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

4

4D Molecular Therapeutics (4DMT)

Status and phase

Active, not recruiting
Phase 2
Phase 1

Conditions

X-Linked Retinitis Pigmentosa

Treatments

Biological: 4D-125 IVT Injection
Other: Observational

Study type

Interventional

Funder types

Industry

Identifiers

NCT04517149
4D-125-C001

Details and patient eligibility

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).

Full 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.

Enrollment

21 patients

Sex

Male

Ages

12+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

21 participants in 3 patient groups

4D-125 Dose Exploration
Experimental group
Description:
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.
Treatment:
Biological: 4D-125 IVT Injection
4D-125 Dose Expansion
Experimental group
Description:
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.
Treatment:
Biological: 4D-125 IVT Injection
Observational
Other group
Description:
Natural History
Treatment:
Other: Observational

Trial contacts and locations

7

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Data sourced from clinicaltrials.gov

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