RECRUITING

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

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Conditions

Tuberous Sclerosis ComplexEpilepsyTSCpreventionmTORsirolimusinfant

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

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

Quick Facts

Study Start:2021-10-13
Study Completion:2026-06-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05104983

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:1 Day to 6 Months
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. 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. 2. Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
  1. 1. Prior history of seizures (clinical or electrographic) at the time of enrollment or identified on baseline EEG.
  2. 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. 3. Has taken any other investigational drug as part of another research study, within 30 days prior to the baseline screening visit.
  4. 4. Has a significant illness or active infection at the time of the baseline screening visit
  5. 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. 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. 7. Prior, planned or anticipated neurosurgery within 3 months of the baseline visit
  8. 8. Has a TSC-associated condition for which mTOR treatment is clinically indicated (i.e. SEGA or AML).
  9. 9. Subjects who are, in the opinion of the investigator, unable to comply with the requirements of the study.

Contacts and Locations

Study Contact

Molly S Griffith, BA
CONTACT
513-636-9669
info@tscsteps.org
Jessica Krefting, RN
CONTACT
256-533-0833
info@tscsteps.org

Principal Investigator

Darcy A Krueger, MD, PhD
PRINCIPAL_INVESTIGATOR
Children's Hospital Medical Center, Cincinnati
Martina Bebin, MD, MPA
PRINCIPAL_INVESTIGATOR
University of Alabama at Birmingham

Study Locations (Sites)

University of Alabama at Birmingham
Birmingham, Alabama, 35294
United States
University of California at Los Angeles
Los Angeles, California, 90095
United States
Stanford University
Palo Alto, California, 94304
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
Washington University -- St. Louis
Saint Louis, Missouri, 63110
United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27510
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
University of Texas HSC at Houston
Houston, Texas, 77030
United States
Seattle Children's Hospital
Seattle, Washington, 98105
United States

Collaborators and Investigators

Sponsor: Darcy Krueger

  • Darcy A Krueger, MD, PhD, PRINCIPAL_INVESTIGATOR, Children's Hospital Medical Center, Cincinnati
  • Martina Bebin, MD, MPA, PRINCIPAL_INVESTIGATOR, University of Alabama at Birmingham

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-10-13
Study Completion Date2026-06-30

Study Record Updates

Study Start Date2021-10-13
Study Completion Date2026-06-30

Terms related to this study

Keywords Provided by Researchers

  • Tuberous Sclerosis Complex
  • TSC
  • epilepsy
  • prevention
  • mTOR
  • sirolimus
  • infant

Additional Relevant MeSH Terms

  • Tuberous Sclerosis Complex
  • Epilepsy