PALV-06

15 evidence topics · 5 sources

Objective

Objective: safety and efficacy of topical QTORIN rapamycin

“determine the safety and efficacy” (opens the source at this quote in a new tab)

Location and study date

PALV-06: study setting

“5 clinical sites in the United States.” (opens the source at this quote in a new tab)

Study completion period

“the 4th quarter of 2022” (opens the source at this quote in a new tab)

Study design

Phase 2 study design

“multicenter, baseline-controlled Phase 2 clinical trial.” (opens the source at this quote in a new tab)

Eligibility criteria

PALV-06: age eligibility

“Participants were ≥6 years old” (opens the source at this quote in a new tab)

Treatment

Treatment and duration

“Participants self-administered topical QTORIN rapamycin once daily for 12 weeks.” (opens the source at this quote in a new tab)

Study outcomes

Rationale for using Treatment-Emergent adverse events as the primary endpoint

Not applicable.

Statistical analysis description

Number of participants (Planned and analyzed)

Enrolled population

Population	Quoted number
Participants enrolled	“12”

Description of analysis sets

Efficacy analysis: nominal significance and paired comparisons

“no adjustment for multiplicity amongst efficacy endpoints” (opens the source at this quote in a new tab)
“paired t-tests vs mean change of 0 as compared to baseline” (opens the source at this quote in a new tab)

Formal analysis-set definitions

No evidence found.

Results

Participant disposition

PALV-06: discontinuations

Information	Source quotation
Discontinuations	“No study participants discontinued”

Baseline characteristics

No evidence found.

Efficacy results

PALV-06: phase 2 efficacy, clinician-reported endpoint

“Efficacy Endpoints”	“Week 12”“Mean”“(n=12)”	“Nominal, Two-sided p-value”
“Clinician Global Impression of Change (CGI-C)”	“2.42”	“<0.0001”

Clinician-rated response: much improved or very much improved

“100% of participants” (opens the source at this quote in a new tab)

Health-related quality of life and patient-reported outcomes

PALV-06: phase 2 efficacy, patient-reported endpoint

“Efficacy Endpoints”	“Week 12”“Mean”“(n=12)”	“Nominal, Two-sided p-value”
“Patient Global Impression of Change (PGI-C)”	“2.08”	“<0.0001”

Additional quality-of-life instrument collected

“dermatology life quality index” (opens the source at this quote in a new tab)

Numerical dermatology life quality index results

No evidence found.

Safety results

PALV-06: phase 2 safety

Information	Source quotation
Serious adverse events	“No serious adverse events”
Most common treatment-related adverse events	“application site pain (n=3, 25.0%), application site pruritus (n=3, 25.0%), and nausea (n=2, 16.7%).”

Study limitations

Summary

PALV-06 enrolled 12 participants in an open-label, baseline-controlled study with 12 weeks of treatment. There was no concurrent comparator. Efficacy testing was exploratory, with nominal p-values and no adjustment for multiplicity. The sample size and duration limit assessment of uncommon adverse events and durability after treatment cessation. The abstract identifies a dermatology life quality index assessment but does not provide its numerical results.