Retinitis PigmentosaPhase 1NCT06936787
An Open-label, Dose-ascending Study of IGT001 for Retinitis Pigmentosa
A Phase 1 study testing IGT001 in people with Retinitis Pigmentosa, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
Trial summary
This is an investigator-initiated, prospective, single-center, open-label, single ascending dose clinical study. The study aims to evaluate the safety, tolerability, and potential efficacy of intravitreal injection of IGT001 in patients with retinitis pigmentosa across different dose groups. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- IGT001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteChina
- Study dates
- 20 Apr 2025to 20 Dec 2027
- Phase
- Phase 1
- 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
- Adults aged ≥ 18 years at the time of signing the informed consent form, regardless of gender
- Clinical diagnosis of retinitis pigmentosa based on medical history and specialized examinations
- Willing to undergo genetic mutation testing, with results confirming known genetic mutations associated with hereditary retinal degeneration and related diseases
- Willing to provide blood samples for HLA typing
- Blood cell counts (hematocrit, hemoglobin, white blood cells, platelets) within the normal range, or outside the normal range but deemed clinically insignificant by the investigator
- Negative screening results for infectious diseases (hepatitis B, hepatitis C, and HIV), with no history of drug abuse (confirmed by negative urine test)
- Female participants of childbearing potential (defined as those who have not undergone surgical sterilization or have not been postmenopausal for at least one year) must undergo a urine pregnancy test at the time of admission (before injection), and the result must be negative.Female or male participants of childbearing potential must use medically approved contraceptive measures, such as abstinence, intrauterine devices (IUDs), or dual barrier methods (e.g., condom plus diaphragm), for at least 30 days before treatment and at least one year after treatment
Who cannot join
- As determined by the investigator, the study eye has concurrent conditions in addition to retinitis pigmentosa that:
- Affect central vision, or
- Increase the safety risk for the participant, or
- Impact efficacy or safety assessments or data collection, or
- Require surgical or medical intervention due to ocular diseases or history, such as retinal detachment or a history of retinal detachment, wet age-related macular degeneration, diabetic macular edema, retinal vein occlusion, uveitis, optic nerve disease, a history of glaucoma or ocular hypertension, severe or grade 3 non-proliferative diabetic retinopathy, proliferative diabetic retinopathy, significant media opacity, or an inability to achieve adequate pupil dilation
- The study eye cannot rule out the differential diagnosis of pseudoretinitis pigmentosa or cancer-associated retinopathies.
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- History of malignant tumors (except for patients in remission for more than five years and those with basal cell carcinoma of the skin), end-stage major organ diseases (including heart failure, severe arrhythmias, stroke or transient ischemic attack, immunosuppressive or autoimmune conditions, severe mental disorders, epilepsy, chronic obstructive pulmonary disease, renal failure, or any chronic systemic disease requiring continuous systemic corticosteroids, anticoagulants, or immunosuppressive therapy)
- History of glaucoma in either eye or a glaucoma diagnosis at screening
- History of ocular hypertension in either eye or a diagnosis of ocular hypertension at screening
- Intraocular pressure (IOP) > 21 mmHg in either eye or current use of any intraocular pressure-lowering medications
- History of allergy, intolerance, or adverse reactions to any procedures involved in the study, including but not limited to a history of allergy/adverse reactions to contrast agents, needle phobia, or hemophobia
- History of ocular surface damage or drug-induced toxicity from medications such as deferoxamine, chloroquine/hydroxychloroquine (Plaquenil), tamoxifen, phenothiazines, or ethambutol
- History of allergy or adverse reactions to products containing dimethyl sulfoxide (DMSO)
- History of allergy to gentamicin
- Inability or unwillingness to comply with any study-related procedures, including genetic testing, blood sample collection, clinical examinations, or procedures requiring pupil dilation, topical anesthesia, or contrast agent injection
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
Shanghai General Hospital, Shanghai Jiao Tong University
Shanghai, 200080
Xiaodong Sun, +86-02163240090 ext. 6822
About this study
Official title: A Prospective, Single-center, Open-label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreal Injection of IGT001 in Adult Patients With Retinitis Pigmentosa
This is an investigator-initiated, prospective, single-center, open-label, single ascending dose clinical study. The study aims to evaluate the safety, tolerability, and potential efficacy of intravitreal injection of IGT001 in patients with retinitis pigmentosa across different dose groups.
IGT001 is administered as a single intravitreal injection in the study eye under local anesthesia. Participants will be discharged on the day of treatment, provided that their visual acuity and intraocular pressure are close to baseline levels, and no clinically significant abnormalities in vital signs are observed compared to pre-injection values. The decision for discharge will be determined by the investigator's assessment.
Read the detailed description
The study's dose groups are divided based on concentration into four cohorts: the low-concentration cohort (0.5 × 10⁶ hRP-50 µL), the medium-concentration cohort (1.0 × 10⁶ hRP-50 µL), the high-concentration cohort (2.0 × 10⁶ hRP-50 µL), and the target-concentration cohort.
Participants in the four cohorts will undergo a one-year follow-up as specified in the protocol. They will also be encouraged to participate in an additional one-year follow-up study to assess the long-term safety and tolerability of IGT001 after completing the main study.
- Conditions
- Retinitis Pigmentosa
- Being tested
- IGT001
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IGT001Drug
Study drugs
| Study group | Role | What they receive |
|---|---|---|
| IGT001 | Experimental | Not described Receives: Drug: IGT001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
AE or SAE
The incidence of adverse events (AE) or serious adverse events (SAE)
Measured: 1 year
The incidence of DLT
DLT
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2025
- Study start20 Apr 2025 (estimated)
- Primary completion20 Dec 2025 (estimated)
- Study completion20 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- 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
- NCT06936787
- Sponsor's protocol ID
- INGEL-01
- Status last verified
- Apr 2025
- First submitted
- 8 Apr 2025
- Last updated
- 27 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- 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 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Single-center, Open-label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreal Injection of IGT001 in Adult Patients With Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT06936787
- Acronym / sponsor ID
- INGEL-01
- Conditions
- Retinitis Pigmentosa
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Study start
- 20 Apr 2025 (estimated)
- Primary completion
- 20 Dec 2025 (estimated)
- Study completion
- 20 Dec 2027 (estimated)
- First posted
- 20 Apr 2025 (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.
This is an investigator-initiated, prospective, single-center, open-label, single ascending dose clinical study. The study aims to evaluate the safety, tolerability, and potential efficacy of intravitreal injection of IGT001 in patients with retinitis pigmentosa across different dose groups.
IGT001 is administered as a single intravitreal injection in the study eye under local anesthesia. Participants will be discharged on the day of treatment, provided that their visual acuity and intraocular pressure are close to baseline levels, and no clinically significant abnormalities in vital signs are observed compared to pre-injection values. The decision for discharge will be determined by the investigator's assessment.
Detailed description
The study's dose groups are divided based on concentration into four cohorts: the low-concentration cohort (0.5 × 10⁶ hRP-50 µL), the medium-concentration cohort (1.0 × 10⁶ hRP-50 µL), the high-concentration cohort (2.0 × 10⁶ hRP-50 µL), and the target-concentration cohort.
Participants in the four cohorts will undergo a one-year follow-up as specified in the protocol. They will also be encouraged to participate in an additional one-year follow-up study to assess the long-term safety and tolerability of IGT001 after completing the main study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IGT001 | Experimental | Drug: IGT001 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Adults aged ≥ 18 years at the time of signing the informed consent form, regardless of gender
- Clinical diagnosis of retinitis pigmentosa based on medical history and specialized examinations
- Willing to undergo genetic mutation testing, with results confirming known genetic mutations associated with hereditary retinal degeneration and related diseases
- Willing to provide blood samples for HLA typing
- Blood cell counts (hematocrit, hemoglobin, white blood cells, platelets) within the normal range, or outside the normal range but deemed clinically insignificant by the investigator
Key exclusion criteria (15 listed)
- As determined by the investigator, the study eye has concurrent conditions in addition to retinitis pigmentosa that:
- The study eye cannot rule out the differential diagnosis of pseudoretinitis pigmentosa or cancer-associated retinopathies.
- History of malignant tumors (except for patients in remission for more than five years and those with basal cell carcinoma of the skin), end-stage major organ diseases (including heart failure, severe arrhythmias, stroke or transient ischemic attack, immunosuppressive or autoimmune conditions, severe mental disorders, epilepsy, chronic obstructive pulmonary disease, renal failure, or any chronic systemic disease requiring continuous systemic corticosteroids, anticoagulants, or immunosuppressive therapy)
- History of glaucoma in either eye or a glaucoma diagnosis at screening
- History of ocular hypertension in either eye or a diagnosis of ocular hypertension at screening
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 |
|---|---|---|
| IGT001Study drugs | Drug | IGT001 |
Primary outcome
AE or SAE
Time frame: 1 year
The incidence of adverse events (AE) or serious adverse events (SAE)
The incidence of DLT
Time frame: 28 days
DLT
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 27 Apr 2025.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing IGT001 in people with Retinitis Pigmentosa, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine 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 7 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: looking for participants now, as of the sponsor's last update on 27 Apr 2025. 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?
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.
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 Xiao Dong Sun, PhD (+86 18017317575, drsunxiaodong@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
AE or SAE (time frame: 1 year). The study lists 2 primary outcomes in all. 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 Apr 2025.