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mTOR inhibitor

TAVT-18 (sirolimus) for Epilepsy (STOP2 Trial)

Phase 1 & 2

Waitlist Available

Led By Darcy Krueger, MD, PhD

Research Sponsored by Children's Hospital Medical Center, Cincinnati

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 and 24 months of age

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new form of sirolimus taken by mouth to help infants with Tuberous Sclerosis Complex. It aims to prevent seizures by calming an overactive pathway in their bodies. Sirolimus has been studied and used for seizure control in pediatric patients with Tuberous Sclerosis Complex (TSC).

Eligible Conditions

Epilepsy
Tuberous Sclerosis

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 and 24 months of age

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 and 24 months of age

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 and 24 months of age for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Efficacy - Time to Seizure Onset

Safety - Adverse Events

Secondary study objectives

Adaptive Behavior Assessed by the the VABS

Age at Seizure Onset

Global Neurodevelopment Assessed by the Bayley Scales of Infant Development

+6 more

Side effects data

From 2022 Phase 1 & 2 trial • 5 Patients • NCT04595513

80%

Hypertriglyceridemia

60%

Rhinitis

60%

Hypercholesterolemia

60%

Tussis (cough)

60%

Otitis Media

40%

Stomatitis (mouth sores/ulcers)

40%

Pyrexia (fever)

40%

Insomnia

40%

Rhinorrhea (Congestion)

40%

Constipation

40%

Excessive Flatulence

20%

Hematochezia (Bloody stool)

20%

Diarrhea

20%

Fussiness

20%

Discomfort/Pain due to Teething

20%

Fatigue

20%

Rash (Allergic reaction)

20%

Lip Lesion

20%

Dermatitis

20%

Eczema

20%

Emesis (vomiting)

20%

Rhinosinusitis (Sinus infection)

20%

Abscess w/Infection

20%

Croup w/Stridor

20%

Respiratory Syncytial Virus (RSV)

20%

Hand-Foot-Mouth Disease

20%

Hypertension

100%

80%

60%

40%

20%

Study treatment Arm

Stage 1 Open Label

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Stage 1 Open LabelExperimental Treatment1 Intervention

Phase I/II, open-label PK and initial safety analysis. TAVT-18 administered orally twice/daily to achieve precision dosing target of 10 ng/ml. Whole blood sirolimus levels are assessed at defined intervals on days 1, 7, and 14. After day 14, participants can elect to continue open-label treatment with TAVT-18 until 12 months of age. Final developmental outcomes are assessed at 24 months of age.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

TAVT-18 (sirolimus)

2020

Completed Phase 2

~10

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