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

September 2, 2017 updated by: Paolo Rama, Ospedale San Raffaele

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
45

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Milan, Italy, 20132
        • Ospedale San Raffaele
      • Milan, Italy
        • Ospedale Sacco

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Typical and atypical forms of retinitis pigmentosa (RP)
  2. Measurable ERG with a significant decrease in the amplitude .
  3. Reduction of the visual field (assessed with Humphrey field analyzer- HFA 24-2) and values of mean deviation> -3 decibel (dB).
  4. 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;
  5. 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
  6. Absence Other ocular confounding diseases
  7. Participant must be medically able to undergo the testing and study procedures required in the flowsheet of exam procedures.

Exclusion Criteria:

  1. Presence of diabetes-related macular edema, or macular edema associated with other causes such as choroideal neovascularization or after the eye surgery.
  2. patients with diabetes mellitus.
  3. Patients who have performed eye surgery in the previous three months.
  4. Evidence of an active eye infection.
  5. previous uveitis or evidence of intraocular inflammation.
  6. History or evidence of glaucoma or elevated intraocular pressure (IOP) greater than or equal to 21 mm Hg at baseline.
  7. abnormalities of the anterior segment or media opacities that prevent the assessment of the posterior segment of te eye
  8. The treatment with corticosteroids (systemic, periocular or intravitreal) from at least 3 months.
  9. The use of any topical medication other than the study drug for the treatment of ocular pathologies.
  10. 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.
  11. Known hypersensitivity to study drug or drugs procedural.
  12. Participation in another clinical study during the same period of this study or within 90 days of the screening visit / baseline.
  13. History of drug use, illegal drugs or alcohol abuse or addiction.

  14. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: study group
rhNGF 180 µg/ml eye drops solution
Drug: rhNGF 180 µg/ml eye drops solution
eye drops
Other Names:
  • recombinant human Nerve Growth Factor
Placebo Comparator: control group
vehicle eye drops solution
Drug: vehicle eye drops
placebo
Other Names:
  • vehicle eye drop solution

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
macular thickness
Time Frame: 28 days of treatment
assessed by ocular coherence tomography (OCT)
28 days of treatment

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
visual field
Time Frame: 28 days of treatment, 1 month, 6 months and 12 months of follow-up
mean deviation
28 days of treatment, 1 month, 6 months and 12 months of follow-up
macular photoreceptors thickness
Time Frame: 28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
assessed by OCT
28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
macular thickness
Time Frame: 1 month,3 months, 6 months and 12 months of follow-up
assessed by OCT
1 month,3 months, 6 months and 12 months of follow-up
electroretinogram (ERG)
Time Frame: 28 days of treatment, 1 month, 6 months and 12 months of follow-up
amplitudes
28 days of treatment, 1 month, 6 months and 12 months of follow-up
visual acuity
Time Frame: 28 days of treatment,1 month,3 months, 6 months and 12 months of follow-up
28 days of treatment,1 month,3 months, 6 months and 12 months of follow-up
contrast sensitivity
Time Frame: 28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
quality of life
Time Frame: 28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
number of cystoid macular edema relapses
Time Frame: through study completion, up to 12 months of follow-up
through study completion, up to 12 months of follow-up
number of drop out for inefficacy of the study treatment
Time Frame: through study completion, up to 12 months of follow-up
through study completion, up to 12 months of follow-up
adverse events
Time Frame: through study completion, up to 12 months of follow-up
through study completion, up to 12 months of follow-up

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Ospedale San Raffaele

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Dompé Farmaceutici S.p.A

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Paolo Rama, MD, Ospedale San Raffaele. Milan

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 1, 2017

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 1, 2017

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 12, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 18, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 20, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 6, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 2, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • RF-2010-2318561

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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