Phase 1, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-015 in Patients with Retinitis Pigmentosa
Details
In this Phase 1, first-in-human, open-label, non-randomized, sequential assignment, dose-escalation study,
approximately 9 eligible patients with RP will be enrolled sequentially in up to 3 dose cohorts of RTx-015. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-015 in the study eye at Visit 3 (Day 0) and be followed for a total of 12 months.
Eligibility
You can join if...
NOTE: This is not a comprehensive list of inclusion and exclusion criteria. Addtional criteria apply.
Inclusion Criteria:
Adults greater than or equal to 18 years of age
Informed consent obtained from the patient
Willing and able to comply with the study visit schedule and all protocol assessments
Diagnosis of retinitis pigmentosa (RP) defined as all of the following:
a. Clinical diagnosis of RP independent of causative mutation
i. If prior genetic testing was performed and the causative mutation is known, this data should be collected at
Screening.
ii. If no prior genetic testing was done or the prior genetic testing did not identify a causative mutation, genetic testing will
be done during Screening, but results are not required for determination of study eligibility.
b. Documentation of <10 microvolts maximal b-wave electroretinography (ERG) on any prior ERG consistent with rod-cone
photoreceptor degeneration
i. If no prior ERG is available, full field ERG should be completed in at least the study eye and interpreted locally as part
of Screening per site procedure.
Study Eye Visual Acuity
a. Best corrected distance visual acuity (BCVA) of LogMAR 1.0 (20/200) up to LogMAR 2.6 (20/8000), measured at the ETF
i. Grating visual acuity will be used when visual acuity cannot be measured by ETDRS chart
b. Study eye and fellow eye BCVA must be within or equal to 0.3logMAR
c. If both eyes are eligible, the worse-seeing eye should be selected as the study eye. If both eyes are eligible and of similar
visual acuity, the study eye may be selected by the Investigator in consultation with the patient.
Presence of retinal ganglion cells and/or retinal nerve fiber layer on SD-OCT testing at Screening confirmed by central image reading center
Exclusion Criteria
Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent or therapy in the past six months
Concurrent participation in another interventional clinical ocular study
Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy
Pre-existing eye conditions in either eye that would preclude the planned treatment or, in the opinion of the Investigator, are significant enough to interfere with the interpretation of study endpoints or procedural complications.
a. Examples may include, but are not limited to glaucoma, refractive error greater than –8D or +6D, amblyopia, diseases
affecting the optic nerve causing significant visual field loss, active uveitis or media opacities sufficient to preclude
examination of the posterior segment.
b. The presence of cystoid macular edema (CME) is not considered an exclusion criterion.
Known steroid responders if their intraocular pressure was not able to be managed effectively with topical pressure-lowering medications after prior use of steroid medications.
Complicating systemic diseases; complicating systemic diseases include those in which the disease itself, or the treatment for the disease, can alter ocular and/or CNS function (e.g., radiation treatment of the orbit; leukemia with CNS/optic nerve involvement).
Any immunological response dysfunction including, immuno-compromising diseases or use of immunosuppressive medications, among others.