High risk infantile haemangioma MedDRA version: 17.1 Level: PT Classification code 10018814 Term: Haemangioma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Trade Name: HEMANGIOL
Pharmaceutical Form: Oral solution
INN or Proposed INN: PROPRANOLOL HYDROCHLORIDE
CAS Number: 318-98-9
Current Sponsor code: V0400SB
Other descriptive name: (2RS)-1-[(1-methyleth
Sponsors
Pierre Fabre Dermatologie represented by IRPF
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age 35-150 days old inclusive, also subject born prematurely but of his/her term equivalent age at inclusion (corrected age) 2. High risk IH in proliferative phase (target hemangioma): - life-threatening IH - peri-orbital, nasal, labial, laryngo-tracheal, limb joints IHs with functional impact or at risk of functional impact - disfiguring IH (IH > 5cm, glabella location, nasal location, philtrum location, central chin location, central cheek location, labial IH with mouth deformities) - ulcerated IH not responding to simple wound care measures, located anywhere on the body 3. If required by national regulations, registered with a social security or health insurance system and/or whose parent(s) or legal guardian(s) was (were) registered with a social security or health insurance system 4. Written informed consent(s) for study participation and the use of the subject?s images obtained according to national regulations from the subject?s parent(s) or guardian(s) prior to performing any study procedures Are the trial subjects under 18? yes Number of subjects for this age range: 45 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: 5. Medically unstable health status that may interfere with his/her ability to complete the study, especially acute broncho-pulmonary abnormality 6. Presence of one or more of the following medical conditions: - Congenital hemangioma - Kasabach-Merritt syndrome - PHACE syndrome - Hepatic hemangioma - Asthma - History of bronchospasm - At risk of hypoglycaemia according to the medical file of the subject - Phaeochromocytoma - Hypotension (SBP/DBP 5.0 mmol/L (only measured for subjects presenting an IH with ulcerated area >= 3 cm) 8. Subject?s target hemangioma treated by LASER therapy within the past month, 9. The subject (and/or the mother if she is breastfeeding the subject) has received at least one of the following prohibited medications within 14 days before first study drug administration: - Corticosteroids by systemic (oral, intra-venous or intra-muscular), intra-lesional or topical route - Imiquimod - Vincristine - Alfa-interferon - Propranolol or other beta-blockers - Cardiovascular treatments: . bradycardia-inducing calcium channel blockers (diltiazem, verapamil, bepridil), . anti-arrhythmics (propafenone, quinidine, amiodarone, lidocaine), . inotropic agents (digitalis glycosides), . dihydropyridines (amlodipine, felodipine, isradipine, lacidipine, lercanidipine, manidipine, nicardipine, nifedipine, nitrendipine, etc?), . antihypertensives (ACE Inhibitors, angiotensin II-receptors antagonists, diuretics, alpha-blockers whatever the indication, centrally-acting antihypertensives, reserpine, etc?) - Drugs inducing orthostatic hypotension (nitrates derivatives, type 5-phosphodiesterase inhibitors, tricyclic antidepressants, antipsychotics, dopaminergic agonists, levodopa, amifostine, baclofen, etc?) - Non-steroid anti-inflammatory drugs (NSAIDs) at anti-inflammatory dose - Enzyme inducers (rifampicin, phenobarbital,?) - Hypoglycaemic agents or drugs able to induce hypoglycaemia - Lipid lowering agents (cholestyramine, colestipol) - Halogenated anesthetic agents, lidocaine (exclusion period shortened to 48 hours if anaesthesia has been performed for diagnosis investigation, e.g. MRI?) 10. Hypersensitivity to any study drug ingredient 11. Hypersensitivity any beta-blocker 12. Medical history of anaphylactic reaction
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To document the efficacy of Hemangiol administered during at least 6 months and up to a maximum of 12 months of age in infants with high risk IH.;Secondary Objective: To document - the safety of Hemangiol. - the persistence of IH response up to 3 months after treatment interruption. - the efficacy of Hemangiol re-administered during up to 6 months in case of hemangioma relapse based on investigator?s judgment. - the impact of Hemangiol on family burden and quality of life linked to the IH.;Primary end point(s): Success, defined by investigator, at the end of initial treatment period. A subject - who is prematurely withdrawn for inefficacy or safety reason with a related AE leading to definitive study drug discontinuation (before the end of initial treatment period), - and/or - receiving (before the end of initial treatment period), prohibited treatments used to treat target IH (systemic [oral, intra-venous or intra-muscular], intra-lesional or topical corticosteroids, imiquimod, vincristine, alfa-interferon, beta-blockers other than the study treatment), in usual conditions for treating IH with appropriate formulation, dose and treatment duration, will be considered for statistical analysis as treatment failure, whatever the evaluation of investigator. Analyses of primary outcome measure: - Primary analysis: number, percentage and 95% CI of success on the FAS. - Supportive analysis: same analysis on the PP set.;Timepoint(s) of evaluation of this end point: End of the initial treatment period: 6 months minimum to 11 months maximum | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): All analyses on the secondary outcome measures will be performed on the FAS. - Success at each visit (defined in the same way than the primary outcome measure): . On the initial treatment period, number (% and 95% CI) of success at each visit . Persistence of effect: on the Follow-Up period, number (% and 95% CI) of success on the subset of FAS who are in success at the end of initial treatment period. . On the period of re-initiation of treatment, number (% and 95% CI) of success on the subset of FAS who needed to be re-treated. - Time to first sustained success from D0 up to the end of study Kaplan-Meier cumulative incidence estimates were provided at each time-point where the success assessment was scheduled for two subsets of FAS: . Subset of patient in FAS with only the initial treatment period. . Subset of patient in FAS with the initial treatment and re-initiation period. - Three-point evolution (improvement/stabilization/worsening) of the target IH compared to baseline evaluation: . On the initial treatment period, qualitative description at each visit . On the Follow-Up period, qualitative description on the subset of FAS who are in improvement at the end of initial treatment period. . On the period of re-initiation of treatment, qualitative description on the subset of FAS who needed to be re-treated. - Time to first sustained improvement (compared to the previous visit) from D0 up to the end of study: .same analyses performed on the time to first sustained success. - Time to first sustained improvement (compared to the baseline) from D0 up to the end of study . same analyses performed on the time to first sustained success. - Re-initiation of treatment: . number (%) of patients needed to be re-treated on the subset of FAS who are in success at the end of initial treatment period. - Investigator?s qualitative assessment of IH characteristics: . description by visit. - Functional impact of ta | — |
Countries
Spain
Contacts
Public ContactClinical Trial Information Desk
PIERRE FABRE MEDICAMENT
Outcome results
None listed