A Phase 2 Study of the Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy

NCT07680322 | Not Yet Recruiting Phase 2 DMC

• 2 other identifiers: CL-078-2012025-522330-31
• Study Type: interventional
• Target: 42
• Locations: 0 countries
• Sites: N/A

Brief Summary

This Phase 2, randomized, double-blind, placebo-controlled study will evaluate the safety, tolerability, and efficacy of oral AV078 in participants with refractory epilepsy due to tuberous sclerosis complex (TSC). Approximately 42 participants will be randomized in a 5:1 ratio to receive AV078 or placebo. The study will include a Screening Period collecting 4 weeks of pre-treatment Baseline data on seizure frequency, and progress to a 12-week Treatment Period, followed by an in person final follow-up visit approximately 2 weeks after the last dose.

Conditions

Tuberous Sclerosis Complex; Epilepsy

Keywords

Tuberous Sclerosis Complex (TSC); Refractory Epilepsy; Drug-Resistant Epilepsy; Seizures; mTORC1 Inhibitor; AV078; mTOR Pathway

Outcome Measures

Primary Outcomes (2)

• Change From Baseline in Seizure Frequency

  Change from Baseline in the number of seizures experienced in participants on active treatment. The number of seizures will be recorded by participants or their caregivers using a seizure diary.

  Baseline (28-day period prior to treatment) to end of treatment (Week 12)

• Clinical Global Impression of Change (CGI-C)

  Change from Baseline in symptoms of TSC, as evaluated by the study doctor.

  Week 12

Study Arms (2)

AV078

EXPERIMENTAL

Participants will receive oral AV078 once daily in addition to their stable background anti-seizure medication regimen for 12 weeks. Dosing will be titrated based on measured drug concentrations to achieve target exposure levels.

Drug: AV078

Placebo

PLACEBO COMPARATOR

Participants will receive matching oral placebo once daily in addition to their stable background anti-seizure medication regimen for 12 weeks. Dose adjustments may be performed in a manner similar to active treatment to maintain blinding.

Drug: Placebo

Interventions

AV078 DRUG

AV078 is a selective inhibitor of mammalian target of rapamycin complex 1 (mTORC1) administered orally once daily. Dosing may be adjusted based on measured drug concentrations to achieve target exposure levels.

AV078

Placebo DRUG

Matching oral placebo administered once daily. The placebo is formulated to match AV078 and may undergo dose adjustments similar to active treatment to maintain blinding.

Also known as: Matching Placebo

Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants aged 12 years and above.
• Diagnosis of TSC based on International TSC Consensus Group criteria.
• History of failure to adequately control seizures despite having been treated by two or more regimens of anti-seizure medications (ASMs).
• Receiving a stable dose of ASMs for at least 4 weeks at the start of the Screening Period and for the duration of the study.

You may not qualify if:

• History of any infection requiring use of antibiotics within the last four weeks.
• Current or history of any clinically significant mental or physical illness or condition other than TSC that the Investigator believes would create significant risk for participation in the study.
• Recent epilepsy surgery/major surgery or planned surgery during the study.
• Treatment with medicines which act in a similar way.
• Treatment with medicines that suppress the immune system.
• Treatment with medicines that may significantly affect the way the body handles the study drug.

Sponsors & Collaborators

• Aeovian Pharmaceuticals, Inc.: lead

MeSH Terms

Conditions

Tuberous Sclerosis; Epilepsy; Drug Resistant Epilepsy; Seizures

Condition Hierarchy (Ancestors)

Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Brain Diseases; Central Nervous System Diseases; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Davis Ryman, MD, PhD

CONTACT

510-961-1148; info@aeovian.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: This is a double-blind study in which participants, investigators, care providers, and outcome assessors are blinded to treatment assignment. Matching placebo is used to maintain blinding, and dose adjustments for placebo may be conducted in a manner similar to active treatment to preserve the blind.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants will be randomized in a 5:1 ratio to receive oral AV078 or matching placebo in parallel treatment arms.

Study Record Dates

• First Submitted: June 25, 2026
• First Posted: July 2, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): December 21, 2027
• Last Updated: July 2, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share