Clinical trial NCT00458575: A Study to Evaluate the Safety of CNTO 2476 in Patients With Advanced Retinitis Pigmentosa
Official title: A Phase I Open Label Non-comparative Study Evaluating the Safety of a Single, Unilateral, Subretinal Administration of CNTO 2476 in Advanced Retinitis Pigmentosa
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Retinitis Pigmentosa
Keywords: Retinitis Pigmentosa
Advanced retinitis pigmentosa (RP)
CNTO 2476
Subretinal
Human umbilical tissue-derived cells (hUTC)
Light perception (LP)
Hand motion (HM)
Lead sponsor: Centocor, Inc.
Sponsor class: INDUSTRY
First posted: 2007-04-11
Enrollment: 7
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The main purpose of this study is to evaluate the preliminary safety and immunogenicity (ability of an antigen to provoke immune response in the human body) of CNTO 2476, administered subretinally, in participants with advanced retinitis pigmentosa (RP; disease of the eye that leads to loss of vision and blindness) with either light perception only (LP) or hand motion (HM).

Detailed description: The study will be an open-label (all people know the identity of the intervention), first in human safety study. Nine adult men and women with advanced RP with LP only (first 5 participants) or not better than HM bilaterally (subsequent 4 participants) in both eyes will be enrolled. Participant 5 may have combination visual acuity of LP in the treated eye and no better than HM in the fellow eye. Treatment will proceed on a cohort basis ie, Cohort I: Participant 1; Cohort II: Participant 2; Cohort III: Participant 3 and 4; Cohort IV: Participant 5, 6, and 7; and Cohort V: Participant 8 and 9. The initial participants for each cohort must have completed at least 4 weeks of post-treatment follow-up before participants in the next cohort may be treated. There are 2 safety assessment phases in this study: the main phase approximately 12 months from the time the participant is eligible to enter the study and the long-term safety follow-up phase of 4 additional years. The total length of the participation in the study could last up to 5 years (including both the main and safety follow-up phases).

Eligibility criteria: Inclusion Criteria:

* Confirmed diagnosis of retinitis pigmentosa that is not part of another systemic disease
* Vision deficit characterized by vision that is not better than either light perception (Group 1) or hand motion (Group 2) in both eyes
* Normal hematology and chemistry lab results
* Participant is suitable candidate for ophthalmologic surgery

Exclusion Criteria:

* Other significant ophthalmologic diseases or any other ophthalmologic condition that interferes with ophthalmologic examination
* Women of childbearing potential
* Ocular hypertension
* Other serious medical conditions

Primary outcomes:
measure: Number of adverse events as a measure of safety and tolerability
timeFrame: Up to 5 years from the time participant is eligible to enter study
measure: Level of anti-CNTO 2476 antibody in blood for immunogenicity testing
timeFrame: Baseline, Days 7 and 15, Weeks 3 and 4, Months 2, 3, 6, 12, 24, 36, 48, and 60
Secondary outcomes:
measure: Change from baseline in retinal structure and visual function
timeFrame: Up to 5 years from the time participant is eligible to enter study
Other outcomes:
Locations:
city: Miami
country: United States
state: Florida
city: Sw Portland
country: United States
state: Oregon
city: Dallas
country: United States
state: Texas
Study officials:
affiliation: Centocor, Inc.
name: Centocor, Inc. Clinical Trial
role: STUDY_DIRECTOR
References:
citation: Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5.