NCT07531511 SLC6A1-NDD Prospective Longitudinal Natural History Study
| NCT ID | NCT07531511 |
| Status | Recruiting |
| Phase | — |
| Sponsor | UCB Biopharma SRL |
| Condition | SLC6A1 Neurodevelopmental Disorder (NDD) |
| Study Type | OBSERVATIONAL |
| Enrollment | 60 participants |
| Start Date | 2026-07-21 |
| Primary Completion | 2029-05-25 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 60 participants in total. It began in 2026-07-21 with a primary completion date of 2029-05-25.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The overall objective of this prospective longitudinal natural history study is to collect clinical data to characterize and evaluate the natural course of SLC6A1-NDD and assess the feasibility of certain assessments for the purpose of conducting future clinical studies in patients with this disease.
Eligibility Criteria
Inclusion Criteria: * Patient with a diagnosis of SLC6A1-NDD characterized by epilepsy, global developmental delay, autism spectrum disorder, or intellectual disability, with a documented history of an SLC6A1 mutation, defined as pathogenic or likely pathogenic by the Investigator. * Patients should not be older than 17 years at time of assent/consent. * Patients under the age of 18 years with legal guardians providing informed consent. Assent will be obtained from any patients judged to have sufficient capacity to provide assent at the discretion of the Investigator. * Patient and patient's caregiver are willing and able to comply with study requirements (including diary completion and visit schedule). Exclusion Criteria: * Patients and their caregivers are unable to complete follow-up visits. * Patients with a history of an alternate diagnosis for disease, including a genetic cause, which is known to contribute to epilepsy or NDD. * Patient is currently receiving an investigational product(s) other than 4-phenylbutyrate or has received an investigational product within 30 days or within \<5 times the half-life of the investigational product, whichever is longer, prior to the Enrollment Visit.
Contact & Investigator
UCB Cares
STUDY DIRECTOR
001 844 599 2273 (UCB)
Frequently Asked Questions
Who can join the NCT07531511 clinical trial?
This trial is open to participants of all sexes, up to 17 Years, studying SLC6A1 Neurodevelopmental Disorder (NDD). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07531511 currently recruiting?
Yes, NCT07531511 is actively recruiting participants. Contact the research team at ucbcares@ucb.com for enrollment information.
Where is the NCT07531511 trial being conducted?
This trial is being conducted at New York, United States, Strasbourg, France, Madrid, Spain.
Who is sponsoring the NCT07531511 clinical trial?
NCT07531511 is sponsored by UCB Biopharma SRL. The principal investigator is UCB Cares at 001 844 599 2273 (UCB). The trial plans to enroll 60 participants.