A Phase 2 interventional study of propranolol and Prednisolone in Hemangioma of Infancy, sponsored by Nancy Bauman. Terminated at 1 site in United States. Open to participants aged 2 Weeks to 6 Months. Per ClinicalTrials.gov, last updated 2016-02-24.
Sponsored by Nancy Bauman · Phase 2, Interventional, and Treatment
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.
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.
141 studies on the registry are indexed under Hemangioma; 16 are open to participants now.
This study's enrollment of 19 is below the median of 50 across 80 interventional studies indexed under Hemangioma.
Browse Hemangioma studies →This is the only study on the registry with Nancy Bauman as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Assessing efficacy and tolerability of propranolol in management of symptomatic hemangiomas
Drug: propranolol
Assessing efficacy and tolerability of prednisolone in management of symptomatic hemangiomas and comparing to propranolol.
Drug: Prednisolone
propranolol 0.5 mg/kg orally, 4 per day - 4-6 months
1.0 mg/kg orally, 2 per day 4-6 months
Also known as: pediapred
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.
Time frame: 4-5 months after initiating therapy
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.
Time frame: enrollment until study close out or withdrawal up to 9 months
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.
Time frame: enrollment until study close out or withdrawal up to 9 months
Growth and Development Adverse Events
Number of Growth and Development AEs in each study arm
Time frame: enrollment to study withdrawal or close out up to 9 months
Pulmonary/Respiratory Adverse Events
Number of pulmonary/respiratory adverse events (CTCAE 22) in each study arm
Time frame: enrollment through study close out or withdrawal, up to 9 months
Allergy/Immunology Adverse Events
Number of allergy/immunology AE per study arm
Time frame: enrollment through study closeout or study withdrawal up to 9 months
Dermatologic Adverse Events
Number of Dermatologic Adverse Events in each study arm.
Time frame: enrollment to study close out or withdrawal up to 9 months
Endocrinologic Adverse Events
Number of Endocrinologic AEs (of which adrenal crisis does not overlap).
Time frame: enrollment to close out or study withdrawal up to 9 months
Gastrointestinal Adverse Events
Number of Gastrointestinal AEs in each arm
Time frame: enrollment to study withdrawal or study close out up to 9 months
Infectious Adverse Events
Number of infectious AEs in each study arm (i.e. conjunctivitis, thrush, fever)
Time frame: enrollment to study withdrawal or close out up to 9 months
Metabolic or Laboratory AEs
Number of Metabolic or Laboratory AEs in each study arm.
Time frame: enrollment to study withdrawal or close out up to 9 months
Vascular Adverse Events
Number of Vascular AEs in each study arm.
Time frame: enrollment to study withdrawal or close out up to 9 months
Constitutional Adverse Events
Number of constitutional AEs in each study arm.
Time frame: enrollment to study close out or withdrawal up to 9 months
| Milestone | Propranolol | Prednisolone |
|---|---|---|
| Started | 11 | 8 |
| Completed | 9 | 2 |
| Not completed | 2 | 6 |
| Withdrew: Adverse event | 0 | 5 |
| Withdrew: Protocol violation | 2 | 0 |
| Withdrew: Physician decision | 0 | 1 |
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.
| mm squared | Propranolol | Prednisolone |
|---|---|---|
| Decrease in Size of Hemangioma (Length x Width) in Square mm | 0.57 (0.34 to 0.80) | 0.63 (0.14 to 1.11) |
All adverse events relating to medication tolerability including: adrenal crisis, growth/development, constitutional (dehydration), allergy/immunology, dermatologic, endocrine, GI, infection, metabolism/labs, pulmonary, vascular.
| Events | Overall Number of Adverse Events in Propranolol | Overall Number of Adverse Events in Prednisolone |
|---|---|---|
| Adverse Events | 34 | 30 |
| Serious Adverse Events | 1 | 11 |
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.
| Serious Adverse Events | Number of Serious Adverse Events in Propranolol | Number of Serious Adverse Events in Prednisolone |
|---|---|---|
| Number of Serious Adverse Events (SAEs) | 1 | 11 |
Number of Growth and Development AEs in each study arm
| Adverse Events | Growth/Developoment AEs Propranolol | Growth/Development AEs Prednisolone |
|---|---|---|
| Growth and Development Adverse Events | 0 | 1 |
Number of pulmonary/respiratory adverse events (CTCAE 22) in each study arm
| Adverse Events | Pulmonary/Respiratory AEs Propranolol | Pulmonary/Respiratory AEs Prednisolone |
|---|---|---|
| Pulmonary/Respiratory Adverse Events | 14 | 4 |
Number of allergy/immunology AE per study arm
| Adverse Events | Allergy/Immunology Events Propranolol | Allergy/Immunology Events Prednisolone |
|---|---|---|
| Allergy/Immunology Adverse Events | 1 | 1 |
Number of Dermatologic Adverse Events in each study arm.
| Adverse Events | Dermatologic AEs Propranolol | Dermatologic AEs Prednisolone |
|---|---|---|
| Dermatologic Adverse Events | 2 | 1 |
Number of Endocrinologic AEs (of which adrenal crisis does not overlap).
| Adverse Events | Endocrine AEs Propranolol | Endocrinologic AEs Prednisolone |
|---|---|---|
| Endocrinologic Adverse Events | 0 | 7 |
Number of Gastrointestinal AEs in each arm
| Adverse Events | Gastrointestinal AEs Propranolol | Gastrointestinal AEs Prednisolone |
|---|---|---|
| Gastrointestinal Adverse Events | 6 | 6 |
Number of infectious AEs in each study arm (i.e. conjunctivitis, thrush, fever)
| Adverse Events | Infectious AEs Propranolol | Infectious AEs Prednisolone |
|---|---|---|
| Infectious Adverse Events | 5 | 3 |
Number of Metabolic or Laboratory AEs in each study arm.
| Adverse Events | Metabolic/Laboratory AEs Propranolol | Metabolic/Laboratory AEs Prednisolone |
|---|---|---|
| Metabolic or Laboratory AEs | 1 | 0 |
Number of Vascular AEs in each study arm.
| Adverse Events | Vascular AEs Propranolol | Vascular AEs Prednisolone |
|---|---|---|
| Vascular Adverse Events | 3 | 4 |
Number of constitutional AEs in each study arm.
| Adverse Events | Constitutional AEs Propranolol | Constitutional AEs Prednisolone |
|---|---|---|
| Constitutional Adverse Events | 2 | 3 |
Collected over Adverse events collected in first 9 months participants treated with medication.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Propranolol | — | 1/11 (9.1%) | 11/11 (100%) |
| Prednisolone | — | 5/8 (62.5%) | 8/8 (100%) |
| Event | Propranolol | Prednisolone |
|---|---|---|
| Failure to thriveMetabolism and nutrition disorders | 0/11 | 5/8 |
| Adrenal CrisisEndocrine disorders | 0/11 | 1/8 |
| DehydrationGeneral disorders | 1/11 | 1/8 |
| Event | Propranolol | Prednisolone |
|---|---|---|
| EndoEndocrine disorders | 0/11 | 6/8 |
| Pulmonary/respiratoryRespiratory, thoracic and mediastinal disorders | 8/11 | 3/8 |
| GastrointerstinalGastrointestinal disorders | 5/11 | 5/8 |
| ConstitutionalGeneral disorders | 2/11 | 3/8 |
| VascularVascular disorders | 3/11 | 1/8 |
| InfectionInfections and infestations | 2/11 | 2/8 |
| DermatologicSkin and subcutaneous tissue disorders | 2/11 | 1/8 |
| AllergyGeneral disorders | 1/11 | 1/8 |
| Growth suppressionMusculoskeletal and connective tissue disorders | 0/11 | 1/8 |
| Metabolic/LabMetabolism and nutrition disorders | 1/11 | 0/8 |
Infants 2 weeks - 6 months of age with symptomatic, proliferating, infantile hemangiomas
| Age, Continuous(months) | Propranolol | Prednisolone | Total |
|---|---|---|---|
| Mean | 4.0 (2.8 to 5.2) | 2.5 (1.7 to 3.4) | 3.4 (1.7 to 5.2) |
| Sex: Female, Male(Participants) | Propranolol | Prednisolone | Total |
|---|---|---|---|
| Female | 8 | 6 | 14 |
| Male | 3 | 2 | 5 |
| Race (NIH/OMB)(Participants) | Propranolol | Prednisolone | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 1 | 0 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 1 | 2 | 3 |
| White | 9 | 6 | 15 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Ethnicity (NIH/OMB)(Participants) | Propranolol | Prednisolone | Total |
|---|---|---|---|
| Hispanic or Latino | 5 | 5 | 10 |
| Not Hispanic or Latino | 6 | 3 | 9 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(participants) | Propranolol | Prednisolone | Total |
|---|---|---|---|
| United States | 11 | 8 | 19 |
| Total surface area (length x width)(mm squared) | Propranolol | Prednisolone | Total |
|---|---|---|---|
| Mean | 5.0 (1.9 to 25.6) | 3.1 (0.8 to 10.5) | 4.2 (0.8 to 25.6) |
| Adjusted total surface area (length x width x proportion of skin involved(mm squared) | Propranolol | Prednisolone | Total |
|---|---|---|---|
| Mean | 5.0 (1.5 to 24.3) | 2.5 (0.2 to 9.4) | 4.0 (0.2 to 24.3) |
| Ulceration(participants) | Propranolol | Prednisolone | Total |
|---|---|---|---|
| Number | 2 | 1 | 3 |
2 further baseline measures are reported on the registry.
This study is terminated, as verified in Jan 2016. You cannot join it, but the record below documents what was studied.
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