Date: 2017-03-01
Type of
information: Initiation of the trial
phase:
Announcement: initiation of the trial
Company: Audentes Therapeutics (USA - CA)
Product:
Action
mechanism:
Disease: Crigler-Najjar Syndrome
Therapeutic
area: Hepatic diseases - Liver diseases
Country:
Trial
details:
- LUSTRO is an international study of approximately 16 to 18 Crigler-Najjar patients one year of age or older. The primary objectives of LUSTRO are to characterize the disease course, natural history, serum bilirubin variability and phototherapy usage of Crigler-Najjar patients. LUSTRO will also assess the burden of disease on Crigler-Najjar patients and caregivers. In addition, LUSTRO has been designed as a run-in study to identify patients for potential enrollment in VALENS, the planned Phase 1 / 2 study of AT342, and is intended to serve as a longitudinal baseline and within-patient control for subjects who enroll in VALENS. As LUSTRO will be conducted at the same sites and using the same assessment tools, LUSTRO will also facilitate certain operational aspects of VALENS. (NCT03078881)
Latest
news:
- • On March 1, 2017, Audentes Therapeutics announced the commencement of LUSTRO, a prospective study designed to characterize the disease course in patients living with Crigler-Najjar Syndrome.
- LUSTRO will evaluate subjects prior to potential enrollment in VALENS, the planned Phase 1 / 2 clinical study to evaluate the safety and preliminary efficacy of AT342, the Audentes product candidate being developed to treat Crigler-Najjar Syndrome. The Investigational New Drug application (IND) for AT342 is active. Audentes plans to report preliminary data from VALENS in the fourth quarter of 2017.
Is
general: Yes
