HemangiomaPhase 2NCT01074437
Corticosteroids With Placebo Versus Corticosteroids With Propranolol Treatment of Infantile Hemangiomas (IH)
A Phase 2 study testing Prednisolone (Corticosteroid) and Propranolol in people with Hemangioma, run by Seattle Children's Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 9 months
- Conditions the study is for
- Hemangioma
- What is being tested?
- Prednisolone (Corticosteroid) and Propranolol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2010to Feb 2013
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 Up to 9 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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)
Show all 11Show fewer
- Wolff-Parkinson-White syndrome (WPW)
- History of unexplained syncope
- Bronchial asthma
- History of impaired renal or liver function
- Diabetes mellitus
Trial locations and contacts
1 site in 1 country.
United States1 site
Seattle Children's Hospital
Seattle, Washington, 98105
About this study
Official title: A Phase II, Randomized, Double-Blind Comparison of Corticosteroid and Corticosteroids With Propranolol Treatment of Infantile Hemangiomas (IH)
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.
Read the detailed description
Infantile hemangiomas (IH) are the most common head and neck pediatric tumors. Presence of these tumors can lead to complications of vision and airway compromise, bleeding and disfigurement. Medical treatment of these lesions has traditionally been focused on stopping new blood vessel growth with corticosteroids. Recent reports and our own experience have demonstrated that significant reduction in IH size and vascularity can also occur through the use of propranolol. Our initial experience with propranolol has demonstrated significant efficacy with fewer side effects than corticosteroids. Despite this experience, the standard of care for initial IH medical therapy remains corticosteroids.
This Trial is a direct comparison of traditional IH therapy with corticosteroids to newer therapy with propranolol and corticosteroids.
- Conditions
- Hemangioma
- Being tested
- Prednisolone (Corticosteroid), Propranolol, Placebo
- Medical subject headings
- Hemangioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Prednisolone (Corticosteroid)Drug
Also called Corticosteroids
Oral liquid prednisolone. Dose: 1-2mg/kg/day for 7 days, and then dose will be slowly reduced and then stopped after 3 weeks.
PropranololDrug
Also called Propranolol hydrochloride (USP/EP) Oral Solution
Oral liquid propranolol will be dosed Oral liquid propranolol will be dosed at 2 mg/kg/day.
PlaceboDrug
Also called Inactive drug, Inactive substance, Inactive medicine
Liquid placebo will be given during the two month treatment trial.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Prednisolone (Corticosteroid); Drug: Propranolol |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1, 2, and 6 months after treatment initiation
Lesion Regression
measure of change in lesion area or volume
Measured: 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.
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.
Measured: 1, 2, and 6 months after treatment initiation
Assess the Safety of Propranolol With Corticosteroids and Corticosteroids Alone in the Treatment of IH.
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.
Measured: 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.
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.
Measured: 1, 2 and 6 months after treatment initiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2010 (estimated)
- Study startFeb 2010
- Primary completionFeb 2012
- Study completionFeb 2013
Why it stopped: Insufficient enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for HemangiomaStudies of the same condition, open and under way.
- More trials run by Seattle Children's HospitalThe sponsor's other registered studies.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Seattle Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jonathan Perkins, Seattle Children's Hospital
- Investigators
- Jonathan A Perkins, DO, Principal investigator, Seattle Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01074437
- Sponsor's protocol ID
- 12901
- Status last verified
- Sep 2018
- First submitted
- 22 Feb 2010
- Last updated
- 4 Oct 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 21493392Truong 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.
PubMed 20674045
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Randomized, Double-Blind Comparison of Corticosteroid and Corticosteroids With Propranolol Treatment of Infantile Hemangiomas (IH)
- ClinicalTrials.gov ID
- NCT01074437
- Acronym / sponsor ID
- 12901
- Conditions
- Hemangioma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Insufficient enrollment
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Seattle Children's HospitalUniversity, hospital or other
- Study start
- Feb 2010
- Primary completion
- Feb 2012 (actual)
- Study completion
- Feb 2013 (actual)
- First posted
- 24 Feb 2010 (estimated)
- Results
- Posted 26 Sep 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Infantile hemangiomas (IH) are the most common head and neck pediatric tumors. Presence of these tumors can lead to complications of vision and airway compromise, bleeding and disfigurement. Medical treatment of these lesions has traditionally been focused on stopping new blood vessel growth with corticosteroids. Recent reports and our own experience have demonstrated that significant reduction in IH size and vascularity can also occur through the use of propranolol. Our initial experience with propranolol has demonstrated significant efficacy with fewer side effects than corticosteroids. Despite this experience, the standard of care for initial IH medical therapy remains corticosteroids.
This Trial is a direct comparison of traditional IH therapy with corticosteroids to newer therapy with propranolol and corticosteroids.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A: Corticosteroid with Placebo | Other | Drug: Prednisolone (Corticosteroid), Drug: PlaceboGroup 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. |
| Group B: Corticosteroid with Propranolol | Other | Drug: Prednisolone (Corticosteroid), Drug: PropranololGroup 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. |
Who may qualify
- Ages
- Up to 9 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (11 listed)
- 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)
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 |
|---|---|---|
| Prednisolone (Corticosteroid)Also: CorticosteroidsOral liquid prednisolone. Dose: 1-2mg/kg/day for 7 days, and then dose will be slowly reduced and then stopped after 3 weeks. | Drug | Group A: Corticosteroid with Placebo, Group B: Corticosteroid with Propranolol |
| PropranololAlso: Propranolol hydrochloride (USP/EP) Oral SolutionOral liquid propranolol will be dosed Oral liquid propranolol will be dosed at 2 mg/kg/day. | Drug | Group B: Corticosteroid with Propranolol |
| PlaceboAlso: Inactive drug, Inactive substance, Inactive medicineLiquid placebo will be given during the two month treatment trial. | Drug | Group A: Corticosteroid with Placebo |
Primary outcome
Compare Changes in IH Size and Vascularity for the Two Treatment Groups
Time frame: 1, 2, and 6 months after treatment initiation
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.
Lesion Regression
Time frame: 12 months
measure of change in lesion area or volume
3 secondary outcomes
Determine Therapeutic Response of IH to Propranolol Among Patients Who Switch to Corticosteroids Plus Propranolol Therapy After Failing to Respond to Corticosteroids Alone.
Time frame: 1, 2, and 6 months after treatment initiation
Assess the Safety of Propranolol With Corticosteroids and Corticosteroids Alone in the Treatment of IH.
Time frame: 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.
Time frame: 1, 2 and 6 months after treatment initiation
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 21493392Cushing 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.
- Background: PubMed 20674045Truong 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.
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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.
Jonathan A Perkins, DO principal investigator
Seattle Children's Hospital
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 Prednisolone (Corticosteroid) and Propranolol in people with Hemangioma, run by Seattle Children's Hospital at 1 site in United States.
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 4 inclusion and 11 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: stopped early, as of the sponsor's last update on 4 Oct 2018. 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 United States. 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?
Yes. Results are posted in the registry (first posted 26 Sep 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Compare Changes in IH Size and Vascularity for the Two Treatment Groups (time frame: 1, 2, and 6 months after treatment initiation). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01074437, which also lists 2 linked publications.
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 4 Oct 2018.