topical timolol maleate for Angioma

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Massachusetts General Hospital, Boston, MA
Angioma
topical timolol maleate - Drug
Eligibility
< 18
All Sexes
Eligible conditions
Select

Study Summary

A Clinical Trial of Pulsed-dye Laser Versus Timolol Topical Solution Versus Observation on the Growth of Hemangioma in Newborn

See full description

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether topical timolol maleate will improve 1 primary outcome and 1 secondary outcome in patients with Angioma. Measurement will happen over the course of 2 years.

2 years
Proportion of lesions that are completely clear or with minimum residual signs
Proportion of parents who consider their children to have a cosmetically acceptable outcome, functional improvement, need for additional treatment and any adverse reactions

Trial Safety

Safety Estimate

1 of 3

Trial Design

3 Treatment Groups

Observation
1 of 3
Pulsed Dye Laser
1 of 3
Topical Timolol
1 of 3
Active Control
Experimental Treatment

This trial requires 126 total participants across 3 different treatment groups

This trial involves 3 different treatments. Topical Timolol Maleate is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are not being studied for commercial purposes.

Pulsed Dye Laser
Device
After verification of eligibility criteria and obtaining informed consent of parent/guardian, infants randomized to the pulsed dye laser arm will receive a series of six weekly to semi-weekly laser treatments treatments for up to 6 treatments with potential for reduced number of treatments if the hemangioma completely resolves. A 595-nm pulsed-dye laser (PDL, V-beam Perfecta, Candela Corp, Wayland, MA) with a dynamic cooling device (DCD) will be utilized for all treatments. This device is cleared by the FDA for clinical treatment of vascular lesions.
Topical Timolol
Drug
After verification of eligibility criteria and obtaining informed consent of parent/guardian, infants randomized to the timolol arm will receive twice daily topical application of a physician-specified amount of timolol maleate 0.5% ophthalmic solution (hereby referred to as topical timolol) for up to six months.
ObservationAfter verification of eligibility criteria and obtaining informed consent of parent/guardian, infants randomized to the observation arm will be followed at study visits according to protocol.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pulsed dye laser
2019
N/A
~270

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 2 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 2 years for reporting.

Who is running the study

Principal Investigator
R. R. A.
Richard Rox Anderson, MD
Massachusetts General Hospital

Closest Location

Massachusetts General Hospital - Boston, MA

Eligibility Criteria

This trial is for patients born any sex aged 18 and younger. There are 9 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Subjects aged less than 3 months, male or female.
Infant with one or more superficial hemangiomas in the preproliferative phase or very early proliferative growth phase.
Absence or minimal appearance of the lesion at birth
More pronounced appearance within 1 month of birth.
Willingness of parent/guardian to participate in the study
Willingness of parent/guardian to receive EXPERIMENTAL treatment
Informed consent agreement signed by the parent/guardian
Willingness of parent/guardian to follow the treatment schedule and post treatment care requirements
Willingness of parent/guardian to not use topical or systemic (oral) TREATMENT medications of the hemangioma other than those prescribed by the investigators during the study period.

Patient Q&A Section

What is topical timolol maleate?

"Topical timolol maleate, a β-adrenergically-selective muscarinic receptor (M) and M signaling antagonist, has potential for the treatment of pathological endothelial cell proliferation in proliferative vitreoretinopathy. Topical timolol maleate induces its activity by an antiangiogenic effect, which may be associated with M receptor suppression." - Anonymous Online Contributor

Unverified Answer

What is angioma?

"Angioma is an uncommon benign vascular tumor. The diagnosis should only be confirmed by biopsy. Patients do well with treatment if they are treated early, usually during childhood." - Anonymous Online Contributor

Unverified Answer

Can angioma be cured?

"Given the heterogeneity and heterogeneity of the angioma, it is hard to state that angiomas can be cured. The presence of angiomas does not mean, let alone dictate, the final nature of a patient. Therefore, the possibility of recurrency is often more realistic than it would have been in some angioma cases and is one to treat cautiously." - Anonymous Online Contributor

Unverified Answer

What are the signs of angioma?

"Although angiomas are usually asymptomatic, palpable nodules may be present in about 20 percent of the population. Clinically significant angiomas occur frequently on the lips and oral mucosa, but it is unclear whether they are due to viral infection or from other causes. There is no reliable clinical parameter that can indicate who will develop an angioma." - Anonymous Online Contributor

Unverified Answer

How many people get angioma a year in the United States?

"Approximately 500,000 people will have angioma in the United States in 2020. Patients with angioma will experience a total of 16,724 lesions before the age 30 years and approximately 36,000 lesions before the age of 40 years. The mean lifetime risk of developing a new onset of lesions, or a new diagnosis of angioma, will be approximately 20% before the age 30 years and approximately 55% before the age of 40 years." - Anonymous Online Contributor

Unverified Answer

What are common treatments for angioma?

"The most common treatment for angioma is laser excision if it is small, or embolization if large. Dermabrasion and surgery are used for larger lesions and may fail." - Anonymous Online Contributor

Unverified Answer

What causes angioma?

"Angiomas can arise from many different causes. One possible contributing cause includes a weakened immune system from an underlying medical condition. Other more traditional causes include bleeding from the tumor or from the vessel supplying the tumor (tumor-suppressing agents, radiation treatment), excessive exposure (e.g., from chemicals in the environment or occupational exposure). A weakened immune system can also worsen the effect of stress or the drug a person is taking. However, the exact cause of most tumors, including angiomas, is still unclear. Tumor-suppressing drugs such as cisplatin can also cause cancer (common side effects include nausea, vomiting, and hair loss)." - Anonymous Online Contributor

Unverified Answer

Does topical timolol maleate improve quality of life for those with angioma?

"In a recent study, findings demonstrated the QOL-associated benefits of topical timolol maleate. In a recent study, findings are a preliminary finding and require further investigation." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets angioma?

"The data were collected by [Census of Diseases in Ontario Statistics Division (DODON)] in Ontario, Canada. There [annual and average mean age (with standard deviation and median value) of Ontario and national population for most common conditions]are the estimates available to us. Please see references to [Ontario Vital Statistics (OVSCD)]#[data-source. OVSCD] for some specifics on how we obtained these values. There is no indication of the source of the following data." - Anonymous Online Contributor

Unverified Answer

What is the latest research for angioma?

"The only known intervention for a large angioma is treatment with radiofrequency. There is a wide variability of treatment success. However, in an angioma exceeding 5 cm, or when the tumor has a substantial fraction of the nose, more recent studies suggest there is no difference in treatment results. The only significant difference is that radiofrequency could be reserved for those with a high-quality life and a small nose or nose with a stable tumor (for those treated conservatively, angiomas have a better treatment outcome). The current recommendation is to perform radiofrequency treatment to the entire tumor, which gives the most successful outcome." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating angioma?

"There have been many research studies on angioma treatment, but there is still a lack of a perfect and conclusive treatment. There are many clinical implications of the research findings. Many times, angioma progresses to the point where it invades blood vessels in other parts of the body, thus negatively affecting other organs and leading to blindness. The ultimate goal will be to find the most efficient and less-dangerous treatment. To make an effective treatment for angiomas, it will be important to understand the mechanisms of tumor cell growth, especially those of angiogenesis. Scientists will need to discover a new way for promoting healthy blood vessels that can prevent tumor cell growth and eventually lead to angioma cure." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving topical timolol maleate?

"Local timolol maleate is well tolerated in all subtypes of glaucoma but there are no data available on the efficacy of timolol maleate in the treatment of aqueous and submacular hemorrhage." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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