A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations

Microcystic Lymphatic Malformation Clinical Trial

— SELVA  

Official 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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06239480
• Other study ID #: PALV-09
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: April 2024
• Est. completion date: January 2026

Study information

• Verified date: January 2024
• Source: Palvella Therapeutics, Inc.
• Contact: Kathy Goin
• Phone: 484-612-2335
• Email: kathy.goin[@]palvellatx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• 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

Related Conditions & MeSH terms

• Congenital Abnormalities
• Lymphangioma
• Lymphatic Abnormalities
• Microcystic Lymphatic Malformation

Intervention

Drug:
• QTORIN 3.9% Rapamycin Anhydrous Gel
QTORIN 3.9% Rapamycin Anhydrous Gel Topical
• Placebo
Placebo Topical Gel

Locations

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	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 Pennsylvania	Philadelphia	Pennsylvania
United States	Mayo Clinic	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Palvella Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

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