Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with tuberous sclerosis complex (RAPIT-trial)

Efficacy of RAD001/everolimus in Autism and NeuroPsychological deficits in children with TSC (RAPIT-trial) - RAPIT

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2010-019519-39-NL

Enrollment

Registered

2011-12-01

Start date

2012-03-14

Completion date

Unknown

Last updated

2025-01-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Tuberous Sclerosis Complex

Interventions

Trade Name: Votubia Product Name: Everolimus Product Code: L04AA18 Pharmaceutical Form: Tablet INN or Proposed INN: Votubia CAS Number: 159351-69-6 Other descriptive name: EVEROLIMUS Concentration uni

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • Children with a definite diagnosis of TSC between 3 and 18 years. • With an IQ estimated =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: • Renal or liver dysfunction • Surgery <6wk before entering the study • Current infection at time of inclusion • Additional diseases or disorders that may influence the endpoints • Allergy for any of the components of the study medication

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective is to determine the effect of Everolimus on the cognition of children with TSC, measured by IQ.;Secondary Objective: We will also evaluate the effect on symptoms of autism spectrum disorder, other (neuro) psychological test parameters, seizure frequency, EEG-abnormalities and specific symptoms of learning disability. Furthermore, we will observe the tolerability of Everolimus in children with TSC.;Primary end point(s): Cognitive functioning: Mullen scales (IQ);Timepoint(s) of evaluation of this end point: IQ measurements will be obtained at inclusion and at the last study visit.	—

Measure

Time frame

Main Objective: The primary objective is to determine the effect of Everolimus on the cognition of children with TSC, measured by IQ.;Secondary Objective: We will also evaluate the effect on symptoms of autism spectrum disorder, other (neuro) psychological test parameters, seizure frequency, EEG-abnormalities and specific symptoms of learning disability. Furthermore, we will observe the tolerability of Everolimus in children with TSC.;Primary end point(s): Cognitive functioning: Mullen scales (IQ);Timepoint(s) of evaluation of this end point: IQ measurements will be obtained at inclusion and at the last study visit.

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Secondary

Measure	Time frame
Secondary end point(s): We will also evaluate the effect on symptoms of autism spectrum disorder, other (neuro) psychological test parameters, seizure frequency, EEG-abnormalities and specific symptoms of learning disability. Furthermore, we will observe the tolerability of Everolimus in children with TSC.;Timepoint(s) of evaluation of this end point: All tests will be executed at inclusion and at the last study visit.	—

Measure

Time frame

Secondary end point(s): We will also evaluate the effect on symptoms of autism spectrum disorder, other (neuro) psychological test parameters, seizure frequency, EEG-abnormalities and specific symptoms of learning disability. Furthermore, we will observe the tolerability of Everolimus in children with TSC.;Timepoint(s) of evaluation of this end point: All tests will be executed at inclusion and at the last study visit.

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Countries

Netherlands

Contacts

Public ContactClinical trials information

Erasmus MC

m.c.y.dewit@erasmusmc.nl+31107036595

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026