Dry Age Related Macular DegenerationPhase 1/2NCT01674829
A PhaseI/IIa Study to Determine the Safety and Tolerability of MA09-hRPE Cells in Patients With Advanced Dry AMD
A Phase 1/2 study testing MA09-hRPE in people with Dry Age Related Macular Degeneration, run by CHABiotech CO., Ltd at 1 site in South Korea.
Trial summary
To evaluate the safety and tolerability of MA09-hRPE cellular therapy in patients with advanced dry AMD To evaluate the safety of the surgical procedures when used to implant MA09-hRPE cells To assess the number of hRPE cells to be transplanted in future studies To evaluate on an exploratory basis potential efficacy… Read more about this study
- Who is it for?
- Ages 55 years and older
- Conditions the study is for
- Dry Age Related Macular Degeneration
- What is being tested?
- MA09-hRPE
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteSouth Korea
- Study dates
- Sep 2012to Sep 2017
- 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 55 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult male or female over 55 years of age. Patient should be in sufficiently good health to reasonably expect survival for at least four years after treatment
- Clinical findings consistent with advanced dry AMD
- GA defined as attenuation or loss of RPE as observed by biomicroscopy, OCT, and FA.
- No evidence of current or prior choroidal neovascularization in the treated eye
- The visual acuity (BCVA) of the eye to receive the transplant found to be eligible for trial based on study protocol
- Electrophysiological findings consistent with advanced dry AMD.
Show all 9Show fewer
- Medically suitable to undergo vitrectomy and subretinal injection.
- Medically suitable for general anesthesia or waking sedation, if needed.
- Medically suitable for transplantation of an embryonic stem cell line
Who cannot join
- Presence of active or inactive CNV in the eye to be treated.
- Presence or history of retinal dystrophy, retinitis pigmentosa, chorioretinitis, central serious choroidopathy, diabetic retinopathy or other retinal vascular or degenerative disease other than ARMD.
- History of optic neuropathy.
- Macular atrophy due to causes other than AMD.
- Presence of glaucomatous optic neuropathy in the study eye, uncontrolled IOP, or use of two or more agents to control IOP (acetazolamide, beta blocker, alpha-1-agonist, prostaglandins, anhydrous carbonic inhibitors)
- Cataract of sufficient severity likely to necessitate surgical extraction within 1 year.
Show all 20Show fewer
- History of retinal detachment repair in the study eye.
- History of myocardial infarction in previous 12 months.
- History of diabetes mellitus.
- History of cognitive impairments or dementia which may impact the patients ability participate in the informed consent process and to appropriately complete evaluations.
- Any immunodeficiency.
- Any abnormalities in laboratory test result.
- Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.
- Current participation in any other clinical trial.
- Participation within previous 6 months in any clinical trial of a drug by ocular or systemic administration.
- Any other sight-threatening ocular disease.
- Ocular lens removal within previous 3 months.
- Ocular surgery in the study eye in the previous 3 months
- If female, pregnancy or lactation.
- Any other medical condition, which, in the Investigator's judgment, will interfere with the patient's ability to comply with the protocol, compromises patient safety, or interferes with the interpretation of the study results.
Trial locations and contacts
1 site in 1 country.
South Korea1 site
CHA Bundang Medical Center
Seongnam-si, Gyeonggi-do, 463-712,
About this study
Official title: A Phase I/IIa, Open-Label, Single-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (MA09-hRPE) Cells in Patients With Advanced Dry Age-related Macular Degeneration(AMD)
To evaluate the safety and tolerability of MA09-hRPE cellular therapy in patients with advanced dry AMD To evaluate the safety of the surgical procedures when used to implant MA09-hRPE cells To assess the number of hRPE cells to be transplanted in future studies To evaluate on an exploratory basis potential efficacy endpoints to be used in future studies of MA09-hRPE cellular therapy.
- Conditions
- Dry Age Related Macular Degeneration
- Being tested
- MA09-hRPE
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MA09-hRPEBiological
Transplantation of MA09-hRPE cells accoding to the assigned dose group
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Biological: MA09-hRPE Cellular therapy Cohort 1 Dose 1 Receives: Biological: MA09-hRPE |
| Cohort 2 | Experimental | Biological: MA09-hRPE Cellular therapy Cohort 2 Dose 2 Receives: Biological: MA09-hRPE |
| Cohort 3 | Experimental | Biological: MA09-hRPE Cellular therapy Cohort 3 Dose 3 Receives: Biological: MA09-hRPE |
| Cohort 4 | Experimental | Biological: MA09-hRPE Cellular therapy Cohort 4 Dose 4 Receives: Biological: MA09-hRPE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of hESC derived RPE cells
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe in the absence of:
- Any grade 2 (NCI grading system) or greater adverse event related to the cell product
- Any evidence that the cells are contaminated with an infectious agent
- Any evidence that the cells show tumorigenic potential
Measured: 12 months
Secondary outcomes (1)
exploratory evaluations for potential efficacy endpoints
Secondary endpoints will be evaluated as exploratory evaluations for potential efficacy endpoints.
- Change in the mean of BCVA
- Autofluorescense photography
- Reading speed
Evidence of successful engraftment will consist of:
- Structural evidence (OCT imaging, fluorescein angiography, slitlamp examination with fundus photography) that cells have been implanted in the correct location
- Electroretinographic evidence (mfERG) showing enhanced activity in the implant location
Measured: 12months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2012 (estimated)
- Study startSep 2012
- Primary completionSep 2017
- Study completionSep 2017
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dry 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
- Sequential
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- CHABiotech CO., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yeong je Seong, Principal investigator, CHA Bundang Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01674829
- Sponsor's protocol ID
- CHA CTP 1101
- Status last verified
- Apr 2024
- First submitted
- 22 Aug 2012
- Last updated
- 9 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/IIa, Open-Label, Single-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (MA09-hRPE) Cells in Patients With Advanced Dry Age-related Macular Degeneration(AMD)
- ClinicalTrials.gov ID
- NCT01674829
- Acronym / sponsor ID
- CHA CTP 1101
- Conditions
- Dry Age Related Macular Degeneration
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 10 participants (actual)
- Lead sponsor
- CHABiotech CO., LtdIndustry (pharma, biotech, devices)
- Study start
- Sep 2012 (actual)
- Primary completion
- Sep 2017 (actual)
- Study completion
- Sep 2017 (actual)
- First posted
- 29 Aug 2012 (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 and tolerability of MA09-hRPE cellular therapy in patients with advanced dry AMD To evaluate the safety of the surgical procedures when used to implant MA09-hRPE cells To assess the number of hRPE cells to be transplanted in future studies To evaluate on an exploratory basis potential efficacy endpoints to be used in future studies of MA09-hRPE cellular therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: MA09-hRPEBiological: MA09-hRPE Cellular therapy Cohort 1 Dose 1 |
| Cohort 2 | Experimental | Biological: MA09-hRPEBiological: MA09-hRPE Cellular therapy Cohort 2 Dose 2 |
| Cohort 3 | Experimental | Biological: MA09-hRPEBiological: MA09-hRPE Cellular therapy Cohort 3 Dose 3 |
| Cohort 4 | Experimental | Biological: MA09-hRPEBiological: MA09-hRPE Cellular therapy Cohort 4 Dose 4 |
Who may qualify
- Ages
- 55 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Adult male or female over 55 years of age. Patient should be in sufficiently good health to reasonably expect survival for at least four years after treatment
- Clinical findings consistent with advanced dry AMD
- GA defined as attenuation or loss of RPE as observed by biomicroscopy, OCT, and FA.
- No evidence of current or prior choroidal neovascularization in the treated eye
- The visual acuity (BCVA) of the eye to receive the transplant found to be eligible for trial based on study protocol
Key exclusion criteria (20 listed)
- Presence of active or inactive CNV in the eye to be treated.
- Presence or history of retinal dystrophy, retinitis pigmentosa, chorioretinitis, central serious choroidopathy, diabetic retinopathy or other retinal vascular or degenerative disease other than ARMD.
- History of optic neuropathy.
- Macular atrophy due to causes other than AMD.
- Presence of glaucomatous optic neuropathy in the study eye, uncontrolled IOP, or use of two or more agents to control IOP (acetazolamide, beta blocker, alpha-1-agonist, prostaglandins, anhydrous carbonic inhibitors)
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 |
|---|---|---|
| MA09-hRPETransplantation of MA09-hRPE cells accoding to the assigned dose group | Biological | Cohort 1, Cohort 2, Cohort 3, Cohort 4 |
Primary outcome
Safety of hESC derived RPE cells
Time frame: 12 months
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe in the absence of: 1. Any grade 2 (NCI grading system) or greater adverse event related to the cell product 2. Any evidence that the cells are contaminated with an infectious agent 3. Any evidence that the cells show tumorigenic potential
1 secondary outcome
exploratory evaluations for potential efficacy endpoints
Time frame: 12months
Results and safety
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2024.
Related trials for Dry Age Related Macular Degeneration
Active, not recruitingPhase 1/2
Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy
Geographic Atrophy
RecruitingPhase 1/2
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Dry Age-Related Macular Degeneration, Geographic Atrophy
Active, not recruitingPhase 1/2
Safety and Efficacy Study of OpRegen for Treatment of Advanced Dry-Form Age-Related Macular Degeneration
Age-Related Macular Degeneration
RecruitingObservational study
A Non-Interventional Study Observing Short-Term Progression in Geographic Atrophy (GA)
Geographic Atrophy Secondary to Age-related Macular Degeneration
Active, not recruitingPhase 3
A Study Investigating the Efficacy and Safety of Intravitreal (IVT) Injections of Vonaprument (Formerly ANX007) in Participants With Dry Age-Related Macular Degeneration (AMD) With Geographic Atrophy (GA)
Geographic Atrophy
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.
Yeong je Seong principal investigator
CHA Bundang Medical Center
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 MA09-hRPE in people with Dry Age Related Macular Degeneration, run by CHABiotech CO., Ltd at 1 site in South Korea.
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 9 inclusion and 20 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: stopped early, as of the sponsor's last update on 9 Apr 2024. 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 South Korea. 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 was stopped early (reason given: Sponsor decision). Results are not available in the source record.
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 of hESC derived RPE cells (time frame: 12 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 9 Apr 2024.