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

Study Purpose

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).

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 1 Day - 6 Months
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. 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). 2. Has a confirmed diagnosis of TSC based on established clinical or genetic criteria.

Exclusion Criteria:

1. Prior history of seizures (clinical or electrographic) at the time of enrollment or identified on baseline EEG. 2. 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. 3. Has taken any other investigational drug as part of another research study, within 30 days prior to the baseline screening visit. 4. Has a significant illness or active infection at the time of the baseline screening visit. 5. Has a history of significant prematurity, defined as gestational age <30 weeks at the time of delivery, or other significant medical complications at birth or during the neonatal period that other than TSC would convey additional risk of seizures or neurodevelopmental delay (i.e. HIE, severe neonatal infection, major surgery, prolonged ventilatory or other life-saving supportive care or procedures). 6. Abnormal laboratory values at baseline (i.e., renal function, liver function, or bone marrow production) that are in the opinion of the investigator clinically significant and may jeopardize the safety of the study subject. 7. Prior, planned or anticipated neurosurgery within 3 months of the baseline visit. 8. Has a TSC-associated condition for which mTOR treatment is clinically indicated (i.e. SEGA or AML). 9. Subjects who are, in the opinion of the investigator, unable to comply with the requirements of the study.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT05104983
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Darcy Krueger
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Darcy A Krueger, MD, PhDMartina Bebin, MD, MPA
Principal Investigator Affiliation Children's Hospital Medical Center, CincinnatiUniversity of Alabama at Birmingham
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Tuberous Sclerosis Complex, Epilepsy
Additional Details

Tuberous Sclerosis Complex (TSC) is caused by genetic mutation in TSC1 or TSC2, resulting in dysregulation of the mechanistic target of rapamycin (mTOR) signaling pathway. Age at time of seizure onset in TSC infants has been linked to long-term neurodevelopmental outcome in this high-risk population. Sirolimus is an mTOR inhibitor used to treat many of the symptoms of TSC, including epilepsy. This will be the first study to truly evaluate a targeted, disease-modifying drug therapy for preventing or delaying seizure onset in TSC using a rational, mechanism-based therapeutic approach.

Arms & Interventions

Arms

Experimental: Sirolimus

Sirolimus

Placebo Comparator: Placebo

Placebo

Interventions

Drug: - Sirolimus

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

Drug: - Placebo

Matching placebo

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

University of Alabama at Birmingham, Birmingham, Alabama

Status

Recruiting

Address

University of Alabama at Birmingham

Birmingham, Alabama, 35294

Site Contact

Jessica Krefting, RN

info@tscsteps.org

513-636-9669

University of California at Los Angeles, Los Angeles, California

Status

Recruiting

Address

University of California at Los Angeles

Los Angeles, California, 90095

Site Contact

Angela Martinez

info@tscsteps.org

513-636-9669

Stanford University, Palo Alto, California

Status

Recruiting

Address

Stanford University

Palo Alto, California, 94304

Site Contact

Jennifer Winterbottom

info@tscsteps.org

513-636-9669

Boston Children's Hospital, Boston, Massachusetts

Status

Recruiting

Address

Boston Children's Hospital

Boston, Massachusetts, 02115

Site Contact

Emine Arcasoy

info@tscsteps.org

513-636-9669

Washington University -- St. Louis, Saint Louis, Missouri

Status

Recruiting

Address

Washington University -- St. Louis

Saint Louis, Missouri, 63110

Site Contact

Olga Novak

info@tscsteps.org

513-636-9669

Chapel Hill, North Carolina

Status

Recruiting

Address

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27510

Site Contact

Hannah Riehl

info@tscsteps.org

513-636-9669

Cincinnati, Ohio

Status

Recruiting

Address

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229

Site Contact

Molly S Griffith, BA

info@tscsteps.org

513-636-9669

University of Texas HSC at Houston, Houston, Texas

Status

Recruiting

Address

University of Texas HSC at Houston

Houston, Texas, 77030

Site Contact

Alexis Rodriguez

info@tscsteps.org

513-636-9669

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