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

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Darcy Krueger
Study ID
NCT05104983
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
1 Day - 6 Months
Healthy Volunteers
Not accepted

Interventions

  • Sirolimus — DRUG
    The investigational drug product to be used in this study is sirolimus, provided in oral suspension.
  • Placebo — DRUG
    Matching placebo

Study Details

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).

Key Dates

Start date
Oct 13, 2021
Status verified
Jan 2026
Primary completion
Jun 30, 2026
Completion
Jun 30, 2027

Study Design

Enrollment
64 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION

Arms

  • Experimental: Sirolimus
    Sirolimus
  • Placebo Comparator: Placebo
    Placebo

Primary Outcome Measure

Efficacy -- time to seizure onset [ Time Frame: 12 months of age ]

Central Contacts

Locations (11)

FacilityCityStateZIPSite coordinators
University of Alabama at BirminghamBirminghamAlabama35294
Jessica Krefting, RN
E. Martina Bebin, MD, MPA (PRINCIPAL_INVESTIGATOR)
University of California at Los AngelesLos AngelesCalifornia90095
Angela Martinez
Rajsekar Rajamaran, MD, MS (PRINCIPAL_INVESTIGATOR)
Stanford UniversityPalo AltoCalifornia94304
Rayann Solidum
[email protected]
Brenda Porter, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital ColoradoAuroraColorado80045
Ryan Osselborn
[email protected]
720-777-0493
Marissa Anthony
[email protected]
720-777-3119
Kevin Ess, MD (PRINCIPAL_INVESTIGATOR)
Lurie Children's Hospital of ChicagoChicagoIllinois60611
Enrique Rojas
[email protected]
312-227-2532
Sofia Mirshed
[email protected]
312-227-4525
Tracy Gertler, MD (PRINCIPAL_INVESTIGATOR)
Boston Children's HospitalBostonMassachusetts02115
Sahana Raman
Mustafa Sahin, MD, PhD (PRINCIPAL_INVESTIGATOR)
Washington University -- St. LouisSt LouisMissouri63110
Olga Novak
Michael Wong, MD, PhD (PRINCIPAL_INVESTIGATOR)
University of North Carolina at Chapel HillChapel HillNorth Carolina27510-
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229
Molly S Griffith, BA
[email protected]
513-636-9669
Darcy A Krueger, MD, PhD (PRINCIPAL_INVESTIGATOR)
University of Texas HSC at HoustonHoustonTexas77030
Mariam Lugo Noguera
Hope Northrup, MD (PRINCIPAL_INVESTIGATOR)
Seattle Children's HospitalSeattleWashington98105
Mikaela Morales
Stephanie Randle, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Birmingham, AL

By condition
Epilepsy
By specialty
NeurologyBrain disorders
By research site
University of Alabama at Birmingham· Birmingham, ALUniversity of California at Los Angeles· Los Angeles, CAStanford University· Palo Alto, CAChildren's Hospital Colorado· Aurora, COLurie Children's Hospital of Chicago· Chicago, ILBoston Children's Hospital· Boston, MA

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