Clinical Assessment Study in Crigler-Najjar Syndrome

See also
Status	Clinical Trial	Phase
Terminated	`NCT03223194` - Gene Transfer Clinical Study in Crigler-Najjar Syndrome	Phase 1
Completed	`NCT00461799` - Orlistat Treatment of Crigler-Najjar Disease	N/A
Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
Recruiting	`NCT03466463` - Gene Therapy for Severe Crigler Najjar Syndrome	N/A
Enrolling by invitation	`NCT03343756` - HepaStem Long-Term Safety Registry

See also

Status

Clinical Trial

Phase

Terminated

NCT03223194 - Gene Transfer Clinical Study in Crigler-Najjar Syndrome

Phase 1

Completed

NCT00461799 - Orlistat Treatment of Crigler-Najjar Disease

N/A

Recruiting

NCT05687474 - Baby Detect : Genomic Newborn Screening

Recruiting

NCT03466463 - Gene Therapy for Severe Crigler Najjar Syndrome

N/A

Enrolling by invitation

NCT03343756 - HepaStem Long-Term Safety Registry

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	NCT03078881
Study type	Observational
Source	Audentes Therapeutics
Contact
Status	Completed
Phase
Start date	March 10, 2017
Completion date	January 23, 2019

Clinical Assessment Study in Crigler-Najjar Syndrome — LUSTRO

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms