Randomized Study of Hormonal Regulation of Infantile Hemangioma

NCT ID: NCT00004436

Last Updated: 2015-03-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 1993-07-31
• Study Completion Date: 2000-06-30

Brief Summary

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.

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.

Conditions

Hemangioma

Study Design

• Allocation Method: RANDOMIZED
• Primary Study Purpose: TREATMENT

Interventions

leuprolide

Intervention Type: DRUG

prednisone

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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

• Minimum Eligible Age: 1 Month
• Maximum Eligible Age: 8 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Boston Children's Hospital

OTHER

Sponsor Role: collaborator

FDA Office of Orphan Products Development

FED

Sponsor Role: lead

Principal Investigators

Lois Hodgson Smith

Role: STUDY_CHAIR

Boston Children's Hospital

Other Identifiers

CH-B-FDR000967

Identifier Type: -

Identifier Source: secondary_id

199/13399

Identifier Type: -

Identifier Source: org_study_id