Neovascular Age-related Macular DegenerationPhase 1/2NCT06141460
Safety and Efficacy of RRG001 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)
A Phase 1/2 study testing RRG001 in people with Neovascular Age-related Macular Degeneration, run by Shanghai Refreshgene Technology Co., Ltd. at 1 site in China.
Trial summary
A Phase I/IIa, Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Neovascular Age-related Macular Degeneration
- What is being tested?
- RRG001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteChina
- Study dates
- 12 Dec 2023to 31 Dec 2030
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects that are willing and able to follow study procedures.
- Patinets ≥50 years old.
- Clinically diagnosed with CNV secondary to nAMD.
- BCVA of the study eye is ≥5 letters and ≤73 letters(Snellen 20/800~20/40), as well as that of the other eye ≥19 letters(Snellen 20/400).
- Responding to anti-VEGF
Who cannot join
- Presence of any other eye diseases other than nAMD in study eye that would affect the treatment or confusing the interpretation of the study results , as judged by the investigator.
- CNV or macular edema in the study eye secondary to any causes other than AMD.
- Uncontrolled glaucoma.
- Uncontrolled hypertension despite medication.
- Pregnant or lactating women or individuals with reproductive potential who are unwilling to take effective contraception during the study period.
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
Tianjin Medical University Eye Hospital
Tianjin, Tianjin Municipality, 300384
Li Xiaorong, Professor, 8602223346434, xiaorli@163.com
About this study
Official title: A Phase I/IIa, Dose-escalation and Dose-extension Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration
A Phase I/IIa, Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration
Read the detailed description
This study adopts a multicenter, single-arm, open-label phase I/IIa seamless study design and comprises two stages, i.e., phase I 3+3 dose escalation study and a phase IIa dose expansion study. Four dose groups are preset for the phase I dose escalation study; 3-6 subjects will be enrolled for each dose group, and it is planned to enroll 12-24 subjects. The phase IIa dose expansion study will be conducted successively at the doses after safety assessments of dose 2 and/or dose 3 in phase I are completed. Twelve subjects are expected to be enrolled for each dose group, with a total of 24 subjects will be enrolled overall. The sponsor will assess whether or not to adjust the dose(s) and sample size for the exploration study based on the results in the phase I exploration study.
- Being tested
- RRG001
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RRG001Drug
Administered via Subretinal injection. Dosage form: injection solution.
| Study group | Role | What they receive |
|---|---|---|
| RRG001 Dose1 | Experimental | Frequency of administration: one time injection. Receives: Drug: RRG001 |
| RRG001 Dose2 | Experimental | Frequency of administration: one time injection. Receives: Drug: RRG001 |
| RRG001 Dose3 | Experimental | Frequency of administration: one time injection. Receives: Drug: RRG001 |
| RRG001 Dose4 | Experimental | Frequency of administration: one time injection. Receives: Drug: RRG001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability
incidence of AEs and SAEs
Measured: Week 52
Secondary outcomes (2)
Efficacy after RRG001 injection
change in best-corrected visual acuity (BCVA)；
Measured: Week 52
Efficacy after RRG001 injection
change in central retinal thickness (CRT)
Measured: Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2023
- Study start12 Dec 2023
- Primary completion15 Apr 2026 (estimated)
- Study completion31 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neovascular Age-related Macular DegenerationStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- dose escalation
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Refreshgene Technology Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06141460
- Sponsor's protocol ID
- RRG001-101
- Status last verified
- Nov 2024
- First submitted
- 10 Nov 2023
- Last updated
- 14 Nov 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/IIa, Dose-escalation and Dose-extension Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration
- ClinicalTrials.gov ID
- NCT06141460
- Acronym / sponsor ID
- RRG001-101
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Shanghai Refreshgene Technology Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 12 Dec 2023 (actual)
- Primary completion
- 15 Apr 2026 (estimated)
- Study completion
- 31 Dec 2030 (estimated)
- First posted
- 21 Nov 2023 (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.
A Phase I/IIa, Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration
Detailed description
This study adopts a multicenter, single-arm, open-label phase I/IIa seamless study design and comprises two stages, i.e., phase I 3+3 dose escalation study and a phase IIa dose expansion study. Four dose groups are preset for the phase I dose escalation study; 3-6 subjects will be enrolled for each dose group, and it is planned to enroll 12-24 subjects. The phase IIa dose expansion study will be conducted successively at the doses after safety assessments of dose 2 and/or dose 3 in phase I are completed. Twelve subjects are expected to be enrolled for each dose group, with a total of 24 subjects will be enrolled overall. The sponsor will assess whether or not to adjust the dose(s) and sample size for the exploration study based on the results in the phase I exploration study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- dose escalation
| Arm | Type | Receives |
|---|---|---|
| RRG001 Dose1 | Experimental | Drug: RRG001Frequency of administration: one time injection. |
| RRG001 Dose2 | Experimental | Drug: RRG001Frequency of administration: one time injection. |
| RRG001 Dose3 | Experimental | Drug: RRG001Frequency of administration: one time injection. |
| RRG001 Dose4 | Experimental | Drug: RRG001Frequency of administration: one time injection. |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Subjects that are willing and able to follow study procedures.
- Patinets ≥50 years old.
- Clinically diagnosed with CNV secondary to nAMD.
- BCVA of the study eye is ≥5 letters and ≤73 letters(Snellen 20/800~20/40), as well as that of the other eye ≥19 letters(Snellen 20/400).
- Responding to anti-VEGF
Key exclusion criteria (5 listed)
- Presence of any other eye diseases other than nAMD in study eye that would affect the treatment or confusing the interpretation of the study results , as judged by the investigator.
- CNV or macular edema in the study eye secondary to any causes other than AMD.
- Uncontrolled glaucoma.
- Uncontrolled hypertension despite medication.
- Pregnant or lactating women or individuals with reproductive potential who are unwilling to take effective contraception during the study period.
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 |
|---|---|---|
| RRG001Administered via Subretinal injection. Dosage form: injection solution. | Drug | RRG001 Dose1, RRG001 Dose2, RRG001 Dose3, RRG001 Dose4 |
Primary outcome
Safety and tolerability
Time frame: Week 52
incidence of AEs and SAEs
2 secondary outcomes
Efficacy after RRG001 injection
Time frame: Week 52
Efficacy after RRG001 injection
Time frame: Week 52
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 14 Nov 2024.
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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 1/2 study testing RRG001 in people with Neovascular Age-related Macular Degeneration, run by Shanghai Refreshgene Technology Co., Ltd. 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 5 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 14 Nov 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 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Xiaorong Li, Master (8602223346434, xiaorong.li@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability (time frame: Week 52). 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 14 Nov 2024.