Gene Transfer Clinical Study in Crigler-Najjar Syndrome (VALENS)
VALENS: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT342, an AAV8-Delivered Gene Transfer Therapy in Crigler-Najjar Syndrome Subjects Aged 1 Year and Older
Researchers at research centers in NY and PA as well as abroad are recruiting patients with Crigler-Najjar Syndrome to trial of a new gene therapy to correct mutations in the UGT1A1 gene. This therapy is a virus engineered to transfer a functional UGT1A1 gene to the patient, so that their bodies can process bilirubin normally.
Participants will undergo a single IV infusion of the investigatory gene therapy, then be assessed at 12 weeks and 18 weeks, with a total observation period of 24 weeks.
- Be 1 years of age or older
- Have a diagnosis of Crigler-Najjar syndrome resulting from a confirmed mutation in the UGT1A1 gene as assessed by a Sponsor-approved testing facility
- Be prescribed daily phototherapy for a minimum of 6 hours within a 24-hour period (daily illumination time).
THE STUDY INVOLVES:
- Screening to confirm eligibility.
- Participants receive a single infusion of the investigatory gene therapy.
- Phototherapy will continue for 12 weeks, when bilirubin levels will be assessed.
- Phototherapy will then discontinue for 4 weeks, when bilirubin levels will be assessed again.
- Total follow up will continue until 24 weeks post step (2).
LOCATIONS AND CONTACTS:
The study sites include
Children’s Hospital at Montefiore in Bronx, NY. Map.
Clinic for Special Children in Strasburg, PA. Map.
Kim Trant, Director of Patient Advocacy | (415) 805-1049 | [email protected]
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