NCT04987463 (ViRap) is a Phase 2/Phase 3 clinical trial of Vigabatrin in Tuberous Sclerosis Complex, sponsored by Katarzyna Kotulska.

Who is sponsoring NCT04987463?

NCT04987463 is sponsored by Katarzyna Kotulska.

What drugs are being tested in NCT04987463?

Interventions in NCT04987463: Vigabatrin, Rapamycin, Placebo, Placebo.

What condition does NCT04987463 study?

NCT04987463 studies Tuberous Sclerosis Complex.

Is NCT04987463 still recruiting?

NCT04987463 is currently status unknown. Last updated 7 February 2024.

Last reviewed 2024-02-07 · How we verify

NCT04987463: ViRap

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Randomized, Placebo-controlled, Double-blind and Double-dummy Clinical Trial Comparing the Safety, Tolerability, and Efficacy of Vigabatrin and Rapamycin in a Preventive Treatment of Infants With Tuberous Sclerosis Complex

Status unknown Phase 2/Phase 3 Last updated 7 February 2024

What this trial tests

Phase 2/Phase 3 trial testing Vigabatrin in Tuberous Sclerosis Complex in 60 participants. Status unknown.

Timeline

7 May 2021

Primary endpoint
1 March 2026

1 March 2026

Quick facts

Lead sponsor	Katarzyna Kotulska
Phase	Phase 2/Phase 3
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	prevention
Enrollment	60
Start date	7 May 2021
Primary completion	1 March 2026
Estimated completion	1 March 2026
Sites	2 locations across Poland

Drugs / interventions tested

Vigabatrin — full drug profile →
Rapamycin — full drug profile →
Placebo
Placebo

Conditions studied

Tuberous Sclerosis Complex — all drugs for Tuberous Sclerosis Complex →

Sponsor

Katarzyna Kotulska

Who can join

Adults 4 Weeks to 16 Weeks, 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.

Occurrence of clinical seizures in the blinded phase of the study,
Time frame: 730 days
Summarized volume of TSC-associated tumors ≥ 125% of initial value within the blinded phase of the study
Time frame: 730 days

Sponsor's own description

The purpose of the study is to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with Tuberous Sclerosis Complex (TSC).

Publications & conference data

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

Perfect match: mTOR inhibitors and tuberous sclerosis complex.
Luo C, Ye WR, Shi W, Yin P, et al · · 2022 · cited 52× · PMID 35246210 · DOI 10.1186/s13023-022-02266-0
Preventative treatment of tuberous sclerosis complex with sirolimus: Phase I safety and efficacy results.
Capal JK, Ritter DM, Franz DN, Griffith M, et al · · 2024 · cited 8× · PMID 39726432 · DOI 10.1002/cns3.20070
Long-term neuropsychologic outcome of pre-emptive mTOR inhibitor treatment in children with tuberous sclerosis complex (TSC) under 4 months of age (PROTECT), a two-arm, randomized, observer-blind, controlled phase IIb national multicentre clinical trial: study protocol.
Driedger JH, Schröter J, PROTECT-Study Group, Syrbe S, et al · · 2025 · cited 5× · PMID 39762914 · DOI 10.1186/s13023-024-03495-1
Mechanistic strategies for secondary prevention of developmental and epileptic encephalopathy in children with tuberous sclerosis complex.
Specchio N, Di Micco V, Scheper M, Aronica E, et al · · 2025 · cited 2× · PMID 40367637 · DOI 10.1016/j.ebiom.2025.105740

Verify or expand the search:

Other trials of Vigabatrin

Trials testing the same drug.

NCT04321395 — Vigabatrin and Insulin Sensitivity · Phase 2 · completed
NCT03421496 — A Study to Assess Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms · Phase 3 · terminated
NCT02299115 — Prednisolone Versus Vigabatrin in the First-line Treatment of Infantile Spasms · Phase 3 · withdrawn

Other recruiting trials for Tuberous Sclerosis Complex

Currently open trials in the same condition.

NCT06160310 — Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry) · recruiting
NCT05104983 — Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study · Phase 2 · recruiting
NCT05467397 — Feasibility of [11C]Acetate-PET in LAM and TSC · Phase 1, PHASE2 · active not recruiting
NCT05044819 — Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution · Phase 4 · active not recruiting
NCT04198181 — The Effectiveness and Safety of Resective Epilepsy Surgery for TRE · active not recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04987463 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Katarzyna Kotulska
Last refreshed: 7 February 2024

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04987463.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing