Age-Related Macular DegenerationPhase 2PORTRAYNCT02563782
Study to Evaluate Sub-retinal Transplantation of Retinal Pigmented Epithelial Cells in Patients With Dry AMD
A Phase 2 study testing Sub-retinal transplantation of MA09-hRPE cells and tacrolimus and mycophenolate mofetil in people with Age-Related Macular Degeneration, run by Astellas Institute for Regenerative Medicine at 1 site in United States.
Trial summary
To evaluate the safety of 3 regimens of short-term, low-dose systemic IMT as rejection prophylaxis prior to and/or following transplant of MA09-hRPE cells. Read more about this study
- Who is it for?
- Ages 50 to 85 years
- Conditions the study is for
- Age-Related Macular Degeneration
- What is being tested?
- Sub-retinal transplantation of MA09-hRPE cells and tacrolimus and mycophenolate mofetil
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Aug 2015to 5 May 2017
- Phase
- Phase 2
- 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 50 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Geographic atrophy (GA) secondary to AMD with no evidence of prior to active choroidal neovascularization (CNV) in the study eye.
- BCVA in the study eye must be between 4 and 58 Early Treatment of Diabetic Retinopathy Study (EDTRS) letters (20/800 to 20/80; 0.025-0.25)
- Subjects must be willing to take IMT and willing to discontinue any medication that has a known strong interaction with tacrolimus or mycophenolate mofetil (MMF)
Who cannot join
- Macular atrophy due to causes other than AMD
- Other sight-threatening ocular disease
- Current or prior history of optic neuropathy, retinal dystrophy, retinitis pigmentosa, chorioretinitis, vasoocclusive disease, retinal vascular disease or retinal degenerative disease OTHER than AMD
- History of uveitis
- History of allergic reaction to sulfa drugs
- Solid organ or bone marrow transplant recipient
Show all 15Show fewer
- History of malignancy within the previous 5 years (except for BCC (basal cell carcinoma), SCC (squamous cell carcinoma) or in-situ cervical)
- History of myocardial infarction with past 12 months
- History of clinically significant cardiac dysrhythmia
- History of diabetes mellitus, bowel disease, tuberculosis
- Prior treatment for non-exudative AMD
- Intraocular, refractive or cataract surgery in the last 12 weeks
- Prior retinal surgery, vitrectomy, macular laser photocoagulation, external beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery (except cataract surgery)
- Receipt of gene transfer of cell transplant therapy in a prior clinical trial
- Participation in any other interventional clinical trial within the last 12 weeks
Trial locations and contacts
1 site in 1 country.
United States1 site
Wills Eye Institute
Philadelphia, Pennsylvania, 19107
About this study
Official title: Ph 2, Double-Masked, Randomized, Parallel, Sham Surgery/Placebo Control, Multi-Center Study to Evaluate Systemic IMT Regimens as Graft Rejection Prophylaxis Following Transplantation of hESC Derived RPE Cells in Patients With AMD
To evaluate the safety of 3 regimens of short-term, low-dose systemic IMT as rejection prophylaxis prior to and/or following transplant of MA09-hRPE cells.
Read the detailed description
This study will be a Phase 2, double-masked, randomized, parallel group, sham surgery/placebo control, multi-center trial. Subjects will be randomized in a 3:1 ratio to either the treatment or control group respectively. Subjects randomized to the treatment group will receive transplantation with 200,000 MA09-hRPE (human embryonic stem cell derived retinal pigmented epithelial)cells in one eye. Subjects randomized to the control group will have a sham surgery without transplantation of MA09-hRPE cells. The study eye must meet all eligibility criteria. If both eyes meet all eligibility criteria, then the study eye will be the eye with the worst Best Corrected Visual Acuity (BCVA) score at screening. If both eyes have identical BCVA scores, then the study eye will be chosen by the Investigator and the subject. There will be 3 cohorts, each with a different regimen of low-dose IMT [tacrolimus and mycophenolate mofetil (MMF)]. Subjects will be randomized to treatment or control within cohorts, defined by severity of BCVA in the study eye at Screening. Enrollment in Cohort 1 and 2 will be concurrent. Enrollment into Cohort 3 will begin once Cohort 2 is fully enrolled.
- Conditions
- Age-Related Macular Degeneration
- Being tested
- Sub-retinal transplantation of MA09-hRPE cells, tacrolimus and mycophenolate mofetil, Placebo tacrolimus and mycophenolate mofetil, Sham Surgery
- Medical subject headings
- Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sub-retinal transplantation of MA09-hRPE cellsBiological
transplantation
tacrolimus and mycophenolate mofetilDrug
Immunosuppressive Agents
Placebo tacrolimus and mycophenolate mofetilDrug
placebo
Sham SurgeryProcedure or surgery
Sham surgery
| Study group | Role | What they receive |
|---|---|---|
| Control group | Sham comparator | Sham surgery and placebo immunosuppressive therapy (IMT) Receives: Drug: Placebo tacrolimus and mycophenolate mofetil; Procedure: Sham Surgery |
| Active Group | Active comparator | Sub-retinal transplantation of MA09-hRPE cells and IMT Receives: Biological: Sub-retinal transplantation of MA09-hRPE cells; Drug: tacrolimus and mycophenolate mofetil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with evidence of graft failure or rejection.
Measured: 18 months
Secondary outcomes (3)
Change in area of geographic atrophy by Optical coherence tomography
Change from baseline to Week 78
Measured: 18 months
Change in area of geographic atrophy by Autofluorescence
Change from baseline to Week 78
Measured: 18 months
Change in average Best Corrected Visual Acuity (BCVA)
Change from baseline to Week 78
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2015 (estimated)
- Study start24 Aug 2015
- Primary completion5 May 2017
- Study completion5 May 2017
Why it stopped: Changes to the study design and the cell line
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 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
- NCT02563782
- Sponsor's protocol ID
- 7317-CL-0002
- Other IDs
- MA09-hRPE AMD 02 PORTRAY
- Acronym
- PORTRAY
- Status last verified
- Jul 2017
- First submitted
- 21 Sep 2015
- Last updated
- 26 Jul 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 26 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ph 2, Double-Masked, Randomized, Parallel, Sham Surgery/Placebo Control, Multi-Center Study to Evaluate Systemic IMT Regimens as Graft Rejection Prophylaxis Following Transplantation of hESC Derived RPE Cells in Patients With AMD
- ClinicalTrials.gov ID
- NCT02563782
- Acronym / sponsor ID
- PORTRAY / 7317-CL-0002
- Conditions
- Age-Related Macular Degeneration
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Changes to the study design and the cell line
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Astellas Institute for Regenerative MedicineIndustry (pharma, biotech, devices)
- Study start
- 24 Aug 2015 (actual)
- Primary completion
- 5 May 2017 (actual)
- Study completion
- 5 May 2017 (actual)
- First posted
- 30 Sep 2015 (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.
To evaluate the safety of 3 regimens of short-term, low-dose systemic IMT as rejection prophylaxis prior to and/or following transplant of MA09-hRPE cells.
Detailed description
This study will be a Phase 2, double-masked, randomized, parallel group, sham surgery/placebo control, multi-center trial. Subjects will be randomized in a 3:1 ratio to either the treatment or control group respectively. Subjects randomized to the treatment group will receive transplantation with 200,000 MA09-hRPE (human embryonic stem cell derived retinal pigmented epithelial)cells in one eye. Subjects randomized to the control group will have a sham surgery without transplantation of MA09-hRPE cells. The study eye must meet all eligibility criteria. If both eyes meet all eligibility criteria, then the study eye will be the eye with the worst Best Corrected Visual Acuity (BCVA) score at screening. If both eyes have identical BCVA scores, then the study eye will be chosen by the Investigator and the subject. There will be 3 cohorts, each with a different regimen of low-dose IMT [tacrolimus and mycophenolate mofetil (MMF)]. Subjects will be randomized to treatment or control within cohorts, defined by severity of BCVA in the study eye at Screening. Enrollment in Cohort 1 and 2 will be concurrent. Enrollment into Cohort 3 will begin once Cohort 2 is fully enrolled.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control group | Sham comparator | Drug: Placebo tacrolimus and mycophenolate mofetil, Procedure: Sham SurgerySham surgery and placebo immunosuppressive therapy (IMT) |
| Active Group | Active comparator | Biological: Sub-retinal transplantation of MA09-hRPE cells, Drug: tacrolimus and mycophenolate mofetilSub-retinal transplantation of MA09-hRPE cells and IMT |
Who may qualify
- Ages
- 50 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Geographic atrophy (GA) secondary to AMD with no evidence of prior to active choroidal neovascularization (CNV) in the study eye.
- BCVA in the study eye must be between 4 and 58 Early Treatment of Diabetic Retinopathy Study (EDTRS) letters (20/800 to 20/80; 0.025-0.25)
- Subjects must be willing to take IMT and willing to discontinue any medication that has a known strong interaction with tacrolimus or mycophenolate mofetil (MMF)
Key exclusion criteria (15 listed)
- Macular atrophy due to causes other than AMD
- Other sight-threatening ocular disease
- Current or prior history of optic neuropathy, retinal dystrophy, retinitis pigmentosa, chorioretinitis, vasoocclusive disease, retinal vascular disease or retinal degenerative disease OTHER than AMD
- History of uveitis
- History of allergic reaction to sulfa drugs
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 |
|---|---|---|
| Sub-retinal transplantation of MA09-hRPE cellstransplantation | Biological | Active Group |
| tacrolimus and mycophenolate mofetilImmunosuppressive Agents | Drug | Active Group |
| Placebo tacrolimus and mycophenolate mofetilplacebo | Drug | Control group |
| Sham SurgerySham surgery | Procedure or surgery | Control group |
Primary outcome
Number of subjects with evidence of graft failure or rejection.
Time frame: 18 months
3 secondary outcomes
Change in area of geographic atrophy by Optical coherence tomography
Time frame: 18 months
Change in area of geographic atrophy by Autofluorescence
Time frame: 18 months
Change in average Best Corrected Visual Acuity (BCVA)
Time frame: 18 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2017.
- Individual participant dataSharing planned: Undecided
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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.
Medical Director study director
Astellas Institute for Regenerative Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sub-retinal transplantation of MA09-hRPE cells and tacrolimus and mycophenolate mofetil in people with Age-Related Macular Degeneration, run by Astellas Institute for Regenerative Medicine at 1 site 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 3 inclusion and 15 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: withdrawn, as of the sponsor's last update on 26 Jul 2017. 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 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. Some participants in this study may receive a placebo. 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 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?
The study was withdrawn before enrolling participants, so there are no results.
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 subjects with evidence of graft failure or rejection. (time frame: 18 months). 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 26 Jul 2017.