Trial Outcomes & Findings for Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations (NCT NCT05050149)

NCT ID: NCT05050149

Last Updated: 2025-01-17

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 12 participants
• Primary outcome timeframe: 4 months
• Results posted on: 2025-01-17

Participant Flow

Participant milestones

Measure	Experimental: PTX-022 PTX-022 QTORIN PTX-022: Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations
Overall Study STARTED	12
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations

Baseline characteristics by cohort

Measure	Experimental: PTX-022 n=12 Participants PTX-022 QTORIN PTX-022: Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations
Age, Categorical <=18 years	5 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	27.3 years STANDARD_DEVIATION 13.56 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · American Indian or Alaska Native	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · Asian	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black or African American	2 Participants n=5 Participants
Race/Ethnicity, Customized Race · Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	8 Participants n=5 Participants
Race/Ethnicity, Customized Race · Other	1 Participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 months

Outcome measures

Measure	Experimental: PTX-022 n=12 Participants PTX-022 QTORIN PTX-022: Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations
Incidence of Treatment-Emergent Adverse Events Skin and Subcutaneous Tissue Disorders	4 participants
Incidence of Treatment-Emergent Adverse Events Subjects with TEAEs	9 participants
Incidence of Treatment-Emergent Adverse Events Gastrointestinal Disorders	2 participants
Incidence of Treatment-Emergent Adverse Events General Disorders and Administration Site Conditions	6 participants
Incidence of Treatment-Emergent Adverse Events Infections and Infestations	2 participants
Incidence of Treatment-Emergent Adverse Events Investigations	1 participants
Incidence of Treatment-Emergent Adverse Events Musculoskeletal and Connective tissue Disorders	1 participants
Incidence of Treatment-Emergent Adverse Events Nervous System Disorders	2 participants
Incidence of Treatment-Emergent Adverse Events Renal and Urinary Disorders	1 participants

Adverse Events

Experimental: PTX-022

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Experimental: PTX-022 n=12 participants at risk PTX-022 QTORIN PTX-022: Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations
Gastrointestinal disorders Abdominal pain	8.3% 1/12 • Adverse events were collected from screening until Month 4
Gastrointestinal disorders Diarrhoea	8.3% 1/12 • Adverse events were collected from screening until Month 4
Gastrointestinal disorders Nausea	16.7% 2/12 • Adverse events were collected from screening until Month 4
General disorders Application Site Discharge	8.3% 1/12 • Adverse events were collected from screening until Month 4
General disorders Application Site Erythema	8.3% 1/12 • Adverse events were collected from screening until Month 4
General disorders Application Site Pain	25.0% 3/12 • Adverse events were collected from screening until Month 4
General disorders Application Site Paraesthesia	8.3% 1/12 • Adverse events were collected from screening until Month 4
General disorders Application Site Pruritus	25.0% 3/12 • Adverse events were collected from screening until Month 4
General disorders Nodule	8.3% 1/12 • Adverse events were collected from screening until Month 4
Infections and infestations Folliculitis	8.3% 1/12 • Adverse events were collected from screening until Month 4
Infections and infestations Nasopharyngitis	8.3% 1/12 • Adverse events were collected from screening until Month 4
Investigations Reticulocyte count increased	8.3% 1/12 • Adverse events were collected from screening until Month 4
Musculoskeletal and connective tissue disorders Joint Swelling	8.3% 1/12 • Adverse events were collected from screening until Month 4
Nervous system disorders Headache	16.7% 2/12 • Adverse events were collected from screening until Month 4
Renal and urinary disorders Micturition urgency	8.3% 1/12 • Adverse events were collected from screening until Month 4
Skin and subcutaneous tissue disorders Blister	8.3% 1/12 • Adverse events were collected from screening until Month 4
Skin and subcutaneous tissue disorders Dermatitis Contact	8.3% 1/12 • Adverse events were collected from screening until Month 4
Skin and subcutaneous tissue disorders Eczema	8.3% 1/12 • Adverse events were collected from screening until Month 4
Skin and subcutaneous tissue disorders Skin Exfoliation	8.3% 1/12 • Adverse events were collected from screening until Month 4
Skin and subcutaneous tissue disorders Urticaria	8.3% 1/12 • Adverse events were collected from screening until Month 4

Additional Information

Christine Kampf, Vice President of Regulatory Affairs

Palvella Therapeutics, Inc.

Phone: 484-844-0865

Email: christine.kampf@palvellatx.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place