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Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

ConditionsTuberous Sclerosis ComplexEpilepsy
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-6
SponsorDarcy Krueger

About this trial

This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants.

This study is supported by research funding from the Office of Orphan Products Division (OOPD) of the US Food and Drug Administration (FDA).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

0-6 months of age at the time of enrollment (subject must be <7 months of chronological age at time of randomization and treatment initiation). Corrected age must be at least 39 weeks (calculated by subtracting the number of weeks born before 40 weeks gestation from the chronological age).

Has a confirmed diagnosis of TSC based on established clinical or genetic criteria

Disqualifiers

Prior history of seizures (clinical or electrographic) at the time of enrollment or identified on baseline EEG.

Has been treated in the past or is currently being treated at the time of enrollment with conventional anticonvulsant medications (AEDs), systemic (oral) mTOR inhibitors (such as rapamycin, sirolimus, or everolimus), ketogenic-related special diet, or another anti-seizure therapeutic agent, device, or procedure.

Has taken any other investigational drug as part of another research study, within 30 days prior to the baseline screening visit.

Has a significant illness or active infection at the time of the baseline screening visit

Trial design

Design model

Parallel

Treatments tested in this trial

  • Sirolimus

    Drug

    The investigational drug product to be used in this study is sirolimus, provided in oral suspension.

  • Placebo

    Drug

    Matching placebo

Treatment groups

64 Participants
are divided into 2 treatment groups
Group A: SirolimusExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Efficacy -- time to seizure onset

Time to seizure onset, comparing sirolimus with placebo

Time frame
12 months of age
2

Safety -- adverse events

Percentage of subjects reporting severe (CTCAE v5.0 grade \>= 3) adverse event (AE) or serious adverse event (SAE), comparing sirolimus with placebo.

Time frame
12 months of age

Secondary outcomes

1

Neurodevelopmental Outcomes

Neurodevelopmental outcomes at the end of treatment, comparing sirolimus with placebo.

Time frame
12 and 24 months of age
2

Quality of Life Outcomes

Patient and caregiver quality of life, comparing sirolimus with placebo.

Time frame
12 and 24 months of age
3

EEG Biomarkers

EEG measures of neuronal connectivity, comparing sirolimus with placebo.

Time frame
12 and 24 months of age
4

MRI Biomarkers

MRI measures of neuronal connectivity, comparing sirolimus with placebo.

Time frame
12 and 24 months of age

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Darcy Krueger

Lead sponsor

Children's Hospital Medical Center, Cincinnati

Sponsor institution

Source ClinicalTrials.gov
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