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Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pierre Fabre Dermatology
ClinicalTrials.gov Identifier:
NCT01056341
First received: January 24, 2010
Last updated: May 21, 2014
Last verified: May 2014
Results First Received: April 9, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Infantile Hemangioma
Interventions: Drug: Propranolol
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment

510 patients were included in the study (informed Consent Form signed). Among these 510 patients, 460 were randomized and 50 were screen failure

  • At least one inclusion criterion not met in 26 cases
  • Parent's decision in 10 cases
  • Other reasons in 15 cases.

One patient was not included for two reasons (Parent's decision and other reason)


Reporting Groups
  Description
Placebo Placebo: Treatment with placebo for 6 months
Propranolol 1mg/kg/d 3 Months Propranolol oral solution 1mg/kg/day for 3 months, then placebo for 3 months
Propranolol 1 mg/kg/d 6 Months Propranolol oral solution 1mg/kg/day for 6 months
Propranolol 3 mg/kg/d 3 Months Propranolol oral solution 3mg/kg/day for 3 months, then placebo for 3 months
Propranolol 3 mg/kg/d 6 Months Propranolol oral solution 3mg/kg/day for 6 months

Participant Flow:   Overall Study
    Placebo     Propranolol 1mg/kg/d 3 Months     Propranolol 1 mg/kg/d 6 Months     Propranolol 3 mg/kg/d 3 Months     Propranolol 3 mg/kg/d 6 Months  
STARTED     55     99     103     101     102  
Interim Analysis     25     41     41     40     43  
COMPLETED     19     63     88     65     88  
NOT COMPLETED     36     36     15     36     14  
Treatment Intolerance                 0                 2                 0                 2                 0  
Lack of Efficacy                 32                 30                 7                 25                 9  
Other reasons                 4                 4                 8                 9                 5  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Among the 460 randomized patients, 4 were not treated: 2 because of parent(s)'/guardian's decision (1 in 1mg/kg/day 6 months arm,1 in 3 mg/kg/day 3 months arm), 2 because of absence of adequate treatment on site (1 in 1 mg/kg/day 3 months arm and 1 in 3mg/kg/day 6 months arm).

Reporting Groups
  Description
Placebo Placebo: Treatment with placebo for 6 months
Propranolol 1mg/kg/d 3 Months Propranolol oral solution 1mg/kg/day for 3 months, then placebo for 3 months
Propranolol 1 mg/kg/d 6 Months Propranolol oral solution 1mg/kg/day for 6 months
Propranolol 3 mg/kg/d 3 Months Propranolol oral solution 3mg/kg/day for 3 months, then placebo for 3 months
Propranolol 3 mg/kg/d 6 Months Propranolol oral solution 3mg/kg/day for 6 months
Total Total of all reporting groups

Baseline Measures
    Placebo     Propranolol 1mg/kg/d 3 Months     Propranolol 1 mg/kg/d 6 Months     Propranolol 3 mg/kg/d 3 Months     Propranolol 3 mg/kg/d 6 Months     Total  
Number of Participants  
[units: participants]
  55     98     102     100     101     456  
Age  
[units: DAYS]
Mean ± Standard Deviation
  103.91  ± 31.06     103.58  ± 33.07     102.65  ± 30.12     107.53  ± 30.14     101.63  ± 31.00     103.85  ± 31.02  
Gender  
[units: participants]
           
Female     38     68     70     79     70     325  
Male     17     30     32     21     31     131  
Region of Enrollment  
[units: participants]
           
United States     6     13     13     11     10     53  
Spain     5     9     14     17     14     59  
Lithuania     1     7     4     1     5     18  
Russian Federation     0     1     0     1     1     3  
Italy     1     0     0     0     1     2  
France     21     25     18     22     28     114  
Czech Republic     2     1     3     1     1     8  
Hungary     1     2     4     2     2     11  
Mexico     0     2     0     3     0     5  
Canada     2     5     6     3     2     18  
Poland     4     4     6     8     4     26  
Romania     2     2     1     0     2     7  
Peru     1     5     9     8     12     35  
Australia     1     5     14     5     7     32  
Germany     8     17     9     17     9     60  
New Zealand     0     0     1     1     3     5  
Infantile hemangioma localization  
[units: participants]
           
FACE     40     71     72     64     71     318  
NONE FACE     15     27     30     36     30     138  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   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.   [ Time Frame: 6 months ]

2.  Primary:   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.   [ Time Frame: 6 months ]

3.  Secondary:   Success/Failure Based on the Investigator Qualitative Assessment of Complete Resolution at W48.   [ Time Frame: 6 months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Dr. Jean-Jacques VOISARD - General Manager -
Organization: Pierre Fabre Dermatologie
phone: +33 5 63 58 88 00
e-mail: jean.jacques.voisard@pierre-fabre.com


Publications:

Responsible Party: Pierre Fabre Dermatology
ClinicalTrials.gov Identifier: NCT01056341     History of Changes
Other Study ID Numbers: V00400 SB 201 Study
Study First Received: January 24, 2010
Results First Received: April 9, 2014
Last Updated: May 21, 2014
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medical Devices
Italy: Ministry of Health
Spain: Ministry of Health
Romania: National Medicines Agency
Peru: Instituto Nacional de Salud
Mexico: Federal Commission for Sanitary Risks Protection
New Zealand: Ministry of Health
Australia: Department of Health and Ageing Therapeutic Goods Administration
Poland: Ministry of Health
Czech Republic: State Institute for Drug Control
Hungary: National Institute of Pharmacy
Lithuania: State Medicine Control Agency - Ministry of Health
Russia: Ministry of Health of the Russian Federation