ChoroideremiaPhase 1NCT04483440
Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
A Phase 1 study testing 4D-110 in people with Choroideremia, run by 4D Molecular Therapeutics at 2 sites in United States.
Trial summary
This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM). Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Choroideremia
- What is being tested?
- 4D-110
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 2 sitesUnited States
- Study dates
- 2 Jun 2020to 31 Aug 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
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing
- Both eyes must have ≥ 34 ETDRS letters (~20/200)
Who cannot join
- Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye
- Patient has previously received any AAV treatment
- Ocular conditions or ocular media opacity in either eye that would preclude the planned treatment (i.e. IVT injection) or interfere with the interpretation of study endpoints (e.g. significant lens opacity)
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Retina Foundation of the Southwest
Dallas, Texas, 75251
Moran Eye Center, University of Utah
Salt Lake City, Utah, 84132
About this study
Official title: Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia
This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).
Read the detailed description
This is an open-label, Phase 1 study to evaluate safety and tolerability as well as preliminary efficacy of a single IVT injection of 4D-110 at three dose levels in male patients with genetically-confirmed CHM.
- Conditions
- Choroideremia
- Being tested
- 4D-110
- Keywords
- CHM, transgene, gene therapy, AAV
- Medical subject headings
- Choroideremia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
4D-110Biological
4D-110 drug product developed for gene therapy which comprises an AAV capsid variant (4D-R100) carrying a transgene encoding a codon-optimized human CHM gene.
| Study group | Role | What they receive |
|---|---|---|
| 4D-110 Dose 1 | Experimental | 4D-110 IVT injection Receives: Biological: 4D-110 |
| 4D-110 Dose 2 | Experimental | 4D-110 IVT injection Receives: Biological: 4D-110 |
| 4D-110 Dose 3 | Experimental | 4D-110 IVT injection Receives: Biological: 4D-110 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency and severity of ocular and systemic adverse events (AEs)
Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2020
- Study start2 Jun 2020
- Primary completion26 May 2027 (estimated)
- Study completion31 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ChoroideremiaStudies of the same condition, open and under way.
- More trials run by 4D Molecular TherapeuticsThe 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
- Non-randomised
- Intervention model
- Sequential
- Model description
- Each dose escalation cohort will initially recruit up to 3 patients to receive a single uniocular intravitreal injection of 4D-110 in a standard 3+3 design. The cohort will be expanded in the event of a dose limiting toxicity (DLT). Once the maximum tolerated dose (MTD)/maximum feasible dose (MFD) has been identified, up to 4 additional patients may be enrolled at that dose level to provide additional safety information.
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- 4D Molecular Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Schonmei Lee, MD, Study director, 4D Molecular Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04483440
- Sponsor's protocol ID
- 4D-110-CP-0001
- Status last verified
- May 2025
- First submitted
- 20 Jul 2020
- Last updated
- 22 May 2025
- Results posted
- No
- 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 22 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia
- ClinicalTrials.gov ID
- NCT04483440
- Acronym / sponsor ID
- 4D-110-CP-0001
- Conditions
- Choroideremia
- Keywords
- CHM, transgene, gene therapy, AAV
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 13 participants (actual)
- Lead sponsor
- 4D Molecular TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 2 Jun 2020 (actual)
- Primary completion
- 26 May 2027 (estimated)
- Study completion
- 31 Aug 2027 (estimated)
- First posted
- 23 Jul 2020 (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 study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).
Detailed description
This is an open-label, Phase 1 study to evaluate safety and tolerability as well as preliminary efficacy of a single IVT injection of 4D-110 at three dose levels in male patients with genetically-confirmed CHM.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Each dose escalation cohort will initially recruit up to 3 patients to receive a single uniocular intravitreal injection of 4D-110 in a standard 3+3 design. The cohort will be expanded in the event of a dose limiting toxicity (DLT). Once the maximum tolerated dose (MTD)/maximum feasible dose (MFD) has been identified, up to 4 additional patients may be enrolled at that dose level to provide additional safety information.
| Arm | Type | Receives |
|---|---|---|
| 4D-110 Dose 1 | Experimental | Biological: 4D-1104D-110 IVT injection |
| 4D-110 Dose 2 | Experimental | Biological: 4D-1104D-110 IVT injection |
| 4D-110 Dose 3 | Experimental | Biological: 4D-1104D-110 IVT injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing
- Both eyes must have ≥ 34 ETDRS letters (~20/200)
Key exclusion criteria (3 listed)
- Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye
- Patient has previously received any AAV treatment
- Ocular conditions or ocular media opacity in either eye that would preclude the planned treatment (i.e. IVT injection) or interfere with the interpretation of study endpoints (e.g. significant lens opacity)
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 |
|---|---|---|
| 4D-1104D-110 drug product developed for gene therapy which comprises an AAV capsid variant (4D-R100) carrying a transgene encoding a codon-optimized human CHM gene. | Biological | 4D-110 Dose 1, 4D-110 Dose 2, 4D-110 Dose 3 |
Primary outcome
Frequency and severity of ocular and systemic adverse events (AEs)
Time frame: 24 months
Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.
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 22 May 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. This study is not recruiting at the moment.
Schonmei Lee, MD study director
4D Molecular Therapeutics
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 4D-110 in people with Choroideremia, run by 4D Molecular Therapeutics at 2 sites in United States.
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 2 inclusion and 3 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: under way, not looking for participants, as of the sponsor's last update on 22 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. 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?
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?
Frequency and severity of ocular and systemic adverse events (AEs) (time frame: 24 months). 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 22 May 2025.