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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Nadolol group | Active Comparator | 40 study participants will take Nadolol (oral liquid suspension) |
|
| Propranolol group | Active Comparator | 40 study paticipants will take Propranolol (oral liquid suspension) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nadolol | Drug | Patients will be administered twice-daily doses of medication as follows: since Day 0- 0.5 mg/kg/day ; since Day 7-1.0 mg/kg/day and since Day 14- 1.5 mg/kg/day. In all subsequent visits the dosage will be adjusted based on the current weight rather than the baseline weight to maintain 1.5 mg/kg/day. Or the dose may be escalated (by 0.5 mg/kg/day at any following study visit) up to 3 mg/kg/day based on the clinical response to maintain the dose that led to at least 75% reduction in the hemangioma size until Week 24, when unblinding happen. At Week 24 paticipants can start weaning by 10% per week or continue with the last dose, depending on the investigator's decision. S/he will be monitored until Week 52. |
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 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 | 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Percent change in IH bulk using VAS at 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 | 4, 12, 52 weeks |
| Time and dose to reach the 50%, 75% and 100% tumor shrinkage |
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Inclusion Criteria:
1-6 months corrected age
Written parental informed consent
At least one of the following:
Exclusion Criteria:
Contraindications to beta-blockers
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)
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| Name | Affiliation | Role |
|---|---|---|
| Elena Pope, MD, MSc | The Hospital for Sick Children | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Hospital for Sick Children | Toronto | Ontario | M5G1X8 | Canada |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34747977 | Derived | Pope E, Lara-Corrales I, Sibbald C, Liy-Wong C, Kanigsberg N, Drolet B, Ma J. Noninferiority and Safety of Nadolol vs Propranolol in Infants With Infantile Hemangioma: A Randomized Clinical Trial. JAMA Pediatr. 2022 Jan 1;176(1):34-41. doi: 10.1001/jamapediatrics.2021.4565. |
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| ID | Term |
|---|---|
| D018324 | Hemangioma, Capillary |
| D006391 | Hemangioma |
| ID | Term |
|---|---|
| D009383 | Neoplasms, Vascular Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| D009248 | Nadolol |
| D011433 | Propranolol |
| ID | Term |
|---|---|
| D050198 | Phenoxypropanolamines |
| D011412 | Propanolamines |
| D000605 | Amino Alcohols |
| D000438 | Alcohols |
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| Propranolol | Drug | Patients will be administered twice-daily doses of medication as follows: since Day 0- 0.5 mg/kg/day ; since Day 7-1.0 mg/kg/day and since Day 14- 1.5 mg/kg/day. In all subsequent visits the dosage will be adjusted based on the current weight rather than the baseline weight to maintain 1.5 mg/kg/day. Or the dose may be increased by investigator, based on clinical response by 0.5 mg/kg/day at any study visit (up to 3 mg/kg/day divided twice a day) to maintain the dose that lead to at least 75% reduction in the hemangioma size until Week 24, when unblinding happen. At Week 24 paticipants can start weaning by 10% per week or continue with the last dose, depending on the investigator's decision. S/he will be monitored until Week 52. |
|
|
Time frame since the baseline and study medication dose, when patient's IH decreased in size by 50%, 75% and 100%. |
| 52 weeks |
| Inter-rater reliability of the VAS scores | Two raters will assess the changes in IH for each study patient ( each visit). We will compare these results to assess inter-rater reliability. | 52 weeks |
| Percentage of patients achieving functional correction at Week 4, 12, 24, 52 | Percentage of patients achieving functional correction at Week 4, 12, 24, 52 | 4,12,24,52 weeks |
| Percent change in the volumetric changes of hemangioma | [(Length + Width)/2]3 X 0.07 | 24 and 52 weeks |
| Percentage of patients with residual changes (telangiectasias, discoloration, fibro-fatty changes, anetoderma) | Percentage of patients with residual changes | 52 weeks |
| Frequency of observed and reported adverse events | Frequency of observed and reported adverse events | 52 weeks |
| D009930 |
| Organic Chemicals |
| D020005 | Propanols |
| D000588 | Amines |
| D009281 | Naphthalenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D011083 | Polycyclic Compounds |