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)
Conditions
Interventions
Product Code: V0400GL01A
Pharmaceutical Form: Gel
INN or Proposed INN: PROPRANOLOL HYDROCHLORIDE
CAS Number: 318-98-9
Current Sponsor co
Sponsors
Pierre Fabre Dermatologie, Represented by Institut de Recherche Pierre Fabre
Eligibility
Sex/Gender
All
Inclusion 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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess propranolol gel efficacy in terms of complete/nearly complete resolution of the Infantile Hemangioma at W12 compared to baseline. ;Timepoint(s) of evaluation of this end point: Week 12; 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 ;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
| Measure | Time frame |
|---|---|
| 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 | — |
Countries
France, Poland, Spain
Contacts
Public ContactClinical Study Manager
Institut de Recherche Pierre Fabre
Outcome results
None listed