Actively Recruiting

Phase 1
Phase 2
Age: 8Years - 45Years
All Genders
NCT05858983

Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy

Led by Frontera Therapeutics · Updated on 2023-05-15

9

Participants Needed

1

Research Sites

365 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.

CONDITIONS

Official Title

Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy

Who Can Participate

Age: 8Years - 45Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Able to understand and sign the informed consent form
  • Female or male aged 8 to 45 years when signing the informed consent form
  • Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy
Not Eligible

You will not qualify if you...

  • Presence of other interfering eye diseases
  • Any systemic or eye disease that may cause vision loss
  • Evidence of uncontrolled concomitant diseases
  • Known active or suspected autoimmune diseases
  • Active systemic infection under treatment
  • Pregnant or lactating women
  • Other conditions deemed unsuitable by the investigator

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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2
3
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Trial Site Locations

Total: 1 location

1

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China, 100142

Actively Recruiting

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Research Team

X

Xinyan Li

CONTACT

M

Minghui Xue

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

3

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