Natural History of Netherton Syndrome
NCT05902663 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 12
Last updated 2025-10-10
Summary
The goal of this non-interventional study (NIS) is to collect real-world data to describe the natural history of Netherton Syndrome (NS).
Conditions
Sponsors & Collaborators
- lead INDUSTRY
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2024-10-09
- Primary Completion
- 2025-03-31
- Completion
- 2025-03-31
Countries
- United States
- Italy
Study Locations
More Related Trials
-
A Natural History Study of Sanfilippo Syndrome Type D
NCT05648851 ·Status: COMPLETED
-
Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB
NCT02293408 ·Status: TERMINATED
-
A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
NCT02435030 ·Status: COMPLETED
-
Natural History Study of Patients With VCP-related Disease
NCT04823143 ·Status: COMPLETED
-
Angelman Syndrome Natural History Study
NCT04507997 ·Status: RECRUITING
-
Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome
NCT05521438 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2/PHASE3
-
Angelman Syndrome Natural History Study-FAST UK
NCT05100810 ·Status: RECRUITING
-
Mucopolysaccharidosis Type II Natural History
NCT03529786 ·Status: COMPLETED
-
Further Delineation of the De Santo Shinawi Syndrome Phenotype Using a Series of Individuals Carrying a Pathogenic Variant of the WAC Gene
NCT06807723 ·Status: RECRUITING
-
Facioscapulohumeral Dystrophy in Children
NCT02625662 ·Status: COMPLETED
-
Natural History Study of ENPP1 Deficiency and the Early-onset Form of ABCC6 Deficiency
NCT05050669 ·Status: COMPLETED
-
Efficacy and Safety of Sirolimus to Vascular Anomalies
NCT03583307 ·Status: COMPLETED ·Phase: NA
-
Natural History Study of Patients With MPS IIIA
NCT02746341 ·Status: COMPLETED
-
Natural History Study of Children With Metachromatic Leukodystrophy
NCT01963650 ·Status: TERMINATED
-
Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
NCT01509768 ·Status: COMPLETED
-
The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)
NCT05705674 ·Status: RECRUITING
-
A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
NCT02293382 ·Status: COMPLETED
-
Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin in Werner Syndrome
NCT05847179 ·Status: NOT_YET_RECRUITING ·Phase: PHASE2
-
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J
NCT06151600 ·Status: RECRUITING
-
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
NCT06078553 ·Status: RECRUITING
-
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
NCT00344331 ·Status: RECRUITING
-
A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome
NCT01894958 ·Status: COMPLETED ·Phase: PHASE2
-
Precision Medicine for Neurocutaneous Syndromes in Western China
NCT07327164 ·Status: COMPLETED
-
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
NCT04470713 ·Status: COMPLETED
-
Mucopolysaccharidosis Type II Observational
NCT04591834 ·Status: WITHDRAWN