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Randomized Study of Hormonal Regulation of Infantile Hemangioma

This study has been completed.
Boston Children’s Hospital
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: October 18, 1999
Last updated: March 24, 2015
Last verified: August 2000

OBJECTIVES: I. Evaluate the clinical efficacy of leuprolide, a gonadotropin-releasing hormone agonist (GnRHa), in treating infants with vision-endangering or large, disfiguring hemangiomas.

II. Assess the impact of GnRHa on growth and development during infancy. III. Assess the safety of GnHRa in these patients.

Condition Intervention
Drug: leuprolide
Drug: prednisone

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 30
Study Start Date: July 1993
Estimated Study Completion Date: June 2000
Detailed Description:

PROTOCOL OUTLINE: This is a randomized, placebo-controlled study. Patients are stratified according to gender and by position of the lesion (periorbital vs nonperiorbital).

All patients receive oral prednisone daily for 3 weeks. Patients are then randomized to receive either placebo or leuprolide IM every 3 weeks, while continuing oral prednisone. Tumors are assessed at 1, 3, and 6 weeks. If the tumor is not responding, the leuprolide will be administered every 2 weeks. Tumors are reassessed at 3 and 6 months, at which point the treatment is stopped. Responding patients are observed every 3 weeks for 3 months. If the tumor begins to grow again, leuprolide may be administered for another 3 months. Patients whose tumors grow rapidly during treatment may crossover to the alternate therapy, repeat the leuprolide or prednisone therapy, or undergo surgical excision.


Ages Eligible for Study:   1 Month to 8 Months   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
  • Presence of hemangioma meeting at least one of the following criteria: Vision-threatening because of induced astigmatism or occlusion of the visual axis or proptosis Severe anatomic distortion compromising function of an organ or creating an unacceptable cosmetic outcome Other complications, e.g., Kasabach-Merritt consumptive coagulopathy, high-output heart failure, etc.
  • No lesions that are clearly regressing before therapy
  • No vascular malformations other than juvenile hemangiomas
  Contacts and Locations
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Please refer to this study by its identifier: NCT00004436

Sponsors and Collaborators
FDA Office of Orphan Products Development
Boston Children’s Hospital
Study Chair: Lois Hodgson Smith Boston Children’s Hospital
  More Information Identifier: NCT00004436     History of Changes
Other Study ID Numbers: 199/13399  CH-B-FDR000967 
Study First Received: October 18, 1999
Last Updated: March 24, 2015
Health Authority: United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:
cardiovascular and respiratory diseases
rare disease

Additional relevant MeSH terms:
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Anti-Inflammatory Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Fertility Agents, Female
Fertility Agents
Reproductive Control Agents processed this record on October 28, 2016