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X-Linked Retinitis Pigmentosa

4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)

Sponsored by 4D Molecular Therapeutics

About this trial

Last updated a year ago

Study ID

4D-125-C001

Status

Active, not recruiting

Type

Interventional

Phase

Phase 1/Phase 2

Placebo

No

Accepting

18-75 Years
12+ Years
Male
Male

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Started 5 years ago

What is this trial about?

This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retinitis pigmentosa (XLRP) due to mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR).

What are the Participation Requirements?

Natural History Key Inclusion Criteria:

- Male, ≥ 6 years of age at the time of informed consent

- Hemizygous non-syndromic RPGR mutation confirmed by genetic testing

Interventional Key Inclusion Criteria:

- Male, ≥12 years of age

- Hemizygous non-syndromic RPGR mutation confirmed by genetic testing

- Phase 1 Dose Exploration: At least one eye amenable to IVT injection and BCVA ≤ 78
ETDRS letters (~20/32) and ≥ 34 ETDRS letters (~20/200)

- Phase 2 Dose Expansion: At least one eye amenable to IVT injection AND both eyes
must have BCVA ≥ 34 ETDRS letters (~20/200)

Key Exclusion Criteria (all cohorts)

- Patient has previously received any AAV treatment

- Pre-existing eye conditions or surgical complications that would preclude
participation in an interventional clinical trial or interfere with the
interpretation of study endpoints

Locations

Location

Status