Official Title: “A Phase II Study of Imiquimod 5 % Cream for the Treatment of Hemangioma in Infancy”

Hemangiomas of infancy, the most common benign tumors of infancy, are congenital or early infancy lesions characterized by a rapid postnatal growth, with high expression of angiogenic stimulators for 9-18 months, followed by slow regression for 5-9 years. Current therapies for the hemangiomas are usually restricted to more severe forms due to the risks of adverse effects, inconvenience and cost. Nevertheless, a substantial amount of the psychological discomfort and morbidity can be caused by untreated hemangiomas, especially those in the face.

Recently, Imiquimod 5% cream has emerged as a safe an effective drug for several skin conditions that benefit from modulation of the activity of the immune system, such as common warts and various forms of the skin pre-cancerous and cancerous lesions. Small case reports series have suggest that it could also be useful in hemangiomas, possibly through the inhibition of the angiogenesis by local IFN production.This is a small, open label study of 16 patients to document the efficacy of the Imiquimod 5% cream in the treatment of hemangioma of infancy (primary outcome). IFN and plasma drug levels, as well as clinical examinations and blood studies, will be carried out to evaluate safety of the treatment (secondary outcome). bFGF and VEGF will be measured in blood and urine in order to study the diagnostic and predictive value of these pro-angiogenic factors in the response of hemangiomas to the treatment with Imiquimod (secondary outcome).

The study is a phase II clinical trial of a once a day application of Imiquimod 5% cream, 3 to 7 times per week for a maximum of four months. The study held at the Dermatology Clinic of Sainte-Justine Hospital, and was completed within a 20 months timeframe after IRB approval.

Study Type: Interventional

Study Design: Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Study Primary Completion Date: September 2006

Intervention(s) in this Clinical Trial

• Drug: Imiquimod 5% cream
  • Imiquimod 5% cream applied topical on hemangioma once a day , 3 to 7 times a week for a maximum of 4 months.

Primary Measures

• To document the efficacy of Imiquimod 5% cream in the treatment of hemangioma of infancy.
  • Time Frame: Cream is applied for 4 months. Visits occured at month 1, 2, 4, and 8.
    Safety Issue?: No

Secondary Measures

• IFN and plasma drug levels, as well as clinical examinations and blood studies, will be carried out to evaluate safety of the treatment.
  • Time Frame: 4 months of treatment. Doage done at each study visits (Month 1, 2 .4 and 8).
    Safety Issue?: Yes
• bFGF and VEGF will be measured in blood and urine in order to study the diagnostic and predictive value of these pro-angiogenic factors in the response of hemangiomas to the treatment with Imiquimod.
  • Time Frame: 4 months of treament with a follow-up at 8 months.
    Safety Issue?: No

Inclusion Criteria:

• Healthy infants aged 2-12 months.
• Superficial or mixed hemangiomas in proliferative phase (growing in size in the last 1-2 months).
• Hemangiomas must be less than 10X10 cm and must not be ulcerated.

Exclusion Criteria:

• Preterm infant (less than 36 weeks of gestation).
• Ulceration of hemangioma prior to treatment.
• Immunosuppression.
• Hemangioma located on the eyelid or perianal region.
• Prior treatment of the hemangioma.
• Concomitant diseases.
• Presence of multiple hemangiomas and/or hemangiomas that would require systemic drug treatment.
• Potential difficulties with follow-up (patient from another town,difficult access to the hospital , etc.).
• History of allergy to any of the components of the drug preparation.
• Hemangiomas more than 10X 10 cm or ulcerated before the start of the treatment.

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: 2 Months

Maximum Age for this Clinical Trial: 12 Months

Are Healthy Volunteers Accepted for this Clinical Trial?: No

Lead Sponsor: St. Justine's Hospital

Overall Clinical Trial Officials and Contacts

Catherine McCuaig, M.D. Principal Investigator CHU Sainte-Justine  

Information obtained from ClinicalTrials.gov on October 10, 2008

Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00601016

Study ID Number: 1-Mccuaig

ClinicalTrials.gov Identifier: NCT00601016

Health Authority: Canada: Health Canada