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QTORIN rapamycin

Phase 3

Microcystic Lymphatic Malformation | Small molecule | Rare Disease |Palvella Therapeutics, Inc.|Last Updated: Mar 27, 2026

Target and mechanism

ModalitySmall molecule

Also known as QTORIN 3.9% rapamycin anhydrous gel, QTORIN 3.9% rapamycin anhydrous, QTORIN 3.9% Rapamycin Anhydrous Gel, QTORIN 3.9% Rapamycin Anhydrous

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment51

FDA Designations

FAST_TRACKBREAKTHROUGH_THERAPYORPHAN_DRUG

Clinical trial landscape

QTORIN rapamycin · 3 trials · 3 indications

Phase 3 2Phase 2 1
NCT06239480SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic MalformationsMicrocystic Lymphatic Malformation
ACTIVE NOT_RECRUITING51 Analytics
NCT05180708A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia CongenitaPachyonychia Congenita
COMPLETED87 Analytics
PHASE3ACTIVE NOT_RECRUITING
SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
Microcystic Lymphatic Malformation
NCT0623948051
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PHASE3COMPLETED
A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita
Pachyonychia Congenita
NCT0518070887
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Study Endpoints

Primary Endpoints

Overall microcystic lymphatic malformations Investigator Global Assessment (mLM-IGA)
At Week 24

A multi-point scale with higher values indicating improvement.

Patient Global Assessment of Activities Difficulty Scale
6 months
Incidence of Treatment-Emergent Adverse Events
Week 12

Secondary Endpoints

Change in the blinded clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS)
At Week 24
Change in the live clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS)
Change from Baseline to Week 24
Overall patient global impression of change (PGI-C)
Change from Baseline to Week 24
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ActiveEXPERIMENTAL -
VehiclePLACEBO_COMPARATOR -
Single-arm, open-labelEXPERIMENTAL -

Interventions

NameTypeDescription
QTORIN 3.9% Rapamycin Anhydrous GelDRUGQTORIN 3.9% Rapamycin Anhydrous Gel
VehicleDRUGMatching vehicle (QTORIN anyhydrous gel)
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Eligibility Criteria

Age Range3 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites15

Key Inclusion Criteria: * Participant must be at least 3 years of age at time of consent/assent * Participant must have a clinically confirmed superficial/cutaneous microcystic lymphatic malformation Key Exclusion Criteria: * Participants who have vascular malformations that interfere with evalua...

Countries:United StatesUnited Kingdom
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