Proliferating Infantile Hemangioma MedDRA version: 12.0 Level: LLT Classification code 10018814 Term: Haemangioma
Conditions
Interventions
Product Name: V0400SB03
Product Code: V0400SB03
Pharmaceutical Form: Oral solution
INN or Proposed INN: PROPRANOLOL HYDROCHLORIDE
CAS Number: 318-98-9
Concentration unit: mg/ml milligram(s)/millilitre
Sponsors
PIERRE FABRE DERMATOLOGIE
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 guardian(s) prior to performing any study procedures - The patient is 35 to 150 days old, inclusive, at inclusion - A facial proliferating IH (target hemangioma) with largest diameter of at least 1.5 cm, requiring systemic therapy is present. Are the trial subjects under 18? yes Number of subjects for this age range: 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 a medically unstable health status that may interfere with his/her ability to complete the study • The patient presents with one or more of the following medical conditions: Congenital hemangioma; Kasabach-Merritt syndrome; bronchial asthma; bronchospasm; hypoglycaemia (or at risk); 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; documented PHACES syndrome with central nervous system involvement • The patient has received at least one of the following medications contra-indicated in association with propranolol within 15 days of randomisation: o Anaesthetic agents (the exclusion period is shortened to 48 hours, if anaesthesia has been performed for diagnosis investigation (e.g. MRI,…) o Anti-arrhythmics o Calcium channel blockers o Chlorpromazine o Cimetidine o Clonidine o Digitalis glycosides o Dihydropyridines o Ergotamine o Hydralazine o Hypoglycaemic agents o Lignocaine o Prostaglandin synthetase inhibiting drugs o Sympathomimetic agents and parenteral adrenaline • The patient has previously been administered systemic or intralesional corticosteroids, vincristine, alfa-interferon, propranolol or other beta-blockers • The patient has previously been administered treatment for IH or surgical procedures have been performed to remove one or more IHs • The patient is known to have an allergy to beta-blockers • One or more of the following types of IH are present: o Life-threatening IH o Function-threatening IH (e.g. those causing impairment of vision, respiratory compromise caused by airway lesions, etc.) o Ulcerated IH (whatever the localisation) with pain and lack of response to simple wound care measures • Diagnosis of the soft tissue tumour as IH is not clinically certain, particularly in the case of sub-dermal lesions • The patient was born prematurely and has not yet reached his/her term equivalent age (e.g. an infant born 2 months prematurely cannot be included before the age of 2 months) • LVEF=40% • The patient is participating in another clinical study or the patient lives in the same household as an infant already participating in this or another study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To identify the appropriate dose and duration of propranolol and demonstrate its superiority over placebo in terms of improvement in the evolution of the target IH over 24 weeks.;Secondary Objective: Safety objective: To document the safety profile of the four regimens of propranolol in the treatment of IH in infants aged 1 to 5 months at inclusion. Long term objective: To study the long-term efficacy and safety of the four regimens of propranolol in the treatment of IH.;Primary end point(s): The primary efficacy criterion is the evolution of target IH from baseline to W24. The binary primary endpoint (improvement versus stabilisation/worsening) is based on four intra-patient blinded centralised independent qualitative assessments of photographs of the target IH at W12, W16, W20 and W24 compared to baseline, W12, W16 and W20, respectively. A treatment success will be defined as a centralised assessment of improvement between at least one pair of visits (W12, W16, W20 or W24 compared to baseline, W12, W16 and W20, respectively) without any assessments of worsening between the paired visits. A treatment failure will be defined as a centralised assessment of worsening between at least one pair of visits (W12, W16, W20 or W24 compared to baseline, W12, W16 or W20, respectively) or a stabilisation between all paired visits. The treatment failure definition will ensure that a worsening of IH between W12 and W24 can be detected even if the IH is in an improved state at W24 compared to baseline. Photographs of the target IH at each visit will be taken by the site investigators based on standardised procedures (see Appendix 17.4). A colour chart will be included in each photograph for colour and size calibration. Photographs will be uploaded to the eCRF for the study and the system will group them by patient-visit. A unique random number will be assigned to each group (photographs of the same patient at different visits will not have the same or consecutive n | — |
Countries
Czech Republic, France, Germany, Hungary, Italy, Lithuania, Spain
Outcome results
None listed