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

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ClinicalTrials.gov Identifier: NCT02609165
Recruitment Status : Completed
First Posted : November 20, 2015
Last Update Posted : September 6, 2017
Sponsor:
Collaborator:
Dompé Farmaceutici S.p.A
Information provided by (Responsible Party):
Paolo Rama, Ospedale San Raffaele

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.

Condition or disease Intervention/treatment Phase
Retinitis Pigmentosa Cystoid Macular Edema Drug: rhNGF 180 µg/ml eye drops solution Drug: vehicle eye drops Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 45 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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 Start Date : May 2015
Actual Primary Completion Date : February 2017
Actual Study Completion Date : February 2017


Arm Intervention/treatment
Active Comparator: study group
rhNGF 180 µg/ml eye drops solution
Drug: rhNGF 180 µg/ml eye drops solution
eye drops
Other Name: recombinant human Nerve Growth Factor

Placebo Comparator: control group
vehicle eye drops solution
Drug: vehicle eye drops
placebo
Other Name: vehicle eye drop solution




Primary Outcome Measures :
  1. macular thickness [ Time Frame: 28 days of treatment ]
    assessed by ocular coherence tomography (OCT)


Secondary Outcome Measures :
  1. visual field [ Time Frame: 28 days of treatment, 1 month, 6 months and 12 months of follow-up ]
    mean deviation

  2. macular photoreceptors thickness [ Time Frame: 28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up ]
    assessed by OCT

  3. macular thickness [ Time Frame: 1 month,3 months, 6 months and 12 months of follow-up ]
    assessed by OCT

  4. electroretinogram (ERG) [ Time Frame: 28 days of treatment, 1 month, 6 months and 12 months of follow-up ]
    amplitudes

  5. visual acuity [ Time Frame: 28 days of treatment,1 month,3 months, 6 months and 12 months of follow-up ]
  6. contrast sensitivity [ Time Frame: 28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up ]
  7. quality of life [ Time Frame: 28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up ]
  8. number of cystoid macular edema relapses [ Time Frame: through study completion, up to 12 months of follow-up ]
  9. number of drop out for inefficacy of the study treatment [ Time Frame: through study completion, up to 12 months of follow-up ]
  10. adverse events [ Time Frame: through study completion, up to 12 months of follow-up ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02609165


Locations
Italy
Ospedale San Raffaele
Milan, Italy, 20132
Ospedale Sacco
Milan, Italy
Sponsors and Collaborators
Ospedale San Raffaele
Dompé Farmaceutici S.p.A
Investigators
Principal Investigator: Paolo Rama, MD Ospedale San Raffaele. Milan

Responsible Party: Paolo Rama, MD, Ospedale San Raffaele
ClinicalTrials.gov Identifier: NCT02609165     History of Changes
Other Study ID Numbers: RF-2010-2318561
First Posted: November 20, 2015    Key Record Dates
Last Update Posted: September 6, 2017
Last Verified: September 2017

Additional relevant MeSH terms:
Edema
Macular Edema
Retinitis
Retinitis Pigmentosa
Cone-Rod Dystrophies
Signs and Symptoms
Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Genetic Diseases, Inborn
Pharmaceutical Solutions
Mitogens
Ophthalmic Solutions
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action