SLC6A1-NDD Prospective Longitudinal Natural History Study
NCT07531511 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 60
Last updated 2026-04-15
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.
Conditions
- SLC6A1 Neurodevelopmental Disorder (NDD)
- Developmental and Epileptic Encephalopathies
Sponsors & Collaborators
-
UCB Biopharma SRL
lead INDUSTRY
Principal Investigators
-
UCB Cares · 001 844 599 2273 (UCB)
Eligibility
- Max Age
- 17 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2026-04-30
- Primary Completion
- 2029-05-25
- Completion
- 2029-05-25
More Related Trials
-
Natural History Studies of Mucopolysaccharidosis III
NCT02037880 ·Status: COMPLETED
-
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
NCT00344331 ·Status: RECRUITING
-
Longitudinal Study of Phenotypic and Developmental Severity in Patients With Dravet Syndrome With SCN1A Gene Mutation
NCT07251673 ·Status: RECRUITING
-
Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients
NCT04098211 ·Status: ACTIVE_NOT_RECRUITING
-
Krabbe Disease Global Patient Registry
NCT02993796 ·Status: RECRUITING
-
Genetic Developmental and Epileptic Encephalopathy Natural History Study for Clinical Trial Readiness
NCT07413211 ·Status: RECRUITING
-
STXBP1 and SYNGAP1 Related Disorders Natural History Study
NCT06555965 ·Status: RECRUITING
-
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
NCT01822184 ·Status: COMPLETED
-
Natural History Study of Patients With MPS IIIA
NCT02746341 ·Status: COMPLETED
-
Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
NCT01938014 ·Status: COMPLETED
-
Registry of Patients Diagnosed With Lysosomal Storage Diseases
NCT05619900 ·Status: RECRUITING
-
Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia
NCT06560346 ·Status: WITHDRAWN
-
Longitudinal Studies to Identify Biomarkers for Sturge-Weber Syndrome
NCT04717427 ·Status: RECRUITING
-
Carisbamate in Adult & Pediatric Subjects With Lennox-Gastaut Syndrome
NCT03731715 ·Status: COMPLETED ·Phase: PHASE1
-
N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
NCT03759665 ·Status: COMPLETED ·Phase: PHASE2
-
Natural History Study of Serious Medical Events in PWS
NCT03718416 ·Status: COMPLETED
-
Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome
NCT03047980 ·Status: COMPLETED ·Phase: PHASE2/PHASE3
-
A Natural History Study of Sanfilippo Syndrome Type D
NCT05648851 ·Status: COMPLETED
-
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
NCT03759639 ·Status: COMPLETED ·Phase: PHASE2
-
A Natural History Study of Angelman Syndrome
NCT07417137 ·Status: RECRUITING
-
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
NCT05163288 ·Status: RECRUITING ·Phase: PHASE3
-
Clinical Utility of Pediatric Whole Exome Sequencing
NCT03525431 ·Status: COMPLETED ·Phase: NA
-
The Brain and Neuropsychological Functioning in Adults With Sapropterin Dihydrochloride Treated Phenylketonuria
NCT02297347 ·Status: COMPLETED
-
Mucopolysaccharidosis Type II Natural History
NCT03529786 ·Status: COMPLETED
-
The Natural History of Infantile Globoid Cell Leukodystrophy
NCT00983879 ·Status: COMPLETED