HemangiomaPhase 2NCT02145884
Topical Timolol Gel for the Treatment of Infantile Hemangiomas
A Phase 2 study testing timolol maleate 0.5% gel in people with Hemangioma, run by Rady Children's Hospital, San Diego at 1 site in United States.
Trial summary
We plan to conduct a study, to see how safe and effective timolol maleate 0.5% gel-forming solution is for infantile hemangiomas (IH) and the response of hemangiomas to timolol maleate 0.5% . Our hypothesis is that timolol will inhibit and possibly reverse growth of appropriate infantile hemangiomas. Read more about this study
- Who is it for?
- Ages 7 days to 6 months
- Conditions the study is for
- Hemangioma
- What is being tested?
- timolol maleate 0.5% gel
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 1 siteUnited States
- Study dates
- May 2014to Nov 2016
- 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 7 days to 6 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Written informed permission for study participation and the use of the patient's images are obtained from the patient's parent(s) or guardian(s),
- The patient is between 7 days and 6 months of age at the time of enrollment,
- and a proliferating HOI not requiring systemic therapy is present anywhere on the body, Multiple hemangiomas may be treated on same child, if the hemangioma meets this criteria.
Who cannot join
- patients who are not otherwise generally healthy;
- at risk for imminent ulceration, life-threatening, function-threatening, or ulcerated;
- patients who have previously received systemic, intra-lesional or topical corticosteroids, imiquimod, vincristine, alpha-interferon, propranolol or other beta blockers;
- patients who have previously been treated for his/her HOI, including any surgical and/or medical procedures;
- patients whose mothers have been breastfeeding the patient while also being treated with beta-blockers, systemic corticosteroids, vincristine or alpha-interferon;
- patients who have previously experienced any anaphylactic reactions; patients with an unclear diagnosis of HOI;
- patients participating in another clinical study or living in the same household as an infant already participating in this study;
- patients born prematurely who have not yet reached his/her term equivalent age; and patients with parent(s) or guardian(s) who cannot be contacted by telephone in case of emergency.
Trial locations and contacts
1 site in 1 country.
United States1 site
Pediatric and Adolescent Dermatology, Rady Children's Hospital/University of California, San Diego
San Diego, California, 92123
About this study
We plan to conduct a study, to see how safe and effective timolol maleate 0.5% gel-forming solution is for infantile hemangiomas (IH) and the response of hemangiomas to timolol maleate 0.5% . Our hypothesis is that timolol will inhibit and possibly reverse growth of appropriate infantile hemangiomas.
Read the detailed description
Subjects will be assigned to open label timolol maleate 0.5%. Parents will be instructed to place 1 to 2 drops twice a day in the center of the hemangioma. Subjects are expected to use the study drug as directed every day for a 4 month period.
Subjects will be assessed at baseline, 2 weeks, 4 weeks, and every 4 weeks thereafter for a total of 5 months. Physical exam, photographs to compare size and color intensity, and a scale of improvement will be completed at each visit. Parents will complete a quality of life questionnaire regarding their child's hemangioma and its impact on quality of life.
Subjects who have evidence of worsening would be removed from the study and transferred to the clinic for evaluation and treatment.
- Conditions
- Hemangioma
- Being tested
- timolol maleate 0.5% gel
- Keywords
- hemangioma, birthmark, strawberry hemangioma, timolol
- Medical subject headings
- Hemangioma, Hemangioma, Cavernous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
timolol maleate 0.5% gelDrug
Also called timoptic
Apply timolol gel 1-2 drops twice a day to lesion
| Study group | Role | What they receive |
|---|---|---|
| timolol maleate 0.5% gel | Experimental | timolol gel 1 to 2 drops twice a day to lesions for 4 months Receives: Drug: timolol maleate 0.5% gel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in size of hemangioma
At each visit, the size of the IH is assessed in three dimensions (length, width, and height) in cm.
Measured: Baseline, week 2, 4, 8, 12, 16
Change in color of hemangioma
At each visit, the color intensity (e.g., barely perceptible; pale pink or mottled pink/red; red with central pallor; dull red; bright red) of the IH is assessed on a 6 point scale.
Measured: Baseline, week 2, 4, 8, 12, 16
Secondary outcomes (1)
Measure the extent of systemic absorption and the factors which influence absorption
A heel stick blood sample is collected to assess for systemic absorption.
Measured: week 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2014 (estimated)
- Study startMay 2014
- Primary completionOct 2016
- Study completionNov 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rady Children's Hospital, San Diego
- Sponsor type
- University, hospital or other
- Collaborators
- Valeant Pharmaceuticals
- Responsible party
- Principal investigator, Sheila Friedlander, Rady Children's Hospital, San Diego
- Investigators
- Sheila F Friedlander, MD, Principal investigator, Rady Children's Hospital/ University of California, San Diego
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02145884
- Sponsor's protocol ID
- PED3560
- Other IDs
- 20132793
- Status last verified
- Feb 2017
- First submitted
- 21 May 2014
- Last updated
- 13 Feb 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Topical Timolol Gel for the Treatment of Infantile Hemangiomas
- ClinicalTrials.gov ID
- NCT02145884
- Acronym / sponsor ID
- PED3560
- Conditions
- Hemangioma
- Keywords
- hemangioma, birthmark, strawberry hemangioma, timolol
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Rady Children's Hospital, San DiegoUniversity, hospital or other
- Collaborators
- Valeant Pharmaceuticals
- Study start
- May 2014 (actual)
- Primary completion
- Oct 2016 (actual)
- Study completion
- Nov 2016 (actual)
- First posted
- 23 May 2014 (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.
We plan to conduct a study, to see how safe and effective timolol maleate 0.5% gel-forming solution is for infantile hemangiomas (IH) and the response of hemangiomas to timolol maleate 0.5% . Our hypothesis is that timolol will inhibit and possibly reverse growth of appropriate infantile hemangiomas.
Detailed description
Subjects will be assigned to open label timolol maleate 0.5%. Parents will be instructed to place 1 to 2 drops twice a day in the center of the hemangioma. Subjects are expected to use the study drug as directed every day for a 4 month period.
Subjects will be assessed at baseline, 2 weeks, 4 weeks, and every 4 weeks thereafter for a total of 5 months. Physical exam, photographs to compare size and color intensity, and a scale of improvement will be completed at each visit. Parents will complete a quality of life questionnaire regarding their child's hemangioma and its impact on quality of life.
Subjects who have evidence of worsening would be removed from the study and transferred to the clinic for evaluation and treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| timolol maleate 0.5% gel | Experimental | Drug: timolol maleate 0.5% geltimolol gel 1 to 2 drops twice a day to lesions for 4 months |
Who may qualify
- Ages
- 7 days to 6 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Written informed permission for study participation and the use of the patient's images are obtained from the patient's parent(s) or guardian(s),
Key exclusion criteria (8 listed)
- patients who are not otherwise generally healthy;
- at risk for imminent ulceration, life-threatening, function-threatening, or ulcerated;
- patients who have previously received systemic, intra-lesional or topical corticosteroids, imiquimod, vincristine, alpha-interferon, propranolol or other beta blockers;
- patients who have previously been treated for his/her HOI, including any surgical and/or medical procedures;
- patients whose mothers have been breastfeeding the patient while also being treated with beta-blockers, systemic corticosteroids, vincristine or alpha-interferon;
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 |
|---|---|---|
| timolol maleate 0.5% gelAlso: timopticApply timolol gel 1-2 drops twice a day to lesion | Drug | timolol maleate 0.5% gel |
Primary outcome
Change in size of hemangioma
Time frame: Baseline, week 2, 4, 8, 12, 16
At each visit, the size of the IH is assessed in three dimensions (length, width, and height) in cm.
Change in color of hemangioma
Time frame: Baseline, week 2, 4, 8, 12, 16
At each visit, the color intensity (e.g., barely perceptible; pale pink or mottled pink/red; red with central pallor; dull red; bright red) of the IH is assessed on a 6 point scale.
1 secondary outcome
Measure the extent of systemic absorption and the factors which influence absorption
Time frame: week 2
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 13 Feb 2017.
- Individual participant dataSharing planned: Yes
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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.
Sheila F Friedlander, MD principal investigator
Rady Children's Hospital/ University of California, San Diego
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 timolol maleate 0.5% gel in people with Hemangioma, run by Rady Children's Hospital, San Diego 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 3 inclusion and 8 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 13 Feb 2017. 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?
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?
Change in size of hemangioma (time frame: Baseline, week 2, 4, 8, 12, 16). 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?
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 13 Feb 2017.