Retinitis PigmentosaPhase 1/2LumosNCT02110225

A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)

A Phase 1/2 study testing rhNGF 60 µg/ml eye drops solution and rhNGF 180 µg/ml eye drops solution in people with Retinitis Pigmentosa, run by Dompé Farmaceutici S.p.A at 5 sites in Italy.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Retinitis Pigmentosa
What is being tested?: rhNGF 60 µg/ml eye drops solution and rhNGF 180 µg/ml eye drops solution
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 50 participants
Where: 5 sitesItaly
Study dates: Jan 2014to Nov 2015
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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

Patients 18 years of age or older.
Patients with typical forms of RP characterized by the following clinical features: classic fundus appearance (i.e. intraretinal pigment deposits, thinning and atrophy of the retinal pigment epithelium (RPE) in the mid- and far peripheral retina, with relative RPE preservation in the macula, waxy pallor of the optic disc, attenuation of the retinal vessels), reduced and delayed ERG responses, visual field constriction
Best corrected distance visual acuity (BCDVA) score of ≥ 48 ETDRS letters (equivalent to 20/100 Snellen, +0.7 LogMar, or 0.2 decimal fraction) in either eye at the time of study enrollment.
Documented evidence of disease progression within the 12 months prior to enrollment in the study as demonstrated by ERG (≥20% decrease in b wave amplitude in scotopic conditions or ≥25% in photopic conditions) and/or visual field testing (≥10% of Goldman Visual Field expressed as area square or ≥3 dB decrease of Humphrey Visual Field Mean Deviation).
Only patients who satisfy all Informed Consent requirements may be included in the study. The patient and/or his/her impartial witness must read, sign and date the Informed Consent document before any study-related procedures are performed. The Informed Consent form signed by patients and/or impartial witness must have been approved by the Ethics Committee (IEC) for the current study.
Patients must have the ability and willingness to comply with study procedures.

Who cannot join

Patients with atypical, early onset (first decade) or syndromic forms of RP (e.g. paravenous, pericentral sector or unilateral RP, Leber's congenital amaurosis, Refsum disease, Usher syndrome, Bardet-Biedl syndrome, etc).
Patients with non-recordable 30 Hz cone ERG in either eye.
Patients with Goldman visual field less than 20º using the V4e target or residual central visual field less than -35 dB as evaluated by the 24-2 program of the Humphrey visual field in either eye.
Evidence of an active ocular infection in either eye.
History of uveitis or evidence of intraocular inflammation in either eye.
History or evidence of glaucoma or an intraocular pressure (IOP) greater than or equal 21 mmHg in either eye at the time of study enrollment.

Show all 12Show fewer

Patients with foveal thickness ≥ 250 micrometers (as evaluated with OCT).
History of cystoid macular oedema or presence of cystoid macular oedema on OCT at the time of study enrolment.
Anterior segment abnormalities or media opacities obscuring the view of the posterior pole in either eye.
History of any ocular surgery (including laser or refractive surgical procedures) in either eye within the 120 days before study enrolment. Ocular surgery will not be allowed during the study treatment period and elective ocular surgery procedures should not be planned during the duration of the follow-up period.
Treatment with corticosteroids (systemic, periocular or intravitreal) or any other non-approved, experimental, investigational or neuroprotectant therapy (systemic, topical, intravitreal) in either eye within 90 days of study enrollment.
Use of any medication other than the study medication for the treatment of ocular diseases with the exception of artificial tears during the study period.

Trial locations and contacts

5 sites in 1 country.

City, postcode or hospital

5 sites in 1 country

Italy5 sites

Azienda Ospedaliero Universitaria Careggi
Florence, 50124
Azienda Ospedaliera San Paolo - U.O. Oculistica
Milan, 20142
A.O. Seconda Università Degli Studi di Napoli - Nuovo Policlinico - UOC Oftalmologia
Naples, 80129
Università Cattolica del Sacro Cuore - Policlinico Gemelli - Istituto di Oftalmologia
Rome, 00168
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia
Rome, 00198

Other trials in Italy

About this study

Official title: A 24 Week Phase Ib/II, Multicenter, Randomized, Controlled, Parallel Group, Dose Ranging Study With a 24 Week Follow-up to Evaluate Safety and Potential Efficacy of 2 Doses (60, 180 µg/ml) of rhNGF Solution vs Vehicle in Patients With RP.

The primary objective of the study is to assess the safety and tolerability of two dose regimens of recombinant human nerve growth factor (rhNGF) eye drops solution administered over 6 months versus a vehicle control in patients with typical retinitis pigmentosa. The secondary objective of this study is to attempt to show a dose response by assessing the potential efficacy of the rhNGF dose regimens for improving or slowing the deterioration of visual function outcomes at 3 and 6 months. During a 6 month follow-up period patients will be monitored to determine if there is evidence of a persistent biological effect after discontinuation of the study treatment.

Read the detailed description

This is a 24-week phase Ib/II, multicenter, randomized, double-masked, vehicle controlled, parallel-group, dose-ranging study with a 24-week follow-up period to evaluate the safety and potential efficacy of two doses (60 μg/ml and 180 μg/ml) of recombinant human nerve growth factor (rhNGF) eye drops solution versus vehicle in patients with typical retinitis pigmentosa (RP).

Conditions: Retinitis Pigmentosa
Being tested: rhNGF 60 µg/ml eye drops solution, rhNGF 180 µg/ml eye drops solution, Placebo
Keywords: Cone-Rod Degenerations, Cone-Rod Dystrophy, Cone-Rod Dystrophy 2, Cone-Rod Retinal Dystrophy, Pigmentary Retinopathy, Retinal Cone-Rod Dystrophy, Rod Cone Dystrophies, Rod-Cone Dystrophy, Tapetoretinal Degeneration
Medical subject headings: Retinitis Pigmentosa, Cone-Rod Dystrophies

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

rhNGF 60 µg/ml eye drops solutionDrug
Also called recombinant human nerve growth factor 60 µg/ml solution
rhNGF 60 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes
rhNGF 180 µg/ml eye drops solutionDrug
Also called recombinant human nerve growth factor 180 µg/ml solution
rhNGF 180 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes
PlaceboDrug
Also called Vehicle
Placebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes

Study groups in NCT02110225
Study group	Role	What they receive
rhNGF 60µg/ml	Experimental	rhNGF 60 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes Receives: Drug: rhNGF 60 µg/ml eye drops solution
rhNGF 180 µg/ml	Experimental	rhNGF 180 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes. Receives: Drug: rhNGF 180 µg/ml eye drops solution
Vehicle	Placebo comparator	Placebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Serious and Non-Serious Adverse Events
Twenty-seven patients of the Safety population experienced at least one treatment-emergent adverse event, 11 patients in the rhNGF 60 μg/ml arm, 13 patients in the rhNGF 180 μg/ml arm and 3 patients in the vehicle arm.
Measured: up to 48 weeks
Change in Ocular Tolerability - VAS
A global ocular discomfort score was determined using a 100 mm Visual Analogue Scale (VAS) on which 0 means no symptoms and 100 means the worst possible discomfort.
Specific ocular symptoms to be assessed with the VAS included: foreign body sensation, burning/stinging, itching, pain, sticky feeling, blurred vision, photophobia.
For ocular tolerability analysis, mixed models for repeated measures were applied using various ocular tolerability parameters as response variable, treatment, visit and treatment by visit interaction as fixed effects, and baseline value as covariate.
Measured: Weeks 1, 2, 6, 12, 24
Change in Best Corrected Distance Visual Acuity (BCDVA) (ETDRS Chart)
Best-Corrected Distance Visual Acuity (BCDVA) was assessed for each eye at each visit using an ETDRS visual acuity chart at 4 meters.
Measured: Weeks 1, 2, 6, 12, 24, 36, 48
Change in Intraocular Pressure (IOP)
Intraocular Pressure was measured using either Goldmann applanation tonometry or a handheld applanation tonometer (e.g. Tonopen) after the instillation of a topical anesthetic.IOP was assessed for each eye at day 0 and at week 2, 12 and 24
Measured: Weeks 2,12 and 24
Number of Participants With Normal or Abnormal Findings by Slit Lamp Examination
Slit Lamp Examination (SLE) (Biomicroscopy) was performed before the instillation of any dilating or anesthetic eye drops or fluorescein agents. SLE was executed to assess eyelids, lashes, conjunctiva, cornea, lens, iris and anterior chamber.
Measured: Day 0; Weeks 1, 2, 6, 12, 24, 36 and 48
External Ocular Examination
External ocular examinations were done to assess, for each eye and at each visit, the motility of extraocular muscles, appearance and function of the eyelids.
Measured: Day 0, Weeks 1, 2, 6, 12, 24
Change in Ocular Tolerability - Dilated Fundus Ophthalmoscopy
Dilated fundus ophthalmoscopy was assessed for each eye evaluating the retina, macula, choroid and optic nerve head.
Measured: Day 0, Weeks 12, 24 and 48
Presence of Anti-NGF Antibodies
Anti-NGF antibodies tests were performed at screening and at the end of treatment
Measured: At Day 0 and at week 24

Secondary outcomes (8)

Change From Baseline in Contrast Sensitivity
Contrast sensitivity was assessed using a Mars chart and is expressed as a log contrast sensitivity (log CS) score given by the log CS value at the lowest contrast numeral just prior to two incorrectly identified numerals, minus a scoring correction. The higher is the number of characters properly read by the patient, the higher is the contrast sensitivity.
Measured: Weeks 12, 24, 36 and 48
Change From Baseline in Humphrey Visual Field 24-2
The Humphrey Visual Field (HVF) analyzer is a tool for measuring the human visual field by providing information regarding the location of any disease processes or lesion(s) throughout the visual pathway.
In particular, Humphrey Visual Field 24-2 was used to assess static perimetry by measuring 24 degrees temporally and 30 degrees nasally and tests 54 points.
The Analyser projects a series of white light stimuli of varying intensities (brightness), throughout a uniformly illuminated bowl. The higher is the number of stimuli perceived by the patient, the better is the retina's ability to detect a stimulus at specific points within the visual field.
Measured: Weeks 12, 24, 36 and 48
Change in Goldmann Visual Field
The Goldmann field exam was performed to assess kinetic perimetry on all enrolled patients.
Measured: Weeks 12, 24, 36 and 48
Fundus Imaging
A recordable fundus image (photo or other electronic format) showing the central 30 degrees was captured through a dilated pupil to document the appearance of the posterior pole.
Measured: Day 0, Weeks 24 and 48
Ocular Coherence Tomography (OCT)
Ocular coherence tomography was performed to evaluate the cross-sectional anatomy of the macula and to document areas of retinal atrophy.
Measured: Day 0, Weeks 12, 24, 36 and 48
Microperimetry
MP1 microperimetry was analyzed to provide a more accurate measurement of retinal sensitivity in the central visual field, even in patients with unstable or extrafoveal fixation.
Measured: Day 0, Weeks 12, 24, 36 and 48
Binocular Estermann Visual Field
Binocular visual field with Estermann grid testing a stimulus array of 120 points spread over an area extending approximately ±75° horizontally, 35° superiorly and 55° inferiorly while the patient looked steadily at the fixation target.
Measured: Day 0, Weeks 12, 24, 36 and 48
Electrorethinogram (ERG)
A full field and 30 Hz flicker ERG was performed according to international standards. Patients were treated with anesthetic and dilating drops prior to the ERG procedure.
Measured: Day 0, Weeks 12, 24, 36 and 48

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jan 2014Nov 2015

First posted10 Apr 2014 (estimated)
Study startJan 2014
Primary completionNov 2015
Study completionNov 2015

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
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 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 50 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02110225
Sponsor's protocol ID: NGF0113
Other IDs: 2013-003029-26
Acronym: Lumos
Status last verified: Dec 2023
First submitted: 8 Apr 2014
Last updated: 18 Apr 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study to Evaluate the Efficacy of a Single Intravitreal Injection of RTx-015 in Participants With Retinitis Pigmentosa (PERCEPT)
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Active, not recruitingPhase 1/2
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All Retinitis Pigmentosa trials

Study at a glance

From the registry record, last updated by the sponsor on 18 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A 24 Week Phase Ib/II, Multicenter, Randomized, Controlled, Parallel Group, Dose Ranging Study With a 24 Week Follow-up to Evaluate Safety and Potential Efficacy of 2 Doses (60, 180 µg/ml) of rhNGF Solution vs Vehicle in Patients With RP.
ClinicalTrials.gov ID: NCT02110225
Acronym / sponsor ID: Lumos / NGF0113
Conditions: Retinitis Pigmentosa
Keywords: Cone-Rod Degenerations, Cone-Rod Dystrophy, Cone-Rod Dystrophy 2, Cone-Rod Retinal Dystrophy, Pigmentary Retinopathy, Retinal Cone-Rod Dystrophy, Rod Cone Dystrophies, Rod-Cone Dystrophy
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 50 participants (actual)
Lead sponsor: Dompé Farmaceutici S.p.AIndustry (pharma, biotech, devices)
Study start: Jan 2014 (actual)
Primary completion: Nov 2015 (actual)
Study completion: Nov 2015 (actual)
First posted: 10 Apr 2014 (estimated)
Results: Posted 11 Jul 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
rhNGF 60µg/ml	Experimental	Drug: rhNGF 60 µg/ml eye drops solutionrhNGF 60 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes
rhNGF 180 µg/ml	Experimental	Drug: rhNGF 180 µg/ml eye drops solutionrhNGF 180 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes.
Vehicle	Placebo comparator	Drug: PlaceboPlacebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (6 listed)

Patients 18 years of age or older.
Patients with typical forms of RP characterized by the following clinical features: classic fundus appearance (i.e. intraretinal pigment deposits, thinning and atrophy of the retinal pigment epithelium (RPE) in the mid- and far peripheral retina, with relative RPE preservation in the macula, waxy pallor of the optic disc, attenuation of the retinal vessels), reduced and delayed ERG responses, visual field constriction
Best corrected distance visual acuity (BCDVA) score of ≥ 48 ETDRS letters (equivalent to 20/100 Snellen, +0.7 LogMar, or 0.2 decimal fraction) in either eye at the time of study enrollment.
Documented evidence of disease progression within the 12 months prior to enrollment in the study as demonstrated by ERG (≥20% decrease in b wave amplitude in scotopic conditions or ≥25% in photopic conditions) and/or visual field testing (≥10% of Goldman Visual Field expressed as area square or ≥3 dB decrease of Humphrey Visual Field Mean Deviation).
Only patients who satisfy all Informed Consent requirements may be included in the study. The patient and/or his/her impartial witness must read, sign and date the Informed Consent document before any study-related procedures are performed. The Informed Consent form signed by patients and/or impartial witness must have been approved by the Ethics Committee (IEC) for the current study.

Key exclusion criteria (12 listed)

Patients with atypical, early onset (first decade) or syndromic forms of RP (e.g. paravenous, pericentral sector or unilateral RP, Leber's congenital amaurosis, Refsum disease, Usher syndrome, Bardet-Biedl syndrome, etc).
Patients with non-recordable 30 Hz cone ERG in either eye.
Patients with Goldman visual field less than 20º using the V4e target or residual central visual field less than -35 dB as evaluated by the 24-2 program of the Humphrey visual field in either eye.
Evidence of an active ocular infection in either eye.
History of uveitis or evidence of intraocular inflammation in either eye.

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 60 µg/ml eye drops solutionAlso: recombinant human nerve growth factor 60 µg/ml solutionrhNGF 60 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes	Drug	rhNGF 60µg/ml
rhNGF 180 µg/ml eye drops solutionAlso: recombinant human nerve growth factor 180 µg/ml solutionrhNGF 180 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes	Drug	rhNGF 180 µg/ml
PlaceboAlso: VehiclePlacebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes	Drug	Vehicle

Primary outcome

Number of Participants With Serious and Non-Serious Adverse Events
Time frame: up to 48 weeks
Twenty-seven patients of the Safety population experienced at least one treatment-emergent adverse event, 11 patients in the rhNGF 60 μg/ml arm, 13 patients in the rhNGF 180 μg/ml arm and 3 patients in the vehicle arm.
Change in Ocular Tolerability - VAS
Time frame: Weeks 1, 2, 6, 12, 24
A global ocular discomfort score was determined using a 100 mm Visual Analogue Scale (VAS) on which 0 means no symptoms and 100 means the worst possible discomfort. Specific ocular symptoms to be assessed with the VAS included: foreign body sensation, burning/stinging, itching, pain, sticky feeling, blurred vision, photophobia. For ocular tolerability analysis, mixed models for repeated measures were applied using various ocular tolerability parameters as response variable, treatment, visit and treatment by visit interaction as fixed effects, and baseline value as covariate.
Change in Best Corrected Distance Visual Acuity (BCDVA) (ETDRS Chart)
Time frame: Weeks 1, 2, 6, 12, 24, 36, 48
Best-Corrected Distance Visual Acuity (BCDVA) was assessed for each eye at each visit using an ETDRS visual acuity chart at 4 meters.
Change in Intraocular Pressure (IOP)
Time frame: Weeks 2,12 and 24
Intraocular Pressure was measured using either Goldmann applanation tonometry or a handheld applanation tonometer (e.g. Tonopen) after the instillation of a topical anesthetic.IOP was assessed for each eye at day 0 and at week 2, 12 and 24
Number of Participants With Normal or Abnormal Findings by Slit Lamp Examination
Time frame: Day 0; Weeks 1, 2, 6, 12, 24, 36 and 48
Slit Lamp Examination (SLE) (Biomicroscopy) was performed before the instillation of any dilating or anesthetic eye drops or fluorescein agents. SLE was executed to assess eyelids, lashes, conjunctiva, cornea, lens, iris and anterior chamber.
External Ocular Examination
Time frame: Day 0, Weeks 1, 2, 6, 12, 24
External ocular examinations were done to assess, for each eye and at each visit, the motility of extraocular muscles, appearance and function of the eyelids.
Change in Ocular Tolerability - Dilated Fundus Ophthalmoscopy
Time frame: Day 0, Weeks 12, 24 and 48
Dilated fundus ophthalmoscopy was assessed for each eye evaluating the retina, macula, choroid and optic nerve head.
Presence of Anti-NGF Antibodies
Time frame: At Day 0 and at week 24
Anti-NGF antibodies tests were performed at screening and at the end of treatment

8 secondary outcomes

Change From Baseline in Contrast Sensitivity
Time frame: Weeks 12, 24, 36 and 48
Change From Baseline in Humphrey Visual Field 24-2
Time frame: Weeks 12, 24, 36 and 48
Change in Goldmann Visual Field
Time frame: Weeks 12, 24, 36 and 48
Fundus Imaging
Time frame: Day 0, Weeks 24 and 48
Ocular Coherence Tomography (OCT)
Time frame: Day 0, Weeks 12, 24, 36 and 48
Microperimetry
Time frame: Day 0, Weeks 12, 24, 36 and 48
Binocular Estermann Visual Field
Time frame: Day 0, Weeks 12, 24, 36 and 48
Electrorethinogram (ERG)
Time frame: Day 0, Weeks 12, 24, 36 and 48

Results and safety

Results posted to the registry on 11 Jul 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
rhNGF 60µg/ml	20	20
rhNGF 180 µg/ml	20	19
Vehicle	10	10

Primary outcome: Number of Participants With Serious and Non-Serious Adverse Events

Time frame: up to 48 weeks. Count of participants. Unit: Participants

Measure	rhNGF 60µg/ml n = 20	rhNGF 180 µg/ml n = 20	Vehicle n = 10
Value	11	13	3

Population analysed: Safety population: all patients who received at least one dose of study medication. The safety population was used to analyze all safety endpoints (primary objective).

Primary outcome: Change in Ocular Tolerability - VAS

Time frame: Weeks 1, 2, 6, 12, 24. Mean (Standard Deviation). Unit: units on a scale

Measure	rhNGF 60µg/ml n = 20	rhNGF 180 µg/ml n = 20	Vehicle n = 10
Foreign body sensation - Left eye, Wk 1 vs Day 0	5.45 (15.23)	4.80 (21.31)	3.50 (11.07)
Foreign body sensation - Right eye, Wk 1 vs Day 0	5.75 (15.41)	10.95 (24.44)	4.30 (14.31)
Foreign body sensation - Left eye, Wk 2 vs Day 0	3.50 (9.05)	-0.90 (13.78)	1.10 (3.14)
Foreign body sensation - Right eye, Wk 2 vs Day 0	3.55 (9.03)	1.50 (4.89)	-0.20 (0.63)
Foreign body sensation - Left eye, Wk 6 vs Day 0	4.05 (11.97)	-0.50 (9.32)	1.70 (4.69)
Foreign body sensation - Right eye, Wk 6 vs Day 0	4.05 (13.62)	2.80 (5.91)	-0.20 (0.63)
Foreign body sensation - Left eye, Wk 12 vs Day 0	2.65 (8.22)	3.05 (15.14)	0.60 (1.58)
Foreign body sensation - Right eye, Wk 12 vs Day 0	2.45 (7.79)	7.90 (17.13)	0.50 (1.58)

Population analysed: Safety population: patients who received at least one dose of study medication. The safety population was used to analyze all safety endpoints (primary objective).

Primary outcome: Change in Best Corrected Distance Visual Acuity (BCDVA) (ETDRS Chart)

Time frame: Weeks 1, 2, 6, 12, 24, 36, 48. Mean (Standard Deviation). Unit: Letters read correctly

Measure	rhNGF 60µg/ml n = 20	rhNGF 180 µg/ml n = 20	Vehicle n = 10
Week 1 vs Day 0 - left eye	0.50 (2.86)	1.65 (2.23)	0.90 (2.81)
Week 1 vs Day 0 - right eye	1.70 (2.27)	2.20 (3.29)	1.70 (3.20)
Week 2 vs Day 0 - left eye	0.95 (2.70)	2.15 (2.30)	2.50 (3.10)
Week 2 vs Day 0 - right eye	1.40 (2.41)	2.55 (4.24)	2.60 (3.60)
Week 6 vs Day 0 - left eye	1.90 (2.73)	1.60 (2.48)	1.80 (3.91)
Week 6 vs Day 0 - right eye	1.50 (2.40)	3.25 (4.56)	2.00 (3.27)
Week 12 vs Day 0 - left eye	2.25 (3.64)	2.50 (2.56)	0.90 (4.46)
Week 12 vs Day 0 - right eye	1.85 (2.23)	2.50 (4.47)	1.10 (4.38)

Population analysed: Safety population: patients who received at least one dose of study medication. The safety population was used to analyze all safety endpoints (primary objective).

Primary outcome: Change in Intraocular Pressure (IOP)

Time frame: Weeks 2,12 and 24. Mean (Standard Deviation). Unit: mmHg

Measure	rhNGF 60µg/ml n = 20	rhNGF 180 µg/ml n = 20	Vehicle n = 10
Week 2 vs Day 0 - left eye	0.15 (1.73)	-0.70 (2.92)	0.30 (2.11)
Week 2 vs Day 0 - right eye	-0.15 (1.93)	-0.40 (3.03)	0.70 (2.11)
Week 12 vs Day 0 - left eye	0.55 (2.24)	-0.45 (3.35)	0.70 (2.50)
Week 12 vs Day 0 - right eye	0.05 (2.06)	-0.25 (3.21)	1.20 (2.35)
Week 24 vs Day 0 - left eye	-0.12 (2.03)	-0.21 (2.90)	0.00 (2.98)
Week 24 vs Day 0 - right eye	0.24 (2.54)	-0.53 (2.52)	0.90 (2.13)

Population analysed: Safety population: patients who received at least one dose of study medication. The safety population was used to analyze all safety endpoints (primary objective).

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: Weeks 1, 2, 6, 12 and 24, plus follow-up at weeks 36 and 48. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
rhNGF 60µg/ml	1 of 20 (5%)	11 of 20 (55%)	0 of 20
rhNGF 180 µg/ml	0 of 20 (0%)	13 of 20 (65%)	0 of 20
Vehicle	0 of 10 (0%)	3 of 10 (30%)	0 of 10

Limitations and caveats (from the sponsor): LImitations and caveats non specified

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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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.

Flavio Mantelli, MD, PhD study director

Dompé farmaceutici S.p.A., Milan

5 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing rhNGF 60 µg/ml eye drops solution and rhNGF 180 µg/ml eye drops solution in people with Retinitis Pigmentosa, run by Dompé Farmaceutici S.p.A at 5 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 6 inclusion and 12 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 18 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Yes. Results are posted in the registry (first posted 11 Jul 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Number of Participants With Serious and Non-Serious Adverse Events (time frame: up to 48 weeks). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02110225.

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 18 Apr 2024.