US-based biotechnology firm Audentes Therapeutics has begun the run-in study (LUSTRO) for Phase I/II clinical trial (VALENS) of AT342 for the treatment of patients with Crigler-Najjar Syndrome.
AT342 is an AAV8 vector containing a functional copy of the UGT1A1 gene designed to treat patients with severely high levels of unconjugated bilirubin in the blood, as well as risk of neurological damage and death.
The international, prospective LUSTRO study will evaluate around 16 to 18 Crigler-Najjar patients aged one or older.
It has been designed as a run-in study to identify patients for potential enrolment in VALENS and to examine the burden of disease on patients and caregivers.
VALENS is the planned Phase I/II clinical trial of AT342.
Audentes senior vice president and chief medical officer Dr Suyash Prasad said: "LUSTRO is designed to further our understanding of the baseline characteristics of patients living with Crigler-Najjar Syndrome, and the burden of disease as experienced by patients, families and caregivers.
"We greatly appreciate the opportunity to work with the Crigler-Najjar community and view the initiation of LUSTRO as an important milestone as we seek to develop an innovative gene therapy for this devastating rare disease."
The primary objectives of LUSTRO are to establish the disease course, natural history, serum bilirubin variability and phototherapy usage of the patients.
The firm intends to conduct the run-in study at the same sites and by using the same assessment tools to enable certain operational aspects of VALENS.
Daily, persistent phototherapy, usually for longer than 12 hours per day is the current standard of care for Crigler-Najjar Syndrome.