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Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants

NCT04987463 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 60

Last updated 2024-02-08

No results posted yet for this study

Summary

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).

Conditions

  • Tuberous Sclerosis Complex

Interventions

DRUG

Vigabatrin

Vigabatrin in capsules administered orally, initially (between V1 and V2) once daily in the evening,and starting from V2 administered two times daily.

DRUG

Rapamycin

Rapamycin in liquid administered orally, in the morning, every other day or daily depending on the patient's body weight. The starting dose of rapamycin will be calculated according to the body weight of the patient measured at V1.

DRUG

Placebo

Placebo in liquid administered orally, once daily, in the morning. The starting dose of placebo in liquid will be calculated according to the body weight of the patient measured at V1.

DRUG

Placebo

Placebo in granules administered orally, initially once daily in the evening,and after reaching the targeted dose administered two times daily.

Sponsors & Collaborators

  • Katarzyna Kotulska

    lead OTHER

Principal Investigators

  • Katarzyna Kotulska-Jozwiak · The Children's Memorial Health Institute

Study Design

Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
4 Weeks
Max Age
16 Weeks
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-05-07
Primary Completion
2026-03-31
Completion
2026-03-31

Countries

  • Poland

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04987463 on ClinicalTrials.gov