Age Related Macular DegenerationEarly Phase 1NCT02868424
Treatment of Age-related Macular Degeneration by Fetal Retinal Pigment Epithelial Cells Transplantation
A Early Phase 1 study testing fRPE cells in people with Age Related Macular Degeneration and Macular Degeneration, run by The First Affiliated Hospital with Nanjing Medical University at 1 site in China.
Trial summary
Early Phase I Study of the Safety and Preliminary Efficacy of Human Fetal Retinal Pigment Epithelial(fRPE) Cells Subretinal Transplantation in Age-Related Macular Degeneration(AMD) Patients Read more about this study
- Who is it for?
- Ages 50 to 90 yearsHealthy volunteers can take part
- Conditions the study is for
- Age Related Macular Degeneration, Macular Degeneration, AMD
- What is being tested?
- fRPE cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 13 Feb 2016to Dec 2018
- Phase
- Early 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 50 to 90 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Diagnosis of dry AMD or non-exudative AMD (geographical atrophy>250μm, involving central fovea of macular; or AMD with fibrous disciform scar and maintained stationary for at least 3 months)
- Experimental eye with best-corrected visual acuity (BCVA) of no more than 20/400 and control eye with BCVA of more than or equal to 20/400
- Patients are in good state
- Patient understand and sign the consent form.
Who cannot join
- Blood routine examination is abnormal (hemoglobin<10gm/dL; blood platelet count<100k/mm³; neutrophil count<1000/mm³)
- Blood biochemistry is abnormal (ALT/AST>1.5; creatinine>1.3mg/dL)
- Experimental eye has optic nerve atrophy caused by glaucoma
- Patients need cataract surgery within a year
- Patients have received cataract surgery and other ocular surgery in recent 3 months
- Experimental eye has retinal detachment, or has received retinal detachment surgery.
Show all 15Show fewer
- Patients with uveitis and other endophthalmitis
- Patients with other ocular disease affecting vision.
- Patients have participated in clinical study of ocular or systemic drug use in recent 6 months.
- Patients with medical history of malignant cancer (except resected basal cell carcinoma and squamous-cell carcinoma).
- Patients with medical history of myocardial infarction
- Patient with diabetes
- Patient with Parkinson disease or Alzheimer's disease
- Patients are under the treatment of immunosuppressive agent (except intermittent, low-dose corticosteroid treatment).
- Patients with other medical conditions that restricts the compliance and safety of patients, or affects experimental results.
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital with Nanjing Medical University
Nanjing, Jiangsu, 210029
About this study
Official title: Early Phase I Study of the Safety and Preliminary Efficacy of Human Fetal Retinal Pigment Epithelial(fRPE) Cells Subretinal Transplantation in Age-Related Macular Degeneration(AMD) Patients
Early Phase I Study of the Safety and Preliminary Efficacy of Human Fetal Retinal Pigment Epithelial(fRPE) Cells Subretinal Transplantation in Age-Related Macular Degeneration(AMD) Patients
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 fRPE cells in subjects with dry Age-Related Macular Degeneration and non-exudative AMD. The investigators will recruit and enroll 6 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 of no more than 20/400 will be determined as experimental eye, which will be divided into 3 groups and undergo subretinal injection of 3 different dosages of fRPE cells (100.000, 200,000 or 500,000) respectively, while the other one (BCVA of more than or equal to 20/400) as control eye, will not receive the surgery.
fRPE cells will be obtained from the fetuses aborted in the Department of Obstetrics and Gynecology in Jiangsu Province Hospital from 2015 to 2018. The obtained fRPE cells will meet specific inclusive/exclusive criteria and conform to Good manufacturing practices (GMP). fRPE 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 fRPE cell transplantation.
- Conditions
- Age Related Macular Degeneration, Macular Degeneration, AMD
- Being tested
- fRPE cells
- Medical subject headings
- Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
fRPE cellsDrug
Also called human fetal retinal pigment epithelial cells
fRPE cells transplantation directly into the subretinal space of one eye (experimental eye) in a single transplant procedure
| Study group | Role | What they receive |
|---|---|---|
| fRPE cells | Experimental | Subretinal transplantation of fRPE cells in experimental eye Receives: Drug: fRPE cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with adverse events
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
Measured: Two years following transplantation
Secondary outcomes (3)
Assessment of standardized ETDRS acuity testing
ETDRS stands for Early Treatment Diabetic Retinopathy Study which is designed to accurately measure visual acuity via ETDRS Scoring.
Measured: Two years following transplantation
Assessment of visual function changes
Assessments will include fluorescein angiography, fundus photography, spectral domain ocular coherence tomography (SD-OCT), microperimetry and multifocal electroretinography.
Measured: Two years following transplantation
Assessment of systemic condition
Items will include vital signs, electrocardiogram, blood and urine routine examination, tumor marker.
Measured: Two years following transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2016 (estimated)
- Study start13 Feb 2016
- Primary completionAug 2018 (estimated)
- Study completionDec 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Age Related Macular DegenerationStudies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital with Nanjing Medical UniversityThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Qinghuai Liu, M.D., Principal investigator, The First Affiliated Hospital with Nanjing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02868424
- Sponsor's protocol ID
- JSPH-fRPE-001
- Status last verified
- May 2017
- First submitted
- 8 Aug 2016
- Last updated
- 16 Jun 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Phase I Study of the Safety and Preliminary Efficacy of Human Fetal Retinal Pigment Epithelial(fRPE) Cells Subretinal Transplantation in Age-Related Macular Degeneration(AMD) Patients
- ClinicalTrials.gov ID
- NCT02868424
- Acronym / sponsor ID
- JSPH-fRPE-001
- Conditions
- Age Related Macular Degeneration, Macular Degeneration, AMD
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (actual)
- Lead sponsor
- The First Affiliated Hospital with Nanjing Medical UniversityUniversity, hospital or other
- Study start
- 13 Feb 2016 (actual)
- Primary completion
- Aug 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 16 Aug 2016 (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.
Early Phase I Study of the Safety and Preliminary Efficacy of Human Fetal Retinal Pigment Epithelial(fRPE) Cells Subretinal Transplantation in Age-Related Macular Degeneration(AMD) Patients
Detailed description
This study is a perspective, single-arm and open-labeled investigation of the safety and preliminary efficacy of unilateral subretinal transplantation of fRPE cells in subjects with dry Age-Related Macular Degeneration and non-exudative AMD. The investigators will recruit and enroll 6 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 of no more than 20/400 will be determined as experimental eye, which will be divided into 3 groups and undergo subretinal injection of 3 different dosages of fRPE cells (100.000, 200,000 or 500,000) respectively, while the other one (BCVA of more than or equal to 20/400) as control eye, will not receive the surgery.
fRPE cells will be obtained from the fetuses aborted in the Department of Obstetrics and Gynecology in Jiangsu Province Hospital from 2015 to 2018. The obtained fRPE cells will meet specific inclusive/exclusive criteria and conform to Good manufacturing practices (GMP). fRPE 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 fRPE cell transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| fRPE cells | Experimental | Drug: fRPE cellsSubretinal transplantation of fRPE cells in experimental eye |
Who may qualify
- Ages
- 50 to 90 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosis of dry AMD or non-exudative AMD (geographical atrophy>250μm, involving central fovea of macular; or AMD with fibrous disciform scar and maintained stationary for at least 3 months)
- Experimental eye with best-corrected visual acuity (BCVA) of no more than 20/400 and control eye with BCVA of more than or equal to 20/400
- Patients are in good state
- Patient understand and sign the consent form.
Key exclusion criteria (15 listed)
- Blood routine examination is abnormal (hemoglobin<10gm/dL; blood platelet count<100k/mm³; neutrophil count<1000/mm³)
- Blood biochemistry is abnormal (ALT/AST>1.5; creatinine>1.3mg/dL)
- Experimental eye has optic nerve atrophy caused by glaucoma
- Patients need cataract surgery within a year
- Patients have received cataract surgery and other ocular surgery in recent 3 months
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 |
|---|---|---|
| fRPE cellsAlso: human fetal retinal pigment epithelial cellsfRPE cells transplantation directly into the subretinal space of one eye (experimental eye) in a single transplant procedure | Drug | fRPE cells |
Primary outcome
Number of subjects with adverse events
Time frame: Two years following transplantation
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
3 secondary outcomes
Assessment of standardized ETDRS acuity testing
Time frame: Two years following transplantation
Assessment of visual function changes
Time frame: Two years following transplantation
Assessment of systemic condition
Time frame: Two years following transplantation
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jun 2017.
- Individual participant dataSharing planned: Yes
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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.
Qinghuai Liu, M.D. principal investigator
The First Affiliated Hospital with Nanjing Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing fRPE cells in people with Age Related Macular Degeneration and Macular Degeneration, run by The First Affiliated Hospital with Nanjing Medical University 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 4 inclusion and 15 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 16 Jun 2017. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 adverse events (time frame: Two years 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 16 Jun 2017.