Stargardt's Macular DystrophyNCT02941991
A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
An observational study of Stargardt's Macular Dystrophy, run by Astellas Institute for Regenerative Medicine at 2 sites in United Kingdom.
Trial summary
The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Stargardt's Macular Dystrophy
- What is being tested?
- hESC-RPE
- Type of treatment
- Injection or infusion
- How many people
- 12 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- 16 Jan 2013to 2 Oct 2019
- Phase
- Observational
- 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
Study population: The study population is by invitation only from previous participants in the core protocol.
Who may be able to join
- Must have been treated with hESC-RPE cell transplant in the core protocol.
- Able to understand and willing to sign the informed consent to participate in the follow-up study.
Who cannot join
- There are no exclusion criteria
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Moorefields Eye Hospital NHS Foundation Trust
London, EC1V2PD
Newcastle on Tyne NHS Foundation Trust
Newcastle upon Tyne, NE7 7DN
About this study
Official title: Follow-up to 5 Years of a Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.
Read the detailed description
This study is a long-term, follow- up of a Phase I/II, open-label, non-randomized, 4-cohort, dose escalation, multi-center clinical trial (referred to as the core trial or core protocol) in which a maximum of 12 SMD patients were transplanted with sequential doses of hESC-RPE cells, starting at a dose of 50,000 hESC-RPE cells transplanted and increasing to a maximum dose of 200,000 hESC-RPE cells transplanted. All patients who participate in the core protocol are eligible for participation in the follow-up protocol. The first visit of this extension protocol will correspond to the last visit of the core protocol, and will take place at 12 months post-cell implantation. Informed consent for this follow-up protocol will be obtained at the first visit which will occur at the 12-month visit of the core protocol. Patients will be evaluated at 18, 24, 36, 48 and 60 months post-transplant (or more frequently as clinically indicated). Follow-up will include obtaining information about ophthalmological findings and events of special interest as defined in the Primary Outcome. At the last visit of this follow up study, whether at 60 months post-transplant or at early discontinuation, patients will be invited to participate in a life-long annual health survey, under a separate protocol, to further monitor long-term safety.
- Conditions
- Stargardt's Macular Dystrophy
- Being tested
- hESC-RPE
- Keywords
- Stargardt's Macular Dystrophy, Retinal Diseases, Macular Degeneration, fundus flavimaculatus, hESC-RPE, ASP7316, juvenile macular dystrophy, SMD
- Medical subject headings
- Retinal Diseases, Macular Degeneration, Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hESC-RPEBiological
Also called ASP7316
sub-retinal injection
| Study group | Role | What they receive |
|---|---|---|
| uniocular subretinal injection of hESC-RPE cells | Cohort 1. 50,000 cells transplanted; Cohort 2. 100,000 cells transplanted; Cohort 3. 150,000 cells transplanted; Cohort 4. 200,000 cells transplanted Receives: Biological: hESC-RPE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product
This will include obtaining information about Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, any Adverse Event (AE) that causes the subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer (irrespective of prior history), ectopic or proliferative cell growth (Retinal pigment epithelium (RPE) or non-RPE) with adverse clinical consequence, unexpected, clinically significant AE possibly related to the cell transplant procedure or the investigational product (hESC-RPE cells), Pregnancy in a female subject or the partner of a male subject and pregnancy outcome.
Measured: 4 years
Secondary outcomes (2)
Incidence of graft failure or rejection
Evidence of graft failure or rejection may consist of: Presence of retinal edema, cystoid macular edema, retinal white dots, retinal hemorrhage, serous retinal detachment, sub-retinal exudates, sub-retinal fibrosis, ocular vascular and/or optic disc leakage, and elevated intraocular pressure or hypotony. Evidence of unanticipated and persistent or increasing non-infectious ocular inflammation (e.g., vasculitis, retinitis, choroiditis, vitritis, pars planitis, anterior segment inflammation/uveitis).
Measured: 4 years
Number of patients with changes in ocular examinations or images
The number of patients with clinically significant absolute values or changes from baseline in Intra-ocular pressure (IOP) and Best Corrected Visual Acuity (BCVA) will be summarized.
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2016 (estimated)
- Study start16 Jan 2013
- Primary completion2 Oct 2019
- Study completion2 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt's Macular DystrophyStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Astellas Institute for Regenerative Medicine
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Astellas Institute for Regenerative Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02941991
- Sponsor's protocol ID
- 7316-CL-0006
- Other IDs
- 2012-002827-14, ACT-hESC-RPE-SMD-01FU EU
- Status last verified
- Dec 2020
- First submitted
- 20 Oct 2016
- Last updated
- 10 Dec 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Follow-up to 5 Years of a Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
- ClinicalTrials.gov ID
- NCT02941991
- Acronym / sponsor ID
- 7316-CL-0006
- Conditions
- Stargardt's Macular Dystrophy
- Keywords
- Stargardt's Macular Dystrophy, Retinal Diseases, Macular Degeneration, fundus flavimaculatus, hESC-RPE, ASP7316, juvenile macular dystrophy, SMD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Astellas Institute for Regenerative MedicineIndustry (pharma, biotech, devices)
- Study start
- 16 Jan 2013 (actual)
- Primary completion
- 2 Oct 2019 (actual)
- Study completion
- 2 Oct 2019 (actual)
- First posted
- 21 Oct 2016 (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 purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.
Detailed description
This study is a long-term, follow- up of a Phase I/II, open-label, non-randomized, 4-cohort, dose escalation, multi-center clinical trial (referred to as the core trial or core protocol) in which a maximum of 12 SMD patients were transplanted with sequential doses of hESC-RPE cells, starting at a dose of 50,000 hESC-RPE cells transplanted and increasing to a maximum dose of 200,000 hESC-RPE cells transplanted. All patients who participate in the core protocol are eligible for participation in the follow-up protocol. The first visit of this extension protocol will correspond to the last visit of the core protocol, and will take place at 12 months post-cell implantation. Informed consent for this follow-up protocol will be obtained at the first visit which will occur at the 12-month visit of the core protocol. Patients will be evaluated at 18, 24, 36, 48 and 60 months post-transplant (or more frequently as clinically indicated). Follow-up will include obtaining information about ophthalmological findings and events of special interest as defined in the Primary Outcome. At the last visit of this follow up study, whether at 60 months post-transplant or at early discontinuation, patients will be invited to participate in a life-long annual health survey, under a separate protocol, to further monitor long-term safety.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| uniocular subretinal injection of hESC-RPE cells | – | Biological: hESC-RPECohort 1. 50,000 cells transplanted; Cohort 2. 100,000 cells transplanted; Cohort 3. 150,000 cells transplanted; Cohort 4. 200,000 cells transplanted |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study population is by invitation only from previous participants in the core protocol.
Key inclusion criteria (2 listed)
- Must have been treated with hESC-RPE cell transplant in the core protocol.
- Able to understand and willing to sign the informed consent to participate in the follow-up study.
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 |
|---|---|---|
| hESC-RPEAlso: ASP7316sub-retinal injection | Biological | uniocular subretinal injection of hESC-RPE cells |
Primary outcome
Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product
Time frame: 4 years
This will include obtaining information about Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, any Adverse Event (AE) that causes the subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer (irrespective of prior history), ectopic or proliferative cell growth (Retinal pigment epithelium (RPE) or non-RPE) with adverse clinical consequence, unexpected, clinically significant AE possibly related to the cell transplant procedure or the investigational product (hESC-RPE cells), Pregnancy in a female subject or the partner of a male subject and pregnancy outcome.
2 secondary outcomes
Incidence of graft failure or rejection
Time frame: 4 years
Number of patients with changes in ocular examinations or images
Time frame: 4 years
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 10 Dec 2020.
- Individual participant dataSharing planned: Undecided
Related trials for Stargardt's Macular Dystrophy
RecruitingObservational study
Rod and Cone Mediated Function in Retinal Disease
Retinal Degeneration, Retinitis Pigmentosa
Active, not recruitingPhase 2
Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration
Stargardt's Disease
RecruitingObservational study
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene
Stargardt, Stargardt's Disease
Not yet recruiting
Feasibility and Tolerability Study of Smart Contact Lens With Healthy Subjects and Patients With Stargardt's Disease
Stargardt Disease
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.
Medical Director study director
Astellas Institute for Regenerative Medicine
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Stargardt's Macular Dystrophy, run by Astellas Institute for Regenerative Medicine at 2 sites 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 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: finished, as of the sponsor's last update on 10 Dec 2020. 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 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.
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?
Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product (time frame: 4 years). 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 10 Dec 2020.