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Gene Therapy Trial Report

Summary

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


NCTID NCT04517149 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication X-Linked Retinitis Pigmentosa (XLRP)
Disease Ontology Term DOID:0110414
Compound Name 4D-125
Sponsor 4D Molecular Therapeutics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 21 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant RPGR
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravitreal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV
Editor Type none
Dose 1 3E11 vg/eye
Dose 2 1E12 vg/eye

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2020-08-14
Completion Date 2029-05
Last Update 2025-03-21

Participation Criteria


Eligible Age >=12 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study MALE
Eligibility Criteria
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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 8
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates January 2025: Sponsor announced they would terminate development of this program

Resources/Links