PARKS

20 evidence topics · 2 sources

Objective

Pharmacokinetics, pharmacodynamics, safety, and tolerability in children and adolescents

“An open-label study to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of epinephrine administered as a single dose of DESF in pediatric subjects weighing ≥30 kg with a personal history of allergic reactions and are at high risk for serious allergic reactions.” (opens the source at this quote in a new tab)

Location and study date

Study dates and number of sites

Field	Quoted record
Study start (actual)	“2024-12-26”
Primary completion (actual)	“2025-03-14”
Study completion (actual)	“2025-05-21”
Number of locations	“This study has 8 locations”

Participating sites in the United States and Canada

“AllerVie Clinical Research” (opens the source at this quote in a new tab)
“UCLA Pediatric Allergy & Immunology” (opens the source at this quote in a new tab)
“Children's Hospital Colorado” (opens the source at this quote in a new tab)
“Texas Children's Hospital - Feigin Center” (opens the source at this quote in a new tab)
“Halton Pediatric Allergy” (opens the source at this quote in a new tab)

Study design

Single-treatment open-label design

“This is a Phase 1, multi-site, open-label, single treatment study conducted in male and female pediatric subjects (age 7-17 years) who have a have a personal history of allergic reactions and are at high risk for serious allergic reactions. This study is designed to evaluate the pharmacokinetics (PK) of epinephrine administered as a single dose of DESF.” (opens the source at this quote in a new tab)

Study schedule and duration

“The expected maximum duration of an individual's participation in this study will be, approximately, 32 days including a 30-day Screening Period, a 1-day Treatment Period (Study Day 1) and a 1-day (±1 day) Post-treatment Follow-up (Study Day 2).” (opens the source at this quote in a new tab)

Eligibility criteria

Key inclusion criteria

“Male and female children ages 7 through 17 years of age at the start of the Screening Period.” (opens the source at this quote in a new tab)
“Participants with a known history of allergic reactions with an active prescription for epinephrine, and who (in the judgement of the investigator) are at high risk for serious allergic reactions (e.g., anaphylaxis).” (opens the source at this quote in a new tab)
“Participant has a body weight ≥30 kg (66 lbs).” (opens the source at this quote in a new tab)
“Participant is within the 5th and 95th percentiles, inclusive, for weight by age (according to CDC growth charts).” (opens the source at this quote in a new tab)

Key exclusion criteria

“Any current or past medical conditions such as significant systemic diseases that, in the investigator's opinion, might significantly affect pharmacodynamic responses to DESF.” (opens the source at this quote in a new tab)
“Known intolerance or hypersensitivity to any component of DESF.” (opens the source at this quote in a new tab)
“Participant has a disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of DESF.” (opens the source at this quote in a new tab)

Treatment

Single 12 mg sublingual dose

“All treated participants will be administered a single, 12 mg, dose of DESF on Study Day 1. No other investigational product will be administered.” (opens the source at this quote in a new tab)

Route of administration

“DESF administered in the sublingual space” (opens the source at this quote in a new tab)

Study outcomes

Rationale for using baseline-corrected maximum epinephrine plasma concentration as the primary endpoint

Registered primary outcome measures

Outcome measure	Quoted description	Quoted time frame
Cmax	“Baseline Corrected Maximum Epinephrine Plasma Concentration”	“0 to 240 minutes post-dose”
AUC0-10	“Baseline Corrected Plasma Epinephrine Concentration-Time Curve from Time Zero to 10 minutes”	“0 to 10 minutes”
AUC0-20	“Baseline Corrected Plasma Epinephrine Concentration-Time Curve from Time Zero to 20 minutes”	“0 to 20 minutes”
AUC0-30	“Baseline Corrected Plasma Epinephrine Concentration-Time Curve from Time Zero to 30 minutes”	“0 to 30 minutes”
AUC0-45	“Baseline Corrected Plasma Epinephrine Concentration-Time Curve from Time Zero to 45 minutes”	“0 to 45 minutes”

Purpose of matching adult exposure to support pediatric labeling

“These data are an important component of the FDA review process and could enable Anaphylm to have a label that includes this pediatric patient population.” (opens the source at this quote in a new tab)

Oral mucosal tolerability assessment

“On Study Day 2, participants will return to the study site for an Oral Mucositis Assessment Scale (OMAS) assessment.” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

Planned and actual enrollment

Field	Quoted record
Planned enrollment	“Up to 24 DESF treated pediatric participants are expected to be enrolled.”
Enrollment (actual)	“32”

Participants who completed the study

“A total of thirty-two patients completed the study.” (opens the source at this quote in a new tab)

Description of analysis sets

No evidence found.

Results

Participant disposition

Completion of the study

“A total of thirty-two patients completed the study.” (opens the source at this quote in a new tab)

Baseline characteristics

Age and weight of the enrolled population

“today announced positive topline results from its pediatric study for Anaphylm™ (epinephrine) sublingual film in patients aged seven to seventeen and weighing greater than thirty kilograms with a personal history of allergic reactions.” (opens the source at this quote in a new tab)

Efficacy results

Pharmacokinetic profile compared with adult studies

“The pediatric study was a multi-site, single treatment study designed to evaluate the PK, pharmacodynamics (PD), safety, and tolerability of Anaphylm. A total of thirty-two patients completed the study. The PK results were consistent with previous adult studies.” (opens the source at this quote in a new tab)

Comparison figure against the adult pivotal study

“Geometric mean baseline adjusted epinephrine concentration over time after administration of Anaphylm 12mg in pediatric subjects (Study AQ109302) compared to adult subjects (Study AQ109301B)” (opens the source at this quote in a new tab)

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

No serious adverse events

“Importantly, Anaphylm was shown to be safe and well-tolerated with no serious adverse events (SAEs) reported.” (opens the source at this quote in a new tab)

Study limitations

Summary: study limitations

The study was open label, gave a single 12 mg dose with no comparator arm, and enrolled 32 participants aged 7 through 17 years who weighed at least 30 kg, so it provides no evidence for younger or lighter children, for whom 0.15 mg injectable products are used. Participants were dosed while well rather than during an allergic reaction. Results have been described only in a company announcement and the registry record carries no posted results, so individual pharmacokinetic parameters, variability, and the Oral Mucositis Assessment Scale findings are not publicly reported.