Macular DegenerationPhase 1NCT05445063
Safety and Efficacy of Autologous Transplantation of iPSC-RPE in the Treatment of Macular Degeneration
A Phase 1 study testing Autologous iPSC-derived RPE in people with Macular Degeneration, run by Beijing Tongren Hospital at 1 site in China.
Trial summary
This project intends to perform autologous transplantation of induced pluripotent stem cell-derived retinal pigment epithelium (iPSC-RPE). The clinical-grade RPE will be transplanted into subretinal space to treat refractory age-related macular degeneration. Read more about this study
- Who is it for?
- Ages 50 to 75 years
- Conditions the study is for
- Macular Degeneration
- What is being tested?
- Autologous iPSC-derived RPE
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- Aug 2022to Dec 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged 50-75 years;
- Clinical diagnosis is consistent with the definition of late dry AMD in the age-related eye disease study (AREDS), with one or more >250 um geographic atrophy in the fovea;
- Clinical diagnosis is wet AMD, but no obvious efficacy after conventional treatment;
- The BCVA of the target eye will be 0.05 to 0.3;
- Voluntary as test subjects, informed consent, regular follow-up on time.
Who cannot join
- One-eyed subjects;
- Macular atrophy caused by other diseases in addition to AMD;
- Suffer from retinitis pigmentosa, choroidal retinitis, central serous choroiditis, diabetic retinopathy, or other retinal vascular and degenerative diseases besides AMD;
- Lens opacities (affecting the central vision), glaucoma, uveitis, retinal detachment, optic neuropathy, and other ocular histories;
- Other intraocular surgery histories besides cataract surgery;
- Combined with severe systemic diseases, such as heart failure, liver disease, renal insufficiency, cor pulmonale, COPD in the previous 12 months;
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- Combined with severe infectious diseases, such as HIV, HBV, HCV, syphilis, tuberculosis, etc;
- Abnormal blood coagulation function or other laboratory tests;
- If female and of childbearing potential, pregnant, breastfeeding, or planning to become pregnant through the study;
- If male, refuse to use barrier and spermicide contraception during the study;
- Malignant tumor and history of malignancy;
- Any immune deficiency;
- Allergy to tacrolimus or other macrolides;
- Any immune deficiency;
- Use glucocorticoids, immunosuppressive drugs, or antipsychotic drugs in the previous 3 months;
- Use anticoagulant, or the platelet function is still not restored to normal after stopping antiplatelet drugs for 10 days;
- A history of addiction to alcoholism or prohibited drugs;
- Be participating in other intervention clinical trials or receiving other study medications;
- Poor compliance, difficulty to complete the study, or refusal to informed consent;
- Some other situations which might increase the risks of the subjects or interfere with clinical trials, such as mental disorders, cognitive dysfunction, etc.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Beijing Tongren Hospitol,Capital Medical University
Beijing, Beijing Municipality, 100730
Zi-Bing Jin, Doctor, +0086-(010)58265913, jinzibing@foxmail.com
About this study
Official title: Safety and Efficacy of Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium in the Treatment of Macular Degeneration
This project intends to perform autologous transplantation of induced pluripotent stem cell-derived retinal pigment epithelium (iPSC-RPE). The clinical-grade RPE will be transplanted into subretinal space to treat refractory age-related macular degeneration. The efficacy and safety of RPE transplants to treat macular degeneration will be monitored and analyzed with results from EDTRS, BCVA, OCT, ERG, microperimetry, and fluorescein angiography, before and after the treatment.
Read the detailed description
The investigators will generate iPSC lines from recruited participants and differentiate the iPSC into RPE. Autologous iPSC-derived RPE will be transplanted into participants eyes by subretinal injections. The safety and efficacy will be closely monitored and analyzed.
- Conditions
- Macular Degeneration
- Being tested
- Autologous iPSC-derived RPE
- Keywords
- Retinal disease, Macular degeneration, Stem Cells, RPE, Safety, Efficacy
- Medical subject headings
- Macular Degeneration, Retinal Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous iPSC-derived RPEBiological
Autologous transplantation of iPSC-derived RPE
| Study group | Role | What they receive |
|---|---|---|
| Participants receiving intervention | Experimental | Participants will receive autologous transplantation of induced pluripotent stem cell-derived retinal pigment epitheliums. Receives: Biological: Autologous iPSC-derived RPE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety measure
Safety will be assessed by Adverse Events (AEs) of special interest in regards to the investigational product. This will include obtaining information about Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, any AE that causes the subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer (irrespective of prior history), ectopic or proliferative cell growth (RPE or non-RPE) with adverse clinical consequence, unexpected, clinically significant AE possibly related to the cell transplant procedure or the investigational product (autologous iPSC-RPE), pregnancy in a female subject or the partner of a male subject and pregnancy outcome.
Measured: 12 months
Secondary outcomes (7)
Best Corrected Visual Acuity (BCVA)
Change in visual acuity will be measured by Early Treatment Diabetic Retinopathy Study (ETDRS) chart.
Measured: 12 months
Optical coherence tomography (OCT) imaging
The number of patients with serious retinal detachment, retinal hemorrhage, and cystoid macular edema, and the change in target treatment areas.
Measured: 12 months
Color and autofluorescence imaging
Change in target treatment areas.
Measured: 12 months
Fluorescein angiography
Change in target treatment areas.
Measured: 12 months
Fundus autofluorescence
Change in target treatment areas.
Measured: 12 months
Microperimetry
Exploratory evaluations for the change of retinal sensitivity from baseline in the region of interest.
Measured: 12 months
Electroretinography (ERG)
Exploratory evaluations for the change of retinal electrophysiology responses from baseline.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2022
- Study startAug 2022 (estimated)
- Primary completionJul 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Macular DegenerationStudies of the same condition, open and under way.
- More trials run by Beijing Tongren HospitalThe sponsor's other registered studies.
- 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
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Tongren Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05445063
- Sponsor's protocol ID
- 2021-214
- Status last verified
- Mar 2022
- First submitted
- 5 Jun 2022
- Last updated
- 6 Jul 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Detailed plan will be made based on institute regulations and funder policies. The investigators will adjust the plan case-by-case accordingly.
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium in the Treatment of Macular Degeneration
- ClinicalTrials.gov ID
- NCT05445063
- Acronym / sponsor ID
- 2021-214
- Conditions
- Macular Degeneration
- Keywords
- Retinal disease, Macular degeneration, Stem Cells, RPE, Safety, Efficacy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Beijing Tongren HospitalUniversity, hospital or other
- Study start
- Aug 2022 (estimated)
- Primary completion
- Jul 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 6 Jul 2022 (actual)
- 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 project intends to perform autologous transplantation of induced pluripotent stem cell-derived retinal pigment epithelium (iPSC-RPE). The clinical-grade RPE will be transplanted into subretinal space to treat refractory age-related macular degeneration. The efficacy and safety of RPE transplants to treat macular degeneration will be monitored and analyzed with results from EDTRS, BCVA, OCT, ERG, microperimetry, and fluorescein angiography, before and after the treatment.
Detailed description
The investigators will generate iPSC lines from recruited participants and differentiate the iPSC into RPE. Autologous iPSC-derived RPE will be transplanted into participants eyes by subretinal injections. The safety and efficacy will be closely monitored and analyzed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants receiving intervention | Experimental | Biological: Autologous iPSC-derived RPEParticipants will receive autologous transplantation of induced pluripotent stem cell-derived retinal pigment epitheliums. |
Who may qualify
- Ages
- 50 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Aged 50-75 years;
- Clinical diagnosis is consistent with the definition of late dry AMD in the age-related eye disease study (AREDS), with one or more >250 um geographic atrophy in the fovea;
- Clinical diagnosis is wet AMD, but no obvious efficacy after conventional treatment;
- The BCVA of the target eye will be 0.05 to 0.3;
- Voluntary as test subjects, informed consent, regular follow-up on time.
Key exclusion criteria (20 listed)
- One-eyed subjects;
- Macular atrophy caused by other diseases in addition to AMD;
- Suffer from retinitis pigmentosa, choroidal retinitis, central serous choroiditis, diabetic retinopathy, or other retinal vascular and degenerative diseases besides AMD;
- Lens opacities (affecting the central vision), glaucoma, uveitis, retinal detachment, optic neuropathy, and other ocular histories;
- Other intraocular surgery histories besides cataract surgery;
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 |
|---|---|---|
| Autologous iPSC-derived RPEAutologous transplantation of iPSC-derived RPE | Biological | Participants receiving intervention |
Primary outcome
Safety measure
Time frame: 12 months
Safety will be assessed by Adverse Events (AEs) of special interest in regards to the investigational product. This will include obtaining information about Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, any AE that causes the subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer (irrespective of prior history), ectopic or proliferative cell growth (RPE or non-RPE) with adverse clinical consequence, unexpected, clinically significant AE possibly related to the cell transplant procedure or the investigational product (autologous iPSC-RPE), pregnancy in a female subject or the partner of a male subject and pregnancy outcome.
7 secondary outcomes
Best Corrected Visual Acuity (BCVA)
Time frame: 12 months
Optical coherence tomography (OCT) imaging
Time frame: 12 months
Color and autofluorescence imaging
Time frame: 12 months
Fluorescein angiography
Time frame: 12 months
Fundus autofluorescence
Time frame: 12 months
Microperimetry
Time frame: 12 months
Electroretinography (ERG)
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2022.
- Individual participant dataSharing planned: No. Detailed plan will be made based on institute regulations and funder policies. The investigators will adjust the plan case-by-case accordingly.
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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.
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 Autologous iPSC-derived RPE in people with Macular Degeneration, run by Beijing Tongren Hospital at 1 site in China.
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 5 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: looking for participants now, as of the sponsor's last update on 6 Jul 2022. 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 China. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xiaohui Zhang (+0086-(010)58265915, zhangxh711@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety measure (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 6 Jul 2022.