Microcystic Lymphatic Malformation Clinical Trial
— SELVAOfficial title:
A Multicenter, Phase 3 Randomized, Double-Blind, Placebo-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 clinician global impression after 24 weeks of treatment with QTORIN 3.9% Rapamycin Anhydrous Gel compared to placebo in approximately 50 participants with microcystic lymphatic malformations.
Status | Not yet recruiting |
Enrollment | 50 |
Est. completion date | January 2026 |
Est. primary completion date | December 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 6 Years and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | University of North Carolina | Chapel Hill | North Carolina |
United States | Cincinnati Children's Hospital | Cincinnati | Ohio |
United States | Cleveland Clinic | Cleveland | Ohio |
United States | Penn State Hershey Medical Center | Hershey | Pennsylvania |
United States | Texas Children's Hospital | Houston | Texas |
United States | University of Iowa | Iowa City | Iowa |
United States | Minnesota Clinical Study Center | New Brighton | Minnesota |
United States | Vascular Birthmark Institute | New York | New York |
United States | Stanford University | Palo Alto | California |
United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Mayo Clinic | Rochester | Minnesota |
Lead Sponsor | Collaborator |
---|---|
Palvella Therapeutics, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Clinician Global Impression of Change (CGI-C) | Comparison from End of Treatment at Week 24 to Baseline | ||
Secondary | Change in the Clinician Microcystic LM Multicomponent Severity Scale | Change from End of Treatment at Week 24 to Baseline | ||
Secondary | Overall patient global impression of change (PGI-C) | End of Treatment at Week 24 | ||
Secondary | Change in overall clinician global impression of change of severity (CGI-S) | Change from end of treatment at Week 24 to Baseline | ||
Secondary | Change in overall patient global impression of severity (PGI-S) | Change from end of treatment at Week 24 to Baseline | ||
Secondary | Incidence and severity of adverse events | From start of treatment to end of treatment at Week 24 |
Status | Clinical Trial | Phase | |
---|---|---|---|
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