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TerminatedNCT03913130INSIGHTUpdated Oct 15, 2024Results posted

Extension Study to Study PQ-110-001 (NCT03140969)

A Phase 1/2 interventional study of QR-110 in Leber Congenital Amaurosis 10, Blindness and Leber Congenital Amaurosis, sponsored by Laboratoires Thea. Terminated at 3 sites in 2 countries. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2024-10-15.

Sponsored by Laboratoires Thea · Phase 1/2, Interventional, and Treatment

Why this study was terminated
Terminated \[Study prematurely terminated due to sponsor decision for reasons unrelated to safety\]
Phase
Phase 1/2
Study type
Interventional
Enrollment
9
Allocation
Not applicable
Ages
6 Years and older
Sex
All
01

Study 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. 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.

02

Conditions studied

  • Leber Congenital Amaurosis 10
  • Blindness
  • Leber Congenital Amaurosis
  • Vision Disorders
  • Sensation Disorders
  • Neurologic Manifestations
  • Eye Diseases
  • Eye Diseases, Hereditary
  • Eye Disorders Congenital
  • Retinal Disease

Keywords

  • LCA10
  • CEP290
  • p.Cys998X
  • c.2991+1655A>G
  • Leber's Congenital Amaurosis
  • Antisense oligonucleotide
  • RNA therapy
03

In context

Blindness

144 studies on the registry are indexed under Blindness; 33 are open to participants now.

This study's enrollment of 9 is below the median of 37 across 101 interventional studies indexed under Blindness.

Browse Blindness studies →

Lead sponsor

Laboratoires Thea is the lead sponsor of 64 studies on the registry; 6 are open to participants now.

Of its 8 completed or terminated interventional studies of FDA-regulated products, 6 (75%) have results posted.

Browse Laboratoires Thea studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • 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 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 for up to 3 months following their last dose QR-110. Acceptable methods of contraception are defined in the protocol. Women of non- may be included without the use of adequate birth control, provided they meet the criteria in the protocol.

Exclusion criteria

Exclusion Criteria:

  • Any 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.
Terms in these criteria, in plain language(4)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraindication

A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.

05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
9 participants (actual)

Study arms

  • Experimental
    Drug QR-110

    First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration

    Drug: QR-110

Interventions

  • DrugQR-110

    First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration

06

What researchers measure

Primary outcomes

  1. Frequency of Ocular AEs

    Frequency of ocular adverse events (AEs)

    Time frame: 24 months

  2. Frequency of Non-ocular AEs

    Frequency of non-ocular AEs

    Time frame: 24 months

Secondary outcomes

  1. Change in BCVA in First Treated Eye

    Change in Best Corrected Visual Acuity (BCVA) in First Treated Eye

    Time frame: 24 months

  2. Change in Mobility Course Score

    Change in Mobility course score

    Time frame: 24 months

  3. Change in Photoreceptor Outer Segment Layer Thickness

    Change in photoreceptor outer segment layer thickness by Optical Coherence Tomography (OCT)

    Time frame: 24 months

  4. Change in OCI

    Change in Oculomotor Instability (OCI)

    Time frame: 24 months

  5. Change in FST Blue

    Change in Full-Field Stimulus Testing (FST) - blue stimuli

    Time frame: 24 months

  6. Change in FST Red

    Change in Full-Field Stimulus Testing (FST) - red stimuli

    Time frame: 24 months

  7. Change in VFQ-25

    Change in Visual Function Questionnaire-25 (VFQ-25) score (adult subjects)

    Time frame: 24 months

  8. Change in CVAQ

    Change in Cardiff Visual Ability Questionnaire for Children (CVAQC) score (pediatric subjects)

    Time frame: 24 months

  9. Change in PLR

    Change in Pupillary Light Reflex (PLR) (latency and amplitude)

    Time frame: 24 months

  10. Change in NIRAF

    Change in Near Infrared AutoFluorescence (NIRAF)

    Time frame: 24 months

  11. Change in BCVA in Treated Contralateral Eye

    Change in Best Corrected Visual Acuity (BCVA) in Treated Contralateral Eye

    Time frame: 24 months

  12. Change in BCVA in Non-Treated Contralateral Eye

    Change in Best Corrected Visual Acuity (BCVA) in Non-Treated Contralateral Eye

    Time frame: 24 months

07

Results

Posted Jan 4, 2023

Participant flow

Participant flow — Overall Study
MilestoneDrug QR-110
Started9
Completed8
Not completed1
Withdrew: Sponsor decision1

Outcome measures

PrimaryFrequency of Ocular AEs

Frequency of ocular adverse events (AEs)

Time frame:
24 months
Reported as:
Count of participants · Participants
Frequency of Ocular AEs
ParticipantsDrug QR-110
Frequency of Ocular AEs7
PrimaryFrequency of Non-ocular AEs

Frequency of non-ocular AEs

Time frame:
24 months
Reported as:
Count of participants · Participants
Frequency of Non-ocular AEs
ParticipantsDrug QR-110
Frequency of Non-ocular AEs6
SecondaryChange in BCVA in First Treated Eye

Change in Best Corrected Visual Acuity (BCVA) in First Treated Eye

Time frame:
24 months
Reported as:
Mean · logMAR
Change in BCVA in First Treated Eye
logMARDrug QR-110
Change in BCVA in First Treated Eye0.03 ± 0.17
SecondaryChange in Mobility Course Score

Change in Mobility course score

Time frame:
24 months

Results for this outcome have not been posted.

SecondaryChange in Photoreceptor Outer Segment Layer Thickness

Change in photoreceptor outer segment layer thickness by Optical Coherence Tomography (OCT)

Time frame:
24 months

Results for this outcome have not been posted.

SecondaryChange in OCI

Change in Oculomotor Instability (OCI)

Time frame:
24 months

Results for this outcome have not been posted.

SecondaryChange in FST Blue

Change in Full-Field Stimulus Testing (FST) - blue stimuli

Time frame:
24 months

Results for this outcome have not been posted.

SecondaryChange in FST Red

Change in Full-Field Stimulus Testing (FST) - red stimuli

Time frame:
24 months

Results for this outcome have not been posted.

SecondaryChange in VFQ-25

Change in Visual Function Questionnaire-25 (VFQ-25) score (adult subjects)

Time frame:
24 months

Results for this outcome have not been posted.

SecondaryChange in CVAQ

Change in Cardiff Visual Ability Questionnaire for Children (CVAQC) score (pediatric subjects)

Time frame:
24 months

Results for this outcome have not been posted.

SecondaryChange in PLR

Change in Pupillary Light Reflex (PLR) (latency and amplitude)

Time frame:
24 months

Results for this outcome have not been posted.

SecondaryChange in NIRAF

Change in Near Infrared AutoFluorescence (NIRAF)

Time frame:
24 months

Results for this outcome have not been posted.

SecondaryChange in BCVA in Treated Contralateral Eye

Change in Best Corrected Visual Acuity (BCVA) in Treated Contralateral Eye

Time frame:
24 months
Reported as:
Mean · logMAR
Change in BCVA in Treated Contralateral Eye
logMARDrug QR-110
Change in BCVA in Treated Contralateral Eye-0.11 ± 0.23
SecondaryChange in BCVA in Non-Treated Contralateral Eye

Change in Best Corrected Visual Acuity (BCVA) in Non-Treated Contralateral Eye

Time frame:
24 months
Reported as:
Mean · logMAR
Change in BCVA in Non-Treated Contralateral Eye
logMARDrug QR-110
Change in BCVA in Non-Treated Contralateral Eye0.05 ± 0.21

Adverse events

Collected over Any event/condition noted once the subject receives their first dose of study drug in the contralateral eye will be captured as an AE. All AEs and SAEs regardless of attribution will be collected until at least 3 months following the last administration of study drug or the subject's EOS visit, whichever is later.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Drug QR-1100/9 (0%)1/9 (11.1%)8/9 (88.9%)
Most frequent serious events
Most frequent serious events
EventDrug QR-110
Lumbar vertebral fractureInjury, poisoning and procedural complications1/9
Most frequent other events
Showing 10 of 33
Most frequent other events
EventDrug QR-110
Posterior capsule opacificationEye disorders3/9
Visual acuity reducedEye disorders3/9
Cataract subcapsularEye disorders2/9
Conjunctival haemorrhageEye disorders2/9
Lenticular opacitiesEye disorders2/9
CoughRespiratory, thoracic and mediastinal disorders2/9
Oropharyngeal painRespiratory, thoracic and mediastinal disorders2/9
CataractEye disorders1/9
Cataract corticalEye disorders1/9
Cystoid macular oedemaEye disorders1/9

Baseline characteristics

Age, Customized
Age, Customized(Years)Drug QR-110
Age (Mean)24.7 ± 12.7
Age, Customized
Age, Customized(Years)Drug QR-110
Age (Median)23.0 (10 to 45)
Age, Customized
Age, Customized(Participants)Drug QR-110
<18 years3
>= 18 Years6
Sex: Female, Male
Sex: Female, Male(Participants)Drug QR-110
Female4
Male5
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Drug QR-110
Hispanic or Latino0
Not Hispanic or Latino9
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Drug QR-110
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American0
White9
More than one race0
Unknown or Not Reported0
Weight (Mean)
Weight (Mean)(kg)Drug QR-110
Mean64.26 ± 23.19
Weight (Median)
Weight (Median)(kg)Drug QR-110
Median61.55 (40.1 to 111.2)

5 further baseline measures are reported on the registry.

08

Study locations

3 sites
  • University of Iowa
    Iowa City, Iowa 52242, United States
  • Scheie Eye Institute, University of Pennsylvania
    Philadelphia, Pennsylvania 19104, United States
  • Ghent University Hospital and Ghent University
    Ghent, B-9000, Belgium
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from May 2019 to primary completion in Oct 2022, 40 months.

Results were first posted in Jan 2023, 3 months after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered
Feb 21, 2019
First posted
Apr 12, 2019
StartActual
May 13, 2019
Primary completionActual
Oct 3, 2022
CompletionActual
Oct 3, 2022
Results first posted
Jan 4, 2023
Last update
Oct 15, 2024
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Oct 15, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03913130
Lead sponsor
Laboratoires Thea
Collaborators
Sepul Bio
Responsible party
Sponsor
First posted
Apr 12, 2019
Start date
May 13, 2019
Primary completion
Oct 3, 2022
Completion
Oct 3, 2022
Results posted
Jan 4, 2023
Last update
Oct 15, 2024

Study contacts

Sepul Bio Chief Medical Officer
study director · Sepul Bio

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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