AN OPEN-LABEL DOSE ESCALATION STUDY OF AN ADENO-ASSOCIATED VIRUS VECTOR (AAV2/2-hRPE65p-hRPE65) FOR GENE THERAPY OF SEVERE EARLY-ONSET RETINAL DEGENERATION - RPE65 gene therapy

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2006-001571-37-GB

Enrollment

Registered

2008-11-20

Start date

2006-12-14

Completion date

Unknown

Last updated

2019-11-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

The condition to be investigated is severe early-onset inherited retinal degeneration due to defects in the gene encoding RPE65 MedDRA version: 13.1 Level: PT Classification code 10038845 Term: Retinal degeneration System Organ Class: 10015919 - Eye disorders

Interventions

Product Name: tgAAG76 Pharmaceutical Form: Suspension for injection Current Sponsor code: tgAAG76 Other descriptive name: AAV vector serotype 2, contain

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. individuals who have severe early-onset retinal dystrophy 2. individuals who are homozygous or compound heterozygous for a mutation(s) in RPE65 3. individuals who are able to give informed consent, with or without the guidance of their parent/guardian where appropriate 4. individuals aged 5 to 30 years Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. individuals who have contraindications for transient immune-suppression (hypertension, diabetes mellitus, tuberculosis, renal impairment, immunocompromise, osteoporosis, gastric ulceration, severe affective disorder) 2. are pregnant or lactating women

Design outcomes

Primary

Measure	Time frame
Main Objective: The main objective is to assess the safety and efficacy of a single subretinal administration of tgAAG76 in up to 12 patients with retinal dystrophy due to mutations in RPE65. ;Secondary Objective: ;Primary end point(s): The primary end point for each subject is defined as any improvement in visual (rod or cone-derived) function, as determined by an array of psychophysical and electrophysiological techniques, that is greater than the test-retest variation for each test.	—

Measure

Time frame

Main Objective: The main objective is to assess the safety and efficacy of a single subretinal administration of tgAAG76 in up to 12 patients with retinal dystrophy due to mutations in RPE65. ;Secondary Objective: ;Primary end point(s): The primary end point for each subject is defined as any improvement in visual (rod or cone-derived) function, as determined by an array of psychophysical and electrophysiological techniques, that is greater than the test-retest variation for each test.

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Countries

United Kingdom

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026