GlaucomaPhase 1/2NCT07770685
A Study About the Safety of ASP2767 and Whether it Helps People With Glaucoma-Related Optic Nerve Damage
A Phase 1/2 study testing ASP2767, Prophylactic Regimen A and Prophylactic Regimen B in people with Glaucoma, run by Astellas Pharma Global Development, Inc. at 1 site in United States.
Trial summary
Open-angle glaucoma is the most common type of glaucoma, a disease that damages the nerves in the eye and can lead to vision loss. Current treatments include eye drops, laser treatment, and surgery that help lower eye pressure. However, they do not work for everyone, and the disease may continue to worsen over time. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Glaucoma
- What is being tested?
- ASP2767, Prophylactic Regimen A and Prophylactic Regimen B
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 156 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Oct 2026to 31 Jul 2030
- Phase
- Phase 1/2
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
- Participant agrees not to participate in another interventional study until the 52-week visit has been completed.
- Participant is able and willing to comply with the study requirements and capable of completing the assessments, including all scheduled visits.
- Participant has open-angle glaucoma (OAG) defined as rate of MD ≤ -0.5 dB/year and consisting of all of the following:
- Presence of glaucomatous VF defects in the study eye with corresponding damage to the optic nerve head (ONH) (confirmed by an independent central image reading center at screening)
- An open iridocorneal drainage angle observed on gonioscopy (Shaffer grade ≥ 3)
- Moderate to advanced VF loss, assessed by standard automated perimetry, and confirmed by the central image reading center and in the study eye:
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- Phase 1 - Dose-Escalation part: advanced VF loss, defined as MD between -12 and -20 dB
- Phase 2 - Dose-Expansion part: moderate to advanced VF loss, defined as MD between -6 and -20 dB
- At least 3 reliable VF tests (≤ 33% FLs and ≤ 15% FP) within the preceding 84 months, confirmed by the central image reading center, and excluding fields obtained before incisional glaucoma or cataract surgeries
- Evidence of VF function in at least 1 quadrant in the study eye
- BCVA ≥ 20/200 (≥ 35 ETDRS letters) at screening and confirmed on day 1 in the study eye
- At least 3 consecutive reliable optical coherence tomography (OCT) scans (peripapillary RNFL, GCC/macula) in the study eye within the preceding 84 months, confirmed by the central image reading center.
- Participant must have an IOP ≤ 21 mmHg on current therapy in the study eye (or participant's IOP is considered well controlled and will not require any additional medical or surgical treatment in the next 12 months).
- Female participant is not pregnant and at least 1 of the following conditions apply:
- Not a women of childbearing potential (WOCBP) o. WOCBP who has a negative serum pregnancy test at screening, or urine pregnancy test on day 1, agrees to follow the contraceptive guidance from the time of informed consent.
- Female participant must not be breastfeeding or lactating starting at screening and throughout the study period.
- Female participant must not donate ova starting at first administration of study intervention and throughout the study period.
- Male participant must agree to use contraception with female partner(s) of childbearing potential (including breastfeeding partner) throughout the treatment period.
- Male participant must agree to remain abstinent or use a condom with pregnant partner(s) for the duration of the pregnancy throughout the investigational period.
- Male participant must not donate sperm during the treatment period (up to week 52).
Who cannot join
- Participant has other optic nerve or retinal degenerative disease-causing vision loss, irrespective of whether it is currently treated or untreated.
- Participant has other known or suspected molecular diagnosis of macular, retinal, or optic nerve disease (e.g., pathogenic mutations in other genes) that could confound the interpretation of the outcome of the study, that could cause a concomitant retinal disease and/or point to an alternate etiology of macular or optic nerve disease.
- Participant has visually significant cataract in the study eye.
- Participant has active infectious conjunctivitis, keratitis, scleritis, or endophthalmitis (mild stable blepharitis is permitted).
- Participant is expected to require a change in IOP-lowering treatment within 6 weeks of screening and/or is anticipated to require a change in IOP-lowering treatment during the study in the study eye.
- Participant has history of
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- closed or narrow angle (Shaffer grade ≤ 2), pigment dispersion, uveitic glaucoma, or congenital glaucoma
- cyclophotocoagulation laser treatment for glaucoma in the study eye
- ocular herpetic disease (including herpes simplex and zoster viruses) or disseminated/visceral herpes zoster infection
- allergy to fluorescein or povidone iodine
- history of steroid response (historic evidence of IOP increase with corticosteroid use)
- Participant has presence of IOI (≥ trace anterior chamber [AC] cell or flare), has active or has a history of idiopathic or autoimmune-associated uveitis in either eye.
- Participant has evidence of corneal opacification or lack of optical clarity in the study eye.
- Participant has refractive error greater than 8 diopters of spherical equivalent at screening in the study eye.
- Participant has choroidal neovascularization, central serous retinopathy, or any other type of retinal degeneration/diseases that may interfere with the study procedures, evaluations and outcome assessments.
- Participant has undergone any laser or intraocular surgery (i.e., cataract surgery or minimally invasive glaucoma surgery) in the study eye within 12 weeks and/or yttrium aluminum garnet capsulotomy within 4 weeks prior to screening.
- Participant has a history of vitrectomy surgery in the study eye.
- Participant has a history of, or currently has, optic neuropathy not due to glaucoma, including traumatic or anterior ischemic optic neuropathy.
- Participant has presence of any other concurrent ocular disease in the study eye that would affect or confound study outcomes.
- Participant has any of the following medical conditions
- diabetes mellitus hemoglobin A1c (HbA1c) value of > 7% at screening, with the following exception: If the HbA1c value is > 7% and the participant has a normal creatinine, has no diabetic retinopathy, or diabetic macular edema, then the participant may be enrolled at the discretion of the investigator after consultation with the medical monitor
- uncontrolled hypertension defined as an average systolic blood pressure > 160 mmHg or an average diastolic blood pressure > 100 mmHg (second set of measurements is permitted if the first average exceeds the values during the same visit). Additional repeat measurement may be obtained within the screening window
- history of malignancy other than basal cell carcinoma, unless it was treated successfully at least 2 years prior to inclusion in the study
- history of multiple sclerosis or other severe autoimmune conditions
- history of uncontrolled hepatitis, pancreatitis, cirrhosis, asthma, liver/kidney/heart failure, or uncontrolled thyroid disease or intracranial hypertension
- Participant is currently receiving systemic steroids (other than those related to the study), chemotherapy, immunosuppressive medications, monoclonal antibodies, or glucagon-like peptide-1 treatment.
- Participant is currently using drugs with known ocular toxicity or confounding effects on VF assessments (including but not limited to hydroxychloroquine [Plaquenil], chloroquine, amiodarone, ethambutol, isoniazid, anticholinergics, sulfonamides [including TMP-SMX/Bactrim], and linezolid) unless these were stopped prior to screening and all possible ocular reactions were completely resolved without sequelae.
- Participant has received any prior treatment including gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or prior IVT treatment for any indication in either eye that may be considered to potentially interfere with the study participation or its conduction.
- Participant is currently participating in an interventional clinical study or has received an investigational agent by ocular or systemic administration within 3 months or 5 half-lives, whichever is longer prior to the screening.
- Participant has any severe acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality may interfere with the study procedures, evaluation and outcome assessments and would make the participant unsuitable for study participation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Ophthalmic Consultants of Boston and Boston Eye Surgery & Laser Center
Boston, Massachusetts, 02114
About this study
Official title: A Phase 1, Multicenter, Open-Label, Ascending-Dose and Phase 2, Randomized, Double-Masked, Sham-Controlled, Clinical Study to Evaluate the Safety and Efficacy of ASP2767 in Participants With Optic Neuropathy Due to Glaucoma
Open-angle glaucoma is the most common type of glaucoma, a disease that damages the nerves in the eye and can lead to vision loss. Current treatments include eye drops, laser treatment, and surgery that help lower eye pressure. However, they do not work for everyone, and the disease may continue to worsen over time. Researchers are looking for better ways to treat open-angle glaucoma.
This is an early development study of ASP2767 in people with open-angle glaucoma. In this study, ASP2767 will be given to humans for the first time. ASP2767 is a type of treatment called gene therapy. Gene therapy uses a tool called a vector. In this study a harmless virus is used as the vector. The vector delivers genes into specific parts of the body like the eyes.
Read more
Once in the eyes these genes help nerve cells in the retina make proteins. These proteins are designed to protect the nerves in the eyes and may help slow down or prevent vision loss.
The main aims of the study are to check the safety of ASP2767 in people with open-angle glaucoma, how well they tolerate it, and to find the highest dose of ASP2767 people can safely receive. In addition, the study will also look at the effect of ASP2767 on vision in people with open-angle glaucoma.
The study has 2 phases. In both phases of the study, ASP2767 will be given as a single injection into one eye only.
In phase 1, small groups of people with open-angle glaucoma will receive ASP2767 starting with lower doses and increasing to higher doses in later groups. The people in the study will also receive prophylactic medicines which will help reduce the risk of inflammation after receiving the ASP2767 injection. Phase 1 is an open-label study. This means that people in the study and the researchers will know which treatment people are getting. Any medical problems people have at each dose level in this study will be recorded. This will help find the highest dose of ASP2767 that people can safely receive, which will also be used in phase 2.
In phase 2, another larger group of people with open-angle glaucoma will be randomly placed in 1 of 3 groups of equal size. This means each person will have an equal chance of being placed in any of the 3 groups. They will receive either the highest dose of ASP2767 that people safely received in phase 1, or a slightly lower dose of ASP2767, or a sham injection. A sham injection is a procedure that mimics the injection but does not deliver ASP2767 into the eye. To reduce the risk of inflammation due to the injection, people in the study will also receive a prophylactic medicine or placebo depending on the group they are in. The placebo will look like the prophylactic medicine and will be given in a similar way but will not have any active medicine in it. Researchers use sham injections and placebo to make sure any effect they see in people who take ASP2767 is actually caused by the drug. Phase 2 is a double-masked study, meaning neither the people in the study, nor the researchers will know who is given which treatment.
All the people in phase 1 and phase 2 will be followed up for about 1 year after receiving the treatment. After receiving their ASP2767 injection, people will visit the clinic on certain days to have health checks. They will also have their vision checked using different eye tests and will give regular blood and urine samples.
- Conditions
- Glaucoma
- Being tested
- ASP2767, Sham ASP2767, Prophylactic Regimen A, Prophylactic Regimen B, Placebo for prophylactic regimen
- Keywords
- Glaucoma, ASP2767, Safety, Tolerability, Optic Neuropathy, Neuroprotection
- Medical subject headings
- Glaucoma, Optic Nerve Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ASP2767Drug
IVT Injection
Sham ASP2767Drug
Mimic IVT injection using empty syringe
Prophylactic Regimen ADrug
Protocol defined
Prophylactic Regimen BDrug
Protocol defined
Placebo for prophylactic regimenDrug
Protocol defined
| Study group | Role | What they receive |
|---|---|---|
| Phase 1: ASP2767 Dose Escalation + Prophylactic Regimen A | Experimental | Participants will receive a single dose of ASP2767 by intravitreal (IVT) injection into the study eye on Day 1 and protocol-defined prophylactic regimen. Receives: Drug: ASP2767; Drug: Prophylactic Regimen A |
| Phase 1: ASP2767 Dose Escalation + Prophylactic Regimen B | Experimental | Participants will receive a single dose of ASP2767 by IVT injection into the study eye on Day 1 and protocol-defined prophylactic regimen. Receives: Drug: ASP2767; Drug: Prophylactic Regimen B |
| Phase 2: ASP2767 High Dose - Dose Expansion + Prophylactic Regimen | Experimental | Participants will receive a single high dose of ASP2767 by IVT injection into the study eye on Day 1 and a prophylactic regimen based on data from Phase 1 Receives: Drug: ASP2767; Drug: Prophylactic Regimen A; Drug: Prophylactic Regimen B |
| Phase 2: ASP2767 Low Dose - Dose Expansion + Prophylactic Regimen | Experimental | Participants will receive a single low dose of ASP2767 by IVT injection into the study eye on Day 1 and a prophylactic regimen based on data from Phase 1 Receives: Drug: ASP2767; Drug: Prophylactic Regimen A; Drug: Prophylactic Regimen B |
| Phase 2: Sham ASP2767 + Placebo Prophylactic Regimen | Sham comparator | Participants will receive a single sham injection on Day 1 and a placebo prophylactic regimen Receives: Drug: Sham ASP2767; Drug: Placebo for prophylactic regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1: Number of participants experiencing dose limiting toxicities (DLTs)
DLT is defined as any adverse event which meets DLT criteria that occurs during the DLT observation period unless it is attributed by the investigator to another clearly identifiable cause (e.g. concomitant medications/procedures or pre-existing medical or ocular conditions for additional context).
Measured: Up to 4 weeks
Phase 1: Number of participants with treatment emergent adverse events (TEAEs)
An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of study intervention This includes events related to the comparator, if applicable, and events related to the (study) procedures.
TEAE is defined as an AE with onset or worsening in severity on or after the date of administration of ASP2767 or sham procedure and up to the end of the follow-up period.
Measured: Up to 52 weeks
Phase 1: Number of participants with serious adverse events (SAEs)
An SAE is defined as any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defects and other medically important situations.
Measured: Up to 52 weeks
Phase 1: Number of participants with adverse events of special interest (AESIs)
AESIs are the predefined AEs which are closely monitored throughout the study.
Measured: Up to 52 weeks
Phase 1: Number of participants with ophthalmic examination abnormalities and or/AEs
Number of participants with ophthalmic examination abnormalities and or/AEs will be reported.
Measured: Up to 52 Weeks
Phase 1: Number of participants with ophthalmic imaging abnormalities and or/AEs
Number of participants with ophthalmic imaging abnormalities and or/AEs will be reported.
Measured: Up to 52 Weeks
Phase 1: Number of participants with vital signs abnormalities and/or AEs
Number of participants with vital signs abnormalities and/or AEs will be reported.
Measured: Up to 52 weeks
Phase 1: Number of participants with laboratory value abnormalities and/or AEs
Number of participants with laboratory value abnormalities and/or AEs will be reported.
Measured: Up to 52 weeks
Phase 1: Change from baseline in best-corrected visual acuity (BCVA)
BCVA will be measured from the ETDRS letters chart.
Measured: Baseline and up to Week 52
Phase 2: Mean rate of change (slope) from baseline in visual field (VF) (24-2) mean deviation (MD)
VF examination will be assessed using standard automated perimetry.
Measured: Baseline and Week 52
Secondary outcomes (21)
Phase 1: Number of participants with positive anti-adeno-associated virus serotype (AAV) antibodies
Serum samples will be collected for testing the presence of Anti-AAV antibodies.
Measured: Up to Week 52
Phase 1: Number of participants with positive anti-transgene antibodies
Serum samples will be collected for testing the presence of Anti-transgene antibodies.
Measured: Up to Week 52
Phase 1: Number of participants with positive AAV interferon gamma (IFN-γ) enzyme-linked immunosorbent spot assay (ELISpot)
Serum samples will be collected, together with blood samples for isolation of peripheral blood mononuclear cells (PBMCs) for IFN-γ ELISpot assays.
Measured: Up to Week 52
Phase 2: Mean rate of change (slope) from baseline in VF (10-2) MD
VF examination will be assessed using standard automated perimetry.
Measured: Baseline and Week 52
Phase 2: Percentage of participants with ≥ 7 dB change in 5 VF points from baseline
VF examination will be assessed using standard automated perimetry.
Measured: Baseline and Week 52
Phase 2: Number of participants with TEAEs
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom or disease (new or exacerbated) temporally associated with the use of study intervention. This includes events related to the comparator, if applicable, and events related to the (study) procedures.
TEAE is defined as an AE with onset or worsening in severity on or after the date of administration of ASP2767 or sham procedure and up to the end of the follow-up period.
Measured: Up to week 52
Phase 2: Number of participants with SAEs
An SAE is defined as any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defects and other medically important situations.
Measured: Up to week 52
Phase 2: Number of participants with AESIs
AESIs are the predefined AEs which are closely monitored throughout the study.
Measured: Up to week 52
Phase 2: Change from baseline in macular thickness: ganglion cell complex (GCC)
Global and sectoral macular thickness of GCC will be measured using the SD-OCT.
Measured: Baseline, Week 24 and 52
Phase 2: Change from baseline in peripapillary retinal nerve fiber layer (RNFL) thickness
Global and sectoral thickness of peripapillary RNFL will be measured using the SD-OCT.
Measured: Baseline, Week 24 and 52
Phase 2: Change from baseline in quantitative contrast sensitivity measurement - area under the log contrast sensitivity function (AULCSF)
AULCSF will be assessed using the manifold platform.
Measured: Baseline, Week 24 and 52
Phase 2: Change from baseline in quantitative contrast sensitivity measurement - contrast acuity
Contrast acuity will be assessed using the manifold platform.
Measured: Baseline, Week 24 and 52
Phase 2: Change from baseline in quantitative contrast sensitivity measurement - photopic contrast sensitivity
Photopic contrast sensitivity will be assessed using the manifold platform.
Measured: Baseline, Week 24 and 52
Phase 2: Change from baseline in quantitative contrast sensitivity measurements - mesopic contrast sensitivity
Mesopic contrast sensitivity will be assessed using the manifold platform.
Measured: Baseline, Week 24 and 52
Phase 2: Change from Baseline in BCVA
BCVA will be measured from the ETDRS letters chart.
Measured: Baseline, Week 24 and 52
Phase 2: Number of Participants with Positive Anti-AAV antibodies
Serum samples will be collected for testing the presence of Anti-AAV antibodies.
Measured: Up to Week 52
Phase 2: Number of Participants with Positive Anti-transgene antibodies
Serum samples will be collected for testing the presence of Anti-transgene antibodies.
Measured: Up to Week 52
Phase 2: Number of participants with Positive AAV IFN-γ ELISpot
Serum samples will be collected, together with blood samples for isolation of PBMCs for IFN-γ ELISpot assays.
Measured: Up to Week 52
Phase 2: Change from baseline in macular thickness: ganglion cell/inner plexiform layer (GCIPL)
Global and sectoral macular thickness of GCIPL will be measured using the SD-OCT.
Measured: Baseline, Week 24 and 52
Phase 2: Change from baseline in macular thickness: ganglion cell layer (GCL)
Global and sectoral macular thickness of GCL will be measured using the SD-OCT.
Measured: Baseline, Week 24 and 52
Phase 2: Change from baseline in macular thickness: inner plexiform layer (IPL)
Global and sectoral macular thickness of IPL will be measured using the SD-OCT.
Measured: Baseline, Week 24 and 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2026
- Study start7 Oct 2026
- Primary completion31 Jul 2030 (estimated)
- Study completion31 Jul 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GlaucomaStudies of the same condition, open and under way.
- More trials run by Astellas Pharma Global Development, Inc.The 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- Phase 1 Dose Escalation: Sequential Phase 2 Dose Expansion: Parallel and Sham Control
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 156 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Astellas Pharma Global Development, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Physician, Study director, Astellas Pharma Global Development, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07770685
- Sponsor's protocol ID
- 2767-CL-0101
- Status last verified
- Oct 2026
- First submitted
- 14 Aug 2026
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as compounds terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.