Retinitis PigmentosaPhase 2NEMONCT02609165
Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema
A Phase 2 study testing rhNGF 180 µg/ml eye drops solution in people with Retinitis Pigmentosa and Cystoid Macular Edema, run by Ospedale San Raffaele at 2 sites in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa, Cystoid Macular Edema
- What is being tested?
- rhNGF 180 µg/ml eye drops solution
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 45 participants
- Where
- 2 sitesItaly
- Study dates
- May 2015to Feb 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
2 sites in 1 country.
About this study
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
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
- Being tested
- rhNGF 180 µg/ml eye drops solution, vehicle eye drops
- Medical subject headings
- Retinitis Pigmentosa, Macular Edema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhNGF 180 µg/ml eye drops solutionDrug
Also called recombinant human Nerve Growth Factor
eye drops
vehicle eye dropsDrug
Also called vehicle eye drop solution
placebo
| Study group | Role | What they receive |
|---|---|---|
| study group | Active comparator | rhNGF 180 µg/ml eye drops solution Receives: Drug: rhNGF 180 µg/ml eye drops solution |
| control group | Placebo comparator | vehicle eye drops solution Receives: Drug: vehicle eye drops |
What is being measured
The primary outcome is the main question the study is designed to answer.
macular thickness
assessed by ocular coherence tomography (OCT)
Measured: 28 days of treatment
Secondary outcomes (10)
visual field
mean deviation
Measured: 28 days of treatment, 1 month, 6 months and 12 months of follow-up
macular photoreceptors thickness
assessed by OCT
Measured: 28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
macular thickness
assessed by OCT
Measured: 1 month,3 months, 6 months and 12 months of follow-up
electroretinogram (ERG)
amplitudes
Measured: 28 days of treatment, 1 month, 6 months and 12 months of follow-up
visual acuity
Measured: 28 days of treatment,1 month,3 months, 6 months and 12 months of follow-up
contrast sensitivity
Measured: 28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
quality of life
Measured: 28 days of treatment, 1 month,3 months, 6 months and 12 months of follow-up
number of cystoid macular edema relapses
Measured: through study completion, up to 12 months of follow-up
number of drop out for inefficacy of the study treatment
Measured: through study completion, up to 12 months of follow-up
adverse events
Measured: through study completion, up to 12 months of follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2015 (estimated)
- Study startMay 2015
- Primary completionFeb 2017
- Study completionFeb 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ospedale San Raffaele
- Sponsor type
- University, hospital or other
- Collaborators
- Dompé Farmaceutici S.p.A
- Responsible party
- Principal investigator, Paolo Rama, Ospedale San Raffaele
- Investigators
- Paolo Rama, MD, Principal investigator, Ospedale San Raffaele. Milan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02609165
- Sponsor's protocol ID
- RF-2010-2318561
- Acronym
- NEMO
- Status last verified
- Sep 2017
- First submitted
- 12 Oct 2015
- Last updated
- 6 Sep 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02609165
- Acronym / sponsor ID
- NEMO / RF-2010-2318561
- Conditions
- Retinitis Pigmentosa, Cystoid Macular Edema
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Ospedale San RaffaeleUniversity, hospital or other
- Collaborators
- Dompé Farmaceutici S.p.A
- Study start
- May 2015
- Primary completion
- Feb 2017 (actual)
- Study completion
- Feb 2017 (actual)
- First posted
- 20 Nov 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| study group | Active comparator | Drug: rhNGF 180 µg/ml eye drops solutionrhNGF 180 µg/ml eye drops solution |
| control group | Placebo comparator | Drug: vehicle eye dropsvehicle eye drops solution |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (19 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rhNGF 180 µg/ml eye drops solutionAlso: recombinant human Nerve Growth Factoreye drops | Drug | study group |
| vehicle eye dropsAlso: vehicle eye drop solutionplacebo | Drug | control group |
Primary outcome
macular thickness
Time frame: 28 days of treatment
assessed by ocular coherence tomography (OCT)
10 secondary outcomes
visual field
Time frame: 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
macular thickness
Time frame: 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
visual acuity
Time frame: 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
quality of life
Time frame: 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
number of drop out for inefficacy of the study treatment
Time frame: through study completion, up to 12 months of follow-up
adverse events
Time frame: through study completion, up to 12 months of follow-up
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Sep 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Paolo Rama, MD principal investigator
Ospedale San Raffaele. Milan
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rhNGF 180 µg/ml eye drops solution in people with Retinitis Pigmentosa and Cystoid Macular Edema, run by Ospedale San Raffaele at 2 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
macular thickness (time frame: 28 days of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Sep 2017.