Retinitis PigmentosaPhase 2/3NCT05158296
Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)
A Phase 2/3 study testing Ultevursen in people with Retinitis Pigmentosa and Usher Syndrome Type 2, run by Laboratoires Thea at 15 sites in 4 countries.
Trial summary
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind
- What is being tested?
- Ultevursen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 7 participants
- Where
- 15 sitesin 4 countries
- Study dates
- 8 Dec 2021to 12 Oct 2022
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female, ≥ 18 years of age OR a minor (12 to < 18 years) with permission from a parent or legal guardian. The lower age limit for pediatric populations is subject to local regulatory and ethics committee requirements.
- An adult willing to comply with the protocol, follow study instructions, attend study visits as required and willing and able to complete all study assessments. OR A minor able to complete all study assessments and comply with the protocol and has a parent or caregiver willing and able to follow study instructions, and attend study visits with the subject as required.
- Clinical presentation consistent with RP with Usher syndrome type 2 or nonsyndromic form of RP (NSRP), based on ophthalmic, audiologic, and vestibular examinations.
- A molecular diagnosis of homozygosity or compound heterozygosity for 1 or more pathogenic exon 13 mutations in the USH2A gene, based on genetic analysis at screening.
- BCVA between ≥30 and ≤68 letters (approximate Snellen equivalent 20/250 - 20/50) in the treatment eye using the mean BCVA reading at screening. Subjects with a mean BCVA between >68 and ≤73 letters will be allowed with documented historic evidence of a BCVA equivalent decline of >5 letters in both eyes.
- BCVA between ≥30 and ≤73 letters (approximate Snellen equivalent 20/250 - 20/40) in the contralateral eye (CE), using the mean BCVA reading at Screening.
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- A difference in mean BCVA readings at Screening between the TE and CE of
- ≤10 letters (based on ETDRS). BCVA differences between eyes that are greater than 10 letters may be allowed however, the Investigator should discuss the case with the Medical Monitor.
- Stable BCVA in the TE and CE, defined as 2 separate BCVA measurements at Screening that fall within ≤ 5 letters for each respective eye.
- A visible EZ layer on SD-OCT in the TE, as determined by the Investigator.
- No limitations to SD-OCT image collection that would prevent high quality, reliable images from being obtained in both eyes, as determined by the Investigator.
- Reliable BCVA, perimetry, and other measurements in both eyes, as described in the Study Reference Manual and Imaging Manual and determined by the Investigator.
- No visually significant ocular media opacities and adequate pupillary dilation to permit good quality retinal imaging in both eyes, as assessed by the Investigator.
Who cannot join
- Presence of additional non-exon 13 USH2A pathogenic mutation(s) on the USH2A allele carrying the exon 13 mutation in subjects who have monoallelic exon-13 mutations.
- Presence of non-exon 13 USH2A pathogenic mutation(s) on both USH2A alleles in subjects who have biallelic exon 13 mutations.
- Presence of pathogenic mutations in genes (other than the USH2A gene) associated with Usher syndrome Type 2 or NSRP, or other inherited retinal degenerative diseases or syndromes. Note: The confirmed presence of homozygous or compound heterozygous known disease-causing mutations in other genes involved in recessive retinal dystrophies (RD), or the confirmed presence of known disease-causing mutations in genes involved in dominant or X-linked retinal dystrophies is exclusionary.
- Presence of any significant ocular (in either eye) or non-ocular disease/disorder (or medication and/or laboratory test abnormalities) which, in the opinion of the Investigator and with concurrence of the Medical Monitor, may either put the subject at risk because of participation in the study, may influence the results of the study, or the subject's ability to participate in the study. This includes but is not limited to a subject who has uncontrolled cystoid macular edema (CME). CME is permissible if stable for 3 months (with or without treatment). Past CME is permissible if resolved for more than 1 month.
- History or presence of ocular herpetic diseases (including herpes simplex virus, varicella zoster or cytomegalovirus) in either eye.
- Presence of any active ocular infection in either eye.
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- Presence of any of the following lens opacities in the study eye: cortical opacity ≥
- +2, posterior subcapsular opacity ≥ +2, or a nuclear sclerosis ≥ +2, and which are: 1) clinically significant in the opinion of the Investigator, 2) would adequately prevent clinical and photographic evaluation of the retina.
- History of amblyopia in either eye that resulted in significant vision loss, in the opinion of the Investigator.
- Receipt within 3 months prior to Screening of any intraocular or periocular surgery (including refractive surgery), or an IVT injection or planned intraocular surgery or procedure in either eye during the course of the study. For YAG laser treatment of a posterior capsular opacity, receipt within 1 month prior to Screening or planned procedure in either eye during the course of the study.
- Current treatment or treatment within the past 12 months with therapies known to influence the immune system (including but not limited to steroid implants, cytostatics, interferons, tumor necrosis factor (TNF)-binding proteins, drugs acting on immunophilins, or antibodies with known impact on the immune system). Subjects that have been treated with systemic steroids within the past 12 months or that require intermittent use of topical steroids may be considered for inclusion following approval by the Medical Monitor.
- A history of glaucoma or an IOP greater than 21 mmHg in either eye that is not controlled with medication or surgery. IOP measurements between 21 and 24 mmHg may be allowed however, the Investigator should discuss the case with the Medical Monitor.
- Use of any investigational drug or device within 90 days or 5 half-lives preceding the first dose of study medication, whichever is longer, or plans to participate in another study of an investigational drug or device during the course of the study.
- Any prior treatment with genetic or stem-cell therapy for ocular or non-ocular disease.
- History of malignancy within 2 years prior to Screening, except adequately treated squamous or basal cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
- Known hypersensitivity to antisense oligonucleotides or any constituents of the injection.
- Pregnant and breastfeeding subjects. Females of childbearing potential and males must comply with using highly effective methods of contraception as defined in the protocol. Women of non-childbearing potential may be included without the use of adequate birth control.
Trial locations and contacts
15 sites in 4 countries.
15 sites in 4 countries
United States10 sites
University of Michigan, Kellogg Eye Center
Ann Arbor, Michigan, 48105
Emory Eye Center
Atlanta, Georgia, 30322
Wilmer Eye Institute, Johns Hopkins Hospital
Baltimore, Maryland, 21287
Center for Clinical Research Operations, Massachusetts Eye and Ear
Boston, Massachusetts, 02114
Retina Foundation of the Southwest
Dallas, Texas, 75231
University of Wisconsin - Madison
Madison, Wisconsin, 53705
University of Miami, Bascom Palmer Eye Institute
Miami, Florida, 33136
Columbia University
New York, New York, 10032
Shiley Eye Institute - UC San Diego
San Diego, California, 92093-0946
University of California, San Francisco
San Francisco, California, 94158
Netherlands2 sites
RadboudUMC
Nijmegen, 6525 GA
Het Oogziekenhuis Rotterdam
Rotterdam, 3011 BH
United Kingdom2 sites
Oxford Eye Hospital
Headington, Oxford, OX3 9DU
Moorfields Eye Hospital
London
Germany1 site
Universitaetsklinikum Tuebingen Department für Augenheilkunde
Tübingen, 72076
About this study
Official title: A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Sirius)
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Read the detailed description
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
The below dose levels of ultevursen will be evaluated with the loading dose administered at Day 1 and maintenance dose administered at Month 3 and every 6 months thereafter:
- Loading dose of 60 µg, maintenance dose of 60 µg
- Loading dose of 180 µg, maintenance dose of 60 µg
Dose levels will include subjects randomized to sham-procedure or treatment with ultevursen.
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary
- Being tested
- Ultevursen, Sham-procedure
- Keywords
- Retinitis Pigmentosa, USH2A, RP, exon 13, RNA therapies, antisense oligonucleotide, exon skipping, Sirius, IVT, mutations in exon 13 of the USH2A gene, NSRP, Non-Syndromic RP, Inherited Retinal Disorders, Usher Syndrome, Intravitreal Injection, ultevursen
- Medical subject headings
- Retinitis Pigmentosa, Usher Syndromes, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Eye Abnormalities, Vision Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UltevursenDrug
Also called RNA antisense oligonucleotide for intravitreal injection
RNA antisense oligonucleotide for intravitreal injection
Sham-procedureOther
Sham-procedure (no experimental drug administered)
| Study group | Role | What they receive |
|---|---|---|
| Ultevursen 60/60 µg | Experimental | 60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter Receives: Drug: Ultevursen |
| Ultevursen 180/60 µg | Experimental | 180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter Receives: Drug: Ultevursen |
| Sham-procedure | Sham comparator | Sham-procedure (no experimental drug administered) on Day 1, Month 3 and every 6 months thereafter Receives: Other: Sham-procedure |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Change From Baseline in Best Corrected Visual Acuity (BCVA)
Mean change from baseline in best corrected visual acuity(BCVA) based on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart
Measured: 18 months of treatment versus sham-procedure
Secondary outcomes (9)
Proportion of Patients Who Maintain Vision Defined by BCVA Loss Less Than 15 Letters (ETDRS)
Proportion of patients who maintain vision defined by BCVA loss less than 15 Letters based on ETDRS
Measured: 27 months
Change From Baseline in Other Analyses of Best Corrected Visual Acuity (BCVA)
Change from baseline in other analyses of Best Corrected Visual Acuity (BCVA)
Measured: 27 months
Change From Baseline in Ellipsoid Zone (EZ) Area and Width as Imaged by Spectral Domain Optical Coherence Tomography (SD-OCT)
Change from baseline in ellipsoid zone (EZ) area and width as imaged by spectral domain optical coherence tomography (SD-OCT)
Measured: 27 months
Change From Baseline in Low Luminance Visual Acuity (LLVA)
Change from baseline in Low Luminance Visual Acuity (LLVA)
Measured: 27 months
Change From Baseline in Microperimetry
Change from baseline in Microperimetry
Measured: 27 months
Change From Baseline in Full-field Stimulus Threshold (FST)
Change from baseline in Full-field Stimulus Threshold (FST)
Measured: 27 months
Change From Baseline in PRO Measures
As assessed by the Veteran Affairs Low Vision Visual Functioning Questionnaire (VA LV VFQ-20), Patient Global Impressions of Severity (PGI-S) and Patient Global Impressions of Change (PGI-C)
Measured: 27 months
Ocular and Non-ocular Adverse Events (AEs)
Ocular and non-ocular adverse events (AEs)
Measured: 27 months
Cmax of Ultevursen in Serum
Maximum concentration (Cmax) of ultevursen in serum
Measured: 27 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2021
- Study start8 Dec 2021
- Primary completion12 Oct 2022
- Study completion12 Oct 2022
Why it stopped: Business decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for 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
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Double-masked, randomized, controlled, multiple-dose study. Subjects will be randomized to one of three treatment groups: 1. Group 1: Ultevursen 180/60 µg (180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter) 2. Group 2: Ultevursen 60/60 µg (60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter; n = 27) 3. Group 3: Sham-procedure (administered on Day 1, Month 3 and every 6 months thereafter; n = 27)
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Laboratoires Thea
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Sepul Bio
- Responsible party
- Sponsor
- Investigators
- Sepul Bio Medical Monitor, Study director, Sepul BioSepul Bio Clinical Operations Director, Study director, Sepul Bio
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05158296
- Sponsor's protocol ID
- PQ-421a-003
- Other IDs
- 2021-002729-74
- Status last verified
- Dec 2022
- First submitted
- 23 Nov 2021
- Last updated
- 12 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Sirius)
- ClinicalTrials.gov ID
- NCT05158296
- Acronym / sponsor ID
- PQ-421a-003
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary
- Keywords
- Retinitis Pigmentosa, USH2A, RP, exon 13, RNA therapies, antisense oligonucleotide, exon skipping, Sirius
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Business decision
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Laboratoires TheaIndustry (pharma, biotech, devices)
- Collaborators
- Sepul Bio
- Study start
- 8 Dec 2021 (actual)
- Primary completion
- 12 Oct 2022 (actual)
- Study completion
- 12 Oct 2022 (actual)
- First posted
- 15 Dec 2021 (actual)
- Results
- Posted 12 Jul 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Detailed description
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
The below dose levels of ultevursen will be evaluated with the loading dose administered at Day 1 and maintenance dose administered at Month 3 and every 6 months thereafter:
- Loading dose of 60 µg, maintenance dose of 60 µg
- Loading dose of 180 µg, maintenance dose of 60 µg
Dose levels will include subjects randomized to sham-procedure or treatment with ultevursen.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Double-masked, randomized, controlled, multiple-dose study. Subjects will be randomized to one of three treatment groups: 1. Group 1: Ultevursen 180/60 µg (180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter) 2. Group 2: Ultevursen 60/60 µg (60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter; n = 27) 3. Group 3: Sham-procedure (administered on Day 1, Month 3 and every 6 months thereafter; n = 27)
| Arm | Type | Receives |
|---|---|---|
| Ultevursen 60/60 µg | Experimental | Drug: Ultevursen60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter |
| Ultevursen 180/60 µg | Experimental | Drug: Ultevursen180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter |
| Sham-procedure | Sham comparator | Other: Sham-procedureSham-procedure (no experimental drug administered) on Day 1, Month 3 and every 6 months thereafter |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- Male or female, ≥ 18 years of age OR a minor (12 to < 18 years) with permission from a parent or legal guardian. The lower age limit for pediatric populations is subject to local regulatory and ethics committee requirements.
- An adult willing to comply with the protocol, follow study instructions, attend study visits as required and willing and able to complete all study assessments. OR A minor able to complete all study assessments and comply with the protocol and has a parent or caregiver willing and able to follow study instructions, and attend study visits with the subject as required.
- Clinical presentation consistent with RP with Usher syndrome type 2 or nonsyndromic form of RP (NSRP), based on ophthalmic, audiologic, and vestibular examinations.
- A molecular diagnosis of homozygosity or compound heterozygosity for 1 or more pathogenic exon 13 mutations in the USH2A gene, based on genetic analysis at screening.
- BCVA between ≥30 and ≤68 letters (approximate Snellen equivalent 20/250 - 20/50) in the treatment eye using the mean BCVA reading at screening. Subjects with a mean BCVA between >68 and ≤73 letters will be allowed with documented historic evidence of a BCVA equivalent decline of >5 letters in both eyes.
Key exclusion criteria (17 listed)
- Presence of additional non-exon 13 USH2A pathogenic mutation(s) on the USH2A allele carrying the exon 13 mutation in subjects who have monoallelic exon-13 mutations.
- Presence of non-exon 13 USH2A pathogenic mutation(s) on both USH2A alleles in subjects who have biallelic exon 13 mutations.
- Presence of pathogenic mutations in genes (other than the USH2A gene) associated with Usher syndrome Type 2 or NSRP, or other inherited retinal degenerative diseases or syndromes. Note: The confirmed presence of homozygous or compound heterozygous known disease-causing mutations in other genes involved in recessive retinal dystrophies (RD), or the confirmed presence of known disease-causing mutations in genes involved in dominant or X-linked retinal dystrophies is exclusionary.
- Presence of any significant ocular (in either eye) or non-ocular disease/disorder (or medication and/or laboratory test abnormalities) which, in the opinion of the Investigator and with concurrence of the Medical Monitor, may either put the subject at risk because of participation in the study, may influence the results of the study, or the subject's ability to participate in the study. This includes but is not limited to a subject who has uncontrolled cystoid macular edema (CME). CME is permissible if stable for 3 months (with or without treatment). Past CME is permissible if resolved for more than 1 month.
- History or presence of ocular herpetic diseases (including herpes simplex virus, varicella zoster or cytomegalovirus) in either eye.
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 |
|---|---|---|
| UltevursenAlso: RNA antisense oligonucleotide for intravitreal injectionRNA antisense oligonucleotide for intravitreal injection | Drug | Ultevursen 180/60 µg, Ultevursen 60/60 µg |
| Sham-procedureSham-procedure (no experimental drug administered) | Other | Sham-procedure |
Primary outcome
Mean Change From Baseline in Best Corrected Visual Acuity (BCVA)
Time frame: 18 months of treatment versus sham-procedure
Mean change from baseline in best corrected visual acuity(BCVA) based on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart
9 secondary outcomes
Proportion of Patients Who Maintain Vision Defined by BCVA Loss Less Than 15 Letters (ETDRS)
Time frame: 27 months
Change From Baseline in Other Analyses of Best Corrected Visual Acuity (BCVA)
Time frame: 27 months
Change From Baseline in Ellipsoid Zone (EZ) Area and Width as Imaged by Spectral Domain Optical Coherence Tomography (SD-OCT)
Time frame: 27 months
Change From Baseline in Low Luminance Visual Acuity (LLVA)
Time frame: 27 months
Change From Baseline in Microperimetry
Time frame: 27 months
Change From Baseline in Full-field Stimulus Threshold (FST)
Time frame: 27 months
Change From Baseline in PRO Measures
Time frame: 27 months
Ocular and Non-ocular Adverse Events (AEs)
Time frame: 27 months
Cmax of Ultevursen in Serum
Time frame: 27 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Sepul Bio Medical Monitor study director
Sepul Bio
Sepul Bio Clinical Operations Director study director
Sepul Bio
15 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Ultevursen in people with Retinitis Pigmentosa and Usher Syndrome Type 2, run by Laboratoires Thea at 15 sites in 4 countries.
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 13 inclusion and 17 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: stopped early, as of the sponsor's last update on 12 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Netherlands, United Kingdom, Germany. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Mean Change From Baseline in Best Corrected Visual Acuity (BCVA) (time frame: 18 months of treatment versus sham-procedure). 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 12 Jul 2024.