Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema

NCT02609165 | Completed Phase 2

• 1 other identifier: RF-2010-2318561
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 2

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.

Conditions

Retinitis Pigmentosa; Cystoid Macular Edema

Outcome Measures

Primary Outcomes (1)

• macular thickness

  assessed by ocular coherence tomography (OCT)

  28 days of treatment

Secondary Outcomes (10)

• 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

• 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

Study Arms (2)

study group

ACTIVE COMPARATOR

rhNGF 180 µg/ml eye drops solution

Drug: rhNGF 180 µg/ml eye drops solution

control group

PLACEBO COMPARATOR

vehicle eye drops solution

Drug: vehicle eye drops

Interventions

rhNGF 180 µg/ml eye drops solution DRUG

eye drops

Also known as: recombinant human Nerve Growth Factor

study group

vehicle eye drops DRUG

placebo

Also known as: vehicle eye drop solution

control group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• Known hypersensitivity to study drug or drugs procedural.
• Participation in another clinical study during the same period of this study or within 90 days of the screening visit / baseline.
• History of drug use, illegal drugs or alcohol abuse or addiction.
• Women of childbearing potential are excluded from participation in the study if they meet one of the following conditions:
• currently pregnant or,

Sponsors & Collaborators

• Ospedale San Raffaele: lead
• Dompé Farmaceutici S.p.A: collaborator

Study Sites (2)

Ospedale San Raffaele

Milan, 20132, Italy

Location

Ospedale Sacco

Milan, Italy

Location

MeSH Terms

Conditions

Retinitis Pigmentosa; Macular Edema

Interventions

cenegermin; Ophthalmic Solutions

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Macular Degeneration

Intervention Hierarchy (Ancestors)

Pharmaceutical Solutions; Solutions; Pharmaceutical Preparations; Therapeutic Uses; Pharmacologic Actions; Chemical Actions and Uses; Specialty Uses of Chemicals

Study Officials

• Paolo Rama, MD

  Ospedale San Raffaele. Milan

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: October 12, 2015
• First Posted: November 20, 2015
• Study Start: May 1, 2015
• Primary Completion: February 1, 2017
• Study Completion: February 1, 2017
• Last Updated: September 6, 2017

Record last verified: 2017-09

Locations

IT (2)