Corticosteroids With Placebo Versus Corticosteroids With Propranolol Treatment of Infantile Hemangiomas (IH)

NCT01074437 | Terminated Phase 2 Results DMC

• 1 other identifier: 12901
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective randomized, double-blind study to compare the clinical efficacy of infantile hemangioma treatment using propranolol with corticosteroids as compared to therapy with corticosteroids and placebo. We hypothesize that a two-month treatment period with propranolol plus corticosteroids is more effective at reducing infantile hemangioma size and vascularity when compared to corticosteroids used without propranolol for the same time period.

Conditions

Hemangioma

Outcome Measures

Primary Outcomes (2)

• Compare Changes in IH Size and Vascularity for the Two Treatment Groups

  This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.

  1, 2, and 6 months after treatment initiation

• Lesion Regression

  measure of change in lesion area or volume

  12 months

Secondary Outcomes (3)

• Determine Therapeutic Response of IH to Propranolol Among Patients Who Switch to Corticosteroids Plus Propranolol Therapy After Failing to Respond to Corticosteroids Alone.

  1, 2, and 6 months after treatment initiation

• Assess the Safety of Propranolol With Corticosteroids and Corticosteroids Alone in the Treatment of IH.

  1, 2 and 6 months after treatment initiation

• Demonstrate How Duplex Scanning to Assess Blood Vessel Density and Qualitative Color Ratings of Cutaneous Lesions From Photographs Can be Used to Measure and Quantify Changes in IH Size and Vascularity in a Clinically Relevant Manner.

  1, 2 and 6 months after treatment initiation

Study Arms (2)

Group A: Corticosteroid with Placebo

OTHER

Group A will receive oral, liquid Prednisolone, which is the standard corticosteroid that we use here at Seattle Children's, and oral liquid placebo. The dose of prednisolone that Group A will receive will be 1-2mg/kg/day for 7 days and then the dose will be slowly reduced and then stopped after 3 weeks. This is a standard dose for IH treatment. Gastric prophylaxis (Zantac) will be given to help prevent any stomach problems associated with prednisolone. This treatment will be given for two months, as is our standard practice.

Drug: Prednisolone (Corticosteroid); Drug: Placebo

Group B: Corticosteroid with Propranolol

OTHER

Group B will receive oral liquid prednisolone, and oral propranolol. As in Group A, the dose of prednisolone will be 1-2mg/kg/day for 7 days and then the dose will be slowly reduced and then stopped after 3 weeks. Oral liquid propranolol will be dosed at 2 mg/kg/day, following initiation in the Cardiology Clinic. Gastric prophylaxis (Zantac) will be given to help prevent any stomach problems associated with prednisolone.

Drug: Prednisolone (Corticosteroid); Drug: Propranolol

Interventions

Prednisolone (Corticosteroid) DRUG

Oral liquid prednisolone. Dose: 1-2mg/kg/day for 7 days, and then dose will be slowly reduced and then stopped after 3 weeks.

Also known as: Corticosteroids

Group A: Corticosteroid with Placebo; Group B: Corticosteroid with Propranolol

Propranolol DRUG

Oral liquid propranolol will be dosed Oral liquid propranolol will be dosed at 2 mg/kg/day.

Also known as: Propranolol hydrochloride (USP/EP) Oral Solution

Group B: Corticosteroid with Propranolol

Placebo DRUG

Liquid placebo will be given during the two month treatment trial.

Also known as: Inactive drug, Inactive substance, Inactive medicine

Group A: Corticosteroid with Placebo

Eligibility Criteria

• Age: Up to 9 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age 0 to \< 9months
• Patients with clinical, radiographic or histologic diagnosis of infantile hemangioma (IH) requiring medical treatment
• IH patients whose parents desire medical treatment for the IH
• Stable cardiac function

You may not qualify if:

• IH patients over 9 months of age.
• Hypersensitivity to propranolol
• Untreated heart failure: If the patient has heart failure associated with the hemangioma, propranolol would be initiated after anti-congestive therapy and under observation as an in-patient.
• Atrioventricular (AV) block
• Resting heart \< 2 SD of normal\*(below)
• Resting blood pressure \< 2 SD of normal\*\*(below)
• Wolff-Parkinson-White syndrome (WPW)
• History of unexplained syncope
• Bronchial asthma
• History of impaired renal or liver function
• Diabetes mellitus

Sponsors & Collaborators

• Seattle Children's Hospital: lead

Study Sites (1)

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Related Publications (2)

• Cushing SL, Boucek RJ, Manning SC, Sidbury R, Perkins JA. Initial experience with a multidisciplinary strategy for initiation of propranolol therapy for infantile hemangiomas. Otolaryngol Head Neck Surg. 2011 Jan;144(1):78-84. doi: 10.1177/0194599810390445.

  PMID: 21493392 BACKGROUND

• Truong MT, Perkins JA, Messner AH, Chang KW. Propranolol for the treatment of airway hemangiomas: a case series and treatment algorithm. Int J Pediatr Otorhinolaryngol. 2010 Sep;74(9):1043-8. doi: 10.1016/j.ijporl.2010.06.001. Epub 2010 Jul 31.

  PMID: 20674045 BACKGROUND

MeSH Terms

Conditions

Hemangioma

Interventions

Prednisolone; Adrenal Cortex Hormones; Propranolol; Solutions

Condition Hierarchy (Ancestors)

Neoplasms, Vascular Tissue; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Phenoxypropanolamines; Propanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Propanols; Amines; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Pharmaceutical Preparations

Limitations and Caveats

Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.

Results Point of Contact

• Title: Jonathan Perkins, Prinicipal Investigator
• Organization: Seattle childrens research institute

jonathan.perkins@seattlechildrens.org

206-987-1927

Study Officials

• Jonathan A Perkins, DO

  Seattle Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 22, 2010
• First Posted: February 24, 2010
• Study Start: February 1, 2010
• Primary Completion: February 1, 2012
• Study Completion: February 1, 2013
• Last Updated: October 4, 2018
• Results First Posted: September 26, 2013

Record last verified: 2018-09

Locations

US (1)