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Crigler-Najjar Syndrome - Healing Genes
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Crigler-Najjar Syndrome

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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


Phase 1 / 2

DESCRIPTION:

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.


PATIENT MUST:

  • 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:

  1. Screening to confirm eligibility.
  2. Participants receive a single infusion of the investigatory gene therapy.
  3. Phototherapy will continue for 12 weeks, when bilirubin levels will be assessed.
  4. Phototherapy will then discontinue for 4 weeks, when bilirubin levels will be assessed again.
  5. 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.
 
Contacts:
Kim Trant, Director of Patient Advocacy  |  (415) 805-1049  |  [email protected]
 
SPONSOR INFORMATION:
Audentes Therapeutics
 
Or go online:
https://clinicaltrials.gov/ct2/show/NCT03223194

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