Angelman Natural History Study - FAST Spain
NCT06115109 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 40
Last updated 2024-04-03
Summary
The goal of this study is to conduct a prospective, longitudinal study to observe the natural clinical progression and disease outcome of AS patients receiving no disease-modified intervention, with the purpose of obtaining data that will be useful for future clinical trials.
Conditions
Interventions
- OTHER
-
Non intervention, Observational study
Non intervention, Observational study
Sponsors & Collaborators
-
Parc Taulí Hospital Universitari
collaborator OTHER -
Puerta de Hierro University Hospital
lead OTHER
Principal Investigators
-
BELEN RUIZ ANTORAN, MD, Ph · Clinical Pharmacology Deparment, Hospital Universitario Puerta de Hierro Majadahonda
Eligibility
- Min Age
- 3 Months
- Max Age
- 99 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2023-12-01
- Primary Completion
- 2024-12-31
- Completion
- 2025-07-01
Countries
- Spain
Study Locations
More Related Trials
-
Natural History Study of Patients With Canavan Disease (CANinform Study)
NCT04126005 ·Status: COMPLETED
-
The Global Angelman Syndrome Registry
NCT05293184 ·Status: RECRUITING
-
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J
NCT06151600 ·Status: RECRUITING
-
Disease Progression in Women With X-linked Adrenoleukodystrophy
NCT06178120 ·Status: RECRUITING
-
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
NCT04880356 ·Status: RECRUITING
-
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
NCT04041102 ·Status: COMPLETED
-
Long-term Extension of GTX-102 in Angelman Syndrome
NCT06415344 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3
-
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
NCT07157254 ·Status: RECRUITING ·Phase: PHASE2
-
German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
NCT06095713 ·Status: ACTIVE_NOT_RECRUITING
-
Assessment of Macrophage Activation syndromE in STill's Disease
NCT06405152 ·Status: COMPLETED
-
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
NCT04259281 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Natural History Study of Serious Medical Events in PWS
NCT03718416 ·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
-
Clinical Study of a Single Ciliopathy: Alström Syndrome
NCT02890550 ·Status: TERMINATED
-
A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
NCT02435030 ·Status: COMPLETED
-
A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)
NCT04428281 ·Status: COMPLETED ·Phase: PHASE1
-
An Observational Study in Children and Adults With Stargardt Disease
NCT06591806 ·Status: RECRUITING
-
Natural History Study of ENPP1 Deficiency and the Early-onset Form of ABCC6 Deficiency
NCT05050669 ·Status: COMPLETED
-
Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia
NCT06560346 ·Status: WITHDRAWN
-
Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)
NCT02824068 ·Status: COMPLETED
-
Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB
NCT02293408 ·Status: TERMINATED
-
An 18-month Prospective Natural History Study to Gain Insight Into FSHD2 Pathophysiology and Disease Progression
NCT06079567 ·Status: RECRUITING ·Phase: NA
-
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
NCT06617429 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3
-
FA Clinical Outcome Measures
NCT03090789 ·Status: ACTIVE_NOT_RECRUITING
-
Fabry Disease Registry & Pregnancy Sub-registry
NCT00196742 ·Status: RECRUITING