Degenerative Myopia With Macular HoleNCT04212624
Clinical Research of Human Retinal Pigment Epithelial (HuRPE) Cell Injection on Atrophy of High Myopia Macular Area
A study testing Human retinal pigment epithelial (HuRPE) cell injection in people with Degenerative Myopia With Macular Hole, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine.
Trial summary
To evaluate the safety and tolerability of human retinal pigment epithelial (HuRPE) cell injection subretinal transplantation for atrophy of high myopia macular area, and to explore the maximum tolerated dose (MTD). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Degenerative Myopia With Macular Hole
- What is being tested?
- Human retinal pigment epithelial (HuRPE) cell injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- No sites listed yet
- Study dates
- Dec 2019to Dec 2021
- Phase
- Interventional
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A patient who is able (or with the assistance of his or her legal representative) to read and understand the informed consent form and is willing to sign a written informed consent;
- age ≥ 18 years old, male or female;
- myopic diopter ≥ -6D or visual axis length ≥ 26mm;
- study the best corrected visual acuity ≥ 5 letters, ≤ 60 letters (using ETDRS eye chart);
- According to the researcher's judgment, the patient's high myopia macular atrophy is the main reason for the patient's vision loss;
- If both eyes meet the inclusion criteria, the selected disease is more severe; the eyes are the same, and the right eye is the study eye.
Who cannot join
- The study eye has intravitreal hemorrhage within 2 months before the group visit;
- the study of the eye involved in the foveal scar, fibrosis, suggesting a serious irreversible vision loss;
- The study eye diagnosis is a pupillary afferent defect (APD);
- the study eye has significant refractive interstitial opacity, and moderate to severe cataract, may cause interference to OCT and fundus examination;
- any eye diagnosed as infectious blepharitis, keratitis, scleritis or conjunctivitis; or there are currently serious systemic infectious diseases;
- Uncontrollable glaucoma, defined as a patient who is adequately treated with medication, with an intraocular pressure still above 25 mmHg, or who has undergone previous or filtering surgery;
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- Study the eye with other diseases that affect vision;
- traumatic macular hole, idiopathic macular hole, combined with other parts of the retinal tear;
- single eye blind patients;
- The study eye is an aphakic eye (except for the intraocular lens);
- Any eye has a history of idiopathic or autoimmune-related uveitis;
- Any eye is diagnosed as an iris neovascularization;
- Received cataract surgery within 2 months prior to enrollment;
- Other intraocular surgery was performed within 3 months prior to the group visit;
- Have an allergic reaction or allergic history to sodium fluorescein, a history of allergies to protein products for therapeutic or diagnostic use, and allergies to two or more drugs and/or non-drug factors, or those with allergic diseases;
- Diabetes patients with uncontrolled blood glucose (fasting blood glucose ≥7.0mmol/L or 2h postprandial blood glucose ≥11.1mmol/L);
- There is a history of surgery within 1 month before screening, or there are currently unhealed wounds, ulcers, fractures, etc.;
- patients with uncontrolled hypertension, defined as the best treatment plan, a single measurement of systolic blood pressure > 180mmHg, two consecutive measurements of systolic blood pressure > 160mmHg or diastolic blood pressure > 100mmHg;
- History of myocardial infarction within 6 months prior to enrollment;
- There is active diffuse intravascular coagulation or obvious bleeding tendency before screening. Anticoagulation or anti-platelet aggregation drugs are used in addition to aspirin/non-steroidal anti-inflammatory drugs within 14 days before screening;
- Any clinical problem that cannot be controlled (such as severe mental, neurological, cardiovascular, respiratory, malignant, Parkinson's, Alzheimer's disease, etc.);
- Women of childbearing age were positive in the screening visit pregnancy test. Women who wish to breastfeed during the study. All women of childbearing age (male and female) did not agree to take effective contraceptive measures (such as intrauterine devices, birth control pills or condoms) during the entire study period and within 30 days of the end of the visit period;
- Patients who participated or are currently participating in other clinical studies within 30 days prior to screening;
- Hemoglobin <100 g/L, blood Plt≤100×109/L, neutrophil count <1.0×109/L, ALT/AST> upper limit of normal value (ULN)×1.5, Cr>1.3 mg/dL;
- Researchers believe that patients who are not eligible for inclusion, or other medical conditions, limit subject compliance, safety, or impact study results.
About this study
To evaluate the safety and tolerability of human retinal pigment epithelial (HuRPE) cell injection subretinal transplantation for atrophy of high myopia macular area, and to explore the maximum tolerated dose (MTD).
Read the detailed description
This study is a perspective, single-arm and open-labeled investigation of the safety and preliminary efficacy of unilateral subretinal transplantation of HuRPE cells in subjects with Macular degeneration caused by high myopia. The investigators will recruit and enroll 9 patients based on specific inclusive/exclusive criteria. Experimental and self-controlled eye will be determined based on best-corrected visual acuity (BCVA). The eye with BCVA between 5 and 60 ETDRS letters will be determined as experimental eye, which will be divided into 3 groups and undergo subretinal injection of 3 different dosages of HuRPE cells (300.000, 500,000 or 1,000,000) respectively, while the other one as control eye, will not receive the surgery.
HuRPE cells will be obtained from Eyecure Therapeutics, Inc.(Jiangsu) located in WuXi, Jiangsu Province. The obtained HuRPE cells will meet its quality standards and conform to Good manufacturing practices (GMP). HuRPE cells will be transplanted by a board-certified vitreoretinal surgeon, which will be administered into the subretinal space of experimental eye through a standard surgical approach.
Immunosuppressive agents will be administered orally to all subjects after transplantation. Dosage and time duration of immunosuppressive agents will be regulated strictly relying on the condition of immune rejection. Subjects will be monitored with ophthalmologic and systemic examinations frequently at regular post-transplant intervals after HuRPE cells transplantation.
- Conditions
- Degenerative Myopia With Macular Hole
- Being tested
- Human retinal pigment epithelial (HuRPE) cell injection
- Keywords
- Human retinal pigment epithelial (HuRPE) cell injection, High myopia, retinal degeneration
- Medical subject headings
- Retinal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human retinal pigment epithelial (HuRPE) cell injectionOther
Human retinal pigment epithelial cell injection Dosage form and specifications: injection; 600,000 cells / branch (200μl), 1 million cells / branch (200μl), 2 million cells / branch (200μl) Transportation and storage: sealed, stored at 2-8 ° C, valid for 6 hours
| Study group | Role | What they receive |
|---|---|---|
| test arm | Experimental | The 25G or 23G conjunctival seamless vitrectomy was performed on the anterior eye to remove the anterior-posterior direction and tangential direction of the rupture hole. The area outside the macular foveal lesion was selected, but 100 μl of HuRPE cell injection was injected within about 2 PD. In combination with the Medone syringe and the combined 35G or 37G needle, the silicone oil injection module is used for injection, and the injection is performed in an amount of 100 μl (depending on the cell concentration). The doses of the three dose groups from low to high were 300,000 cells, 500,000 cells, and 1 million cells, respectively, in a single injection. Receives: Other: Human retinal pigment epithelial (HuRPE) cell injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with Local and systemic adverse events
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
Measured: six months following transplantation
Secondary outcomes (3)
Mean change of Best-Corrected Visual Acuity (BCVA)
Best-corrected visual acuity (BCVA) will be assessed in a sitting position using manifest refraction and ETDRS-like visual acuity testing charts. higher scores means improvement of patient's visual acuity.
Measured: six months following transplantation
Evidence of successful engraftment
Structural evidence (OCT imaging, fluorescein angiography, slit-lamp examination with fundus photography) that HuRPE cells have been implanted in the correct location.
Measured: six months following transplantation
Change in NEI VFQ-25 Total Score
National eye institute 25-item visual function questionnaire (NEI VFQ-25) is a condition-specific measure which was designed to capture the specific impact of vision loss on health-related quality of life (HRQoL). The calculation for NEI VFQ-25 sub-scale scores and total score was performed according to the "NEI VFQ-25 Scoring Algorithm - August 2000". The NEI VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. All items are scored so that a high score represents better functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. In this format scores represent the achieved percentage of the total possible score, e.g. a score of 50 represents 50% of the highest possible score.
Measured: six months following transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Dec 2019
- Study startDec 2019 (estimated)
- Primary completionDec 2021 (estimated)
- Study completionDec 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Degenerative Myopia With Macular HoleStudies of the same condition, open and under way.
- More trials run by Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Eyecure Therapeutics Inc.
- Responsible party
- Principal investigator, Xiaodong Sun, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04212624
- Sponsor's protocol ID
- JR-HuRPE-HMML-02
- Status last verified
- Dec 2019
- First submitted
- 23 Oct 2019
- Last updated
- 27 Dec 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Research of Human Retinal Pigment Epithelial (HuRPE) Cell Injection on Atrophy of High Myopia Macular Area
- ClinicalTrials.gov ID
- NCT04212624
- Acronym / sponsor ID
- JR-HuRPE-HMML-02
- Conditions
- Degenerative Myopia With Macular Hole
- Keywords
- Human retinal pigment epithelial (HuRPE) cell injection, High myopia, retinal degeneration
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Collaborators
- Eyecure Therapeutics Inc.
- Study start
- Dec 2019 (estimated)
- Primary completion
- Dec 2021 (estimated)
- Study completion
- Dec 2021 (estimated)
- First posted
- 27 Dec 2019 (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.
To evaluate the safety and tolerability of human retinal pigment epithelial (HuRPE) cell injection subretinal transplantation for atrophy of high myopia macular area, and to explore the maximum tolerated dose (MTD).
Detailed description
This study is a perspective, single-arm and open-labeled investigation of the safety and preliminary efficacy of unilateral subretinal transplantation of HuRPE cells in subjects with Macular degeneration caused by high myopia. The investigators will recruit and enroll 9 patients based on specific inclusive/exclusive criteria. Experimental and self-controlled eye will be determined based on best-corrected visual acuity (BCVA). The eye with BCVA between 5 and 60 ETDRS letters will be determined as experimental eye, which will be divided into 3 groups and undergo subretinal injection of 3 different dosages of HuRPE cells (300.000, 500,000 or 1,000,000) respectively, while the other one as control eye, will not receive the surgery.
HuRPE cells will be obtained from Eyecure Therapeutics, Inc.(Jiangsu) located in WuXi, Jiangsu Province. The obtained HuRPE cells will meet its quality standards and conform to Good manufacturing practices (GMP). HuRPE cells will be transplanted by a board-certified vitreoretinal surgeon, which will be administered into the subretinal space of experimental eye through a standard surgical approach.
Immunosuppressive agents will be administered orally to all subjects after transplantation. Dosage and time duration of immunosuppressive agents will be regulated strictly relying on the condition of immune rejection. Subjects will be monitored with ophthalmologic and systemic examinations frequently at regular post-transplant intervals after HuRPE cells transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| test arm | Experimental | Other: Human retinal pigment epithelial (HuRPE) cell injectionThe 25G or 23G conjunctival seamless vitrectomy was performed on the anterior eye to remove the anterior-posterior direction and tangential direction of the rupture hole. The area outside the macular foveal lesion was selected, but 100 μl of HuRPE cell injection was injected within about 2 PD. In combination with the Medone syringe and the combined 35G or 37G needle, the silicone oil injection module is used for injection, and the injection is performed in an amount of 100 μl (depending on the cell concentration). The doses of the three dose groups from low to high were 300,000 cells, 500,000 cells, and 1 million cells, respectively, in a single injection. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- A patient who is able (or with the assistance of his or her legal representative) to read and understand the informed consent form and is willing to sign a written informed consent;
- age ≥ 18 years old, male or female;
- myopic diopter ≥ -6D or visual axis length ≥ 26mm;
- study the best corrected visual acuity ≥ 5 letters, ≤ 60 letters (using ETDRS eye chart);
- According to the researcher's judgment, the patient's high myopia macular atrophy is the main reason for the patient's vision loss;
Key exclusion criteria (25 listed)
- The study eye has intravitreal hemorrhage within 2 months before the group visit;
- the study of the eye involved in the foveal scar, fibrosis, suggesting a serious irreversible vision loss;
- The study eye diagnosis is a pupillary afferent defect (APD);
- the study eye has significant refractive interstitial opacity, and moderate to severe cataract, may cause interference to OCT and fundus examination;
- any eye diagnosed as infectious blepharitis, keratitis, scleritis or conjunctivitis; or there are currently serious systemic infectious diseases;
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 |
|---|---|---|
| Human retinal pigment epithelial (HuRPE) cell injectionHuman retinal pigment epithelial cell injection Dosage form and specifications: injection; 600,000 cells / branch (200μl), 1 million cells / branch (200μl), 2 million cells / branch (200μl) Transportation and storage: sealed, stored at 2-8 ° C, valid for 6 hours | Other | test arm |
Primary outcome
Number of subjects with Local and systemic adverse events
Time frame: six months following transplantation
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
3 secondary outcomes
Mean change of Best-Corrected Visual Acuity (BCVA)
Time frame: six months following transplantation
Evidence of successful engraftment
Time frame: six months following transplantation
Change in NEI VFQ-25 Total Score
Time frame: six months following transplantation
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Dec 2019.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Human retinal pigment epithelial (HuRPE) cell injection in people with Degenerative Myopia With Macular Hole, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine.
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 6 inclusion and 25 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: status not confirmed, as of the sponsor's last update on 27 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
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?
Number of subjects with Local and systemic adverse events (time frame: six months following transplantation). 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 27 Dec 2019.