Single-patient emergency use: germany

8 evidence topics · 1 source

Objective

Location and study date

Treating center location

“Munich, Germany” (opens the source at this quote in a new tab)

Exact date of relutrigine initiation

No evidence found.

Study design

Eligibility criteria

Treatment

Adjunctive relutrigine dose

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

Study outcomes

Rationale for using seizure frequency as the reported outcome

Not applicable.

Statistical analysis description

Number of participants (Planned and analyzed)

Case population

“A preterm infant” (opens the source at this quote in a new tab)

Description of analysis sets

Not applicable.

Results

Participant disposition

Baseline characteristics

Gestational age

“29+4 weeks gestation” (opens the source at this quote in a new tab)

Efficacy results

Seizure frequency after adjunctive relutrigine

“as low as 2 seizures per hour” (opens the source at this quote in a new tab)

Emergency-use case: antisense oligonucleotide treatment combined with precision sodium channel modulation

“Continued improvement in clinical status permitted a previously unattainable reduction in carbamazepine dosage.” (opens the source at this quote in a new tab)

Health-related quality of life and patient-reported outcomes

Safety results

Safety after adjunctive relutrigine

“no new safety findings” (opens the source at this quote in a new tab)

Study limitations

Summary

This is one emergency-use case, not a randomized study. Relutrigine was used in a treatment course that also included an antisense oligonucleotide and carbamazepine. The report therefore does not isolate the effect of relutrigine from other treatment changes, and the reduction in carbamazepine dosage should not be interpreted as proof of efficacy for relutrigine alone.