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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01056341




Registration number
NCT01056341
Ethics application status
Date submitted
24/01/2010
Date registered
26/01/2010
Date last updated
10/12/2015

Titles & IDs
Public title
Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy
Scientific title
A Randomised, Controlled, Multidose, Multicentre, Adaptive Phase II/III Study in Infants With Proliferating Infantile Hemangiomas (IHs) Requiring Systemic Therapy to Compare 4 Regimens of Propranolol (1 or 3 mg/kg/Day for 3 or 6 Months) to Placebo (Double Blind).
Secondary ID [1] 0 0
V00400 SB 201 Study
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Infantile Hemangioma 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Skin 0 0 0 0
Other skin conditions

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Propranolol
Treatment: Drugs - Placebo

Experimental: Propranolol oral solution -

Placebo comparator: Placebo -


Treatment: Drugs: Propranolol
Propranolol (1 or 3 mg/kg/day for 3 or 6 months)

Treatment: Drugs: Placebo
Treatment with placebo for 6 months
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Interim Analysis : Complete/Nearly Complete Resolution of the Target Infantile Hemangioma at Week 24 Compared to Baseline Based on the Intra-patient Blinded Centralized Independent Qualitative Assessments of Week 24 Photographs.
Timepoint [1] 0 0
6 months
Primary outcome [2] 0 0
Primary Analysis : Complete/Nearly Complete Resolution of the Target Infantile Hemangioma at W24 Compared to Baseline Based on the Intra-patient Blinded Centralized Independent Qualitative Assessments of W24 Photographs.
Timepoint [2] 0 0
6 months
Secondary outcome [1] 0 0
Success/Failure Based on the Investigator Qualitative Assessment of Complete Resolution at W48.
Timepoint [1] 0 0
6 months

Eligibility
Key inclusion criteria
* Proliferating IH (target hemangioma) requiring systemic therapy anywhere on the body except on the diaper area with largest diameter of at least 1.5 cm
Minimum age
35 Days
Maximum age
150 Days
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- The patient presents with one or more of the following medical conditions: Congenital hemangioma; Kasabach-Merritt syndrome; bronchial asthma; bronchospasm; hypoglycaemia (< 40 mg/dl or at risk); untreated phaeochromocytoma; hypotension (< 50/30 mmHg); second or third degree heart block; cardiogenic shock; metabolic acidosis; bradycardia (< 80 bpm); 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 previously been treated for IH, including any surgical and/or medical procedures (e.g. laser therapy)
* The patient is known to have a hypersensitivity to propranolol and/or any other beta-blockers
* One or more of the following types of IH are present:

* Life-threatening IH
* Function-threatening IH (e.g. those causing impairment of vision, respiratory compromise caused by airway lesions, etc.)
* Ulcerated IH (whatever the localisation) with pain and lack of response to simple wound care measures
* 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 (left ventricular systolic function) =40% and/or cardiomyopathy and/or hereditary arrhythmia disorder

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/01/2010
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/11/2013
Sample size
Target
Accrual to date
Final
512
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Eastern Clinical Research Unit - Box Hill Hospital - Box Hill
Recruitment hospital [2] 0 0
Royal Children's Hospital - Melbourne
Recruitment hospital [3] 0 0
Sydney Children's Hospital - Randwick
Recruitment postcode(s) [1] 0 0
- Box Hill
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment postcode(s) [3] 0 0
- Randwick
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Oregon
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
Canada
State/province [9] 0 0
Montreal
Country [10] 0 0
Canada
State/province [10] 0 0
Toronto
Country [11] 0 0
Czech Republic
State/province [11] 0 0
Brno
Country [12] 0 0
Czech Republic
State/province [12] 0 0
Prague
Country [13] 0 0
France
State/province [13] 0 0
Bordeaux
Country [14] 0 0
France
State/province [14] 0 0
Lyon
Country [15] 0 0
France
State/province [15] 0 0
Nantes
Country [16] 0 0
France
State/province [16] 0 0
Nice
Country [17] 0 0
France
State/province [17] 0 0
Paris
Country [18] 0 0
France
State/province [18] 0 0
St-Etienne
Country [19] 0 0
France
State/province [19] 0 0
Toulouse
Country [20] 0 0
France
State/province [20] 0 0
Tours
Country [21] 0 0
Germany
State/province [21] 0 0
Freiburg
Country [22] 0 0
Germany
State/province [22] 0 0
Hamburg
Country [23] 0 0
Germany
State/province [23] 0 0
Kiel
Country [24] 0 0
Germany
State/province [24] 0 0
München
Country [25] 0 0
Hungary
State/province [25] 0 0
Budapest
Country [26] 0 0
Italy
State/province [26] 0 0
Bari
Country [27] 0 0
Italy
State/province [27] 0 0
Milano
Country [28] 0 0
Lithuania
State/province [28] 0 0
Vilnius
Country [29] 0 0
Mexico
State/province [29] 0 0
Mexico CIty
Country [30] 0 0
New Zealand
State/province [30] 0 0
Auckland
Country [31] 0 0
New Zealand
State/province [31] 0 0
Hamilton
Country [32] 0 0
Peru
State/province [32] 0 0
Lima
Country [33] 0 0
Poland
State/province [33] 0 0
Gdansk
Country [34] 0 0
Poland
State/province [34] 0 0
Krakow
Country [35] 0 0
Poland
State/province [35] 0 0
Lodz
Country [36] 0 0
Poland
State/province [36] 0 0
Warszawa
Country [37] 0 0
Romania
State/province [37] 0 0
Bucharest
Country [38] 0 0
Romania
State/province [38] 0 0
Iasi
Country [39] 0 0
Romania
State/province [39] 0 0
Timisoara
Country [40] 0 0
Russian Federation
State/province [40] 0 0
Moscow
Country [41] 0 0
Russian Federation
State/province [41] 0 0
St-Peterburg
Country [42] 0 0
Spain
State/province [42] 0 0
A Coruna
Country [43] 0 0
Spain
State/province [43] 0 0
Barcelona
Country [44] 0 0
Spain
State/province [44] 0 0
Madrid
Country [45] 0 0
Spain
State/province [45] 0 0
Sevilla
Country [46] 0 0
Spain
State/province [46] 0 0
Valencia

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pierre Fabre Dermatology
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
There is an unsatisfied medical need for a first-line treatment of proliferating IHs with a good benefit/risk profile. Based on the recent findings of encouraging results obtained with propranolol in a series of infants with severe Infantile Hemangioma (IH), propranolol is expected to be of significant benefit in the management of the condition. The present study has been designed to confirm efficacy of propranolol in severe IH by demonstrating superiority over placebo and to document the safety profile of propranolol in this indication.
Trial website
https://clinicaltrials.gov/study/NCT01056341
Trial related presentations / publications
Leaute-Labreze C, Dumas de la Roque E, Hubiche T, Boralevi F, Thambo JB, Taieb A. Propranolol for severe hemangiomas of infancy. N Engl J Med. 2008 Jun 12;358(24):2649-51. doi: 10.1056/NEJMc0708819. No abstract available.
Sans V, de la Roque ED, Berge J, Grenier N, Boralevi F, Mazereeuw-Hautier J, Lipsker D, Dupuis E, Ezzedine K, Vergnes P, Taieb A, Leaute-Labreze C. Propranolol for severe infantile hemangiomas: follow-up report. Pediatrics. 2009 Sep;124(3):e423-31. doi: 10.1542/peds.2008-3458. Epub 2009 Aug 10.
Leaute-Labreze C, Hoeger P, Mazereeuw-Hautier J, Guibaud L, Baselga E, Posiunas G, Phillips RJ, Caceres H, Lopez Gutierrez JC, Ballona R, Friedlander SF, Powell J, Perek D, Metz B, Barbarot S, Maruani A, Szalai ZZ, Krol A, Boccara O, Foelster-Holst R, Febrer Bosch MI, Su J, Buckova H, Torrelo A, Cambazard F, Grantzow R, Wargon O, Wyrzykowski D, Roessler J, Bernabeu-Wittel J, Valencia AM, Przewratil P, Glick S, Pope E, Birchall N, Benjamin L, Mancini AJ, Vabres P, Souteyrand P, Frieden IJ, Berul CI, Mehta CR, Prey S, Boralevi F, Morgan CC, Heritier S, Delarue A, Voisard JJ. A randomized, controlled trial of oral propranolol in infantile hemangioma. N Engl J Med. 2015 Feb 19;372(8):735-46. doi: 10.1056/NEJMoa1404710.
Public notes

Contacts
Principal investigator
Name 0 0
Christine Labreze, MD
Address 0 0
Hopital de Bordeaux
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01056341