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

• Register: ClinicalTrials.gov
• Last refreshed on: 29 April 2024
• Main ID: NCT02110225
• Date of registration: 08/04/2014
• Prospective Registration: No
• Primary sponsor: Dompé Farmaceutici S.p.A
• Public title: A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP) Lumos
• Scientific 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.
• Date of first enrolment: January 2014
• Target sample size: 50
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02110225
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

Italy

Contacts

• Name: Flavio Mantelli, MD, PhD
• Affiliation: Dompé farmaceutici S.p.A., Milan

Key inclusion & exclusion 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, 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.

- 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Retinitis Pigmentosa

Intervention(s)

Drug: rhNGF 180 µg/ml eye drops solution

Drug: rhNGF 60 µg/ml eye drops solution

Drug: Placebo

Primary Outcome(s)

Change in Best Corrected Distance Visual Acuity (BCDVA) (ETDRS Chart) [Time Frame: Weeks 1, 2, 6, 12, 24, 36, 48]

Change in Intraocular Pressure (IOP) [Time Frame: Weeks 2,12 and 24]

Change in Ocular Tolerability - VAS [Time Frame: Weeks 1, 2, 6, 12, 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]

Change in Ocular Tolerability - Dilated Fundus Ophthalmoscopy [Time Frame: Day 0, Weeks 12, 24 and 48]

Number of Participants With Serious and Non-Serious Adverse Events [Time Frame: up to 48 weeks]

Presence of Anti-NGF Antibodies [Time Frame: At Day 0 and at week 24]

External Ocular Examination [Time Frame: Day 0, Weeks 1, 2, 6, 12, 24]

Secondary Outcome(s)

Fundus Imaging [Time Frame: Day 0, Weeks 24 and 48]

Ocular Coherence Tomography (OCT) [Time Frame: Day 0, Weeks 12, 24, 36 and 48]

Change From Baseline in Contrast Sensitivity [Time Frame: Weeks 12, 24, 36 and 48]

Electrorethinogram (ERG) [Time Frame: Day 0, Weeks 12, 24, 36 and 48]

Change From Baseline in Humphrey Visual Field 24-2 [Time Frame: Weeks 12, 24, 36 and 48]

Binocular Estermann Visual Field [Time Frame: Day 0, Weeks 12, 24, 36 and 48]

Change in Goldmann Visual Field [Time Frame: Weeks 12, 24, 36 and 48]

Microperimetry [Time Frame: Day 0, Weeks 12, 24, 36 and 48]

Secondary ID(s)

2013-003029-26

NGF0113

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 11/07/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02110225