Ignite Creation Date:
2024-05-06 @ 2:30 PM
Last Modification Date:
2024-10-26 @ 1:32 PM
Study NCT ID:
NCT04339764
Status:
RECRUITING
Last Update Posted:
2024-06-18
First Post:
2020-04-08
Brief Title:
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Sponsor:
National Eye Institute NEI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Phase IIIa Trial for Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Status:
RECRUITING
Status Verified Date:
2024-06-14
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Background
Age-related macular degeneration is a common eye disease in people over 50 The dry form of the disease can worsen into geographic atrophy causing blind spots Researchers want to learn if replacing older eye cells with younger ones can help treat this disease
Objective
To test the safety of putting cells inside the eye as a possible future treatment for dry age-related macular degeneration
Eligibility
People ages 55 and older who have geographic atrophy with loss of vision People who have had wet macular degeneration in study eye are NOT eligible
Design
Participants will be screened with
Medical history
Physical exam
Blood and urine tests
Eye exam
Eye photos
Fluorescein angiography An intravenous IV line is placed in an arm vein A dye is injected A camera takes pictures of the dye as it flows through the eyes blood vessels
Electroretinography An electrode is taped to participants forehead They sit in the dark After 30 minutes numbing eye drops and contact lenses are placed in their eyes They watch flashing lights
Tuberculosis test
Chest X-ray
Electrocardiography Sticky pads are placed on participants chest to record the hearts electrical activity
Participants will have at least 14 study visits over 5 and a half years They will repeat screening tests
Participants will have retinal pigment epithelium RPE transplantation surgery in one eye For this cells from participants blood are turned into RPE cells These cells are placed in their eye through a cut in their retina They will get dilating eye drops an IV line and anesthesia that may make them sleep A gas bubble will be put in their eye to help it heal Participants will receive immunosuppressive medications to avoid transplant rejection
Participants will be contacted yearly for up to 15 years
Age-related macular degeneration is a common eye disease in people over 50 The dry form of the disease can worsen into geographic atrophy causing blind spots Researchers want to learn if replacing older eye cells with younger ones can help treat this disease
Objective
To test the safety of putting cells inside the eye as a possible future treatment for dry age-related macular degeneration
Eligibility
People ages 55 and older who have geographic atrophy with loss of vision People who have had wet macular degeneration in study eye are NOT eligible
Design
Participants will be screened with
Medical history
Physical exam
Blood and urine tests
Eye exam
Eye photos
Fluorescein angiography An intravenous IV line is placed in an arm vein A dye is injected A camera takes pictures of the dye as it flows through the eyes blood vessels
Electroretinography An electrode is taped to participants forehead They sit in the dark After 30 minutes numbing eye drops and contact lenses are placed in their eyes They watch flashing lights
Tuberculosis test
Chest X-ray
Electrocardiography Sticky pads are placed on participants chest to record the hearts electrical activity
Participants will have at least 14 study visits over 5 and a half years They will repeat screening tests
Participants will have retinal pigment epithelium RPE transplantation surgery in one eye For this cells from participants blood are turned into RPE cells These cells are placed in their eye through a cut in their retina They will get dilating eye drops an IV line and anesthesia that may make them sleep A gas bubble will be put in their eye to help it heal Participants will receive immunosuppressive medications to avoid transplant rejection
Participants will be contacted yearly for up to 15 years
Detailed Description:
Age-related macular degeneration AMD is a leading cause of vision loss among the elderly There are limited treatment options for geographic atrophy GA the advanced stage of dry AMD in which cells of the neurosensory retina and associated retinal pigment epithelium RPE gradually degenerate and die Advances in stem cell biology allowing differentiation of pluripotent cells into RPE in vitro make feasible a cell-based strategy for potential treatment of AMD and recent methods for induced pluripotent stem cell iPSC generation offer promise of individualized autologous therapy Such an approach involves generation of iPSC from somatic cells taken from a patient with GA differentiation of iPSC into RPE grown as a monolayer on a thin scaffold in vitro and transplantation of the RPEscaffold construct into a small region in the subretinal space of the same patient with a goal of rescuing the overlying neurosensory retina from further degeneration
Objective To evaluate the safety and feasibility of subretinal transplantation of iPSC-derived RPE grown as a monolayer on a biodegradable poly lactic-co-glycolic acid PLGA scaffold as a potential autologous cell-based therapy for GA associated with AMD
Study Population Five participants will undergo RPE transplantation in one eye Eligible eyes will have GA best-corrected visual acuity BCVA between 20100 and inclusive of counting fingers CF and a fellow eye that has same or better BCVA If the National Eye Institute NEI Data and Safety Monitoring Committee DSMC gives clearance to proceed based on review of data from the first cohort a second cohort of up to seven additional participants with GA BCVA between 2080 and CF inclusive in the eye being considered for RPE transplantation and same or better visual acuity in the other eye may undergo the procedure to gather additional safety and potential efficacy data useful for planning future studies Up to 20 participants may be enrolled to allow for screening failures for participants withdrawing from the study prior to RPE transplantation or cases where the RPE cell transplantation does not occur due to intraoperative surgical considerations
Design In this phase IIIa prospective single-arm multi-center clinical trial participants will undergo subretinal transplantation of autologous iPSC-derived RPE in one eye and will be followed for five years after surgery
Outcome Measures The primary outcome measure is the safety of RPEPLGA transplantation as determined by assessment of visual acuity change and summary of adverse events at 12 months after RPEPLGA transplantation Secondary outcome measures include visual acuity change and adverse event reporting at 24 and 60 months and changes in the following at 12 24 and 60 months as compared with baseline assessed in the transplanted region and compared where applicable with other areas in the macula andor with corresponding regions in the fellow eye retinal sensitivity and fixation parameters assessed by microperimetry multifocal electroretinography mfERG responses macular structure on cross-sectional and en face imaging by optical coherence tomography OCT macular features on color single-wavelength reflectance and fundus autofluorescence FAF photography and fluorescein angiography FA Some NEI and JHU participants may undergo imaging of photoreceptorRPE features using adaptive-optics-assisted macular imaging under a separate NEI protocol eg 15-EI-0020
Objective To evaluate the safety and feasibility of subretinal transplantation of iPSC-derived RPE grown as a monolayer on a biodegradable poly lactic-co-glycolic acid PLGA scaffold as a potential autologous cell-based therapy for GA associated with AMD
Study Population Five participants will undergo RPE transplantation in one eye Eligible eyes will have GA best-corrected visual acuity BCVA between 20100 and inclusive of counting fingers CF and a fellow eye that has same or better BCVA If the National Eye Institute NEI Data and Safety Monitoring Committee DSMC gives clearance to proceed based on review of data from the first cohort a second cohort of up to seven additional participants with GA BCVA between 2080 and CF inclusive in the eye being considered for RPE transplantation and same or better visual acuity in the other eye may undergo the procedure to gather additional safety and potential efficacy data useful for planning future studies Up to 20 participants may be enrolled to allow for screening failures for participants withdrawing from the study prior to RPE transplantation or cases where the RPE cell transplantation does not occur due to intraoperative surgical considerations
Design In this phase IIIa prospective single-arm multi-center clinical trial participants will undergo subretinal transplantation of autologous iPSC-derived RPE in one eye and will be followed for five years after surgery
Outcome Measures The primary outcome measure is the safety of RPEPLGA transplantation as determined by assessment of visual acuity change and summary of adverse events at 12 months after RPEPLGA transplantation Secondary outcome measures include visual acuity change and adverse event reporting at 24 and 60 months and changes in the following at 12 24 and 60 months as compared with baseline assessed in the transplanted region and compared where applicable with other areas in the macula andor with corresponding regions in the fellow eye retinal sensitivity and fixation parameters assessed by microperimetry multifocal electroretinography mfERG responses macular structure on cross-sectional and en face imaging by optical coherence tomography OCT macular features on color single-wavelength reflectance and fundus autofluorescence FAF photography and fluorescein angiography FA Some NEI and JHU participants may undergo imaging of photoreceptorRPE features using adaptive-optics-assisted macular imaging under a separate NEI protocol eg 15-EI-0020
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 20-EI-0052 | None | None | None |