Age Related Macular DegenerationPhase 1/2NCT02903576
Stem Cell Therapy for Outer Retinal Degenerations
A Phase 1/2 study testing injection of hESC-RPE in suspension and injection hESC-RPE seeded in a substrate in people with Age Related Macular Degeneration and Stargardt's Disease, run by Federal University of São Paulo at 1 site in Brazil.
Trial summary
This study is a Phase I/II , open label,non randomized, prospective study to determine the safety of human embryonic stem cell derived Retinal pigmented epithelium (hESC RPE) sub retinal injections versus hESC RPE seeded on a polymeric substrate implanted in the sub retinal space, Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Age Related Macular Degeneration, Stargardt's Disease, Exudative Age-related Macular Degeneration
- What is being tested?
- injection of hESC-RPE in suspension and injection hESC-RPE seeded in a substrate
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteBrazil
- Study dates
- Aug 2015to Jun 2019
- Phase
- Phase 1/2
- 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 18 to 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with AMD ( Dry , Wet after failure of treatment, disciform scars)
- Patients with Stargardt's Disease BCVA on the selected eye: worse than 20/200
Who cannot join
- Other ophthalmological diseases( Glaucoma, Diabetic Retinopathy, Previous retinal surgery, uveitis)
- Systemic diseases with contraindication for surgical procedures with local anaesthesia
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Stem Cell Derived Retinal Pigmented Epithelium Implantation in Patients With Outer Retinal Degenerations: Phase I/II Clinical Trial
This study is a Phase I/II , open label,non randomized, prospective study to determine the safety of human embryonic stem cell derived Retinal pigmented epithelium (hESC RPE) sub retinal injections versus hESC RPE seeded on a polymeric substrate implanted in the sub retinal space,
Read the detailed description
To determine whether the surgical implantation of a human embryonic stem cell-derived retinal pigmented epithelium (hESC-RPE) monolayer seeded onto a polymeric versus hESC-RPE injections into the sub retinal space is a safe procedure.
6 Patients will receive hESC-RPE cell injections (100000 cells) in the sub retinal space (2 Dry Age-related macular degeneration (AMD), 2 Wet AMD with disciform scar and 2 with Stargardt's disease).
Also 5 patients Dry AMD, 5 patients with Wet AMD with disciform scar and 5 patients with Stargardt's disease with receive a subretinal implantation of the hESC-RPE seeded in a monolayer in a polymeric substrate.
Patients will be enrolled sequentially, and after the procedure the patients will be followed for 1 year.
- Conditions
- Age Related Macular Degeneration, Stargardt's Disease, Exudative Age-related Macular Degeneration
- Being tested
- injection of hESC-RPE in suspension, injection hESC-RPE seeded in a substrate
- Medical subject headings
- Macular Degeneration, Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
injection of hESC-RPE in suspensionProcedure or surgery
Also called Human Embryonic Stem Cell - Retinal Pigmented Epithelium
The first six patients will receive a sub retinal injection of hESC-RPE in solution after a complete pars plana vitrectomy to access safety of the cell implant alone.
injection hESC-RPE seeded in a substrateProcedure or surgery
Also called Human Embryonic Stem Cell seeded in a polymeric substrate
Fifteen patients will receive a sub-retinal implantation of embryonic stem cell derived retinal pigmented epithelium seeded in a polymeric substrate to access safety of substrate seeded with RPE
| Study group | Role | What they receive |
|---|---|---|
| injection of hESC-RPE in suspension | Active comparator | 6 patients will receive cell suspension injections on the sub retinal space prior to the surgeries, to access safety Receives: Procedure: injection of hESC-RPE in suspension |
| injection hESC-RPE seeded in a substrate | Active comparator | 15 patients will receive a sub-retinal implantation of a polymeric scaffold seeded hesc- RPE in monolayer Receives: Procedure: injection hESC-RPE seeded in a substrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events of sub retinal implantation of stem cell derived retinal pigmented epithelium in the sub retinal space.
Incidence of surgical related side effects: Retinal detachment, Ocular inflammation, Increase in intraocular pressure, Infection( endophthalmitis), Loss of vision due to surgical, related complications
Measured: 1 year
Secondary outcomes (1)
Incidence of side effects related to the treatment itself( injection and implantation of sub retinal stem cell related RPE)
Inflammation/rejection Cell migration/differentiation Tumor formation Proliferative vitreoretinopathy/Retinal detachment Implant migration
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2016 (estimated)
- Study startAug 2015
- Primary completionJan 2019
- Study completionJun 2019
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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Federal University of São Paulo
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Rubens Belfort Jr., Federal University of São Paulo
- Investigators
- Rubens Belfort, MD, Study chair, Federal University of São Paulo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02903576
- Sponsor's protocol ID
- 12018712.5.0000.5505
- Status last verified
- Aug 2017
- First submitted
- 7 Mar 2016
- Last updated
- 22 Jul 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: scientific publication in a peer review journal
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Stem Cell Derived Retinal Pigmented Epithelium Implantation in Patients With Outer Retinal Degenerations: Phase I/II Clinical Trial
- ClinicalTrials.gov ID
- NCT02903576
- Acronym / sponsor ID
- 12018712.5.0000.5505
- Conditions
- Age Related Macular Degeneration, Stargardt's Disease, Exudative Age-related Macular Degeneration
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Federal University of São PauloUniversity, hospital or other
- Study start
- Aug 2015
- Primary completion
- Jan 2019 (actual)
- Study completion
- Jun 2019 (actual)
- First posted
- 16 Sep 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.
This study is a Phase I/II , open label,non randomized, prospective study to determine the safety of human embryonic stem cell derived Retinal pigmented epithelium (hESC RPE) sub retinal injections versus hESC RPE seeded on a polymeric substrate implanted in the sub retinal space,
Detailed description
To determine whether the surgical implantation of a human embryonic stem cell-derived retinal pigmented epithelium (hESC-RPE) monolayer seeded onto a polymeric versus hESC-RPE injections into the sub retinal space is a safe procedure.
6 Patients will receive hESC-RPE cell injections (100000 cells) in the sub retinal space (2 Dry Age-related macular degeneration (AMD), 2 Wet AMD with disciform scar and 2 with Stargardt's disease).
Also 5 patients Dry AMD, 5 patients with Wet AMD with disciform scar and 5 patients with Stargardt's disease with receive a subretinal implantation of the hESC-RPE seeded in a monolayer in a polymeric substrate.
Patients will be enrolled sequentially, and after the procedure the patients will be followed for 1 year.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| injection of hESC-RPE in suspension | Active comparator | Procedure: injection of hESC-RPE in suspension6 patients will receive cell suspension injections on the sub retinal space prior to the surgeries, to access safety |
| injection hESC-RPE seeded in a substrate | Active comparator | Procedure: injection hESC-RPE seeded in a substrate15 patients will receive a sub-retinal implantation of a polymeric scaffold seeded hesc- RPE in monolayer |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patients with AMD ( Dry , Wet after failure of treatment, disciform scars)
- Patients with Stargardt's Disease BCVA on the selected eye: worse than 20/200
Key exclusion criteria (2 listed)
- Other ophthalmological diseases( Glaucoma, Diabetic Retinopathy, Previous retinal surgery, uveitis)
- Systemic diseases with contraindication for surgical procedures with local anaesthesia
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 |
|---|---|---|
| injection of hESC-RPE in suspensionAlso: Human Embryonic Stem Cell - Retinal Pigmented EpitheliumThe first six patients will receive a sub retinal injection of hESC-RPE in solution after a complete pars plana vitrectomy to access safety of the cell implant alone. | Procedure or surgery | injection of hESC-RPE in suspension |
| injection hESC-RPE seeded in a substrateAlso: Human Embryonic Stem Cell seeded in a polymeric substrateFifteen patients will receive a sub-retinal implantation of embryonic stem cell derived retinal pigmented epithelium seeded in a polymeric substrate to access safety of substrate seeded with RPE | Procedure or surgery | injection hESC-RPE seeded in a substrate |
Primary outcome
Incidence of Treatment-Emergent Adverse Events of sub retinal implantation of stem cell derived retinal pigmented epithelium in the sub retinal space.
Time frame: 1 year
Incidence of surgical related side effects: Retinal detachment, Ocular inflammation, Increase in intraocular pressure, Infection( endophthalmitis), Loss of vision due to surgical, related complications
1 secondary outcome
Incidence of side effects related to the treatment itself( injection and implantation of sub retinal stem cell related RPE)
Time frame: 1 year
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 Jul 2020.
- Individual participant dataSharing planned: Yes. scientific publication in a peer review journal
Related trials for Age Related Macular Degeneration
Active, not recruitingPhase 4
A Study to Determine the Efficacy, Safety, and Durability of Faricimab in Participants With Neovascular Age-Related Macular Degeneration
Neovascular Age-related Macular Degeneration (nAMD)
Active, not recruitingObservational study
A Study to Learn How Avacincaptad Pegol (Izervay™) is Used in Clinical Practice in People Who Have Geographic Atrophy
Geographic Atrophy, Macular Degeneration
RecruitingPhase 3
Extension Study for the Port Delivery System With Ranibizumab (Portal)
Neovascular Age-Related Macular Degeneration
Not yet recruitingObservational study
A Case-Control Study Revealing Intraocular Microenvironment Characteristics in Patients With Ocular Diseases Based on Multi-Omics Analysis
Age- Related Macular Degeneration, Diabetic Retinopathy
Active, not recruitingPhase 2
Safety and Efficacy of ADVM-022 in Treatment-Experienced Patients With Neovascular Age-related Macular Degeneration [LUNA]
Neovascular Age-related Macular Degeneration
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.
Rubens Belfort, MD study chair
Federal University of São Paulo
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing injection of hESC-RPE in suspension and injection hESC-RPE seeded in a substrate in people with Age Related Macular Degeneration and Stargardt's Disease, run by Federal University of São Paulo at 1 site in Brazil.
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 2 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 Jul 2020. 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 Brazil. 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?
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 of Treatment-Emergent Adverse Events of sub retinal implantation of stem cell derived retinal pigmented epithelium in the sub retinal space. (time frame: 1 year). 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 22 Jul 2020.