NCT07072403

Brief Summary

Complicated vascular anomalies have diverse etiologies and variable clinical manifestations, and no standardized treatment protocol has been established. Since most patients present with diffuse lesions that are difficult to resect surgically, identifying effective therapeutic strategies is of critical importance. This study aims to evaluate the safety and efficacy of systemic trametinib therapy in patients with complicated vascular anomalies.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at P25-P50 for phase_1

Timeline
16mo left

Started Jul 2025

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress39%
Jul 2025Sep 2027

First Submitted

Initial submission to the registry

July 7, 2025

Completed
Same day until next milestone

Study Start

First participant enrolled

July 7, 2025

Completed
11 days until next milestone

First Posted

Study publicly available on registry

July 18, 2025

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2027

Expected
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2027

Last Updated

July 22, 2025

Status Verified

July 1, 2025

Enrollment Period

2 years

First QC Date

July 7, 2025

Last Update Submit

July 17, 2025

Conditions

Vascular Anomalies

Keywords

complicated vascular anomalies

Outcome Measures

Primary Outcomes (1)

  • The proportion of patients achieving an objective response at month 12

    Objective response was defined as ≥20% reduction in lesion volume compared to that at baseline.

    12 months

Secondary Outcomes (5)

  • The proportion of patients achieving an objective response at month 6

    6 months

  • lesion responses

    6 and 12 months

  • Quality of life (QOL) in patients

    12 months

  • Disease sequelae

    12 months

  • Frequency of adverse events

    12 months

Study Arms (1)

Oral Trametinib

EXPERIMENTAL

Patients will receive oral trametinib once daily

Drug: Trametinib tablet

Interventions

Trametinib tabletDRUG

Drug is supplied in 0.5 mg and 2 mg tablets

Oral Trametinib

Eligibility Criteria

Age1 Year+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Presenting a complicated vascular anomalies vascular anomalies with the following characteristics:
  • Male and female;
  • Between 1 and 18 years of age;
  • Complicated vascular anomalies vascular anomalies diagnosis was confirmed by local investigators and by consensus of our multidisciplinary vascular anomaly group at the West China Hospital of Sichuan University based on:
  • Biopsy; Compatible MRI findings; History and clinical features.

You may not qualify if:

  • Patients have allergy or contraindication to MEK inhibitor
  • Exposure to chemotherapy, embolization, corticosteroids, propranolol, sclerotherapy or any other investigational agents within 1 weeks before enrolment on study;
  • Patients had a history of a major surgery within 2 weeks before enrollment;
  • Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of enrollment;
  • Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of trametinib.
  • Concurrent severe and/or uncontrolled medical diseases that could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe malnutrition, chronic liver or renal disease, active upper gastrointestinal tract ulceration).
  • Patients with inadequate liver function:
  • Total bilirubin higher than or equal to 1.5 × the upper limit of the normal (ULN) for age and alanine aminotransferase and aspartate aminotransferase higher than or equal to 2.5 × the ULN for age.
  • Patients with inadequate renal function:
  • years of age maximum serum creatinine (mg/dL) of 0.8; 6-10 years of age maximum serum creatinine (mg/dL) of 1.0; 11-14 years of age maximum serum creatinine (mg/dL) of 1.2;
  • Adequate bone marrow function:
  • Absolute neutrophil count lower than 1 × 109/L;
  • History of a malignancy within 5 years;
  • HIV infection or known immunodeficiency;
  • Patients with an inability to participate in or follow-up during the study treatment and assessment plan;
  • +1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

West China Hospital of Sichuan University

Chengdu, Sichuan, 610041, China

Location

Related Publications (5)

  • Croteau SE, Kozakewich HP, Perez-Atayde AR, Fishman SJ, Alomari AI, Chaudry G, Mulliken JB, Trenor CC 3rd. Kaposiform lymphangiomatosis: a distinct aggressive lymphatic anomaly. J Pediatr. 2014 Feb;164(2):383-8. doi: 10.1016/j.jpeds.2013.10.013. Epub 2013 Nov 16.

    PMID: 24252784BACKGROUND

  • Zhou Z, Qiu T, Zhou J, Zhang Z, Gong X, Zhang X, Lan Y, Yang C, Zhang Y, Xiang S, Ji Y. Clinical features and current management experience in Gorham-Stout disease: a systematic review. Orphanet J Rare Dis. 2025 Mar 19;20(1):134. doi: 10.1186/s13023-025-03649-9.

    PMID: 40102890BACKGROUND

  • Foster JB, Li D, March ME, Sheppard SE, Adams DM, Hakonarson H, Dori Y. Kaposiform lymphangiomatosis effectively treated with MEK inhibition. EMBO Mol Med. 2020 Oct 7;12(10):e12324. doi: 10.15252/emmm.202012324. Epub 2020 Sep 7.

    PMID: 32894644BACKGROUND

  • Li D, Sheppard SE, March ME, Battig MR, Surrey LF, Srinivasan AS, Matsuoka LS, Tian L, Wang F, Seiler C, Dayneka J, Borst AJ, Matos MC, Paulissen SM, Krishnamurthy G, Nriagu B, Sikder T, Casey M, Williams L, Rangu S, O'Connor N, Thomas A, Pinto E, Hou C, Nguyen K, Pellegrino da Silva R, Chehimi SN, Kao C, Biroc L, Britt AD, Queenan M, Reid JR, Napoli JA, Low DM, Vatsky S, Treat J, Smith CL, Cahill AM, Snyder KM, Adams DM, Dori Y, Hakonarson H. Genomic profiling informs diagnoses and treatment in vascular anomalies. Nat Med. 2023 Jun;29(6):1530-1539. doi: 10.1038/s41591-023-02364-x. Epub 2023 Jun 1.

    PMID: 37264205BACKGROUND

  • Barclay SF, Inman KW, Luks VL, McIntyre JB, Al-Ibraheemi A, Church AJ, Perez-Atayde AR, Mangray S, Jeng M, Kreimer SR, Walker L, Fishman SJ, Alomari AI, Chaudry G, Trenor Iii CC, Adams D, Kozakewich HPW, Kurek KC. A somatic activating NRAS variant associated with kaposiform lymphangiomatosis. Genet Med. 2019 Jul;21(7):1517-1524. doi: 10.1038/s41436-018-0390-0. Epub 2018 Dec 13.

    PMID: 30542204BACKGROUND

MeSH Terms

Conditions

Vascular Malformations

Interventions

trametinib

Condition Hierarchy (Ancestors)

Cardiovascular AbnormalitiesCardiovascular DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Yi Ji

    West China Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Clinical Professor

Study Record Dates

First Submitted

July 7, 2025

First Posted

July 18, 2025

Study Start

July 7, 2025

Primary Completion (Estimated)

July 1, 2027

Study Completion (Estimated)

September 1, 2027

Last Updated

July 22, 2025

Record last verified: 2025-07

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)