Other Clinical Trial - Natural History Study in Pediatric Patients With

See also
Status	Clinical Trial	Phase
Recruiting	`NCT01238250` - Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight
Recruiting	`NCT05232630` - Fenfluramine for the Treatment of Different Types of Developmental and Epileptic Encephalopathies: a Pilot Trial Exploring Epileptic and Non-epileptic Outcomes	Phase 4
Not yet recruiting	`NCT06356233` - Phenotyping and Identification of Biological Markers in STXBP1 Encephalopathy
Recruiting	`NCT05161494` - Gait in Rare Diseases

See also

Status

Clinical Trial

Phase

Recruiting

NCT01238250 - Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

Recruiting

NCT05232630 - Fenfluramine for the Treatment of Different Types of Developmental and Epileptic Encephalopathies: a Pilot Trial Exploring Epileptic and Non-epileptic Outcomes

Phase 4

Not yet recruiting

NCT06356233 - Phenotyping and Identification of Biological Markers in STXBP1 Encephalopathy

Recruiting

NCT05161494 - Gait in Rare Diseases

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT05462054
Study type	Observational
Source	Capsida Biotherapeutics, Inc.
Contact	Medical Monitor
Email	clinicaltrials@capsida.com
Status	Not yet recruiting
Phase
Start date	July 2023
Completion date	January 2026

Natural History Study in Pediatric Patients With STXBP1 Encephalopathy With Epilepsy

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms