Clinical trial NCT06273111: Topical Simvastatin for Treating Infantile Hemangioma
Official title: A Pilot Study Using Topical Statins to Treat Children With Infantile Hemangioma
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: EARLY_PHASE1
Conditions: Hemangioma Skin
Lead sponsor: Stanford University
Sponsor class: OTHER
Collaborators: name: Stanford University
First posted: 2024-02-22
Enrollment: 12
Sex: ALL
Minimum age (years): 3
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a 24-week, open-label pilot study to evaluate the safety and preliminary efficacy of 5% simvastatin ointment in treating 12 children with superficial IH.

The primary objective:

To evaluate the safety and tolerability of topical treatment with 5% simvastatin ointment for superficial IH over 24 weeks.

The secondary objective:

1.1 To evaluate the efficacy of 5% simvastatin ointment when topical treatment is administered twice daily for 24 weeks. Evaluation is performed at each clinic visit via investigator global assessment (IGA) based on standardized 3D digital photography and hemangioma activity score (HAS).

1.2 To evaluate the impact of 5% simvastatin ointment on quality of life using the IH-QoL questionnaire.


Eligibility criteria: Inclusion Criteria:

* Healthy children aged between 3 months and 5 years.
* Newly diagnosed superficial IH or experiencing rebound growth with a superficial component.
* Participants must possess at least one IH lesion with the longest diameter equal to or greater than 2 cm, located on any part of the body except the lips.
* Participants must not have received any of the following treatments for their IH:

  * Topical medical therapy within the past 2 weeks.
  * Systemic medical therapy within the past 3 months.
  * Laser treatment within the past 6 weeks.
* Written informed consent from the parent(s)/guardian(s) of minor participants must be obtained before any study procedure is performed.

Exclusion Criteria:

* IH is primarily characterized as subcutaneous, and deep, with minimal cutaneous involvement for evaluation.
* IH with active ulceration.
* IH to be treated involving the lips mainly.
* Participants with concurrent skin conditions that may impede accurate clinical assessment of the IH.
* Participants with hereditary or metabolic disorders requiring systemic statin therapy.
* Participants who are allergic to statins, or other ingredients present in the topical medication.

Primary outcomes:
measure: Number of Participants with Treatment-Related Adverse Events
timeFrame: Baseline through week 24
Secondary outcomes:
measure: Percentage of participants achieved a 50% HAS reduction at week 12 compared to baseline
timeFrame: Baseline through week 12
measure: Percentage of participants achieved a 75% HAS reduction from baseline by the end of the study (week 24) compared to baseline.
timeFrame: Baseline through week 24
measure: Percentage of targeted hemangiomas with complete or nearly complete resolution (IGA 0 or 1), defined as a minimal degree of telangiectasia, skin thickening, and no definitive palpable cutaneous texture changes.
timeFrame: Baseline through week 24
measure: Percentage of targeted hemangiomas that are stabilized (no noticeable change to baseline) or continue to progress.
timeFrame: Baseline through week 24
measure: Percentage of participants with a significant improvement in quality-of-life, defined as > 50% reduction in the IH-QoL questionnaire score from baseline to week 24.
timeFrame: Baseline through week 24
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University
state: California
Study officials:
affiliation: Stanford University
name: Joyce Teng, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: