Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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

Tracking Information
First Submitted Date  ICMJE April 8, 2014
First Posted Date  ICMJE April 10, 2014
Last Update Posted Date July 26, 2018
Actual Study Start Date  ICMJE January 2014
Actual Primary Completion Date November 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 24, 2018)
Number of Participants with Serious and Non-Serious Adverse Events [ Time Frame: up to 48 weeks ]
Safety evaluation
Original Primary Outcome Measures  ICMJE
 (submitted: April 9, 2014)
Number of Participants with Serious and Non-Serious Adverse Events [ Time Frame: up to 48 weeks ]
Change History Complete list of historical versions of study NCT02110225 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 9, 2014)
  • 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.
  • 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
  • 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
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)
Official Title  ICMJE 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.
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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Retinitis Pigmentosa
Intervention  ICMJE
  • 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
  • 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
  • Drug: Placebo
    Placebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes
    Other Name: Vehicle
Study Arms  ICMJE
  • Experimental: rhNGF 60µg/ml
    rhNGF 60 µg/ml eye drops solution, one drop 3 times a day for 24 weeks in both eyes
    Intervention: Drug: rhNGF 60 µg/ml eye drops 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.
    Intervention: Drug: rhNGF 180 µg/ml eye drops solution
  • Placebo Comparator: Vehicle
    Placebo eye drops solution, one drop 3 times a day for 24 weeks in both eyes.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 9, 2014)
50
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE November 2015
Actual Primary Completion Date November 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Italy
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02110225
Other Study ID Numbers  ICMJE NGF0113
2013-003029-26 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Dompé Farmaceutici S.p.A
Study Sponsor  ICMJE Dompé Farmaceutici S.p.A
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Flavio Mantelli, MD, PhD Dompé farmaceutici S.p.A., Milan
PRS Account Dompé Farmaceutici S.p.A
Verification Date July 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP