Leber Congenital Amaurosis, Retinitis PigmentosaEarly Phase 1NCT03566147
Treatment of RP and LCA by Primary RPE Transplantation
A Early Phase 1 study testing Human primary Retinal Pigment Epithelial (HuRPE) cells in people with Leber Congenital Amaurosis, Retinitis Pigmentosa, run by Eyecure Therapeutics Inc. at 1 site in China.
Trial summary
Early Phase I Study of the Safety and Preliminary Efficacy of Human primary Retinal Pigment Epithelial (HuRPE) Cells Subretinal Transplantation in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) Patients Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Leber Congenital Amaurosis, Retinitis Pigmentosa
- What is being tested?
- Human primary Retinal Pigment Epithelial (HuRPE) cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2018to 31 Mar 2020
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of RP or LCA;
- Experimental eye with best-corrected visual acuity (BCVA) of no more than 35 letters or visual field of less than 10 degree;
- 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
- Experimental eye has retinal detachment, or has received retinal detachment surgery.
- Patients with uveitis and other endophthalmitis
- Patients with other ocular disease affecting vision.
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- 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, 1 recruiting
- Recruiting
Beijing Tongren Hospital
Beijing
Jingxue Zhang, Dr, 18610772626, jingxuezh@163.com
About this study
Official title: Treatment of Retinitis Pigmentosa and Leber Congenital Amaurosis by Primary Retinal Pigment Epithelial Cells Transplantation
Early Phase I Study of the Safety and Preliminary Efficacy of Human primary Retinal Pigment Epithelial (HuRPE) Cells Subretinal Transplantation in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) Patients
- Being tested
- Human primary Retinal Pigment Epithelial (HuRPE) cells
- Medical subject headings
- Leber Congenital Amaurosis, Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human primary Retinal Pigment Epithelial (HuRPE) cellsBiological
HuRPE 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.
| Study group | Role | What they receive |
|---|---|---|
| low dose group | Experimental | 300,000 HuRPE cells Receives: Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells |
| middle dose group | Experimental | 500,000 HuRPE cells Receives: Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells |
| high dose group | Experimental | 1,000,000 HuRPE cells Receives: Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
best-corrected visual acuity (BCVA)
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 posted21 Jun 2018
- Study start1 Aug 2018 (estimated)
- Primary completion31 Dec 2019 (estimated)
- Study completion31 Mar 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leber Congenital Amaurosis, Retinitis PigmentosaStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eyecure Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Beijing Tongren Hospital
- Responsible party
- Sponsor
- Investigators
- Ningli Wang, Principal investigator, Beijing Tongren Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03566147
- Sponsor's protocol ID
- TR-RPE-RP/LCA
- Status last verified
- Jun 2018
- First submitted
- 12 Jun 2018
- Last updated
- 9 Aug 2018
- 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 9 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Retinitis Pigmentosa and Leber Congenital Amaurosis by Primary Retinal Pigment Epithelial Cells Transplantation
- ClinicalTrials.gov ID
- NCT03566147
- Acronym / sponsor ID
- TR-RPE-RP/LCA
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Eyecure Therapeutics Inc.Industry (pharma, biotech, devices)
- Collaborators
- Beijing Tongren Hospital
- Study start
- 1 Aug 2018 (estimated)
- Primary completion
- 31 Dec 2019 (estimated)
- Study completion
- 31 Mar 2020 (estimated)
- First posted
- 21 Jun 2018 (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.
Early Phase I Study of the Safety and Preliminary Efficacy of Human primary Retinal Pigment Epithelial (HuRPE) Cells Subretinal Transplantation in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) Patients
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| low dose group | Experimental | Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells300,000 HuRPE cells |
| middle dose group | Experimental | Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells500,000 HuRPE cells |
| high dose group | Experimental | Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells1,000,000 HuRPE cells |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of RP or LCA;
- Experimental eye with best-corrected visual acuity (BCVA) of no more than 35 letters or visual field of less than 10 degree;
- Patient understand and sign the consent form.
Key exclusion criteria (13 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
- Experimental eye has retinal detachment, or has received retinal detachment surgery.
- Patients with uveitis and other endophthalmitis
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 primary Retinal Pigment Epithelial (HuRPE) cellsHuRPE 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. | Biological | high dose group, low dose group, middle dose group |
Primary outcome
best-corrected visual acuity (BCVA)
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Aug 2018.
- 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.
Ningli Wang principal investigator
Beijing Tongren Hospital
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 Human primary Retinal Pigment Epithelial (HuRPE) cells in people with Leber Congenital Amaurosis, Retinitis Pigmentosa, run by Eyecure Therapeutics Inc. 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 3 inclusion and 13 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 9 Aug 2018. 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?
Contact Jingxue Zhang, Dr (86-18610772626, jingxuezh@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
best-corrected visual acuity (BCVA) (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 Aug 2018.