Stargardt's Macular DystrophyPhase 1NCT01625559
Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)
A Phase 1 study testing MA09-hRPE in people with Stargardt's Macular Dystrophy, run by CHABiotech CO., Ltd at 1 site in South Korea.
Trial summary
The purpose of this study is to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Stargardt's Macular Dystrophy
- What is being tested?
- MA09-hRPE
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteSouth Korea
- Study dates
- Sep 2012to Apr 2015
- Phase
- Phase 1
- 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 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult male or female over 20 years of age.
- Clinical diagnosis of advanced SMD.
- The visual acuity of the eye to receive the transplant will be no better than hand movement.
- The visual acuity of the eye that is not to receive the transplant will be no better than 24 (20/320) Early Treatment of Diabetic Retinopathy Study (ETDRS) letters.
Who cannot join
- History of malignancy.
- History of myocardial infarction in previous 12 months.
- History of diabetes mellitus.
- Any immunodeficiency.
- Any current immunosuppressive therapy other than intermittent or low dose cortico steroids.
- Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.
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- 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.
- Any chronic ocular medications. Any history of retinal vascular disease (compromised blood-retinal barrier). Glaucoma. Uveitis or other intraocular inflammatory disease. Significant lens opacities or other media opacity. Ocular lens removal within previous 3 months
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, Open-Label, 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 Stargardt's Macular Dystrophy(SMD)
The purpose of this study is to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD
Read the detailed description
- to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD
- to evaluate the preliminary efficacy of MA09-hPRE in patients with SMD
- Conditions
- Stargardt's Macular Dystrophy
- 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
MA09-hRPE cells
| Study group | Role | What they receive |
|---|---|---|
| MA09-hRPE administration | Experimental | Biological: MA09-hRPE Cellular therapy Receives: Biological: MA09-hRPE |
What is being measured
The primary outcome is the main question the study is designed to answer.
safety and tolerance of transplantation
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated 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: 18 months
Secondary outcomes (1)
Evidence of successful engraftment
Evidence of successful engraftmentEvidence of successful engraftment will consist of:
Structural evidence (OCT imaging, fluorescein angiography, autofluorescense photography, slit-lamp examination with fundus photography) that cells have been implanted in the correct location Electroretinographic evidence (mfERG) showing enhanced activity in the implant location
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 posted21 Jun 2012 (estimated)
- Study startSep 2012
- Primary completionApr 2015
- Study completionApr 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- CHABiotech CO., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Wonkyung Song, Principal investigator, CHA Bundang Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01625559
- Sponsor's protocol ID
- CHA_CTP_0903
- Status last verified
- Feb 2024
- First submitted
- 18 Jun 2012
- Last updated
- 10 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Sung Y, Lee MJ, Choi J, Jung SY, Chong SY, Sung JH, Shim SH, Song WK. Long-term safety and tolerability of subretinal transplantation of embryonic stem cell-derived retinal pigment epithelium in Asian Stargardt disease patients. Br J Ophthalmol. 2021 Jun;105(6):829-837. doi: 10.1136/bjophthalmol-2020-316225. Epub 2020 Jul 29.
PubMed 32727729
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Open-Label, 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 Stargardt's Macular Dystrophy(SMD)
- ClinicalTrials.gov ID
- NCT01625559
- Acronym / sponsor ID
- CHA_CTP_0903
- Conditions
- Stargardt's Macular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- CHABiotech CO., LtdIndustry (pharma, biotech, devices)
- Study start
- Sep 2012
- Primary completion
- Apr 2015 (actual)
- Study completion
- Apr 2015 (actual)
- First posted
- 21 Jun 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.
The purpose of this study is to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD
Detailed description
- to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD
- to evaluate the preliminary efficacy of MA09-hPRE in patients with SMD
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MA09-hRPE administration | Experimental | Biological: MA09-hRPEBiological: MA09-hRPE Cellular therapy |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Adult male or female over 20 years of age.
- Clinical diagnosis of advanced SMD.
- The visual acuity of the eye to receive the transplant will be no better than hand movement.
- The visual acuity of the eye that is not to receive the transplant will be no better than 24 (20/320) Early Treatment of Diabetic Retinopathy Study (ETDRS) letters.
Key exclusion criteria (10 listed)
- History of malignancy.
- History of myocardial infarction in previous 12 months.
- History of diabetes mellitus.
- Any immunodeficiency.
- Any current immunosuppressive therapy other than intermittent or low dose cortico steroids.
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-hRPEMA09-hRPE cells | Biological | MA09-hRPE administration |
Primary outcome
safety and tolerance of transplantation
Time frame: 18 months
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated 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
Evidence of successful engraftment
Time frame: 18 months
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 Apr 2024.
- Derived from this study: PubMed 32727729Sung Y, Lee MJ, Choi J, Jung SY, Chong SY, Sung JH, Shim SH, Song WK. Long-term safety and tolerability of subretinal transplantation of embryonic stem cell-derived retinal pigment epithelium in Asian Stargardt disease patients. Br J Ophthalmol. 2021 Jun;105(6):829-837. doi: 10.1136/bjophthalmol-2020-316225. Epub 2020 Jul 29.
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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.
Wonkyung Song 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 study testing MA09-hRPE in people with Stargardt's Macular Dystrophy, 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 4 inclusion and 10 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 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 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.
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?
safety and tolerance of transplantation (time frame: 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01625559, which also lists 1 linked publication.
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 Apr 2024.