Retinitis PigmentosaPhase 1NCT00458575
A Study to Evaluate the Safety of CNTO 2476 in Patients With Advanced Retinitis Pigmentosa
A Phase 1 study testing CNTO 2476 in people with Retinitis Pigmentosa, run by Centocor, Inc. at 3 sites in United States.
Trial 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)… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- CNTO 2476
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 3 sitesUnited States
- Study dates
- Apr 2007to Aug 2013
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Other significant ophthalmologic diseases or any other ophthalmologic condition that interferes with ophthalmologic examination
- Women of childbearing potential
- Ocular hypertension
- Other serious medical conditions
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Study site
Dallas, Texas
Study site
Miami, Florida
Study site
Southwest Portland, Oregon
About this study
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
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).
Read the 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
- Being tested
- CNTO 2476
- Keywords
- Retinitis Pigmentosa, Advanced retinitis pigmentosa (RP), CNTO 2476, Subretinal, Human umbilical tissue-derived cells (hUTC), Light perception (LP), Hand motion (HM)
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CNTO 2476Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| CNTO 2476 | Experimental | 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. Receives: Drug: CNTO 2476 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events as a measure of safety and tolerability
Measured: Up to 5 years from the time participant is eligible to enter study
Level of anti-CNTO 2476 antibody in blood for immunogenicity testing
Measured: Baseline, Days 7 and 15, Weeks 3 and 4, Months 2, 3, 6, 12, 24, 36, 48, and 60
Secondary outcomes (1)
Change from baseline in retinal structure and visual function
This will be assessed by optical coherence tomography (OCT), fluorescein angiography (FA), fundus photography, and visual field sensitivity (FFST).
Measured: Up to 5 years from the time participant is eligible to enter study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2007 (estimated)
- Study startApr 2007
- Primary completionAug 2013
- Study completionAug 2013
Why it stopped: Internal Business Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centocor, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Centocor, Inc. Clinical Trial, Study director, Centocor, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00458575
- Sponsor's protocol ID
- CR013210
- Other IDs
- CNTO2476RPG1001
- Status last verified
- Jun 2014
- First submitted
- 10 Apr 2007
- Last updated
- 26 Jun 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 30194931
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00458575
- Acronym / sponsor ID
- CR013210
- 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)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Internal Business Decision
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Centocor, Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2007
- Primary completion
- Aug 2013 (actual)
- Study completion
- Aug 2013 (actual)
- First posted
- 11 Apr 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CNTO 2476 | Experimental | Drug: CNTO 2476Participants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (4 listed)
- Other significant ophthalmologic diseases or any other ophthalmologic condition that interferes with ophthalmologic examination
- Women of childbearing potential
- Ocular hypertension
- Other serious medical conditions
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CNTO 2476Participant 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. | Drug | CNTO 2476 |
Primary outcome
Number of adverse events as a measure of safety and tolerability
Time frame: Up to 5 years from the time participant is eligible to enter study
Level of anti-CNTO 2476 antibody in blood for immunogenicity testing
Time frame: Baseline, Days 7 and 15, Weeks 3 and 4, Months 2, 3, 6, 12, 24, 36, 48, and 60
1 secondary outcome
Change from baseline in retinal structure and visual function
Time frame: Up to 5 years from the time participant is eligible to enter study
Results and safety
The study was stopped early (reason given: Internal Business Decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2014.
- Derived from this study: PubMed 30194931Davis 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Centocor, Inc. Clinical Trial study director
Centocor, Inc.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CNTO 2476 in people with Retinitis Pigmentosa, run by Centocor, Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Jun 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Internal Business Decision). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adverse events as a measure of safety and tolerability (time frame: Up to 5 years from the time participant is eligible to enter study). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00458575, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2014.