Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema (NEMO)
Primary Purpose
Retinitis Pigmentosa, Cystoid Macular Edema
Status
Completed
Phase
Phase 2
Locations
Italy
Study Type
Interventional
Intervention
rhNGF 180 µg/ml eye drops solution
vehicle eye drops
Sponsored by
About this trial
This is an interventional treatment trial for Retinitis Pigmentosa
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.
- 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,
- have a positive urine pregnancy test at screening / baseline or,
- They plan to become pregnant during the treatment period of the study or,
- They are breast-feeding or,
- They are not available to use highly effective measures of birth control, such as: the oral hormonal contraceptives and / or mechanical barrier methods - spermicide in conjunction with a barrier such as a condom or diaphragm or during the 'entire course of the study and 30 days after treatment periods provided.
Sites / Locations
- Ospedale San Raffaele
- Ospedale Sacco
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Placebo Comparator
Arm Label
study group
control group
Arm Description
rhNGF 180 µg/ml eye drops solution
vehicle eye drops solution
Outcomes
Primary Outcome Measures
macular thickness
assessed by ocular coherence tomography (OCT)
Secondary Outcome Measures
visual field
mean deviation
macular photoreceptors thickness
assessed by OCT
macular thickness
assessed by OCT
electroretinogram (ERG)
amplitudes
visual acuity
contrast sensitivity
quality of life
number of cystoid macular edema relapses
number of drop out for inefficacy of the study treatment
adverse events
Full Information
NCT ID
NCT02609165
First Posted
October 12, 2015
Last Updated
September 2, 2017
Sponsor
Ospedale San Raffaele
Collaborators
Dompé Farmaceutici S.p.A
1. Study Identification
Unique Protocol Identification Number
NCT02609165
Brief Title
Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema
Acronym
NEMO
Official 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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2017
Overall Recruitment Status
Completed
Study Start Date
May 2015 (undefined)
Primary Completion Date
February 2017 (Actual)
Study Completion Date
February 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Ospedale San Raffaele
Collaborators
Dompé Farmaceutici S.p.A
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa, Cystoid Macular Edema
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
45 (Actual)
8. Arms, Groups, and Interventions
Arm Title
study group
Arm Type
Active Comparator
Arm Description
rhNGF 180 µg/ml eye drops solution
Arm Title
control group
Arm Type
Placebo Comparator
Arm Description
vehicle eye drops solution
Intervention Type
Drug
Intervention Name(s)
rhNGF 180 µg/ml eye drops solution
Other Intervention Name(s)
recombinant human Nerve Growth Factor
Intervention Description
eye drops
Intervention Type
Drug
Intervention Name(s)
vehicle eye drops
Other Intervention Name(s)
vehicle eye drop solution
Intervention Description
placebo
Primary Outcome Measure Information:
Title
macular thickness
Description
assessed by ocular coherence tomography (OCT)
Time Frame
28 days of treatment
Secondary Outcome Measure Information:
Title
visual field
Description
mean deviation
Time Frame
28 days of treatment, 1 month, 6 months and 12 months of follow-up
Title
macular photoreceptors thickness
Description
assessed by OCT
Time Frame
28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
Title
macular thickness
Description
assessed by OCT
Time Frame
1 month,3 months, 6 months and 12 months of follow-up
Title
electroretinogram (ERG)
Description
amplitudes
Time Frame
28 days of treatment, 1 month, 6 months and 12 months of follow-up
Title
visual acuity
Time Frame
28 days of treatment,1 month,3 months, 6 months and 12 months of follow-up
Title
contrast sensitivity
Time Frame
28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
Title
quality of life
Time Frame
28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
Title
number of cystoid macular edema relapses
Time Frame
through study completion, up to 12 months of follow-up
Title
number of drop out for inefficacy of the study treatment
Time Frame
through study completion, up to 12 months of follow-up
Title
adverse events
Time Frame
through study completion, up to 12 months of follow-up
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
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,
have a positive urine pregnancy test at screening / baseline or,
They plan to become pregnant during the treatment period of the study or,
They are breast-feeding or,
They are not available to use highly effective measures of birth control, such as: the oral hormonal contraceptives and / or mechanical barrier methods - spermicide in conjunction with a barrier such as a condom or diaphragm or during the 'entire course of the study and 30 days after treatment periods provided.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Paolo Rama, MD
Organizational Affiliation
Ospedale San Raffaele. Milan
Official's Role
Principal Investigator
Facility Information:
Facility Name
Ospedale San Raffaele
City
Milan
ZIP/Postal Code
20132
Country
Italy
Facility Name
Ospedale Sacco
City
Milan
Country
Italy
12. IPD Sharing Statement
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Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema
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