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

Status Active, not recruiting

Clinical Trial 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).

Clinical Trial Description

This Phase 1/2 study will gather data in an observational phase Natural History Cohort to further characterize and evaluate natural disease progression in male patients with genetically-confirmed X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR). The study will also evaluate the safety and tolerability, as assessed by frequency and severity of ocular and systemic adverse events, as well as preliminary clinical efficacy of a single intravitreal (IVT) injection of 4D-125 at two dose levels in this patient population in one or both eyes (the contralateral eye dose provided the subject is eligible and provides consent). 4D-125 has been developed as a gene replacement therapy for XLRP. After receiving 4D-125, patients will be followed for 24 months with continued safety follow-up and 36 additional months of long-term follow-up. Secondary endpoints will assess preliminary efficacy measures over time after 4D-125 administration. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04517149
Study type	Interventional
Source	4D Molecular Therapeutics
Contact
Status	Active, not recruiting
Phase	Phase 1/Phase 2
Start date	June 9, 2020
Completion date	May 2029

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See also
Status	Clinical Trial	Phase
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Completed	`NCT03116113` - A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112	Phase 1/Phase 2
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Enrolling by invitation	`NCT03584165` - Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa	Phase 3
Active, not recruiting	`NCT04312672` - Long-term Follow-up Gene Therapy Study for RPGR- XLRP
Recruiting	`NCT04868916` - An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa
Completed	`NCT03252847` - Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)	Phase 1/Phase 2
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Active, not recruiting	`NCT04794101` - Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene	Phase 3
Active, not recruiting	`NCT04671433` - Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene	Phase 3
Completed	`NCT03314207` - Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
Active, not recruiting	`NCT03316560` - Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations	Phase 1/Phase 2
Active, not recruiting	`NCT06333249` - A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)	Phase 2