Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)Phase 1/2CHORDNCT05788536
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations
A Phase 1/2 study testing DB-OTO in people with Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF), run by Regeneron Pharmaceuticals at 17 sites in 5 countries.
Trial summary
Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat pediatric and adult participants who have severe-to profound and profound hearing loss due to changes in the otoferlin gene. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
- What is being tested?
- DB-OTO
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 17 sitesin 5 countries
- Study dates
- 27 Jun 2023to 25 Feb 2032
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Willingness to provide written informed consent (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) and willingness to comply with trial protocol
- Willingness to consent to genetic testing for the participant (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) in order to evaluate a panel of hearing loss-related genes
- Willingness to consent to vaccinations for the participant (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) in accordance with the country-specific, age-appropriate immunization schedule, as described in the protocol
- Participant able to perform all necessary assessments to qualify for enrollment and dosing in the corresponding cohort at the time the participant or parent/legal guardian signing the informed consent form (and participant providing assent, when applicable)
- Presence of biallelic, likely pathogenic or pathogenic OTOF variants
- No clinically significant laboratory findings on clinical laboratory tests at time of Screening as described in the protocol
Show all 11Show fewer
- Audiological Criteria:
- Investigator diagnoses the participant with profound sensorineural hearing loss (SNHL; >90 dB HL) based on behavioral and physiologic measurements (ABR) of inner ear function
- Outer hair cell presence is confirmed via presence of otoacoustic emissions (≥6 dBSNR) at ≥3 frequencies from 1 to 8 kHz in the ear(s) to be infused with DB-OTO. Alternatively, for participants >24 months of age, outer hair cell presence can be confirmed via presence of the cochlear microphonic in the ear(s) to be infused with DB-OTO.
- No evidence from measures of hearing loss that show a dependence on body temperature
- From study start and for the duration of the short-term follow-up period (48 weeks): Female participants of childbearing potential and fertile males, must agree to use highly effective contraception. Female participants must agree not to become pregnant. Fertile male participants must agree not to father a child or donate sperm, for 48 weeks and in cases of early withdrawal, for at least 12 months after DB-OTO administration.
Who cannot join
- History of prior treatment with gene therapy
- Surgical anatomy that would preclude or meaningfully impact the planned surgical approach as indicated by medical imaging (eg, Computed Tomography [CT] or Magnetic Resonance Imaging [MRI]) in the ear(s) to be infused with DB-OTO
- History or presence of other permanent or untreatable hearing loss conditions
- Prior or current history of malignancies
- Prior or current history of meningitis
- History or presence of cochlear implants in the ear(s) to be infused with DB-OTO
- History of risk factor(s) for auditory neuropathy not caused by OTOF pathogenic variants including but not limited to: prematurity, low birth weight, hyperbilirubinemia, Neonatal Intensive Care Unit (NICU) admission, and/or low Apgar scores as described in the protocol
- Note: Other protocol-defined inclusion/exclusion criteria apply
Trial locations and contacts
17 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
17 sites in 5 countries
United States9 sites, 9 recruiting
- Recruiting
Boston Children's Hospital - Main
Boston, Massachusetts, 02115
Eliot Shearer, Eliot.Shearer@childrens.harvard.edu
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
John Greinwald, Jr., john.greinwald@cchmc.edu
- Recruiting
Nemours Children s Clinic
Jacksonville, Florida, 32207
Evie Landry, evie.Landry@nemours.org
- Recruiting
University of California Los Angeles Medical Center
Los Angeles, California, 90024
Akira Ishiyama, ishiyama@g.ucla.edu
- Recruiting
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Sarah Mleziva, smleziva@mcw.edu
- Recruiting
Columbia University Irving Medical Center
New York, New York, 10032
Lawrence Lustig, lustig@columbia.edu
- Recruiting
Nemours Childrens Hospital
Orlando, Florida, 32827
Cedric Pritchett, cedric.Pritchett@nemours.org
- Recruiting
Rady Children's Hospital
San Diego, California, 92123
Daniela Carvalho, dcarvalho@rchsd.org
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Jay Rubenstein, rubinj@uw.edu
Spain4 sites, 3 recruiting
- Recruiting
Hospital Sant Joan de Deu
Barcelona, 08950
Oliver Haag, oliver.haag@sjd.es
- Withdrawn
Hospital Universitario Materno Infantil en las Palmas de Gran Canaria
Las Palmas de Gran Canaria, 35016
- Recruiting
Ramon y Cajal University Hospital
Madrid, 28034
Ruben Polo, rubenpolo1979@gmail.com
- Recruiting
Clinica Universidad de Navarra- Pamplona
Pamplona, Navarre, 31008
Manuel Jesus Manrique Rodriguez, mmanrique@unav.es
United Kingdom2 sites, 2 recruiting
- Recruiting
Addenbrooke's Hospital, Cambridge University Hospitals NHS FT
Cambridge, CB2 0QQ
Manohar Bance, mlb59@cam.ac.uk
- Recruiting
Great Ormond Street Hospital For Children NHS Foundation Trust
London, WC1N 3JH
Robert Nash, r.nash@ucl.ac.uk
Germany1 site, 1 recruiting
- Recruiting
University Hospital Tubingen
Tübingen, 72076
Hubert Lowenheim, hubert.loewenheim@uni-tuebingen.de
Japan1 site, 1 recruiting
- Recruiting
Shinshu University Hospital
Matsumoto-shi, Nagano, 390-8621
Yutaka Takumi, takumi@shinshu-u.ac.jp
About this study
Official title: A Phase 1/2, Open-Label, Multicenter Trial With a Single Ascending Dose Cohort With Unilateral Intracochlear Injection Followed by a Bilateral Injection Expansion Cohort to Evaluate the Safety, Tolerability, and Efficacy of DB-OTO in Children and Infants With Biallelic hOTOF Mutations
Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat pediatric and adult participants who have severe-to profound and profound hearing loss due to changes in the otoferlin gene.
The purpose of this study is to:
- Learn about the safety of DB-OTO
- Determine how well DB-OTO is tolerated (does not cause ongoing discomfort)
- Evaluate the efficacy of DB-OTO (how well DB-OTO works)
Read the detailed description
Former Sponsor Decibel Therapeutics
- Being tested
- DB-OTO
- Keywords
- DB-OTO, Gene Therapy, Congenital Hearing Loss, Sensorineural Hearing Loss, Auditory Neuropathy, Pediatric, Cochlear Implant, Otoferlin, Deaf, Hard of hearing, Hearing impaired, Hearing disorder, Fully implantable hearing aid, Child, Infant, Adolescents, Young Adults, Adults, CHORD
- Medical subject headings
- Hearing Loss, Sensorineural, Auditory neuropathy, Deafness, Autosomal Recessive 9, Hearing Loss, Hearing Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DB-OTOGenetic
Also called Otarmeni™, Otoferlin
Administered per the protocol
| Study group | Role | What they receive |
|---|---|---|
| DB-OTO - Part A | Experimental | Unilateral intracochlear dosing Receives: Genetic: DB-OTO |
| DB-OTO - Part B | Experimental | Bilateral intracochlear dosing using the dose selected based on safety and efficacy data from Part A Receives: Genetic: DB-OTO |
| DB-OTO - Part C | Experimental | Unilateral or bilateral intracochlear dosing Receives: Genetic: DB-OTO |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
Measured: Up to week 104
Achievement of a hearing sensitivity threshold of ≤70 dB assessed by average Pure Tone Audiometry (PTA)
Measured: Up to week 104
Secondary outcomes (27)
Auditory Brainstem Response (ABR) to click stimulus at ≤90 dB normalized Hearing Level (nHL)
Measured: Up to week 48
Achievement of hearing sensitivity threshold of ≤45 dB assessed by average PTA
Measured: Up to week 104
Achievement of hearing sensitivity threshold of ≤25 dB assessed by average PTA
Measured: Up to week 104
Achievement of a score ≥3 on the Early Speech Perception (ESP) test
Measured: At week 104
Achievement of a score of 4 on the ESP test
Measured: At week 104
Speech perception scores by age-appropriate tests
Measured: Up to week 104
Speech Awareness Threshold (SAT): achievement of a threshold of ≤70 dB
Measured: Up to week 48
SAT: achievement of a threshold of ≤45 dB
Measured: Up to week 48
SAT: achievement of threshold of ≤25 dB
Measured: Up to week 48
Severity in speech perception ability assessed by Global Impression scales (clinician and parent/legal guardian)
Measured: At week 104
Change in speech perception ability assessed by Global Impression scales (clinician and parent/legal guardian)
Measured: At week 104
Average PTA threshold in the subset of participants who achieved an average PTA threshold ≤70 dB
Measured: Up to week 104
Average PTA threshold in the subset of participants who achieved an average PTA threshold >70 dB but ≤85 dB
Measured: Up to week 48
Achievement of a hearing sensitivity threshold improvement of ≤85 dB HL, as assessed by PTA
Measured: Up to week 48
Achievement of a hearing sensitivity threshold improvement of ≥10 dB from baseline
Measured: Up to week 48
Time to an average PTA threshold ≤70 dB
Measured: Up to week 104
Persistence of an average PTA threshold ≤70 dB
Measured: Up to week 104
Incidence of participants who regress to >70 dB after having achieved average PTA threshold
Measured: Up to week 104
Presence of ABR to click at ≤90 dB nHL
Measured: At week 104
Change from baseline on LittlEARS®
Measured: At week 104
Change in scores on LittlEARS®
Measured: At week 104
Change from baseline on Auditory Skills Checklist
Measured: At week 104
Change in scores on Auditory Skills Checklist
Measured: At week 104
Change from baseline on MacArthur-Bates Communicative Development Inventories (MB-CDI)
Measured: At week 104
Change in scores on MB-CDI
Measured: At week 104
Clinical assessments of speech production: articulation
Measured: At week 104
Clinical assessments of speech production: speech intelligibility
Measured: At week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2023
- Study start27 Jun 2023
- Primary completion26 Feb 2029 (estimated)
- Study completion25 Feb 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)Studies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe 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
- Non-randomised
- Intervention model
- Sequential
- Model description
- DB-OTO will be administered as a single intracochlear infusion into one ear (Part A), both ears (Part B), one or both ears (Part C). For bilateral infusions (Part B and C), participants will receive DB-OTO in 1 surgical session.
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05788536
- Sponsor's protocol ID
- DB-OTO-001
- Other IDs
- 2022-000079-38, 2024-511342-40-00
- Acronym
- CHORD
- Status last verified
- Aug 2026
- First submitted
- 15 Mar 2023
- Last updated
- 5 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Publications
Valayannopoulos V, Bance M, Carvalho DS, Greinwald JH Jr, Harvey SA, Ishiyama A, Landry EC, Lowenheim H, Lustig LR, Manrique M, Nash R, Polo R, Pritchett CV, Rubinstein JT, Shearer AE, Del Castillo I, Anderson JJ, Corrales CE, Quigley TM, Riggs WJ, Weber P, Wilson G, Irvin SC, Hassan HE, Chen Y, Liu R, Drummond MC, Sabin LR, Musser BJ, Yancopoulos GD, Kyratsous CA, Herman GA, Baras A, Whitton JP; CHORD Study Group. DB-OTO Gene Therapy for Inherited Deafness. N Engl J Med. 2026 Mar 12;394(11):1074-1083. doi: 10.1056/NEJMoa2400521. Epub 2025 Oct 12.
PubMed 41085057
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Open-Label, Multicenter Trial With a Single Ascending Dose Cohort With Unilateral Intracochlear Injection Followed by a Bilateral Injection Expansion Cohort to Evaluate the Safety, Tolerability, and Efficacy of DB-OTO in Children and Infants With Biallelic hOTOF Mutations
- ClinicalTrials.gov ID
- NCT05788536
- Acronym / sponsor ID
- CHORD / DB-OTO-001
- Keywords
- DB-OTO, Gene Therapy, Congenital Hearing Loss, Sensorineural Hearing Loss, Auditory Neuropathy, Pediatric, Cochlear Implant, Otoferlin
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 27 Jun 2023 (actual)
- Primary completion
- 26 Feb 2029 (estimated)
- Study completion
- 25 Feb 2032 (estimated)
- First posted
- 29 Mar 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.
Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat pediatric and adult participants who have severe-to profound and profound hearing loss due to changes in the otoferlin gene.
The purpose of this study is to:
- Learn about the safety of DB-OTO
- Determine how well DB-OTO is tolerated (does not cause ongoing discomfort)
- Evaluate the efficacy of DB-OTO (how well DB-OTO works)
Detailed description
Former Sponsor Decibel Therapeutics
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- DB-OTO will be administered as a single intracochlear infusion into one ear (Part A), both ears (Part B), one or both ears (Part C). For bilateral infusions (Part B and C), participants will receive DB-OTO in 1 surgical session.
| Arm | Type | Receives |
|---|---|---|
| DB-OTO - Part A | Experimental | Genetic: DB-OTOUnilateral intracochlear dosing |
| DB-OTO - Part B | Experimental | Genetic: DB-OTOBilateral intracochlear dosing using the dose selected based on safety and efficacy data from Part A |
| DB-OTO - Part C | Experimental | Genetic: DB-OTOUnilateral or bilateral intracochlear dosing |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Willingness to provide written informed consent (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) and willingness to comply with trial protocol
- Willingness to consent to genetic testing for the participant (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) in order to evaluate a panel of hearing loss-related genes
- Willingness to consent to vaccinations for the participant (by at least one parent/legal guardian for pediatric participants, and with participant to provide assent, when applicable, or by the adult participant) in accordance with the country-specific, age-appropriate immunization schedule, as described in the protocol
- Participant able to perform all necessary assessments to qualify for enrollment and dosing in the corresponding cohort at the time the participant or parent/legal guardian signing the informed consent form (and participant providing assent, when applicable)
- Presence of biallelic, likely pathogenic or pathogenic OTOF variants
Key exclusion criteria (8 listed)
- History of prior treatment with gene therapy
- Surgical anatomy that would preclude or meaningfully impact the planned surgical approach as indicated by medical imaging (eg, Computed Tomography [CT] or Magnetic Resonance Imaging [MRI]) in the ear(s) to be infused with DB-OTO
- History or presence of other permanent or untreatable hearing loss conditions
- Prior or current history of malignancies
- Prior or current history of meningitis
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 |
|---|---|---|
| DB-OTOAlso: Otarmeni™, OtoferlinAdministered per the protocol | Genetic | DB-OTO - Part A, DB-OTO - Part B, DB-OTO - Part C |
Primary outcome
Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to week 104
Achievement of a hearing sensitivity threshold of ≤70 dB assessed by average Pure Tone Audiometry (PTA)
Time frame: Up to week 104
27 secondary outcomes
Auditory Brainstem Response (ABR) to click stimulus at ≤90 dB normalized Hearing Level (nHL)
Time frame: Up to week 48
Achievement of hearing sensitivity threshold of ≤45 dB assessed by average PTA
Time frame: Up to week 104
Achievement of hearing sensitivity threshold of ≤25 dB assessed by average PTA
Time frame: Up to week 104
Achievement of a score ≥3 on the Early Speech Perception (ESP) test
Time frame: At week 104
Achievement of a score of 4 on the ESP test
Time frame: At week 104
Speech perception scores by age-appropriate tests
Time frame: Up to week 104
Speech Awareness Threshold (SAT): achievement of a threshold of ≤70 dB
Time frame: Up to week 48
SAT: achievement of a threshold of ≤45 dB
Time frame: Up to week 48
SAT: achievement of threshold of ≤25 dB
Time frame: Up to week 48
Severity in speech perception ability assessed by Global Impression scales (clinician and parent/legal guardian)
Time frame: At week 104
Change in speech perception ability assessed by Global Impression scales (clinician and parent/legal guardian)
Time frame: At week 104
Average PTA threshold in the subset of participants who achieved an average PTA threshold ≤70 dB
Time frame: Up to week 104
Average PTA threshold in the subset of participants who achieved an average PTA threshold >70 dB but ≤85 dB
Time frame: Up to week 48
Achievement of a hearing sensitivity threshold improvement of ≤85 dB HL, as assessed by PTA
Time frame: Up to week 48
Achievement of a hearing sensitivity threshold improvement of ≥10 dB from baseline
Time frame: Up to week 48
Time to an average PTA threshold ≤70 dB
Time frame: Up to week 104
Persistence of an average PTA threshold ≤70 dB
Time frame: Up to week 104
Incidence of participants who regress to >70 dB after having achieved average PTA threshold
Time frame: Up to week 104
Presence of ABR to click at ≤90 dB nHL
Time frame: At week 104
Change from baseline on LittlEARS®
Time frame: At week 104
Change in scores on LittlEARS®
Time frame: At week 104
Change from baseline on Auditory Skills Checklist
Time frame: At week 104
Change in scores on Auditory Skills Checklist
Time frame: At week 104
Change from baseline on MacArthur-Bates Communicative Development Inventories (MB-CDI)
Time frame: At week 104
Change in scores on MB-CDI
Time frame: At week 104
Clinical assessments of speech production: articulation
Time frame: At week 104
Clinical assessments of speech production: speech intelligibility
Time frame: At week 104
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 Aug 2026.
- Derived from this study: PubMed 41085057Valayannopoulos V, Bance M, Carvalho DS, Greinwald JH Jr, Harvey SA, Ishiyama A, Landry EC, Lowenheim H, Lustig LR, Manrique M, Nash R, Polo R, Pritchett CV, Rubinstein JT, Shearer AE, Del Castillo I, Anderson JJ, Corrales CE, Quigley TM, Riggs WJ, Weber P, Wilson G, Irvin SC, Hassan HE, Chen Y, Liu R, Drummond MC, Sabin LR, Musser BJ, Yancopoulos GD, Kyratsous CA, Herman GA, Baras A, Whitton JP; CHORD Study Group. DB-OTO Gene Therapy for Inherited Deafness. N Engl J Med. 2026 Mar 12;394(11):1074-1083. doi: 10.1056/NEJMoa2400521. Epub 2025 Oct 12.
- Individual participant dataSharing planned: Yes. All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Related trials for Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
RecruitingPhase 1/2
A Clinical Trial of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
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.
Clinical Trial Management study director
Regeneron Pharmaceuticals
17 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing DB-OTO in people with Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF), run by Regeneron Pharmaceuticals at 17 sites in 5 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 11 inclusion and 8 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 5 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, Spain, United Kingdom, Germany, Japan. 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 Clinical Trials Administrator (844-734-6643, clinicaltrials@regeneron.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of Treatment-Emergent Adverse Events (TEAEs) (time frame: Up to week 104). 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?
On the ClinicalTrials.gov record for NCT05788536, which also lists 1 linked publication.
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 Aug 2026.