SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations

Phase 3RecruitingNCT06239480

Palvella Therapeutics, Inc.40 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Microcystic Lymphatic Malformation

Interventions

QTORIN 3.9% Rapamycin Anhydrous Gel

Eligibility

Sex: ALLMin age: 3 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: * 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 Key Exclusion Criteria: * 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

Locations (15)

Children's Hospital of Orange County

Irvine, California, United States

RECRUITING

Stanford University

Palo Alto, California, United States

RECRUITING

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

RECRUITING

Minnesota Clinical Study Center

New Brighton, Minnesota, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, United States

RECRUITING

Vascular Birthmark Institute

New York, New York, United States

RECRUITING

University of North Carolina

Chapel Hill, North Carolina, United States

RECRUITING

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, United States

RECRUITING

Penn State Hershey Medical Center

Hershey, Pennsylvania, United States

RECRUITING

...and 5 more locations

Outcomes

Primary Outcomes

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

A multi-point scale with higher values indicating improvement.

Time frame: At Week 24

Secondary Outcomes

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