Clinical trial
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
Name
PALV-09
Description
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.
Trial arms
Trial start
2024-04-01
Estimated PCD
2025-12-01
Trial end
2026-01-01
Status
Not yet recruiting
Phase
Early phase I
Treatment
QTORIN 3.9% Rapamycin Anhydrous Gel
QTORIN 3.9% Rapamycin Anhydrous Gel Topical
Arms:
Active
Placebo
Placebo Topical Gel
Arms:
Placebo
Size
50
Primary endpoint
Clinician Global Impression of Change (CGI-C)
Comparison from End of Treatment at Week 24 to Baseline
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
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 50, 'type': 'ESTIMATED'}}
Updated at
2024-02-02
1 organization
3 products
1 indication
Organization
Palvella TherapeuticsProduct
PlaceboIndication
Microcystic Lymphatic Malformation