Age Related Macular DegenerationPhase 1NCT01691261
A Study of Implantation of Retinal Pigment Epithelium in Subjects with Acute Wet Age Related Macular Degeneration
A Phase 1 study testing PF-05206388 in people with Age Related Macular Degeneration, run by Moorfields Eye Hospital NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
Phase 1 trial of retinal pigment epithelium replacement in subjects with wet age-related macular degeneration in whom there is rapidly progressing vision loss Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Age Related Macular Degeneration
- What is being tested?
- PF-05206388
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 14 Oct 2021to 24 Jun 2024
- 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 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and /or post-menopausal female subjects aged 60 years or above.
- Diagnosis of wet Age-related Macular Degeneration (AMD) plus rapid recent vision decline
- An informed consent document signed and dated by the subject or a legal representative.
Who cannot join
- Pregnant females; breastfeeding females; and females of childbearing potential.
- Treatment with an investigational drug within 30 days (or as determined by the local requirement, whichever is longer) or 5 half-lives preceding the first dose of study medication.
- Current or previous significant other ocular disease in the study eye, as determined by the investigator.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Moorfields Eye Hospital NHS Foundation Trust
London, EC1V 2PD
About this study
Official title: Phase 1, Open-label, Safety and Feasibility Study of Implantation of Pf-05206388 (human Embryonic Stem Cell Derived Retinal Pigment Epithelium (rpe) Living Tissue Equivalent) in Subjects with Acute Wet Age Related Macular Degeneration and Recent Rapid Vision Decline
Phase 1 trial of retinal pigment epithelium replacement in subjects with wet age-related macular degeneration in whom there is rapidly progressing vision loss
Read the detailed description
Phase 1, open-label, safety and feasibility study of implantation of PF-05206388 (human embryonic stem cell derived retinal pigment epithelium) in subjects with wet age related macular degeneration and rapid vision loss
- Conditions
- Age Related Macular Degeneration
- Being tested
- PF-05206388
- Medical subject headings
- Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PF-05206388Biological
PF-05206388 will be provided as a Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane. The membrane is approximately 6 mm x 3 mm and will contain a confluent layer of RPE cells, at a nominal dose of 17 mm2. The implant is intended to be life-long.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | PF-05206388 Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane Receives: Biological: PF-05206388 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events.
The number of Adverse Events (AE) and Serious Adverse Events (SAE) noted during the study and an assessment of whether they are trial product related
Measured: 52 weeks
Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more at Week 24.
The number of patients with a difference between the baseline BCVA and BCVA at 24 weeks in ETDRS letters, where the differences is 15 letters or more, as a percentage of the total number of cases.
Measured: 24 weeks
Secondary outcomes (9)
Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more
The number of patients with a difference between the baseline BCVA and BCVA at weeks 1,2,4,8, 12,16, 36, 52 in ETDRS letters, where the differences is 15 letters or more, as a percentage of the total number of cases.
Measured: Weeks 1,2,4,8, 12,16, 36, 52
Mean change of best corrected visual acuity (BCVA) from baseline by study visit.
The mean difference between the baseline BCVA and final BCVA in ETDRS letters for all cases
Measured: 52 weeks
Position of PF-05206388 by serial biomicroscopic evaluation.
Measurement of movement in millimetre and rotation in degrees measure relative to baseline, at day 2 and weeks 1, 2, 4, 8, 10, 12, 16, 24, 36 and 52
Measured: Day 2 and Weeks 1, 2, 4, 8, 10, 12, 16, 24, 36, 52
Position and presence of pigmented RPE cells by serial fundus photography
The subjective reporting of area of pigmentation as a % with cross reference to the OCT at weeks 2, 4, 8, 10, 12, 16, 24, 36 and 52
Measured: Weeks 2, 4, 8, 10, 12, 16, 24, 36, 52
Mean change from baseline in contrast sensitivity by Pelli Robson test
The mean difference between the baseline BCVA and final BCVA in Pelli Robson letters read, across all subjects
Measured: Weeks 24, 52
Change in liver and renal function by blood tests and liver ultrasound .
Record of any abnormalities in liver and renal function on blood testing and any abnormalities detected on the liver ultrasound.
Measured: Weeks 24 and 52
Change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography.
Assessment and noting of abnormalities on Funded fluorescine angiography at weeks 4, 8, 12, 24 and 52.
Measured: Weeks 4, 8, 12, 24 and 52
Change in central 30 degree of visual function by Humphrey Field test.
Recording and reporting of any changes on the central 30 degree field on the automated Humphrey Field test at weeks 4, 8, 12, 24 and 52
Measured: Weeks 4, 8, 12, 24 and 52
Change in thickness of RPE layer by B-mode orbital ultrasound.
Recording of any changes in thickness of RPE layer by B-mode orbital ultrasound carried out by the ocular oncologist or medical physicist at weeks 4, 8, 16, 24, 36, 52.
Measured: Weeks 4, 8, 16, 24, 36, 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2012 (estimated)
- Study start14 Oct 2021
- Primary completion10 Oct 2023
- Study completion24 Jun 2024
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 Age Related Macular DegenerationStudies 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
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Moorfields Eye Hospital NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- University College, London
- Responsible party
- Sponsor
- Investigators
- Moorfields, Study director, Moorfields Eye Hospital NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01691261
- Sponsor's protocol ID
- DACL1010
- Other IDs
- 2011-005493-37
- Status last verified
- Nov 2024
- First submitted
- 19 Sep 2012
- Last updated
- 22 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
Publications
Soomro T, Georgiadis O, Coffey PJ, da Cruz L. Safety, structure and function five years after hESC-RPE patch transplantation in acute neovascular AMD with submacular haemorrhage. Graefes Arch Clin Exp Ophthalmol. 2024 Sep;262(9):3057-3060. doi: 10.1007/s00417-024-06463-4. Epub 2024 Apr 5. No abstract available.
PubMed 38578329
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1, Open-label, Safety and Feasibility Study of Implantation of Pf-05206388 (human Embryonic Stem Cell Derived Retinal Pigment Epithelium (rpe) Living Tissue Equivalent) in Subjects with Acute Wet Age Related Macular Degeneration and Recent Rapid Vision Decline
- ClinicalTrials.gov ID
- NCT01691261
- Acronym / sponsor ID
- DACL1010
- Conditions
- Age Related Macular Degeneration
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Moorfields Eye Hospital NHS Foundation TrustUniversity, hospital or other
- Collaborators
- University College, London
- Study start
- 14 Oct 2021 (actual)
- Primary completion
- 10 Oct 2023 (actual)
- Study completion
- 24 Jun 2024 (actual)
- First posted
- 24 Sep 2012 (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.
Phase 1 trial of retinal pigment epithelium replacement in subjects with wet age-related macular degeneration in whom there is rapidly progressing vision loss
Detailed description
Phase 1, open-label, safety and feasibility study of implantation of PF-05206388 (human embryonic stem cell derived retinal pigment epithelium) in subjects with wet age related macular degeneration and rapid vision loss
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Biological: PF-05206388PF-05206388 Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male and /or post-menopausal female subjects aged 60 years or above.
- Diagnosis of wet Age-related Macular Degeneration (AMD) plus rapid recent vision decline
- An informed consent document signed and dated by the subject or a legal representative.
Key exclusion criteria (3 listed)
- Pregnant females; breastfeeding females; and females of childbearing potential.
- Treatment with an investigational drug within 30 days (or as determined by the local requirement, whichever is longer) or 5 half-lives preceding the first dose of study medication.
- Current or previous significant other ocular disease in the study eye, as determined by the investigator.
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 |
|---|---|---|
| PF-05206388PF-05206388 will be provided as a Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane. The membrane is approximately 6 mm x 3 mm and will contain a confluent layer of RPE cells, at a nominal dose of 17 mm2. The implant is intended to be life-long. | Biological | Treatment |
Primary outcome
Incidence and severity of adverse events.
Time frame: 52 weeks
The number of Adverse Events (AE) and Serious Adverse Events (SAE) noted during the study and an assessment of whether they are trial product related
Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more at Week 24.
Time frame: 24 weeks
The number of patients with a difference between the baseline BCVA and BCVA at 24 weeks in ETDRS letters, where the differences is 15 letters or more, as a percentage of the total number of cases.
9 secondary outcomes
Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more
Time frame: Weeks 1,2,4,8, 12,16, 36, 52
Mean change of best corrected visual acuity (BCVA) from baseline by study visit.
Time frame: 52 weeks
Position of PF-05206388 by serial biomicroscopic evaluation.
Time frame: Day 2 and Weeks 1, 2, 4, 8, 10, 12, 16, 24, 36, 52
Position and presence of pigmented RPE cells by serial fundus photography
Time frame: Weeks 2, 4, 8, 10, 12, 16, 24, 36, 52
Mean change from baseline in contrast sensitivity by Pelli Robson test
Time frame: Weeks 24, 52
Change in liver and renal function by blood tests and liver ultrasound .
Time frame: Weeks 24 and 52
Change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography.
Time frame: Weeks 4, 8, 12, 24 and 52
Change in central 30 degree of visual function by Humphrey Field test.
Time frame: Weeks 4, 8, 12, 24 and 52
Change in thickness of RPE layer by B-mode orbital ultrasound.
Time frame: Weeks 4, 8, 16, 24, 36, 52
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2024.
- Derived from this study: PubMed 38578329Soomro T, Georgiadis O, Coffey PJ, da Cruz L. Safety, structure and function five years after hESC-RPE patch transplantation in acute neovascular AMD with submacular haemorrhage. Graefes Arch Clin Exp Ophthalmol. 2024 Sep;262(9):3057-3060. doi: 10.1007/s00417-024-06463-4. Epub 2024 Apr 5. No abstract available.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: No. Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
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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.
Moorfields study director
Moorfields Eye Hospital NHS Foundation Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PF-05206388 in people with Age Related Macular Degeneration, run by Moorfields Eye Hospital NHS Foundation Trust at 1 site in United Kingdom.
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 3 inclusion and 3 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: finished, as of the sponsor's last update on 22 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence and severity of adverse events. (time frame: 52 weeks). 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 NCT01691261, 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 22 Nov 2024.