Nerve Growth Factor Eye Drops as a Novel Treatment for Vision Loss in Patients With Retinitis Pigmentosa: From Preclinical to Clinical Phase II Trial

Ospedale San Raffaele2 sites in 1 country45 target enrollmentMay 2015

ConditionsRetinitis Pigmentosa Cystoid Macular Edema

InterventionsrhNGF 180 µg/ml eye drops solution vehicle eye drops

DrugsrhNGF 180 µg/ml eye drops solution vehicle eye drops

Overview

Phase: Phase 2
Intervention: rhNGF 180 µg/ml eye drops solution
Conditions: Retinitis Pigmentosa
Sponsor: Ospedale San Raffaele
Enrollment: 45
Locations: 2
Primary Endpoint: macular thickness
Status: Completed
Last Updated: 8 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This study aims at investigating the therapeutic potential of recombinant human Nerve Growth Factor ( rhNGF ) eye drops treatment in patients with Retinitis Pigmentosa (RP) associated with cystoid macular edema (CME) in a phase II, randomized, double-masked, controlled clinical trial.

Registry

clinicaltrials.gov

Start Date

May 2015

End Date

February 2017

Last Updated

8 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Ospedale San Raffaele

Responsible Party

Principal Investigator

Paolo Rama

Ospedale San Raffaele

Eligibility Criteria

Inclusion Criteria

•Typical and atypical forms of retinitis pigmentosa (RP)
•Measurable ERG with a significant decrease in the amplitude .
•Reduction of the visual field (assessed with Humphrey field analyzer- HFA 24-2) and values of mean deviation\> -3 decibel (dB).
•RP associated with CME documented by OCT (macular thickness greater than 250 uM) present for at least three months and not in treatment for at least one month;
•Best corrected visual acuity (BCVA) between 20/100 (2/10 or 0.1 logMAR) and 20/25 (8/10 or 0.7 logMAR) and best corrected visual acuity for near not exceeding II character
•Absence Other ocular confounding diseases
•Participant must be medically able to undergo the testing and study procedures required in the flowsheet of exam procedures.

Exclusion Criteria

•Presence of diabetes-related macular edema, or macular edema associated with other causes such as choroideal neovascularization or after the eye surgery.
•patients with diabetes mellitus.
•Patients who have performed eye surgery in the previous three months.
•Evidence of an active eye infection.
•previous uveitis or evidence of intraocular inflammation.
•History or evidence of glaucoma or elevated intraocular pressure (IOP) greater than or equal to 21 mm Hg at baseline.
•abnormalities of the anterior segment or media opacities that prevent the assessment of the posterior segment of te eye
•The treatment with corticosteroids (systemic, periocular or intravitreal) from at least 3 months.
•The use of any topical medication other than the study drug for the treatment of ocular pathologies.
•Presence or history of any systemic or ocular disease or condition that may significantly limit of visual acuity or visual field, hindering the effectiveness of treatment in the study or its evaluation, or could interfere with the interpretation of The study results, or may be judged by the investigator incompatible with the visit program or with the procedures in the study.

Arms & Interventions

study group

rhNGF 180 µg/ml eye drops solution

Intervention: rhNGF 180 µg/ml eye drops solution

control group

vehicle eye drops solution

Intervention: vehicle eye drops

Outcomes

Primary Outcomes

macular thickness

Time Frame: 28 days of treatment

assessed by ocular coherence tomography (OCT)

Secondary Outcomes

macular thickness(1 month,3 months, 6 months and 12 months of follow-up)
electroretinogram (ERG)(28 days of treatment, 1 month, 6 months and 12 months of follow-up)
visual acuity(28 days of treatment,1 month,3 months, 6 months and 12 months of follow-up)
contrast sensitivity(28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up)
quality of life(28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up)
visual field(28 days of treatment, 1 month, 6 months and 12 months of follow-up)
macular photoreceptors thickness(28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up)
number of cystoid macular edema relapses(through study completion, up to 12 months of follow-up)
number of drop out for inefficacy of the study treatment(through study completion, up to 12 months of follow-up)
adverse events(through study completion, up to 12 months of follow-up)