Macular Degenerative DiseasePhase 1/2NCT03167203
A Safety Surveillance Study in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy
A Phase 1/2 study testing Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells in people with Macular Degenerative Disease, run by Astellas Institute for Regenerative Medicine at 2 sites in 2 countries.
Trial summary
The purpose of this study is to is to evaluate the occurrence of late onset (i.e., greater than 5 years after treatment) adverse events of special interest (AESI) in participants who have received sub-retinal transplant of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cells in an… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Macular Degenerative Disease
- What is being tested?
- Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 8 Jan 2018to 31 Mar 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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
- Participant was treated with hESC-RPE cell therapy in an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial in macular degenerative disease.
- Participant is able to understand.
Who cannot join
- There are no exclusion criteria.
Trial locations and contacts
2 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
2 sites in 2 countries
About this study
Official title: A Safety Surveillance Study of Events of Special Interest Occurring in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy
The purpose of this study is to is to evaluate the occurrence of late onset (i.e., greater than 5 years after treatment) adverse events of special interest (AESI) in participants who have received sub-retinal transplant of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cells in an AIRM-sponsored clinical trial. The events of special interest are adverse events (AEs) that are presumed to have a potential causal relationship to the hESC-RPE cells.
Read the detailed description
Participants will be contacted by the Patient Outreach Center (POC) on an annual basis to complete a questionnaire about the occurrence of AESI, as defined in the Outcome Measure section.
The questionnaire will be completed directly by the participant (via a secure web based platform) or will be administered via telephone by a POC staff member. In the event of the occurrence of a AESI, study participant will be encouraged to contact the POC as soon as possible, rather than wait for the annual questionnaire completion. Follow up data is inclusive of the 1 year in the core trial, 4 years in the long term follow up trial and 10 years in this long term safety follow up. Participation for United Kingdom participants will be life-long.
- Conditions
- Macular Degenerative Disease
- Being tested
- Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells
- Keywords
- hESC-RPE, Macular Degenerative Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial CellsBiological
Also called ASP7316
Participants who have received sub-retinal transplant of hESC-RPE cells in an AIRM sponsored clinical trial
| Study group | Role | What they receive |
|---|---|---|
| hESC-RPE cells | Experimental | Participants previously enrolled into an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial and treated with Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial (hESC-RPE) cells Receives: Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events (AEs) that are ophthalmologic, neurologic, infectious or hematologic
Adverse events (AEs) will be coded using Medical Dictionary for Regulatory Activities (MedDRA). Participant self-reported via an annual questionnaire of adverse events that are ophthalmologic, neurologic, infectious or hematologic.
Measured: Up to 15 Years
Number of new diagnoses of an immune-mediated disorder
Any new diagnosis of an immune-mediated disorder will be participant self-reported via an annual questionnaire.
Measured: Up to 15 Years
Incidents of new cancer, irrespective of prior history
Any new cancer, irrespective of prior history, will be participant self-reported via an annual questionnaire.
Measured: Up to 15 Years
Incidents of hESC-RPE cell proliferation
Occurrence of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cell proliferation will be participant self-reported via an annual questionnaire.
Measured: Up to 15 Years
Incidents of ectopic tissue (RPE or non-RPE) formation
Occurrence of ectopic tissue (retinal pigment epithelial [RPE] or non-RPE) will be participant self-reported via an annual questionnaire
Measured: Up to 15 Years
Number of participant reported pregnancies or pregnancy of participant's partner
Occurrence of pregnancy will be participant self-reported via an annual questionnaire
Measured: Up to 15 Years
Number of reported pregnancy outcome(s)
Occurrence of pregnancy outcomes will be participant self-reported via an annual questionnaire
Measured: Up to 15 Years
All cause death
All causes of death will be collected via an annual questionnaire (through participant-designated secondary contacts)
Measured: Up to 15 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2017
- Study start8 Jan 2018
- Primary completion31 Mar 2029 (estimated)
- Study completion31 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Macular Degenerative DiseaseStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Astellas Institute for Regenerative Medicine
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Astellas Institute for Regenerative Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03167203
- Sponsor's protocol ID
- 7316-CL-0007
- Other IDs
- 2016-005245-23, RPE-SSS-001, 2016
- Status last verified
- Jul 2026
- First submitted
- 24 May 2017
- Last updated
- 13 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as products terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.
Study at a glance
From the registry record, last updated by the sponsor on 13 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Safety Surveillance Study of Events of Special Interest Occurring in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy
- ClinicalTrials.gov ID
- NCT03167203
- Acronym / sponsor ID
- 7316-CL-0007
- Conditions
- Macular Degenerative Disease
- Keywords
- hESC-RPE, Macular Degenerative Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Astellas Institute for Regenerative MedicineIndustry (pharma, biotech, devices)
- Study start
- 8 Jan 2018 (actual)
- Primary completion
- 31 Mar 2029 (estimated)
- Study completion
- 31 Mar 2029 (estimated)
- First posted
- 25 May 2017 (actual)
- 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 purpose of this study is to is to evaluate the occurrence of late onset (i.e., greater than 5 years after treatment) adverse events of special interest (AESI) in participants who have received sub-retinal transplant of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cells in an AIRM-sponsored clinical trial. The events of special interest are adverse events (AEs) that are presumed to have a potential causal relationship to the hESC-RPE cells.
Detailed description
Participants will be contacted by the Patient Outreach Center (POC) on an annual basis to complete a questionnaire about the occurrence of AESI, as defined in the Outcome Measure section.
The questionnaire will be completed directly by the participant (via a secure web based platform) or will be administered via telephone by a POC staff member. In the event of the occurrence of a AESI, study participant will be encouraged to contact the POC as soon as possible, rather than wait for the annual questionnaire completion. Follow up data is inclusive of the 1 year in the core trial, 4 years in the long term follow up trial and 10 years in this long term safety follow up. Participation for United Kingdom participants will be life-long.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| hESC-RPE cells | Experimental | Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial CellsParticipants previously enrolled into an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial and treated with Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial (hESC-RPE) cells |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Participant was treated with hESC-RPE cell therapy in an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial in macular degenerative disease.
- Participant is able to understand.
Key exclusion criteria (1 listed)
- There are no exclusion criteria.
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 |
|---|---|---|
| Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial CellsAlso: ASP7316Participants who have received sub-retinal transplant of hESC-RPE cells in an AIRM sponsored clinical trial | Biological | hESC-RPE cells |
Primary outcome
Number of adverse events (AEs) that are ophthalmologic, neurologic, infectious or hematologic
Time frame: Up to 15 Years
Adverse events (AEs) will be coded using Medical Dictionary for Regulatory Activities (MedDRA). Participant self-reported via an annual questionnaire of adverse events that are ophthalmologic, neurologic, infectious or hematologic.
Number of new diagnoses of an immune-mediated disorder
Time frame: Up to 15 Years
Any new diagnosis of an immune-mediated disorder will be participant self-reported via an annual questionnaire.
Incidents of new cancer, irrespective of prior history
Time frame: Up to 15 Years
Any new cancer, irrespective of prior history, will be participant self-reported via an annual questionnaire.
Incidents of hESC-RPE cell proliferation
Time frame: Up to 15 Years
Occurrence of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cell proliferation will be participant self-reported via an annual questionnaire.
Incidents of ectopic tissue (RPE or non-RPE) formation
Time frame: Up to 15 Years
Occurrence of ectopic tissue (retinal pigment epithelial [RPE] or non-RPE) will be participant self-reported via an annual questionnaire
Number of participant reported pregnancies or pregnancy of participant's partner
Time frame: Up to 15 Years
Occurrence of pregnancy will be participant self-reported via an annual questionnaire
Number of reported pregnancy outcome(s)
Time frame: Up to 15 Years
Occurrence of pregnancy outcomes will be participant self-reported via an annual questionnaire
All cause death
Time frame: Up to 15 Years
All causes of death will be collected via an annual questionnaire (through participant-designated secondary contacts)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jul 2026.
- Individual participant dataSharing planned: Yes. Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as products terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.
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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.
Medical Monitor study director
Astellas Institute for Regenerative Medicine
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells in people with Macular Degenerative Disease, run by Astellas Institute for Regenerative Medicine at 2 sites in 2 countries.
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 2 inclusion and 1 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: enrolling by invitation, as of the sponsor's last update on 13 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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 (AEs) that are ophthalmologic, neurologic, infectious or hematologic (time frame: Up to 15 Years). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Jul 2026.