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
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
51
QTORIN 3.9% Rapamycin Anhydrous Gel
Children's Hospital of Orange County
Irvine, California, 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
...and 5 more locations
Overall microcystic lymphatic malformations Investigator Global Assessment (mLM-IGA)
A multi-point scale with higher values indicating improvement.
Time frame: At Week 24
Change in the blinded clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS)
A multi-point scale with lower values indicating improvement.
Time frame: At Week 24
Change in the live clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS)
A multi-point scale with lower values indicating improvement.
Time frame: Change from Baseline to Week 24
Overall patient global impression of change (PGI-C)
A multi-point scale with lower values indicating improvement.
Time frame: Change from Baseline to Week 24
Change in Overall Clinician Global Impression of Severity (CGI-S)
A multi-point scale with lower values indicating improvement.
Time frame: Change from Baseline to Week 24
Change in Overall Patient Global Impression of Severity (PGI-S)
A multi-point scale with lower values indicating improvement.
Time frame: Change from Baseline to Week 24
Incidence and severity of adverse events
Time frame: From Baseline to Week 24
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