Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (TSC-STEPS)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT05104983|
Recruitment Status : Recruiting
First Posted : November 3, 2021
Last Update Posted : September 6, 2022
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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||Intervention/treatment||Phase|
|Tuberous Sclerosis Complex Epilepsy||Drug: Sirolimus Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||64 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||This trial will employ a randomized, double-blind, placebo-controlled multisite design to evaluate the safety and efficacy of early sirolimus treatment to prevent or delay seizure onset in TSC infants.|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study|
|Actual Study Start Date :||October 13, 2021|
|Estimated Primary Completion Date :||June 30, 2025|
|Estimated Study Completion Date :||June 30, 2026|
The investigational drug product to be used in this study is sirolimus, provided in oral suspension.
Placebo Comparator: Placebo
- Efficacy -- time to seizure onset [ Time Frame: 12 months of age ]Time to seizure onset, comparing sirolimus with placebo
- Safety -- adverse events [ Time Frame: 12 months of age ]Percentage of subjects reporting severe (CTCAE v5.0 grade >= 3) adverse event (AE) or serious adverse event (SAE), comparing sirolimus with placebo.
- Neurodevelopmental Outcomes [ Time Frame: 12 and 24 months of age ]Neurodevelopmental outcomes at the end of treatment, comparing sirolimus with placebo.
- Quality of Life Outcomes [ Time Frame: 12 and 24 months of age ]Patient and caregiver quality of life, comparing sirolimus with placebo.
- EEG Biomarkers [ Time Frame: 12 and 24 months of age ]EEG measures of neuronal connectivity, comparing sirolimus with placebo.
- MRI Biomarkers [ Time Frame: 12 and 24 months of age ]MRI measures of neuronal connectivity, comparing sirolimus with placebo.
- Sirolimus Precision Dosing [ Time Frame: 12 months of age ]Validate the feasibility and effectiveness of sirolimus precision dosing in infants with TSC
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||1 Day to 6 Months (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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
- 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
- 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).
- 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.
- Prior, planned or anticipated neurosurgery within 3 months of the baseline visit
- Has a TSC-associated condition for which mTOR treatment is clinically indicated (i.e. SEGA or AML).
- Subjects who are, in the opinion of the investigator, unable to comply with the requirements of the study.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05104983
|Contact: Molly S Griffith, BAemail@example.com|
|Contact: Jessica Krefting, RNfirstname.lastname@example.org|
|United States, Alabama|
|University of Alabama at Birmingham||Recruiting|
|Birmingham, Alabama, United States, 35294|
|Contact: Jessica Krefting, RN|
|Principal Investigator: E. Martina Bebin, MD, MPA|
|United States, California|
|University of California at Los Angeles||Recruiting|
|Los Angeles, California, United States, 90095|
|Contact: Angela Martinez|
|Principal Investigator: Rajsekar Rajamaran, MD, MS|
|Palo Alto, California, United States, 94304|
|Contact: Jennifer Winterbottom|
|Principal Investigator: Brenda Porter, MD|
|United States, Massachusetts|
|Boston Children's Hospital||Recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: Emine Arcasoy|
|Principal Investigator: Mustafa Sahin, MD, PhD|
|United States, Missouri|
|Washington University -- St. Louis||Recruiting|
|Saint Louis, Missouri, United States, 63110|
|Contact: Olga Novak|
|Principal Investigator: Michael Wong, MD, PhD|
|United States, North Carolina|
|University of North Carolina at Chapel Hill||Recruiting|
|Chapel Hill, North Carolina, United States, 27510|
|Contact: Hannah Riehl|
|Principal Investigator: Jamie Capal, MD|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center||Recruiting|
|Cincinnati, Ohio, United States, 45229|
|Contact: Molly S Griffith, BA 513-636-9669 email@example.com|
|Principal Investigator: Darcy A Krueger, MD, PhD|
|United States, Texas|
|University of Texas HSC at Houston||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Alexis Rodriguez|
|Principal Investigator: Hope Northrup, MD|
|Principal Investigator:||Darcy A Krueger, MD, PhD||Children's Hospital Medical Center, Cincinnati|
|Principal Investigator:||Martina Bebin, MD, MPA||University of Alabama at Birmingham|
|Responsible Party:||Darcy Krueger, IND Sponsor/Lead Principal Investigator, Children's Hospital Medical Center, Cincinnati|
|Other Study ID Numbers:||
1R01FD007275 ( U.S. FDA Grant/Contract )
|First Posted:||November 3, 2021 Key Record Dates|
|Last Update Posted:||September 6, 2022|
|Last Verified:||September 2022|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Tuberous Sclerosis Complex
Central Nervous System Diseases
Nervous System Diseases
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Physiological Effects of Drugs