Nerve Growth Factor eye drops as a novel treatment for patients with Retinitis Pigmentosa.

• Register: EUCTR
• Last refreshed on: 23 March 2015
• Main ID: EUCTR2014-000385-22-IT
• Date of registration: 12/05/2014
• Prospective Registration: Yes
• Primary sponsor: Ospedale San Raffaele di Milano
• Public title: Nerve Growth Factor eye drops as a novel treatment for patients with Retinitis Pigmentosa.
• Scientific title: Nerve Growth Factor eye drops as a novel treatment for vision loss in patients with Retinitis Pigmentosa: from preclinical to clinical Phase II trial. - NEMO
• Date of first enrolment: 07/10/2014
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-000385-22
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Italy

Contacts

• Name: Cornea and Ocular surface unit
• Affiliation: Ospedale San Raffaele di Milano

• Name: Cornea and Ocular surface unit
• Affiliation: Ospedale San Raffaele di Milano

Key inclusion & exclusion 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:
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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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

Therapeutic area: Diseases [C] - Eye Diseases [C11]

Intervention(s)

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 (RHNGF)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 0.18-
Pharmaceutical form of the placebo: Eye drops, solution
Route of administration of the placebo: Ocular use

Primary Outcome(s)

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

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

Timepoint(s) of evaluation of this end point: after one month of experimental treatment

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 Outcome(s)

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

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

Secondary ID(s)

RF-2010-2318561

Source(s) of Monetary Support

Italian Ministry of Health