Active — Not RecruitingPhase 1/2

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

United States21 participantsStarted 2020-06-09

Plain-language summary

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).

Who can participate

Age range12 Years

SexMALE

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Incidence and severity of TEAEs and serious adverse events (SAEs), including clinically significant changes in safety parameters

Timeframe: 24 months to 60 Months

Trial details

NCT IDNCT04517149

Sponsor4D Molecular Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-06

View on ClinicalTrials.gov More X-Linked Retinitis Pigmentosa trials