NCT06239480

Brief Summary

SELVA: A Multicenter, Phase 3 Baseline-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations The main purpose of this study is to assess the change in microcystic lymphatic malformations IGA after 24 weeks of treatment with QTORIN 3.9% Rapamycin Anhydrous Gel in approximately 40 participants with microcystic lymphatic malformations. Efficacy will be evaluated at 24 weeks and patients have the option of continuing on treatment for \>24 weeks.

Trial Health

75
On Track

Trial Health Score

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

Enrollment
51

participants targeted

Target at below P25 for phase_3

Timeline
1mo left

Started Jul 2024

Geographic Reach
1 country

15 active sites

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 Progress92%
Jul 2024Jul 2026

First Submitted

Initial submission to the registry

January 16, 2024

Completed
17 days until next milestone

First Posted

Study publicly available on registry

February 2, 2024

Completed
6 months until next milestone

Study Start

First participant enrolled

July 31, 2024

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 14, 2026

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2026

Expected
Last Updated

March 27, 2026

Status Verified

March 1, 2026

Enrollment Period

1.5 years

First QC Date

January 16, 2024

Last Update Submit

March 26, 2026

Conditions

Microcystic Lymphatic Malformation

Keywords

Lymphangioma circumscriptumLymphatic malformationVascular malformationTopicalSirolimusRapamycinCongenital malformationNon-infective disorder of lymphatic vessels and lymph nodes

Outcome Measures

Primary Outcomes (1)

  • Overall microcystic lymphatic malformations Investigator Global Assessment (mLM-IGA)

    A multi-point scale with higher values indicating improvement.

    At Week 24

Secondary Outcomes (6)

  • Change in the blinded clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS)

    At Week 24

  • Change in the live clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS)

    Change from Baseline to Week 24

  • Overall patient global impression of change (PGI-C)

    Change from Baseline to Week 24

  • Change in Overall Clinician Global Impression of Severity (CGI-S)

    Change from Baseline to Week 24

  • Change in Overall Patient Global Impression of Severity (PGI-S)

    Change from Baseline to Week 24

  • +1 more secondary outcomes

Study Arms (1)

Active

EXPERIMENTAL
Drug: QTORIN 3.9% Rapamycin Anhydrous Gel

Interventions

QTORIN 3.9% Rapamycin Anhydrous GelDRUG

QTORIN 3.9% Rapamycin Anhydrous Gel

Active

Eligibility Criteria

Age3 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Participant must be at least 3 years of age at time of consent/assent
  • Participant must have a clinically confirmed superficial/cutaneous microcystic lymphatic malformation

You may not qualify if:

  • Participants who have vascular malformations that interfere with evaluating the target microcystic lymphatic malformation
  • Participants with complicated vascular anomalies with severe systemic symptoms that require systemic therapy
  • The participant's treatment area is mainly in any wet mucosa or within the orbital rim
  • Participants who are pregnant or planning to become pregnant

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (15)

Children's Hospital of Orange County

Irvine, California, 92612, United States

Location

Stanford University

Palo Alto, California, 94305, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30342, United States

Location

Minnesota Clinical Study Center

New Brighton, Minnesota, 55112, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Vascular Birthmark Institute

New York, New York, 10016, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27516, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Penn State Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Vanderbilt Children's Hospital

Nashville, Tennessee, 37232, United States

Location

University of Texas, Dell Children's

Austin, Texas, 78723, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

University of Utah Health

Salt Lake City, Utah, 84113, United States

Location

MeSH Terms

Conditions

LymphangiomaLymphatic AbnormalitiesVascular MalformationsCongenital Abnormalities

Condition Hierarchy (Ancestors)

Neoplasm, Lymphatic TissueNeoplasms by Histologic TypeNeoplasmsLymphatic DiseasesHemic and Lymphatic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCardiovascular AbnormalitiesCardiovascular Diseases

Study Officials

  • Joyce Teng

    Stanford University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 16, 2024

First Posted

February 2, 2024

Study Start

July 31, 2024

Primary Completion

January 14, 2026

Study Completion (Estimated)

July 1, 2026

Last Updated

March 27, 2026

Record last verified: 2026-03

Locations

US(15)