Efficacy, Safety and Pharmacokinetics of Topical Timolol in Infants With Infantile Hemangioma (IH)

NCT ID: NCT02913612

Last Updated: 2024-03-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 105 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-05-05
• Study Completion Date: 2020-10-20

Brief Summary

The purpose of this study is to assess the safety and efficacy of Timolol 0.25% and 0.5% doses.

Detailed Description

Primary: Describe the efficacy of 0.25% and 0.5% topical timolol maleate Gel-forming solution (GFS) as assessed through Infantile Hemangioma (IH) changes in volume.

Secondary: Describe the safety of topical timolol maleate GFS for treatment of IH.

Conditions

Infantile Hemangioma

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

0.25% Timolol Treatment

Subjects assigned to this arm will be randomized to 0.25% timolol for 180 days. If during the 180 days the subject is considered a treatment failure, the subject will be unblinded. If the subject is on 0.25% timolol they will be changed to 0.5% timolol.

Group Type: EXPERIMENTAL

0.25% Timolol Maleate Gel Forming Solution

Intervention Type: DRUG

50:50 Randomized 0.25% Timolol Maleate Gel Forming Solution

0.5% Timolol Treatment

Subjects assigned to this arm will be randomized to 0.5% timolol for 180 days. If during the 180 days the subject is considered a treatment failure, the subject will be unblinded. If the subject is on 0.5% timolol the treating physician will decide to either continue 0.5% timolol or withdraw the subject and begin an alternative treatment.

Group Type: EXPERIMENTAL

0.5% Timolol Maleate Gel Forming Solution

Intervention Type: DRUG

50:50 Randomized 0.5% Timolol Maleate Gel Forming Solution

Non-Intervention Group

Subjects assigned to this group will not receive treatment. The subject will only be photographed on the same schedule as the intervention group.

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

0.25% Timolol Maleate Gel Forming Solution

50:50 Randomized 0.25% Timolol Maleate Gel Forming Solution

Intervention Type: DRUG

0.5% Timolol Maleate Gel Forming Solution

50:50 Randomized 0.5% Timolol Maleate Gel Forming Solution

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Documented informed consent from legal guardian

2. 0-84 days postnatal age at time of first study dose or when enrolled into the non-intervention cohort.

3. Clinical diagnosis of superficial cutaneous or mucosal infantile hemangioma (must include all of the following):

1. Superficial lesion in the dermis

2. Thin <2 mm in thickness

3. Small >=5 cm at its longest dimension and <=10cm2

4. Involves skin or keratinized mucosa

Exclusion Criteria

1. History of previous treatment with any pharmacologic or laser therapy for IH

2. Ongoing therapy with an oral beta blocker or oral corticosteroid (e.g., cardiac arrhythmia, adrenal insufficiency, upper airway obstruction, tetralogy of fallot (TOF), hypertension, reactive airways disease)

3. IH that requires systemic therapy (defined by dynamic complication scale >3)

4. IH of the non-keratinized mucosa

5. Infants with more than one hemangioma that requires therapy

6. Hemodynamically significant cardiovascular disease, as determined by the investigator

7. Known allergy to beta blockers or vehicle

8. Heart rate <100 beats per minute at screening visit

9. Known prenatal or postnatal diagnosis of 2nd/3rd degree atrioventricular block

10. History of Reactive Airways Disease (RAD)

11. Any condition which would make the participant, in the opinion of the investigator unsuitable for the study.

• Maximum Eligible Age: 84 Days
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Emmes Company, LLC

INDUSTRY

Sponsor Role: collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

Kanecia Obie Zimmerman

OTHER

Sponsor Role: lead

Responsible Party

Kanecia Obie Zimmerman

Principal Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Kanecia Zimmerman, MD, MHS

Role: PRINCIPAL_INVESTIGATOR

Duke Clinical Research Institute

Kristin Holland, MD

Role: STUDY_CHAIR

Medical College of Wisconsin

Locations

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Indiana University Health

Indianapolis, Indiana, United States

Johns Hopkins Medical Center

Baltimore, Maryland, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Mayo Clinic

Rochester, Minnesota, United States

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, United States

Columbia University Medical Center

New York, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital of Philadephia

Philadelphia, Pennsylvania, United States

The University of Texas Medical School at Houston

Houston, Texas, United States

DCOL Center for Clinical Research

Longview, Texas, United States

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Countries

United States

References

Drolet BA, Boakye-Agyeman F, Harper B, Holland K, Lewandowski A, Stefanko N, Melloni C; Pediatric Trials Network Steering Committee (See Acknowledgments for a listing of committee members.). Systemic timolol exposure following topical application to infantile hemangiomas. J Am Acad Dermatol. 2020 Mar;82(3):733-736. doi: 10.1016/j.jaad.2019.02.029. Epub 2019 Feb 18. No abstract available.

Reference Type: BACKGROUND

PMID: 30790601 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

Pro00068212

Identifier Type: -

Identifier Source: org_study_id