NCT07377032RecruitingPhase 3
TAP-GRIN: Interventional Study on Patients With GRIN-related Neurodevelopmental Disorders
L-serine Supplementation in Patients With GRIN-related Neurodevelopmental Disorders: Multicentre Protocol for an Aggregated Series of Randomised, Placebo-controlled N-of-1 Trials
Source: ClinicalTrials.gov · Last updated 29 Jan 2026
Brief summary (registry, in English)
The goal of this clinical study is to find out whether L-serine dietary supplementation helps improve overall clinical functioning in children and young adults (2-30 years) with GRIN-related neurodevelopmental disorders (GRIN-NDD) caused by loss-of-function (LoF) variants in GRIN1, GRIN2A, GRIN2B, or GRIN2D. It will also assess the safety and tolerability of L-serine. The main questions it aims to answer are: Does L-serine improve overall clinical status, measured mainly by the Clinical Global Impression-Severity (CGI-S) score? Does L-serine improve behaviour, cognition, adaptive functioning, motor skills, sleep, and (in those with epilepsy) seizure frequency and EEG findings? What side effects or medical problems occur during L-serine compared with placebo? Do neurophysiological measures (including TMS-EMG/TMS-EEG) change with treatment and potentially act as biomarkers of response? Researchers will compare L-serine to a placebo (maltodextrin powder with similar appearance/texture) using a randomised, double-blind, placebo-controlled "n-of-1" approach, where each participant receives both treatments in alternating periods. Results from multiple single-patient trials will then be combined (aggregated) to estimate the overall treatment effect across the study population. Participants will: Complete a 4-week baseline period with assessments (and seizure diary use where applicable) Receive L-serine and placebo in alternating 3-month periods within each cycle (minimum 2 cycles, up to 4 cycles; each cycle lasts 6 months) Take the assigned study product by mouth 3 times per day at 500 mg/kg/day (maximum 30 g/day for participants ≥60 kg) Have the first 7 days of each 3-month period treated as washout, with data from that week not analysed Attend regular clinic visits for clinical exams, safety labs, and standardized assessments of global status, behaviour/cognition, motor function, and sleep If they have epilepsy: keep a seizure diary and undergo EEG assessments after each treatment period In some sites (Italy and France): undergo TMS-based neurophysiology testing Optionally, a subset may join a cellular biomarker substudy (blood collection to generate iPSC-derived neuronal models and organoids) to explore treatment effects in variant-specific lab models.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 40 (estimated)
- First submitted
- 19 Jan 2026
- Start date
- 29 Aug 2025
- Primary completion date
- Jun 2028
- Lead sponsor
- Meyer Children's Hospital IRCCS
- Diseases
- Epilepsy
- NCT number
- NCT07377032
What you commit to
- Study type: Interventional · Phase 3
- Planned participants: 40
- From 29 Aug 2025 to the primary completion date, Jun 2028
- 3 sites in 3 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Participation in another clinical trial involving an investigational medicinal product within the previous 6 months.
Other illness
- Known hypersensitivity or intolerance to L-serine, placebo, or any excipients used in the study formulations.
- Presence of a clinically significant unstable medical condition (other than epilepsy) that, in the investigator's judgement, may place the participant at increased risk or interfere with study participation.
- Any other significant disease or disorder that may compromise participant safety, affect study outcomes, or impair the participant's ability to complete the study procedures.
Age
- Age younger than 2 years at screening.
Other
- Inadequate supervision by parent(s) or caregiver(s), as judged by the investigator.
- Female participants who are pregnant or breastfeeding.
- Presence of a GRIN1, GRIN2A, GRIN2B, or GRIN2D variant for which a clear loss-of-function effect cannot be demonstrated.
Who the study is looking for
- Clinical diagnosis of a GRIN-related neurodevelopmental disorder (GRIN-NDD)
- Presence of a pathogenic or likely pathogenic loss-of-function (LoF) variant in GRIN1, GRIN2A, GRIN2B, or GRIN2D
- Parent(s), caregiver(s), or legally authorised representative(s) have been informed of the nature of the study and have provided written informed consent.
- Participants who are able to do so have provided written informed consent or assent, according to local regulations and cognitive capacity.
- Parent(s)/caregiver(s) are willing and able to comply with study procedures and visits, in the opinion of the investigator.
- Participants who have previously received L-serine supplementation are willing to discontinue L-serine for at least one week prior to the baseline observation period.
Age in the registry: 2 years to 30 years · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Hospices Civils de Lyon Coordinator of this site? Confirm its status | Lyon | France | Not yet recruiting |
| Meyer Children's Hospital Coordinator of this site? Confirm its status | Florence | Italy | Recruiting |
| Children's Memorial Health Institute Coordinator of this site? Confirm its status | Warsaw | Poland | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.