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Also known as 4D-125 IVT Injection, 4D-125 IVT
4D-125 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| 4D-125 Dose Exploration | EXPERIMENTAL | Dose 1 and Dose 2 4D-125 will be administered at the assigned dose level as a single dose, IVT injection on Day 1. The contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent. |
| 4D-125 Dose Expansion | EXPERIMENTAL | 4D-125 will be administered at the assigned dose level as a single dose, IVT injection on Day 1 in both adults and pediatric participants. For adult participants only, the contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent. |
| Observational | OTHER | Natural History |
| Name | Type | Description |
|---|---|---|
| 4D-125 IVT Injection | BIOLOGICAL | 4D-125 drug product developed for gene therapy, which comprises an AAV capsid variant (4D-R100) carrying a codon-optimized human Retinitis Pigmentosa GTPase Regulator transgene. |
| Observational | OTHER | Natural History |
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 test...
4D-125 is an investigational gene therapy being studied for the treatment of X-Linked Retinitis Pigmentosa (XLRP), a rare inherited retinal disease that causes progressive vision loss. It is designed to be administered by intravitreal injection into the eye. 4D-125 has not been approved by the FDA and remains in clinical development.
4D-125 is being developed by 4D Molecular Therapeutics, Inc., which trades on the Nasdaq under the ticker symbol FDMT. The company is the sponsor of the clinical program evaluating 4D-125 in patients with X-Linked Retinitis Pigmentosa.
4D-125 is in Phase 1 clinical development. The program consists of one active trial that is no longer recruiting participants. 4D-125 is investigational and has not received FDA approval for any indication.
4D-125 is being evaluated in the Phase 1 trial NCT04517149, titled "4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)." The study has enrolled 21 participants, is conducted in the United States, and is currently active but not recruiting. It enrolls male patients aged 12 years and older.
Yes. 4D-125 IVT Injection and 4D-125 IVT are alternative names for the same investigational therapy, 4D-125. The IVT designation refers to intravitreal administration, the route by which the therapy is delivered into the eye during the Phase 1 XLRP trial.