Single-patient emergency use: new zealand

8 evidence topics · 1 source

Objective

Location and study date

New Zealand case: treatment initiation

“An infant with SCN2A-DEE began receiving relutrigine in February 2024 at age 4 months” (opens the source at this quote in a new tab)

Study design

Eligibility criteria

Treatment

Starting daily dose

“0.5 mg/kg/day” (opens the source at this quote in a new tab)

Study outcomes

Rationale for using motor seizure frequency as the reported outcome

Not applicable.

Statistical analysis description

Number of participants (Planned and analyzed)

Single treated infant

“An infant with SCN2A-DEE began receiving relutrigine in February 2024 at age 4 months” (opens the source at this quote in a new tab)

Description of analysis sets

Not applicable.

Results

Participant disposition

Baseline characteristics

Diagnosis and age at treatment initiation

“An infant with SCN2A-DEE began receiving relutrigine in February 2024 at age 4 months” (opens the source at this quote in a new tab)

Efficacy results

Single-patient emergency use: SCN2A-DEE, New Zealand

“>50% reduction in seizures at 3mg/kg/day” (opens the source at this quote in a new tab)

Health-related quality of life and patient-reported outcomes

Clinical observation of alertness and responsiveness

“the patient has been more alert and awake, responding to, and looking towards, familiar voices.” (opens the source at this quote in a new tab)

Safety results

Reported adverse-event findings

“no drug-related or severe adverse events.” (opens the source at this quote in a new tab)

Study limitations

Summary

This report describes one participant treated from four months of age. The observations are uncontrolled, and the reported seizure reduction at 3 mg/kg/day cannot establish comparative effectiveness or population-level safety. Alertness and responsiveness are clinical observations, not a randomized assessment of health-related quality of life.