Immunity Against AAV in Crigler Najjar Patient

Clinical Trial Summary

The proposed research protocol aims at addressing these points by pre-screening CN patients for their AAV serology in link with their medical history and current medical status. A first objective is to assess the presence of neutralizing AAV antibodies in the serum of CN patients.

See also
Status	Clinical Trial	Phase
Completed	`NCT02051049` - Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)
Completed	`NCT01765283` - Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN)	Phase 1/Phase 2
Recruiting	`NCT02356978` - New Phototherapy Device to Treat Patients With Crigler-Najjar Disease	N/A

See also

Status

Clinical Trial

Phase

Completed

NCT02051049 - Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)

Completed

NCT01765283 - Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN)

Phase 1/Phase 2

Recruiting

NCT02356978 - New Phototherapy Device to Treat Patients With Crigler-Najjar Disease

N/A

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	NCT02302690
Study type	Observational
Source	Genethon
Contact
Status	Completed
Phase	N/A
Start date	November 2014
Completion date	June 2017

Immunity Against AAV in Crigler Najjar Patient

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms