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NCT05104983 | RECRUITING | Tuberous Sclerosis Complex

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

Darcy Krueger

Information provided by (Responsible Party):

Darcy Krueger

Brief Summary:

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).

Condition or disease

Tuberous Sclerosis Complex

Epilepsy

Intervention/treatment

Sirolimus

Placebo

Phase

PHASE2

Detailed Description:

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.

Study Type : INTERVENTIONAL
Estimated Enrollment : 64 participants
Masking : TRIPLE
Primary Purpose : PREVENTION
Official Title : Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Actual Study Start Date : 2021-10-13
Estimated Primary Completion Date : 2025-12-31
Estimated Study Completion Date : 2027-06-30

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 1 Day to 6 Months
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
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.
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

Location Details

NCT05104983

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How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

United States, alabama

University of Alabama at Birmingham

Birmingham, alabama, United States, 35294

RECRUITING

United States, California

University of California at Los Angeles

Los Angeles, California, United States, 90095

RECRUITING

United States, California

Stanford University

Palo Alto, California, United States, 94304

RECRUITING

United States, Colorado

Children's Hospital Colorado

Aurora, Colorado, United States, 80045

RECRUITING

United States, Illinois

Lurie Children's Hospital of Chicago

Chicago, Illinois, United States, 60611

RECRUITING

United States, Massachusetts

Boston Children's Hospital

Boston, Massachusetts, United States, 02115

RECRUITING

United States, Missouri

Washington University -- St. Louis

St Louis, Missouri, United States, 63110

RECRUITING

United States, North Carolina

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States, 27510

RECRUITING

United States, Ohio

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

RECRUITING

United States, Texas

University of Texas HSC at Houston

Houston, Texas, United States, 77030

RECRUITING

United States, Washington

Seattle Children's Hospital

Seattle, Washington, United States, 98105