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Synaptic Plasticity and Cognitive Function in RASopathies

NCT ID: NCT03504501

Last Updated: 2023-11-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-03-22

Study Completion Date

2023-10-31

Brief Summary

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The project is targeting cognitive impairment, one of the main health problems of patients with RAS pathway disorders. The aim of this study is to translate findings of animal studies to humans. This has been done by the applicants successfully for Lovastatin in Nf1. This result will be transferred to patients with Noonan Syndrome. lamotrigine is most likely a more effective and promising substance improving synaptic plasticity and consecutive cognitive function. It is expected that both substances are improving synaptic plasticity as well as alertness and changes in alertness may be a precondition for improvement of cognition.

Detailed Description

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Conditions

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Impaired Synaptic Plasticity Impaired Cognition

Keywords

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RASopathies, neurofibromatosis type 1, noonan syndrome, synaptic plasticity, transcranial magnetic stimulation

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Monocenter, randomized, double-blind, parallel-group, placebo controlled, cross-over design with a series of three experiments (Noonan Syndrome: 2 experiments; Neurofibromatosis type 1 1 experiment) and n=14 participants per experiments
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Exp. I: Noonan Syndrome - Lovastatin

200 mg Lovastatin daily for four days / Lovastatin-placebo (cross-over) prior to transcranial magnetic stimulation and test of attentional performance

Group Type EXPERIMENTAL

Lovastatin

Intervention Type DRUG

oral application prior to transcranial magnetic stimulation intervention

Exp. II: Noonan Syndrome - Lamotrigine

300 mg Lamotrigine single dose / Lamotrigine-placebo prior to transcranial magnetic stimulation and test of attentional performance

Group Type EXPERIMENTAL

Lamotrigine

Intervention Type DRUG

oral application prior to transcranial magnetic stimulation intervention

Exp. III: Neurofibromatosis Type 1 - Lamotrigine

300 mg Lamotrigine single dose / Lamotrigine-placebo prior to transcranial magnetic stimulation and test of attentional performance

Group Type EXPERIMENTAL

Lamotrigine

Intervention Type DRUG

oral application prior to transcranial magnetic stimulation intervention

Interventions

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Lovastatin

oral application prior to transcranial magnetic stimulation intervention

Intervention Type DRUG

Lamotrigine

oral application prior to transcranial magnetic stimulation intervention

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Group 1: NS, Group 2: NF1 (both genetically assured)
2. Age ≥16 years
3. The adolescent (≥16) and legal guardian who are capable to give their consent and understand the aim and rationale of the study. In case of doubts, an independent medical practitioner will evaluate the capacity to consent.
4. Signed informed consent if ≥ 16 years and legal guardian.
5. Persons who are ≥ 18 years old and capable to give their consent and understand the aim and rationale of the study. In case of doubts, an independent medical practitioner will evaluate the capacity to consent.
6. Signed informed consent if ≥ 18 years.
7. Male participants and female participants who are not capable of bearing children or who use a method of contraception that is medically approved by the health authority of the respective country.

Exclusion Criteria

1. Epilepsy
2. Medication with known CNS effects
3. Severe mental retardation
4. Side effects during previous medication with and contraindications for LTG and/or LOV and/or TMS
5. Psychiatric diseases
6. Previous history of allergic reactions with LTG and LOV medications
7. Potentially unreliable patients
8. Patients who are not suitable for the study in the opinion of the investigator
9. Pregnancy (incl. positive urine pregnancy test)
10. Persons who are incapable of giving consent or do not understand the aim or rationale of the study.
Minimum Eligible Age

16 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Technical University of Munich

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Technical University Munich

Munich, , Germany

Site Status

Countries

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Germany

References

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Mainberger F, Jung NH, Zenker M, Wahllander U, Freudenberg L, Langer S, Berweck S, Winkler T, Straube A, Heinen F, Granstrom S, Mautner VF, Lidzba K, Mall V. Lovastatin improves impaired synaptic plasticity and phasic alertness in patients with neurofibromatosis type 1. BMC Neurol. 2013 Oct 2;13:131. doi: 10.1186/1471-2377-13-131.

Reference Type BACKGROUND
PMID: 24088225 (View on PubMed)

Jung NH, Egert-Schwender S, Schossow B, Kehl V, Wahllander U, Brich L, Janke V, Blankenstein C, Zenker M, Mall V. Improvement of synaptic plasticity and cognitive function in RASopathies-a monocentre, randomized, double-blind, parallel-group, placebo-controlled, cross-over clinical trial (SynCoRAS). Trials. 2023 Jun 6;24(1):383. doi: 10.1186/s13063-023-07392-z.

Reference Type DERIVED
PMID: 37280688 (View on PubMed)

Other Identifiers

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2016-005022-10

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

SYN-1748-MAL-0030-I

Identifier Type: -

Identifier Source: org_study_id