic treatment.
? The patient is known to have one or more of the following medical conditions: bronchial asthma; bronchospasm; hypoglycaemia; untreated phaeochromocytoma; hypotension; second or third degree heart block; cardiogenic shock; metabolic acidosis; bradycardia; severe peripheral arterial circulatory disturbances; Raynaud?s phenomenon; sick sinus syndrome; uncontrolled heart failure or Prinzmetal?s angina.
? The patient has received at least one of the following prohibited medications within 14 days before randomisation:
- Cardiovascular treatments: anti-arrhythmics, calcium channel blockers, ACE inhibitors, inotropic agents, vasodilators (hydralazine hydrochloride?), clonidine?
- Hypoglycaemic agents or drugs able to induce hypoglycaemia.
- Non-steroid anti-inflammatory drugs (NSAIDs) at anti-inflammatory dose.
- Sympathomimetic agents and parenteral adrenaline.

? The patient has previously been administered systemic, intralesional or topical corticosteroids, vincristine, alfa-interferon, imiquimod, propranolol or other beta-blockers.
? 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.
? The patient?s mother has been breastfeeding the patient while she was also being treated with beta-blockers (including propranolol) or, she has been breastfeeding the patient within 14 days of randomisation while she was also being treated with systemic (oral, intra-venous or intra-muscular) corticosteroids, vincristine or alfa-interferon.
? The patient is known to have a hypersensitivity to propranolol and/or any other beta-blockers and/or any ingredient of the test product.
? Patient with congenital hemangioma
? Patient with ulcerated IH.
? Patient with heart rate < 80 bpm at inclusion
? Patient with blood pressure < 50/30 mmHg at inclusion
? Patient with known renal impairment.
? Patient with known cardiac conditions that may predispose to heart block.
? The patient is participating in another clinical study or has received treatment with known remnant effects or undergone investigation liable to interfere with the present clinical trial.
? The patient has sibling already participating to this clinical study
? Parent(s) or legal guardian(s) who cannot be contacted by telephone in case of emergency.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Proliferating localised uncomplicated infantile hemangioma not requiring systemic therapy
MedDRA version: 14.0 Level: LLT Classification code 10019400 Term: Hemangioma of skin and subcutaneous tissue System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

Intervention(s)

Product Code: V0400GL01A
Pharmaceutical Form: Gel
INN or Proposed INN: PROPRANOLOL HYDROCHLORIDE
CAS Number: 318-98-9
Current Sponsor code: V0400
Concentration unit: mg/g milligram(s)/gram
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Gel
Route of administration of the placebo: Cutaneous use

Primary Outcome(s)

Primary end point(s): The primary efficacy criterion is the evolution of IH from baseline to W12. The binary primary endpoint (success/failure) will be evaluated based on the intra-patient blinded centralised independent qualitative assessments of W12 photographs of the IH compared to baseline. A treatment success will be defined as a centralised assessment of complete/nearly complete resolution of the IH at W12 compared to baseline, where nearly complete resolution is defined as a minimal degree of telangiectasis, erythema, skin thickening, soft tissue swelling and/or distortion of anatomical landmarks.

Secondary Objective: Efficacy objectives:
To assess propranolol gel efficacy in terms of complete/nearly complete resolution of the IH at W4, W8 and Week 24 compared to baseline
To evaluate the parent(s) or guardian(s) on-site qualitative assessments
To assess the persistence of efficacy 12 weeksafter the end of treatment.

Safety objective:
To assess the safety profile and the local tolerance of the propranolol gel

Timepoint(s) of evaluation of this end point: Week 12

Main Objective: To assess propranolol gel efficacy in terms of complete/nearly complete resolution of the Infantile Hemangioma at W12 compared to baseline.

Secondary Outcome(s)

Secondary end point(s): Secondary efficacy criteria:
Centralised assessments of complete/nearly complete resolution of the IH at W4, W8 and W24 compared to baseline,.
? Investigator on-site qualitative assessments
- At each scheduled post-baseline visit compared to baseline categorical endpoints for complete/nearly complete resolution
- At week 4, week 8, week 12 and week 24 compared to baseline categorical endpoints for IH evolution (4-points scale: complete resolution, improvement, stabilisation, worsening)
- At each scheduled post-baseline visit compared to the previous schedule visit, categorical endpoints for IH evolution (4-points scale: complete resolution, improvement, stabilisation, worsening)
? Parent(s) or guardian(s) on-site qualitative assessments at each scheduled post-baseline visit compared to the previous scheduled visit
- Categorical endpoints for IH evolution (4-points scale: complete resolution, improvement, stabilisation, worsening)

Persistence of efficacy criteria:
? Persistence of complete/nearly complete resolution of the IH at W24 compared to W12 based on centralised assessments.
? Investigator qualitative assessments by categorical endpoints for hemangioma evolution (4-points scale: complete resolution, improvement, stabilisation, worsening) at Week 24 (3 months after end of treatment).

Safety criteria:
? Safety endpoints will be based on the following evaluations:
- Adverse events (AE)
- Height, weight, head circumference, temperature, heart rate, blood pressure, respiratory rate, pulmonary auscultation, liver palpation and global physical examinations (all visits)
? Local tolerability
- Very good tolerance : no subjective sign or physical sign of local side effects
- Good tolerance : transitory subjective signs, without physical sign or necessary modification of the frequency of product application
- Poor tolerance : persisting subjective signs or physical signs of local side effects, leading to modification of the frequency of the product application but no treatment discontinuation
- Very poor tolerance: subjective and/or physical signs leading to treatment discontinuation.

Timepoint(s) of evaluation of this end point: Secondary efficacy criteria: W4, W8, W12 and W24 compared to baseline
Persistence of efficacy criteria: W24 compared to W12

Secondary ID(s)

2011-003144-50-FR

V00400GL2011A

Source(s) of Monetary Support

PIERRE FABRE DERMATOLOGIE

Ethics review

Status: Approved
Approval date: 15/12/2011
Contact:

Results

• Results available: Yes
• Date Posted: 08/07/2016
• Date Completed: 09/05/2013
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-003144-50/results