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Hemangioma of InfancyPhase 2NCT00967226

Propranolol Versus Prednisolone for Treatment of Symptomatic Hemangiomas

A Phase 2 study testing propranolol and Prednisolone in people with Hemangioma of Infancy, run by Nancy Bauman at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Hemangiomas are relatively common lesions in infants. Most go away spontaneously after one year of life and do not need treatment. Others require treatment because they cause significant symptoms such as pain, or difficulty with breathing, eating or ambulating. Read more about this study

Who is it for?
Ages 2 weeks to 6 months
Conditions the study is for
Hemangioma of Infancy
What is being tested?
propranolol and Prednisolone
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
1 siteUnited States
Study dates
Jul 2009to Dec 2014
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 2 weeks to 6 months
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • infants with symptomatic hemangiomas

Who cannot join

  • asthma
  • diabetes
  • hypertension
  • hypotension
  • hypoglycemia
  • liver failure
  • previous treatment for hemangiomas

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20111

Other trials in United States

About this study

Official title: Propranolol vs Prednisolone for Infant Hemangiomas-A Clinical and Molecular Study

Hemangiomas are relatively common lesions in infants. Most go away spontaneously after one year of life and do not need treatment. Others require treatment because they cause significant symptoms such as pain, or difficulty with breathing, eating or ambulating. Steroids have classically been used to treat hemangiomas and help to shrink them in 1/3 - 2/3 of patients. Unfortunately, steroids have many side effects in babies so physicians have sought other ways to treat them. Recently, the use of propranolol, a heart medication, was serendipitously found to reduce the size of hemangiomas. It appears to have many fewer side effects than steroids but it is not yet known if it works as well as steroids. This study seeks to compare the effect and the side effects of propranolol versus steroids for treating hemangiomas that cause symptoms in infants.

Read the detailed description

Infants with symptomatic hemangiomas will be enrolled. Magnetic resonance imaging will be completed before starting medication if the extent of the hemangioma is not evident on clinical examination alone. Infants will be randomized to receive either propranolol or steroids for 4-6 months. Hemangioma response will be measured and compared monthly as will tolerability of the medications. Additionally, urine specimens will be collected at each visit to determine if markers are present that can predict response to therapy.

Additionally, any hemangiomas that are excised will be examined for genetic markers to aid in predicting response to therapy.

Keywords
hemangioma
Medical subject headings
Hemangioma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • propranololDrug

    propranolol 0.5 mg/kg orally, 4 per day - 4-6 months

  • PrednisoloneDrug

    Also called pediapred

    1.0 mg/kg orally, 2 per day 4-6 months

Study groups in NCT00967226
Study groupRoleWhat they receive
propranolol for treatment of hemangiomasExperimentalAssessing efficacy and tolerability of propranolol in management of symptomatic hemangiomas
Receives: Drug: propranolol
PrednisoloneActive comparatorAssessing efficacy and tolerability of prednisolone in management of symptomatic hemangiomas and comparing to propranolol.
Receives: Drug: Prednisolone

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Decrease in Size of Hemangioma (Length x Width) in Square mm

    A priori primary outcome was proportional change in the total surface area as measured by lesion's outer margin length x width at baseline minus the same measure at 4 months with surrogate data used at 5 months if 4 months not available.

    Measured: 4-5 months after initiating therapy

Secondary outcomes (12)

  1. Tolerability of Medication

    All adverse events relating to medication tolerability including: adrenal crisis, growth/development, constitutional (dehydration), allergy/immunology, dermatologic, endocrine, GI, infection, metabolism/labs, pulmonary, vascular.

    Measured: enrollment until study close out or withdrawal up to 9 months

  2. Number of Serious Adverse Events (SAEs)

    Number of serious adverse events experienced by the participants in each treatment arm within the categories adrenal crisis, growth/development, constitutional. Serious adverse events are defined as events that result in death, require either inpatient hospitalization or the prolongation of hospitalization, are life-threatening, result in a persistent or significant disability/incapacity, or result in a congenital anomaly/birth defect. Other important medical events, based upon appropriate medical judgment, may also be considered Serious Adverse Events if a trial participant's health is at risk and intervention is required to prevent an outcome mentioned.

    Measured: enrollment until study close out or withdrawal up to 9 months

  3. Growth and Development Adverse Events

    Number of Growth and Development AEs in each study arm

    Measured: enrollment to study withdrawal or close out up to 9 months

  4. Pulmonary/Respiratory Adverse Events

    Number of pulmonary/respiratory adverse events (CTCAE 22) in each study arm

    Measured: enrollment through study close out or withdrawal, up to 9 months

  5. Allergy/Immunology Adverse Events

    Number of allergy/immunology AE per study arm

    Measured: enrollment through study closeout or study withdrawal up to 9 months

  6. Dermatologic Adverse Events

    Number of Dermatologic Adverse Events in each study arm.

    Measured: enrollment to study close out or withdrawal up to 9 months

  7. Endocrinologic Adverse Events

    Number of Endocrinologic AEs (of which adrenal crisis does not overlap).

    Measured: enrollment to close out or study withdrawal up to 9 months

  8. Gastrointestinal Adverse Events

    Number of Gastrointestinal AEs in each arm

    Measured: enrollment to study withdrawal or study close out up to 9 months

  9. Infectious Adverse Events

    Number of infectious AEs in each study arm (i.e. conjunctivitis, thrush, fever)

    Measured: enrollment to study withdrawal or close out up to 9 months

  10. Metabolic or Laboratory AEs

    Number of Metabolic or Laboratory AEs in each study arm.

    Measured: enrollment to study withdrawal or close out up to 9 months

  11. Vascular Adverse Events

    Number of Vascular AEs in each study arm.

    Measured: enrollment to study withdrawal or close out up to 9 months

  12. Constitutional Adverse Events

    Number of constitutional AEs in each study arm.

    Measured: enrollment to study close out or withdrawal up to 9 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Aug 2009 (estimated)
  2. Study startJul 2009
  3. Primary completionJan 2013
  4. Study completionDec 2014

Why it stopped: Serious adverse events with prednisolone, primarily temporary growth retardation, \<5th percentile.

Resources

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
Single blind
Who is masked
Outcomes assessor
Enrolment
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00967226
Sponsor's protocol ID
IRB 4502
Other IDs
NIH grant number 10179326
Status last verified
Jan 2016
First submitted
26 Aug 2009
Last updated
24 Feb 2016
Data monitoring committee
No

Publications

  • Perez RS, Mora PC, Rodriguez JD, Sanchez FR, de Torres Jde L. [Treatment of infantile hemangioma with propranolol]. An Pediatr (Barc). 2010 Feb;72(2):152-4. doi: 10.1016/j.anpedi.2009.05.019. Epub 2009 Jul 23. No abstract available. Spanish.

    PubMed 19631595

  • Denoyelle F, Leboulanger N, Enjolras O, Harris R, Roger G, Garabedian EN. Role of Propranolol in the therapeutic strategy of infantile laryngotracheal hemangioma. Int J Pediatr Otorhinolaryngol. 2009 Aug;73(8):1168-72. doi: 10.1016/j.ijporl.2009.04.025. Epub 2009 May 29.

    PubMed 19481268

  • Leaute-Labreze C, Dumas de la Roque E, Hubiche T, Boralevi F, Thambo JB, Taieb A. Propranolol for severe hemangiomas of infancy. N Engl J Med. 2008 Jun 12;358(24):2649-51. doi: 10.1056/NEJMc0708819. No abstract available.

    PubMed 18550886

  • Bauman NM, McCarter RJ, Guzzetta PC, Shin JJ, Oh AK, Preciado DA, He J, Greene EA, Puttgen KB. Propranolol vs prednisolone for symptomatic proliferating infantile hemangiomas: a randomized clinical trial. JAMA Otolaryngol Head Neck Surg. 2014 Apr;140(4):323-30. doi: 10.1001/jamaoto.2013.6723.

    PubMed 24526257

  • Patel NJ, Bauman NM. How should propranolol be initiated for infantile hemangiomas: inpatient versus outpatient? Laryngoscope. 2014 Jun;124(6):1279-81. doi: 10.1002/lary.24363. Epub 2013 Dec 17. No abstract available.

    PubMed 24347141

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing propranolol and Prednisolone in people with Hemangioma of Infancy, run by Nancy Bauman 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 1 inclusion and 7 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 24 Feb 2016. 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 24 Feb 2016). 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?

Decrease in Size of Hemangioma (Length x Width) in Square mm (time frame: 4-5 months after initiating therapy). 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 NCT00967226, which also lists 5 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 24 Feb 2016.

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