A Study to Evaluate the Safety of CNTO 2476 in Patients With Advanced Retinitis Pigmentosa

NCT ID: NCT00458575

Last Updated: 2014-06-26

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-04-30
• Study Completion Date: 2013-08-31

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).

Conditions

Retinitis Pigmentosa

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

CNTO 2476

Participants 1 to 4: advanced retinitis pigmentosa (RP) with light perception only (LP); Participant 5: combination visual acuity of LP in the treated eye and no better than hand motion (HM) in the fellow eye; and Participants 6 to 9: advanced RP with hand motion (HM) will receive different dose levels of CNTO 2476.

Group Type: EXPERIMENTAL

CNTO 2476

Intervention Type: DRUG

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.

Interventions

CNTO 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.

Intervention Type: DRUG

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Centocor, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Centocor, Inc. Clinical Trial

Role: STUDY_DIRECTOR

Centocor, Inc.

Locations

Miami, Florida, United States

Southwest Portland, Oregon, United States

Dallas, Texas, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 30194931 (View on PubMed)

Other Identifiers

CNTO2476RPG1001

Identifier Type: OTHER

Identifier Source: secondary_id

CR013210

Identifier Type: -

Identifier Source: org_study_id