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

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ClinicalTrials.gov Identifier: NCT02110225
Recruitment Status : Completed
First Posted : April 10, 2014
Last Update Posted : July 26, 2018
Sponsor:
Information provided by (Responsible Party):
Dompé Farmaceutici S.p.A

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Retinitis Pigmentosa Drug: rhNGF 60 µg/ml eye drops solution Drug: rhNGF 180 µg/ml eye drops solution Drug: Placebo Phase 1 Phase 2

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).

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 50 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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.
Actual Study Start Date : January 2014
Actual Primary Completion Date : November 2015
Actual Study Completion Date : November 2015


Arm Intervention/treatment
Experimental: rhNGF 60µg/ml
rhNGF 60 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes
Drug: rhNGF 60 µg/ml eye drops solution
rhNGF 60 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes
Other Name: recombinant human nerve growth factor 60 µg/ml solution

Experimental: rhNGF 180 µg/ml
rhNGF 180 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes.
Drug: rhNGF 180 µg/ml eye drops solution
rhNGF 180 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes
Other Name: recombinant human nerve growth factor 180 µg/ml solution

Placebo Comparator: Vehicle
Placebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes.
Drug: Placebo
Placebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes
Other Name: Vehicle




Primary Outcome Measures :
  1. Number of Participants with Serious and Non-Serious Adverse Events [ Time Frame: up to 48 weeks ]
    Safety evaluation


Secondary Outcome Measures :
  1. Best Corrected Distance Visual Acuity (BCDVA) (ETDRS chart) [ Time Frame: Day 0, Week 12, 24, 36, 48 ]
    Efficacy as measured by Best Corrected Distance Visual Acuity (BCDVA) test results will be analyzed by means of analysis of variance, including terms for treatment, time (Day 0, Week 12, 24, 36, 48), and treatment by time interaction.

  2. Change in ocular tolerability - VAS [ Time Frame: Weeks 1, 2, 6, 12, 24 ]
    Ocular tolerability as measured by the visual analogue scale (VAS), will be analyzed by means of analysis of variance, including terms for treatment, time (Weeks 1, 2, 6, 12, 24), and treatment by time interaction

  3. Change in ocular tolerability - Dilated fundus ophthalmoscopy [ Time Frame: Day 0, Weeks 12, 24 and 48 ]
    Ocular tolerability as measured by fundus appearance, will be analyzed by means of analysis of variance, including terms for treatment, time (Day 0, Weeks 12, 24 and 48), and treatment by time interaction

  4. Presence of Anti-NGF antibodies [ Time Frame: At day 0 and at week 24 ]
    Anti-NGF antibodies and laboratory tests are performed at screening and at the end of treatment and shift tables will be used to present shifts from within/outside the normal range between screening and end of treatment



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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:

  • 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.

Exclusion Criteria:

  • Patients with atypical, early onset (first decade) or syndromic forms of RP (e.g. paravenous, pericentral sector or unilateral RP, Leber's congenital amaurosis, Resfum 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.
  • 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.

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): NCT02110225


Locations
Italy
Azienda Ospedaliero Universitaria Careggi
Florence, Italy, 50124
Azienda Ospedaliera San Paolo - U.O. Oculistica
Milano, Italy, 20142
A.O. Seconda Università Degli Studi di Napoli - Nuovo Policlinico - UOC Oftalmologia
Naples, Italy, 80129
Università Cattolica del Sacro Cuore - Policlinico Gemelli - Istituto di Oftalmologia
Rome, Italy, 00168
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia
Rome, Italy, 00198
Sponsors and Collaborators
Dompé Farmaceutici S.p.A
Investigators
Study Director: Flavio Mantelli, MD, PhD Dompé farmaceutici S.p.A., Milan

Responsible Party: Dompé Farmaceutici S.p.A
ClinicalTrials.gov Identifier: NCT02110225     History of Changes
Other Study ID Numbers: NGF0113
2013-003029-26 ( EudraCT Number )
First Posted: April 10, 2014    Key Record Dates
Last Update Posted: July 26, 2018
Last Verified: July 2018

Keywords provided by Dompé Farmaceutici S.p.A:
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

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