Infantile HemangiomaPhase 2NCT01010308
Nadolol for Proliferating Infantile Hemangiomas
A Phase 2 study testing Nadolol in people with Infantile Hemangioma, run by The Hospital for Sick Children at 1 site in Canada.
Trial summary
The purpose of this study is to explore the efficacy and safety of Nadolol in hemangiomas of infancy. The secondary objective is to assess the feasibility of conducting a randomized controlled trial comparing nadolol with corticosteroids and propranolol. Read more about this study
- Who is it for?
- Ages 1 month to 1 year
- Conditions the study is for
- Infantile Hemangioma
- What is being tested?
- Nadolol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteCanada
- Study dates
- Nov 2009to Feb 2012
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 1 year
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Intervention Group
- Infants aged 1 month to 1 year of age with head and neck hemangiomas currently causing /or with impending function loss (e.g. vision, airway obstruction, feeding, etc), or hemangiomas currently causing/or with potential for facial disfigurement.
- Historical Control Group
- Infants aged 1 month to 1 year of age with head and neck hemangiomas that received treatment with systemic propranolol in the past 2 years
- Angiogenesis Marker Control Group
- Infants aged 1 month to 1 year attending dermatology clinic
Who cannot join
- Intervention Group
- Patients with PHACES syndrome (proven) or suspected PHACES (plaque like hemangioma awaiting imaging).
- Children with history of hypersensitivity to beta blockers
- Children with personal history or family history of a first degree relative with asthma
- Children with known renal impairment
- Children with known cardiac conditions which may predispose to heart blocks
Show all 13Show fewer
- Personal history of hypoglycemia
- Children on medications that may interact with beta blockers
- Historical Control Group:
- No digital photography available documenting IHs progression
- Angiogenesis Marker Control Group:
- Children with IH
- Children on beta blocker or systemic corticosteroids
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Nadolol for Proliferating Infantile Hemangiomas: A Prospective Open Label Study With a Historical Control
The purpose of this study is to explore the efficacy and safety of Nadolol in hemangiomas of infancy.
The secondary objective is to assess the feasibility of conducting a randomized controlled trial comparing nadolol with corticosteroids and propranolol.
Read the detailed description
Systemic corticosteroids are currently the most frequent used medication for treatment of problematic infantile hemangiomas (IH's). Since June 2008, systemic propranolol has been an important addition to the therapeutic options for problematic IH, allowing decreased dependence on the systemic corticosteroids. So far, we have found excellent response with propranolol with minimal short-term side effects. Studies, which compared nadolol and propranolol in children with other conditions, suggest that nadolol is safer and more efficacious than propranolol. In addition, it has better dosing schedules and less central nervous system (CNS) penetration, making it suitable even for patients with suspected or proven PHACES syndrome.
- Conditions
- Infantile Hemangioma
- Being tested
- Nadolol
- Keywords
- infantile hemangioma, nadolol
- Medical subject headings
- Hemangioma, Capillary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NadololDrug
Nadolol will be administered orally at home starting at 0.5 mg/kg/day divided into 2 doses. Weekly, if BP and heart rate are acceptable, the dose will be increased by 0.5 mg/kg/day up to 2 mg/kg/day.
| Study group | Role | What they receive |
|---|---|---|
| Intervention Group: | Experimental | The patients in this study are infants aged 1 month to 1 year of age with head and neck hemangiomas currently causing /or with impending function loss (e.g. vision, airway obstruction, feeding, etc), or hemangiomas currently causing/or with potential for facial disfigurement
Infants aged 1 month to 1 year of age with head and neck hemangiomas that received treatment with systemic propranolol in the past 2 years as a control group Receives: Drug: Nadolol |
| Historical control group | No intervention | Ten infants (1-12 months of age) treated with propranolol will be identified from a Dermatology Database. Patients will be considered as controls if they were treated with propranolol before 1 year of age and had digital photography documentation of their hemangioma. |
| Angiogenesis marker control group | No intervention | The angiogenesis marker control group will consist of 6 -10 patients seen in the Dermatology clinic for conditions other than IH and not receiving corticosteroids or beta blockers. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects with at least 75% improvement in the extent of the hemangioma
Measured: Baseline, 6months
Secondary outcomes (3)
The proportion of subjects with at least 50% improvement in the extent of the hemangiomas
The proportion of subjects with at least 50% improvement in the extent of the hemangiomas
Measured: Baseline and 6 months
The percentage of patients with >75% improvement in the Nadolol group compared to a historical cohort of patients receiving propranolol.
The percentage of patients with >75% improvement in the Nadolol group compared to a historical cohort of patients receiving propranolol.
Measured: 6 months
The correlation between the changes in the levels of angiogenesis markers and clinical response to treatment.
The correlation between the changes in the levels of angiogenesis markers and clinical response to treatment.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2009 (estimated)
- Study startNov 2009
- Primary completionMay 2011
- Study completionFeb 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile HemangiomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Elena Pope, The Hospital for Sick Children
- Investigators
- Elena Pope, MD, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01010308
- Sponsor's protocol ID
- 1000014079
- Status last verified
- Aug 2021
- First submitted
- 8 Nov 2009
- Last updated
- 19 Aug 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nadolol for Proliferating Infantile Hemangiomas: A Prospective Open Label Study With a Historical Control
- ClinicalTrials.gov ID
- NCT01010308
- Acronym / sponsor ID
- 1000014079
- Conditions
- Infantile Hemangioma
- Keywords
- infantile hemangioma, nadolol
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Study start
- Nov 2009
- Primary completion
- May 2011 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 10 Nov 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to explore the efficacy and safety of Nadolol in hemangiomas of infancy.
The secondary objective is to assess the feasibility of conducting a randomized controlled trial comparing nadolol with corticosteroids and propranolol.
Detailed description
Systemic corticosteroids are currently the most frequent used medication for treatment of problematic infantile hemangiomas (IH's). Since June 2008, systemic propranolol has been an important addition to the therapeutic options for problematic IH, allowing decreased dependence on the systemic corticosteroids. So far, we have found excellent response with propranolol with minimal short-term side effects. Studies, which compared nadolol and propranolol in children with other conditions, suggest that nadolol is safer and more efficacious than propranolol. In addition, it has better dosing schedules and less central nervous system (CNS) penetration, making it suitable even for patients with suspected or proven PHACES syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Intervention Group: | Experimental | Drug: NadololThe patients in this study are infants aged 1 month to 1 year of age with head and neck hemangiomas currently causing /or with impending function loss (e.g. vision, airway obstruction, feeding, etc), or hemangiomas currently causing/or with potential for facial disfigurement Infants aged 1 month to 1 year of age with head and neck hemangiomas that received treatment with systemic propranolol in the past 2 years as a control group |
| Historical control group | No intervention | –Ten infants (1-12 months of age) treated with propranolol will be identified from a Dermatology Database. Patients will be considered as controls if they were treated with propranolol before 1 year of age and had digital photography documentation of their hemangioma. |
| Angiogenesis marker control group | No intervention | –The angiogenesis marker control group will consist of 6 -10 patients seen in the Dermatology clinic for conditions other than IH and not receiving corticosteroids or beta blockers. |
Who may qualify
- Ages
- 1 month to 1 year
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Intervention Group
- Infants aged 1 month to 1 year of age with head and neck hemangiomas currently causing /or with impending function loss (e.g. vision, airway obstruction, feeding, etc), or hemangiomas currently causing/or with potential for facial disfigurement.
- Historical Control Group
- Infants aged 1 month to 1 year of age with head and neck hemangiomas that received treatment with systemic propranolol in the past 2 years
- Angiogenesis Marker Control Group
Key exclusion criteria (13 listed)
- Intervention Group
- Patients with PHACES syndrome (proven) or suspected PHACES (plaque like hemangioma awaiting imaging).
- Children with history of hypersensitivity to beta blockers
- Children with personal history or family history of a first degree relative with asthma
- Children with known renal impairment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NadololNadolol will be administered orally at home starting at 0.5 mg/kg/day divided into 2 doses. Weekly, if BP and heart rate are acceptable, the dose will be increased by 0.5 mg/kg/day up to 2 mg/kg/day. | Drug | Intervention Group: |
Primary outcome
Proportion of subjects with at least 75% improvement in the extent of the hemangioma
Time frame: Baseline, 6months
3 secondary outcomes
The proportion of subjects with at least 50% improvement in the extent of the hemangiomas
Time frame: Baseline and 6 months
The percentage of patients with >75% improvement in the Nadolol group compared to a historical cohort of patients receiving propranolol.
Time frame: 6 months
The correlation between the changes in the levels of angiogenesis markers and clinical response to treatment.
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elena Pope, MD principal investigator
The Hospital for Sick Children
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Nadolol in people with Infantile Hemangioma, run by The Hospital for Sick Children at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of subjects with at least 75% improvement in the extent of the hemangioma (time frame: Baseline, 6months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2021.