NCT02849457

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex (PREVeNT Trial) A Randomized, Double-blind, Placebo-controlled Seizure Prevention Clinical Trial for Infants With TSC

Completed Phase 2 Results posted Last updated 22 July 2024

What this trial tests

Phase 2 trial testing Early Vigabatrin in Tuberous Sclerosis Complex in 84 participants. Completed in 5 May 2023.

Timeline

1 December 2016

Primary endpoint
26 April 2023

5 May 2023

Quick facts

Lead sponsor	Martina Bebin
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	prevention
Enrollment	84
Start date	1 December 2016
Primary completion	26 April 2023
Estimated completion	5 May 2023
Sites	13 locations across United States

Drugs / interventions tested

• Early Vigabatrin — full drug profile →
• Delayed Vigabatrin (Placebo) — full drug profile →

Conditions studied

• Tuberous Sclerosis Complex — all drugs for Tuberous Sclerosis Complex →

Sponsor

Martina Bebin — full company profile →

Who can join

Adults 1 Day to 6 Months, any sex, with Tuberous Sclerosis Complex. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Cognitive Assessment Scores and Developmental Impact
  Time frame: 24 months
  The primary outcome measure will be the standardized Cognitive scale scores on the Bayley Scales of Infant and Toddler Development- Third Edition at 24 months. The Cognitive scale Composite score is a standard score derived from the observed and elicited performance of the child on cognitive assessment tasks, with a mean of 100 and standard deviation of 10. The range for the Cognitive scale Compos

Sponsor's own description

Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to the onset of their first seizure

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Presentation and Diagnosis of Tuberous Sclerosis Complex in Infants.
   Davis PE, Filip-Dhima R, Sideridis G, Peters JM, et al · · 2017 · cited 91× · PMID 29101226 · DOI 10.1542/peds.2016-4040
2. Repurposed molecules for antiepileptogenesis: Missing an opportunity to prevent epilepsy?
   Klein P, Friedman A, Hameed MQ, Kaminski RM, et al · · 2020 · cited 73× · PMID 32196665 · DOI 10.1111/epi.16450
3. A clinical update on tuberous sclerosis complex-associated neuropsychiatric disorders (TAND).
   de Vries PJ, Wilde L, de Vries MC, Moavero R, et al · · 2018 · cited 73× · PMID 30117265 · DOI 10.1002/ajmg.c.31637
4. Scalp EEG spikes predict impending epilepsy in TSC infants: A longitudinal observational study.
   Wu JY, Goyal M, Peters JM, Krueger D, et al · · 2019 · cited 56× · PMID 31691264 · DOI 10.1111/epi.16379
5. Tuber Locations Associated with Infantile Spasms Map to a Common Brain Network.
   Cohen AL, Mulder BPF, Prohl AK, Soussand L, et al · · 2021 · cited 47× · PMID 33410532 · DOI 10.1002/ana.26015
6. Update on Drug Management of Refractory Epilepsy in Tuberous Sclerosis Complex.
   van der Poest Clement E, Jansen FE, Braun KPJ, Peters JM. · · 2020 · cited 44× · PMID 31912454 · DOI 10.1007/s40272-019-00376-0
7. Review of the treatment options for epilepsy in tuberous sclerosis complex: towards precision medicine.
   Schubert-Bast S, Strzelczyk A. · · 2021 · cited 34× · PMID 34349839 · DOI 10.1177/17562864211031100
8. Prevention of premature death and seizures in a Depdc5 mouse epilepsy model through inhibition of mTORC1.
   Klofas LK, Short BP, Zhou C, Carson RP. · · 2020 · cited 34× · PMID 32280987 · DOI 10.1093/hmg/ddaa068