SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
- Conditions
- Microcystic Lymphatic Malformation
- Interventions
- Registration Number
- NCT06239480
- Lead Sponsor
- Palvella Therapeutics, Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 40
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Active QTORIN 3.9% Rapamycin Anhydrous Gel -
- Primary Outcome Measures
Name Time Method Overall microcystic lymphatic malformations Investigator Global Assessment (mLM-IGA) At Week 24 A multi-point scale with higher values indicating improvement.
- Secondary Outcome Measures
Name Time Method Overall patient global impression of change (PGI-C) Change from Baseline to Week 24 A multi-point scale with lower values indicating improvement.
Change in Overall Patient Global Impression of Severity (PGI-S) Change from Baseline to Week 24 A multi-point scale with lower values indicating improvement.
Incidence and severity of adverse events From Baseline to Week 24 Change in Overall Clinician Global Impression of Severity (CGI-S) Change from Baseline to Week 24 A multi-point scale with lower values indicating improvement.
Change in the blinded clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS) At Week 24 A multi-point scale with lower values indicating improvement.
Change in the live clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS) Change from Baseline to Week 24 A multi-point scale with lower values indicating improvement.
Trial Locations
- Locations (15)
Children's Hospital of Orange County
🇺🇸Irvine, California, United States
University of Utah Health
🇺🇸Salt Lake City, Utah, United States
Stanford University
🇺🇸Palo Alto, California, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Minnesota Clinical Study Center
🇺🇸New Brighton, Minnesota, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Vascular Birthmark Institute
🇺🇸New York, New York, 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
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Vanderbilt Children's Hospital
🇺🇸Nashville, Tennessee, United States
University of Texas, Dell Children's
🇺🇸Austin, Texas, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States