Retinitis Pigmentosa
Conditions
Keywords
Retinitis Pigmentosa, Advanced retinitis pigmentosa (RP), CNTO 2476, Subretinal, Human umbilical tissue-derived cells (hUTC), Light perception (LP), Hand motion (HM)
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).
Interventions
DRUGCNTO 2476
Participant 1 will receive 5.6x100000; Participants 2, 3, 5, 6, and 7 will receive 6.0x10000; Participant 4 will receive 3.0x100000; and Participants 8 and 9 will receive 1.2x100000 of CNTO 2476 as a single dose of viable cells in phosphate buffered saline (PBS) in a total volume of 100 μL injected subretinally.
Sponsors
Centocor, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of adverse events as a measure of safety and tolerability | Up to 5 years from the time participant is eligible to enter study |
| Level of anti-CNTO 2476 antibody in blood for immunogenicity testing | Baseline, Days 7 and 15, Weeks 3 and 4, Months 2, 3, 6, 12, 24, 36, 48, and 60 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in retinal structure and visual function | Up to 5 years from the time participant is eligible to enter study | This will be assessed by optical coherence tomography (OCT), fluorescein angiography (FA), fundus photography, and visual field sensitivity (FFST). |
Countries
United States
Outcome results
None listed