NCT03646240

ABI-009 (Nab-rapamycin) for Surgically-Refractory Epilepsy (RaSuRE)

Phase 1 Study of ABI-009 (Nab-rapamycin) for Surgically-Refractory Epilepsy (RaSuRE)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Seattle Children's Hospital · Academic / Other
• Sex: All
• Age: 3 Years – 26 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective, single-center, phase 1 safety study to investigate the safety, tolerability, seizure control, and quality of life in participants with medically-refractory epilepsy who failed epilepsy surgery. These participants will have continued seizures despite being at least 3 months post-epilepsy surgery (resective surgery with an intent to cure).

Detailed description

Seizures that are refractory to both medical and surgical therapy increase the risk of morbidity and mortality in children with epilepsy. At this point in time, options for these children are sparse and suboptimal. This hypothesis-driven phase 1 study aims to evaluate the use of a mammalian target of rapamycin \[mTOR\] inhibitor, ABI-009, in this subset of challenging participants. The underlying hypotheses being tested in trial are: (1) ABI-009 is a safe and well-tolerated medication in children who have medically-refractory epilepsy and have failed epilepsy surgery, and (2) The addition of ABI-009 therapy to the current clinical standard of continued antiepileptic medications results in improved epilepsy control. This is unique among trials of anti-epileptic medications in that it also studies mTOR inhibition in a non-Tuberous Sclerosis Complex (TSC) specific population for whom few additional effective therapies exist. Upon enrollment, participants will be continued and observed on their pre-existing, clinically prescribed antiepileptic drug (AED) regimen for 1 month. At the 1-month mark, participants will receive weekly ABI-009 intravenously at different dose levels in cohorts of 3 participants each using the standard 3+3 dose-finding design. ABI-009 will be continued for a total of 3 weeks. ABI-009 will then be discontinued and the participants will be observed for an additional 3 months. The investigators intend an expansion of the maximum tolerated dose (MTD) cohort to an estimated additional 6 participants for a maximum possible enrollment of 18 participants.

Conditions

• Epilepsy Intractable

Interventions

Type	Name	Description
DRUG	ABI-009	For dose finding, ABI-009 will start at 5 mg/m2/dose IV, once a week for three weeks, in cohorts of 3 participants each using the standard 3+3 dose-finding design

Timeline

• Start date: 2018-07-31
• Primary completion: 2022-08-22
• Completion: 2022-12-31
• First posted: 2018-08-24
• Last updated: 2023-05-06

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study