Nadolol Versus Propranolol in Children With Infantile Hemangiomas

Phase 3

Completed

• Conditions: Infantile Hemangioma
• Interventions: Drug: Nadolol; Drug: Propranolol

• Registration Number: NCT02505971
• Lead Sponsor: The Hospital for Sick Children

Brief Summary

The purpose of this study is to assess the efficacy and safety of oral propranolol versus nadolol in patients with Infantile Hemangiomas (IH) in a randomized, controlled, double-blinded study.

Detailed Description

The study objective is to compare the efficacy and safety of oral propranolol in comparison with oral nadolol in patients with IH. Patients will be randomly assigned to either propranolol or dose equivalent nadolol. The duration of the study will be 24 weeks, however, patient will be monitored for up to 1 year post study enrolment. Both efficacy and safety will be closely monitored and captured.

Study Timeline

• Study First Submitted: 2015-07-21
• Study First Submitted QC: 2015-07-21
• Study First Posted: 2015-07-22
• Study Start: 2015-09
• Primary Completion: 2020-04
• Study Completion: 2020-06-30
• Status Verified: 2020-10
• Last Update Submitted: 2020-10-27
• Last Update Posted: 2020-10-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 74

Inclusion Criteria

• 1-6 months corrected age

• Written parental informed consent

• At least one of the following:

  • Size: hemangioma >1.5 cm on the face or >3 cm on other body parts
  • Causing or with potential for functional impairment (e.g. amblyogenic IH, ulcerated hemangioma)
  • Causing or with potential for cosmetic disfigurement (e.g. nasal tip, glabella location)

Exclusion Criteria

• Contraindications to beta-blockers

  • Hypotension
  • Bradycardia
  • Hypoglycemia
  • Cardiac disease associated with decreased ejection fraction and/or > second degree heart block
  • Bronchospasm (including bronchial asthma)
  • Allergic rhinitis

• Corrected gestational age less than 1 month at screening

• Patients with PHACES cerebral arteriopathy at risk of stroke

• Patients and/or breastfeeding mothers receiving treatment with anti-arrhythmic agents, calcium channel blockers, ACE inhibitors, inotropic agents, vasodilators, hypoglycemic agents, neuroleptics, antiacids, benzodiazepines, thyroxine, warfarin

• Patients treated with an oral beta-blocker or other agent (e.g. systemic steroids, vincristine) within 2 weeks from randomization

• Patients treated with topical timolol within 1 week from randomization

• Vascular tumors other than infantile hemangioma (e.g. pyogenic granuloma, hemangioendothelioma)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Nadolol group	Nadolol	40 study participants will take Nadolol (oral liquid suspension)
Propranolol group	Propranolol	40 study paticipants will take Propranolol (oral liquid suspension)

Primary Outcome Measures

Name	Time	Method
The change in the bulk (size/extent) and color of the infantile hemangioma (IH)at Week 24 compared to baseline using Visual Analog Scale (VAS).	24 weeks	A 100 mm visual analog scale (VAS) will be used to quantify changes in the visible bulk (size/extent) and color of the lesion by comparing clinical photographs at 24 weeks versus baseline

Secondary Outcome Measures

Name	Time	Method
Time and dose to reach the 50%, 75% and 100% tumor shrinkage	52 weeks	Time frame since the baseline and study medication dose, when patient's IH decreased in size by 50%, 75% and 100%.
Percent change in the volumetric changes of hemangioma	24 and 52 weeks	\[(Length + Width)/2\]3 X 0.07
Percentage of patients with residual changes (telangiectasias, discoloration, fibro-fatty changes, anetoderma)	52 weeks	Percentage of patients with residual changes
Frequency of observed and reported adverse events	52 weeks	Frequency of observed and reported adverse events
Percent change in IH bulk using VAS at 4, 12, 52 weeks	4, 12, 52 weeks	A 100 mm visual analog scale (VAS) will be used to quantify changes in the visible bulk (size/extent) of the lesion by comparing clinical photographs at weeks 4, 12, and 52 versus baseline
Inter-rater reliability of the VAS scores	52 weeks	Two raters will assess the changes in IH for each study patient ( each visit). We will compare these results to assess inter-rater reliability.
Percentage of patients achieving functional correction at Week 4, 12, 24, 52	4,12,24,52 weeks	Percentage of patients achieving functional correction at Week 4, 12, 24, 52

Trial Locations

Locations (1)

The Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada