Clinical trial NCT06239480: SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Microcystic Lymphatic Malformation
Keywords: Lymphangioma circumscriptum
Lymphatic malformation
Vascular malformation
Topical
Sirolimus
Rapamycin
Congenital malformation
Non-infective disorder of lymphatic vessels and lymph nodes
Lead sponsor: Palvella Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: FDA Office of Orphan Products Development
First posted: 2024-02-02
Enrollment: 40
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Key Inclusion Criteria:

* Participant must be at least 6 years of age at time of consent
* 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

Primary outcomes:
measure: Overall microcystic lymphatic malformations Investigator Global Assessment (mLM-IGA)
timeFrame: At Week 24
Secondary outcomes:
measure: Change in the blinded clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS)
timeFrame: At Week 24
measure: Change in the live clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS)
timeFrame: Change from Baseline to Week 24
measure: Overall patient global impression of change (PGI-C)
timeFrame: Change from Baseline to Week 24
measure: Change in Overall Clinician Global Impression of Severity (CGI-S)
timeFrame: Change from Baseline to Week 24
measure: Change in Overall Patient Global Impression of Severity (PGI-S)
timeFrame: Change from Baseline to Week 24
measure: Incidence and severity of adverse events
timeFrame: From Baseline to Week 24
Other outcomes:
Locations:
city: Irvine
country: United States
facility: Children's Hospital of Orange County
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: New Brighton
country: United States
facility: Minnesota Clinical Study Center
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: New York
country: United States
facility: Vascular Birthmark Institute
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Hershey
country: United States
facility: Penn State Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt Children's Hospital
state: Tennessee
city: Austin
country: United States
facility: University of Texas, Dell Children's
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health
state: Utah
Study officials:
affiliation: Stanford University
name: Joyce Teng
role: PRINCIPAL_INVESTIGATOR
References: