Retinitis pigmentosa with cystoid macular edema MedDRA version: 17.0 Level: LLT Classification code 10054467 Term: Macular edema System Organ Class: 100000004853 MedDRA version: 17.0 Level: PT Classification code 10038914 Term: Retinitis pigmentosa System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: recombinant human Nerve Growth Factor (rhNGF)
Pharmaceutical Form: Eye drops, solution
INN or Proposed INN: not yet assigned
Other descriptive name: RECOMBINANT HUMAN NERVE GROWTH FACTOR
Sponsors
Ospedale San Raffaele di Milano
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients between 18 and 60 years of age. 2. Patients with typical orms of RP characterized by the following clinical features: a) classic fundus appearance (i.e. intraretinal pigment deposits,thinning and atrophy of the RPE in the mid- and far peripheral retina, with relative RPE preservation in the macula, waxy pallor of the optic disc, attenuation of the retinal vessels) b) reduced and delayed ERG responses c) visual field constriction and/or atypical forms of RP: The atypical form of RP is characterized by early onset (first decade) or syndromic forms of RP (eg paravenous sector pericentral or unilateral RP, Leber congenital amaurosis, Resfum disease, Usher syndrome, Bardet - Biedl, etc. ). 3. presence of macular edema documented by OCT (macular thickness greater than 250 um) lasting for at least 3 months and not in treatment for at least 1 month; 4 Best corrected distance visual acuity (BCDVA) between 20/100 (2/10 or 0.1 logMAR) and 20/25 (8/10 or +0.7 logMAR) , near vision lower than second character in either eye. 5. Absence of other confounding ocular diseases. 6. Only patients who satisfy all Informed Consent requirements may be included in the study. The patient and/or his/her impartial witness must read, sign and date the Informed Consent document before any studyrelated procedures are performed. The Informed Consent form signed by patients and/or impartial witness must have been approved by the IEC for the current study. 7. Patients must have the ability and willingness to comply with study procedures. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 45 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Presence of macular edema associated with diabetes , choroidal neovascularization or after eye surgery. 2 . Patients with diabetes mellitus 3 . History of any ocular surgery (including laser or refractive surgical procedures) in either eye within the 90 days before study enrolment. Ocular surgery will not be allowed during the study treatment period and elective ocular surgery procedure. 4. Evidence of an active ocular infection. 5. History of uveitis or evidence of intraocular inflammation in either eye. 6. History or evidence of glaucoma or an IOP greater than or equal 21 mmHg in either eye at the time of study enrollment 7. Anterior segment abnormalities or media opacities obscuring the view of the posterior pole in either eye. 8. Treatment with corticosteroids (systemic, periocular or intravitreal) ) in either eye within 90 days of study enrollment. 9. Use of any topical medication other than the study medication for the treatment of ocular diseases with the exception of artificial tears during the study period. 10. Presence or history of any ocular or systemic disorder or condition that might significantly limit visual acuity or the visual field, hinder the efficacy of the study treatment or its evaluation, could possibly interfere with the interpretation of study results, or could be judged by the investigator to be incompatible with the study visit schedule or conduct of trail procedures. 11. Known hypersensitivity to one of the components of the study or procedural medications. 12. Participation in another clinical study at the same time as the present study or within 90 days of screening/baseline visit. 13. History of drug, medication or alcohol abuse or addiction. 14. Females of childbearing potential (those who are not surgically sterilized or post-menopausal for at least 1 year) are excluded from participation in the study if they meet any one of the following conditions: a. are currently pregnant or, b. have a positive result on the urine pregnancy test at the Screening/Baseline Visit or, c. intend to become pregnant during the study treatment period or, d. are breast-feeding or, e. not willing to use highly effective birth control measures, such as: Hormonal contraceptives – oral, implanted, transdermal, or injected and/or mechanical barrier methods – spermicide in conjunction with a barrier such as a condom or diaphragm or IUD during the entire course of and 30 days after the study treatment periods.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to assess the efficacy of rhNGF eye drops solution administered over 1 month versus a vehicle control in patients with macular edema associated with retinitis pigmentosa;Secondary Objective: The secondary objective of this study is to assess safety and tolerability of hNGF eye drops solution administered over 1 month versus a vehicle control in patients with macular edema associated with retinitis pigmentosa;Primary end point(s): The primary endpoint will be to evaluate the effects of NGF eye drops on macular thickness in patients with RP associated with macular edema. Macular thickness greater than 250µm will be considered significant. Treatment will be considered effective if it will induce an improvement of at least one of the following parameters: 1) macular thickness less than 250µm; 2) visual acuity of at least 20 EDTRS letters; 3) near visual acuity of at least 1 line;Timepoint(s) of evaluation of this end point: after one month of experimental treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoints of this study are to evaluate differences between the two treatment groups of the following parameters: - Best-corrected visual acuity for distance - best-corrected visual acuity for near -contrast sensitivity - visual-field -OCT - ERG -Quality of Life (NEI-VFQ) - Number of drop out for inefficacy -number of recurrence of macular edema ;Timepoint(s) of evaluation of this end point: secondary endpoints will be evaluated at the end of experimental treatment (1 month) and at 3, 6 and 12 months of follow-up | — |
Countries
Italy
Contacts
Public ContactCornea and Ocular surface unit
Ospedale San Raffaele di Milano
Outcome results
None listed