Leber Congenital Amaurosis 10Phase 1/2INSIGHTNCT03913130
Extension Study to Study PQ-110-001 (NCT03140969)
A Phase 1/2 study testing QR-110 in people with Leber Congenital Amaurosis 10 and Blindness, run by Laboratoires Thea at 3 sites in 2 countries.
Trial summary
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis
- What is being tested?
- QR-110
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 13 May 2019to 3 Oct 2022
- Phase
- Phase 1/2
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects who completed participation in the study PQ-110-001 and who may derive benefit from continued treatment with QR 110, as assessed by the Investigator, in consultation with the Medical Monitor
- Persistence of detectable outer nuclear layer (ONL) in the area of the macula in the opinion of the Investigator, as determined by OCT.
- Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging, as assessed by the Investigator.
- An adult (≥ 18 years) willing and able to provide informed consent for participation OR a minor (6 to < 18 years) with a parent or legal guardian willing and able to provide written permission for the subject's participation prior to performing any study related procedures, and pediatric subjects able to provide age-appropriate assent for study participation.
- Female subjects who have reached menarche and male subjects must either practice true abstinence in accordance with their preferred and usual lifestyle, or agree to use acceptable, highly effective methods of contraception for up to 3 months following their last dose QR-110. Acceptable methods of contraception are defined in the protocol. Women of non-childbearing potential may be included without the use of adequate birth control, provided they meet the criteria in the protocol.
Who cannot join
- Any contraindication to IVT injection according to the Investigator's clinical judgment and international guidelines (Avery 2014).
- Safety issue during study PQ-110-001 that may compromise subject safety when continued dosing, as determined by the Investigator, and in consultation with the Medical Monitor.
- Any ocular or systemic disease or condition (including medications and laboratory test abnormalities) that could compromise subject safety or interfere with assessment of efficacy and safety, as determined by the Investigator and in consultation with the Medical Monitor.
- Pregnant or breast-feeding female.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
University of Iowa
Iowa City, Iowa, 52242
Scheie Eye Institute, University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
Official title: An Open-Label, Extension Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Study PQ-110-002 will provide long-term safety, tolerability, pharmacokinetic (PK), and efficacy data of QR-110.
Read the detailed description
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject.
Subjects will be given the opportunity to enroll into this extension study for continued dosing if available data support current and/or future benefits for the subject. The Investigator, in consultation and agreement with the Medical Monitor, will decide on enrollment of each individual subject, as well as on dosing of the first treated eye and treatment initiation of the contralateral eye. Continued subject treatment in this study is desirable, but cannot be guaranteed, since it will depend on the risks and benefit of further treatment on a case-by-case basis, as discussed and agreed upon with the Medical Monitor.
The contralateral eye and the first treated eye will be injected 3 months apart. The injection interval of 3 months between both eyes will limit burden for the subjects, with a 3 month-visit frequency during the course of the study. This between-eye interval could be adapted if safety data are supportive, and for logistic reasons, and in agreement with the Medical Monitor.
The same safety monitoring protocol and efficacy assessments will apply to both eyes.
QR-110 will be administered via intravitreal (IVT) injection.
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations
- Being tested
- QR-110
- Keywords
- LCA10, CEP290, p.Cys998X, c.2991+1655A>G, Leber's Congenital Amaurosis, Antisense oligonucleotide, RNA therapy
- Medical subject headings
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QR-110Drug
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
| Study group | Role | What they receive |
|---|---|---|
| Drug QR-110 | Experimental | First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration Receives: Drug: QR-110 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Ocular AEs
Frequency of ocular adverse events (AEs)
Measured: 24 months
Frequency of Non-ocular AEs
Frequency of non-ocular AEs
Measured: 24 months
Secondary outcomes (12)
Change in BCVA in First Treated Eye
Change in Best Corrected Visual Acuity (BCVA) in First Treated Eye
Measured: 24 months
Change in Mobility Course Score
Change in Mobility course score
Measured: 24 months
Change in Photoreceptor Outer Segment Layer Thickness
Change in photoreceptor outer segment layer thickness by Optical Coherence Tomography (OCT)
Measured: 24 months
Change in OCI
Change in Oculomotor Instability (OCI)
Measured: 24 months
Change in FST Blue
Change in Full-Field Stimulus Testing (FST) - blue stimuli
Measured: 24 months
Change in FST Red
Change in Full-Field Stimulus Testing (FST) - red stimuli
Measured: 24 months
Change in VFQ-25
Change in Visual Function Questionnaire-25 (VFQ-25) score (adult subjects)
Measured: 24 months
Change in CVAQ
Change in Cardiff Visual Ability Questionnaire for Children (CVAQC) score (pediatric subjects)
Measured: 24 months
Change in PLR
Change in Pupillary Light Reflex (PLR) (latency and amplitude)
Measured: 24 months
Change in NIRAF
Change in Near Infrared AutoFluorescence (NIRAF)
Measured: 24 months
Change in BCVA in Treated Contralateral Eye
Change in Best Corrected Visual Acuity (BCVA) in Treated Contralateral Eye
Measured: 24 months
Change in BCVA in Non-Treated Contralateral Eye
Change in Best Corrected Visual Acuity (BCVA) in Non-Treated Contralateral Eye
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 posted12 Apr 2019
- Study start13 May 2019
- Primary completion3 Oct 2022
- Study completion3 Oct 2022
Why it stopped: Terminated \[Study prematurely terminated due to sponsor decision for reasons unrelated to safety\]
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 Leber Congenital Amaurosis 10Studies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Laboratoires Thea
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Sepul Bio
- Responsible party
- Sponsor
- Investigators
- Sepul Bio Chief Medical Officer, Study director, Sepul Bio
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03913130
- Sponsor's protocol ID
- PQ-110-002
- Other IDs
- 2018-003500-40, 2018
- Acronym
- INSIGHT
- Status last verified
- Oct 2024
- First submitted
- 21 Feb 2019
- Last updated
- 15 Oct 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 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Extension Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
- ClinicalTrials.gov ID
- NCT03913130
- Acronym / sponsor ID
- INSIGHT / PQ-110-002
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations
- Keywords
- LCA10, CEP290, p.Cys998X, c.2991+1655A>G, Leber's Congenital Amaurosis, Antisense oligonucleotide, RNA therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated \[Study prematurely terminated due to sponsor decision for reasons unrelated to safety\]
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Laboratoires TheaIndustry (pharma, biotech, devices)
- Collaborators
- Sepul Bio
- Study start
- 13 May 2019 (actual)
- Primary completion
- 3 Oct 2022 (actual)
- Study completion
- 3 Oct 2022 (actual)
- First posted
- 12 Apr 2019 (actual)
- Results
- Posted 4 Jan 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Study PQ-110-002 will provide long-term safety, tolerability, pharmacokinetic (PK), and efficacy data of QR-110.
Detailed description
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject.
Subjects will be given the opportunity to enroll into this extension study for continued dosing if available data support current and/or future benefits for the subject. The Investigator, in consultation and agreement with the Medical Monitor, will decide on enrollment of each individual subject, as well as on dosing of the first treated eye and treatment initiation of the contralateral eye. Continued subject treatment in this study is desirable, but cannot be guaranteed, since it will depend on the risks and benefit of further treatment on a case-by-case basis, as discussed and agreed upon with the Medical Monitor.
The contralateral eye and the first treated eye will be injected 3 months apart. The injection interval of 3 months between both eyes will limit burden for the subjects, with a 3 month-visit frequency during the course of the study. This between-eye interval could be adapted if safety data are supportive, and for logistic reasons, and in agreement with the Medical Monitor.
The same safety monitoring protocol and efficacy assessments will apply to both eyes.
QR-110 will be administered via intravitreal (IVT) injection.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Drug QR-110 | Experimental | Drug: QR-110First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Subjects who completed participation in the study PQ-110-001 and who may derive benefit from continued treatment with QR 110, as assessed by the Investigator, in consultation with the Medical Monitor
- Persistence of detectable outer nuclear layer (ONL) in the area of the macula in the opinion of the Investigator, as determined by OCT.
- Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging, as assessed by the Investigator.
- An adult (≥ 18 years) willing and able to provide informed consent for participation OR a minor (6 to < 18 years) with a parent or legal guardian willing and able to provide written permission for the subject's participation prior to performing any study related procedures, and pediatric subjects able to provide age-appropriate assent for study participation.
- Female subjects who have reached menarche and male subjects must either practice true abstinence in accordance with their preferred and usual lifestyle, or agree to use acceptable, highly effective methods of contraception for up to 3 months following their last dose QR-110. Acceptable methods of contraception are defined in the protocol. Women of non-childbearing potential may be included without the use of adequate birth control, provided they meet the criteria in the protocol.
Key exclusion criteria (4 listed)
- Any contraindication to IVT injection according to the Investigator's clinical judgment and international guidelines (Avery 2014).
- Safety issue during study PQ-110-001 that may compromise subject safety when continued dosing, as determined by the Investigator, and in consultation with the Medical Monitor.
- Any ocular or systemic disease or condition (including medications and laboratory test abnormalities) that could compromise subject safety or interfere with assessment of efficacy and safety, as determined by the Investigator and in consultation with the Medical Monitor.
- Pregnant or breast-feeding female.
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 |
|---|---|---|
| QR-110First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration | Drug | Drug QR-110 |
Primary outcome
Frequency of Ocular AEs
Time frame: 24 months
Frequency of ocular adverse events (AEs)
Frequency of Non-ocular AEs
Time frame: 24 months
Frequency of non-ocular AEs
12 secondary outcomes
Change in BCVA in First Treated Eye
Time frame: 24 months
Change in Mobility Course Score
Time frame: 24 months
Change in Photoreceptor Outer Segment Layer Thickness
Time frame: 24 months
Change in OCI
Time frame: 24 months
Change in FST Blue
Time frame: 24 months
Change in FST Red
Time frame: 24 months
Change in VFQ-25
Time frame: 24 months
Change in CVAQ
Time frame: 24 months
Change in PLR
Time frame: 24 months
Change in NIRAF
Time frame: 24 months
Change in BCVA in Treated Contralateral Eye
Time frame: 24 months
Change in BCVA in Non-Treated Contralateral Eye
Time frame: 24 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 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 Chief Medical Officer study director
Sepul Bio
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing QR-110 in people with Leber Congenital Amaurosis 10 and Blindness, run by Laboratoires Thea at 3 sites in 2 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 5 inclusion and 4 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 15 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Belgium. 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?
Yes. Results are posted in the registry (first posted 4 Jan 2023). 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?
Frequency of Ocular AEs (time frame: 24 months). 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 15 Oct 2024; AzTrials last refreshed its copy on 1 Nov 2026.