To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)NCT06024057
An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA
A study testing LX101 in people with To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA), run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine.
Trial summary
To evaluate the scaling clinical trail of AAV2-RPE65 gene therapy agent (LX101) in patients with congenital amaurosis (LCA). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)
- What is being tested?
- LX101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- No sites listed yet
- Study dates
- 1 Sep 2023to 30 Sep 2038
- Phase
- Interventional
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1) Participants in the main study "Exploratory clinical study evaluating AAV2-RPE65 gene therapy formulation (LX101) in patients with congenital amaurosis (LCA) 2) Willingness to participate in the extended study, subject and/or their guardian signing a written informed consent and complying with a long-term follow-up protocol
Who cannot join
- Have any of the following eye conditions:
- Subjects with SAE in the "Exploratory clinical Study Evaluating AAV2-RPE65 gene therapy agent (LX101) in patients with congenital Amaurosis (LCA)
- pre-existing eye conditions (such as glaucoma, cornea, or severe lens opacity) that the investigator determines may interfere with planned surgery or with interpretation of the study endpoint;
- Screening subjects who had undergone any internal eye surgery in the six months prior to the visit;
- Inability or unwillingness to meet the requirements of the study;
- Have any of the following systemic conditions:
Show all 9Show fewer
- 6) Known allergy to the drug planned for use in the study; 7) Have a history of surgical operations within 1 month before enrollment, and/or present unhealed wounds, ulcers, fractures, etc.; Present infectious disease requiring oral, intramuscular or intravenous administration; 8) Baseline laboratory values for complex systemic diseases or clinically significant abnormalities; 9) Complex systemic diseases include diseases in which the disease itself or the treatment of the disease can alter eye function. For example, the treatment of malignant tumors can affect central nervous system function (e.g., orbital radiation therapy; Leukemia with central/optic nerve involvement); 10) Subjects with a history of immune-damaging diseases due to the possibility of opportunistic infection (e.g., CMV retinitis); Subjects with diabetes or sickle cell disease will be excluded if they have any manifestations of advanced retinopathy, such as macular edema or proliferative changes. Subjects with juvenile rheumatoid arthritis have an increased risk of infection due to poor postoperative wound healing; 11) Participants who cannot discontinue Viagra (Sildenafil) or related compounds used to treat erectile dysfunction during the study period; 12) Subjects who cannot discontinue hydroxychloroquine, Chloroquine, meralide, or any related retinal toxic compound during the study; 13) Any other conditions in which the potential subject is not allowed to complete a follow-up examination during the study, and the investigator believes that there are conditions that make the potential subject unfit for the study.
- Fertile subjects with any of the following conditions:
- 14) Those who do not use effective contraceptive measures; 15) Pregnant and lactating women.
About this study
Official title: An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy (LX101) in Patients With Leber's Congenital Amaurosis (LCA)
To evaluate the scaling clinical trail of AAV2-RPE65 gene therapy agent (LX101) in patients with congenital amaurosis (LCA).
Read the detailed description
This study evaluated the overall safety and initial efficacy of RPE65 mutant congenital amaurosis (RPE65-LCA) in subjects treated with a single, subretinal injection of LX101 (containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65) in the second eye 1 year after treatment.
- Being tested
- LX101
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LX101Drug
LX101 (Containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65 encoding human RPE65 gene).Specification: 0.2mL/bottle.Route of administration: subretinal injection, injection volume 0.3 mL/ eye
| Study group | Role | What they receive |
|---|---|---|
| LX101 | Experimental | Not described Receives: Drug: LX101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Local and systemic safety assessment
Eye and non-eye adverse events
Measured: 52（+4）weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2023
- Study start1 Sep 2023 (estimated)
- Primary completion30 Sep 2028 (estimated)
- Study completion30 Sep 2038 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)Studies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiaodong Sun, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06024057
- Sponsor's protocol ID
- SHGH-LX101-EX
- Status last verified
- Aug 2023
- First submitted
- 29 Aug 2023
- Last updated
- 5 Sep 2023
- Results posted
- 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 5 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy (LX101) in Patients With Leber's Congenital Amaurosis (LCA)
- ClinicalTrials.gov ID
- NCT06024057
- Acronym / sponsor ID
- SHGH-LX101-EX
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Study start
- 1 Sep 2023 (estimated)
- Primary completion
- 30 Sep 2028 (estimated)
- Study completion
- 30 Sep 2038 (estimated)
- First posted
- 5 Sep 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.
To evaluate the scaling clinical trail of AAV2-RPE65 gene therapy agent (LX101) in patients with congenital amaurosis (LCA).
Detailed description
This study evaluated the overall safety and initial efficacy of RPE65 mutant congenital amaurosis (RPE65-LCA) in subjects treated with a single, subretinal injection of LX101 (containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65) in the second eye 1 year after treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LX101 | Experimental | Drug: LX101 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- 1) Participants in the main study "Exploratory clinical study evaluating AAV2-RPE65 gene therapy formulation (LX101) in patients with congenital amaurosis (LCA) 2) Willingness to participate in the extended study, subject and/or their guardian signing a written informed consent and complying with a long-term follow-up protocol
Key exclusion criteria (9 listed)
- Have any of the following eye conditions:
- 6) Known allergy to the drug planned for use in the study; 7) Have a history of surgical operations within 1 month before enrollment, and/or present unhealed wounds, ulcers, fractures, etc.; Present infectious disease requiring oral, intramuscular or intravenous administration; 8) Baseline laboratory values for complex systemic diseases or clinically significant abnormalities; 9) Complex systemic diseases include diseases in which the disease itself or the treatment of the disease can alter eye function. For example, the treatment of malignant tumors can affect central nervous system function (e.g., orbital radiation therapy; Leukemia with central/optic nerve involvement); 10) Subjects with a history of immune-damaging diseases due to the possibility of opportunistic infection (e.g., CMV retinitis); Subjects with diabetes or sickle cell disease will be excluded if they have any manifestations of advanced retinopathy, such as macular edema or proliferative changes. Subjects with juvenile rheumatoid arthritis have an increased risk of infection due to poor postoperative wound healing; 11) Participants who cannot discontinue Viagra (Sildenafil) or related compounds used to treat erectile dysfunction during the study period; 12) Subjects who cannot discontinue hydroxychloroquine, Chloroquine, meralide, or any related retinal toxic compound during the study; 13) Any other conditions in which the potential subject is not allowed to complete a follow-up examination during the study, and the investigator believes that there are conditions that make the potential subject unfit for the study.
- 14) Those who do not use effective contraceptive measures; 15) Pregnant and lactating women.
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 |
|---|---|---|
| LX101LX101 (Containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65 encoding human RPE65 gene).Specification: 0.2mL/bottle.Route of administration: subretinal injection, injection volume 0.3 mL/ eye | Drug | LX101 |
Primary outcome
Local and systemic safety assessment
Time frame: 52（+4）weeks
Eye and non-eye adverse events
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 5 Sep 2023.
- Individual participant dataSharing planned: No
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.
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 LX101 in people with To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA), 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 1 inclusion and 9 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: opening soon, as of the sponsor's last update on 5 Sep 2023. 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?
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?
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?
Local and systemic safety assessment (time frame: 52（+4）weeks). 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 5 Sep 2023; AzTrials last refreshed its copy on 31 Oct 2026.