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Gene Transfer Clinical Study in Crigler-Najjar Syndrome

NCT03223194 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2022-05-18

No results posted yet for this study

Summary

This is a Phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and preliminary efficacy of AT342 in subjects with Crigler-Najjar aged ≥1 year. Subjects will receive a single dose of AT342 and will be followed for safety and efficacy for 5 years.

Conditions

  • Crigler-Najjar Syndrome

Interventions

GENETIC

AT342

AT342 is an AAV8 vector containing a functional copy of the UGT1A1 gene.

Sponsors & Collaborators

  • Audentes Therapeutics

    lead INDUSTRY

Principal Investigators

  • Suyash Prasad, M.D. · Audentes Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
1 Year
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-09-08
Primary Completion
2021-02-11
Completion
2021-02-11
FDA Drug
Yes

Countries

  • United States
  • Israel
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03223194 on ClinicalTrials.gov