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Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked Achromatopsia (Colourbridge)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02610582
Recruitment Status : Recruiting
First Posted : November 20, 2015
Last Update Posted : December 8, 2022
Sponsor:
Collaborators:
University Hospital Tuebingen
Ludwig-Maximilians - University of Munich
Information provided by (Responsible Party):
STZ eyetrial

Study Description
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Brief Summary:
The purpose of this study is to proof the safety and efficacy of a single bilateral subretinal injection of rAAV.hCNGA3 in adult and minor patients with CNGA3-linked achromatopsia.

Condition or disease Intervention/treatment Phase
Achromatopsia Drug: rAAV.hCNGA3 Phase 1 Phase 2

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Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 14 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Masking Description: open label
Primary Purpose: Treatment
Official Title: Safety and Efficacy of a Bilateral Single Subretinal Injection of rAAV.hCNGA3 in Adult and Minor Patients With CNGA3-linked Achromatopsia Investigated in a Randomized, Wait List Controlled, Observer-masked Trial
Actual Study Start Date : November 2015
Estimated Primary Completion Date : April 2023
Estimated Study Completion Date : April 2027

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Safety

Arms and Interventions
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Arm Intervention/treatment
Treatment arm
single subretinal injection of 1x10e11 vector genome particles of rAAV.hCNGA3 in each eye at different time-points
 Drug: rAAV.hCNGA3
Single subretinal injection of rAAV.hCNGA3
Waiting group Arm
Waiting group will serve as comparator group first and will receive the treatment at a later timepoint.
 Drug: rAAV.hCNGA3
Single subretinal injection of rAAV.hCNGA3


Outcome Measures
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Primary Outcome Measures :
  1. Safety (AE). Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment [ Time Frame: Day 0 - Day 365 ]
    Safety as the primary endpoint will be assessed by clinical examination of ocular inflammation (slit lamp, fundus biomicroscopy, fundus photography. Systemic safety will be assessed by vital signs, routine clinical chemistry testing (including CRP, ESR) and full/differential blood counts. Immunopathology essays will include specific enzyme-linked immunosorbent assays for humoral antibodies against rAAV8 capsid protein. Biodistribution will be monitored by polymerase chain reaction studies on rAAV8 genome in blood, urine, saliva and tear fluid.


Secondary Outcome Measures :
  1. Efficacy measures. Number of Participants With improved Visual Function. [ Time Frame: Day 14 - Day 365 ]
    The investigation of treatment effects as reflected by patient/parent reported outcomes and the efficacy of the intervention on visual function, as well as the evaluation of retinal imaging (safety) are secondary aims of the trial.


Eligibility Criteria
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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria (Both Eyes)

  • clinical diagnosis of achromatopsia
  • 6-12 years of age
  • ≥ 18 years of age
  • confirmed mutation in CNGA3
  • BCVA ≥ 20/400
  • a minimal outer nuclear layer thickness of 10µm at 3° eccentricity (normal = 38±6µm)
  • ability to understand and willingness to consent to study protocol
  • no infection with Human Immundeficiency Virus (HIV)
  • Male patients must agree to use condoms during the first 6 months post treatment.
  • Female patients of childbearing potential must agree to use an effective method of birth control during the first 6 months post treatment.
  • negative pregnancy test in women with childbearing potential (a woman who is two years post-menopausal or surgically sterile is not considered to be of childbearing potential)

Exclusion Criteria

  • any other retinopathy due to other diseases e.g. (but not limited to) arterial hypertension, trauma or acquired inflammatory diseases (uveitis serology) , retinopathy of the premature
  • systemic conditions (e.g. coronary heart disease, congenital/genetic conditions, autoimmune disorders) which may affect study participation or outcome measures
  • current or recent participation in other study or administration of biologic agent within the last three months
  • recent (6 months) ocular surgery, intravitreal or subretinal implantation of a medical device
  • known sensitivity to any compound used in the study
  • contraindications to systemic immunosuppression
  • subject/partner of childbearing potential unwilling to use adequate contraception for six months after dosing
  • nursing or pregnant female subject women
  • any other cause that, in the investigator's opinion, renders potential subjects not suitable for the study
  • mutations in another achromatopsia gene
  • contraindications in view of the planned surgery (e.g. anaemia Hb<8g/dl, severe coagulopathy, severe blood pressure fluctuations) including intolerance and contraindications to general anaesthesia
  • ocular opacity and mature cataract
  • ocular infection with herpes simplex virus in medical history
  • history of ocular malignancies
  • disorders of the internal retina (e.g. retinal detachment in the patients history)
  • glaucoma defined as damage of the optic nerve
  • vascular retinal occlusion
  • history of poorly controlled (HbA1c > 7%) Diabetes Mellitus type 1 or type 2
  • patients treated with systemic corticoids within 14 days prior inclusion
  • systemic illness or medically significant abnormal laboratory values >3 UNL in blood analysis including renal and hepatic functions at inclusion
  • absence of vision on the other contralateral eye
  • contraindication to pharmacological mydriasis (e.g. history of angle block glaucoma)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02610582


Contacts
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Contact: Barbara J Wilhelm, Prof. +49 7071 2984898 Barbara.Wilhelm@stz-eyetrial.de
Contact: Dominik Fischer, Prof. +49 7071 298 37 21 dominik.fischer@uni-tuebingen.de

Locations
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Germany
University Hospital Tuebingen, Center for Ophthalmology Recruiting
Tuebingen, Germany, 72076
Contact: Manuel D Fischer, MD, PhD    ++40 7071 29 84894    dominik.fischer@med.uni-tuebingen.de   
Contact: Barbara J Wilhelm, MD, PhD    ++40 7071 29 84898    barbara.wilhelm@stz-eyetrial.de   
Sponsors and Collaborators
STZ eyetrial
University Hospital Tuebingen
Ludwig-Maximilians - University of Munich
Investigators
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Principal Investigator: Dominik Fischer, Prof. University Hospital Tuebingen, Center for Ophthalmology
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: STZ eyetrial
ClinicalTrials.gov Identifier: NCT02610582    
Other Study ID Numbers: RDC-CNGA3-01
2014-001874-32 ( EudraCT Number )
096/2015 AMG1 ( Other Identifier: Ethics Committee Tübingen )
First Posted: November 20, 2015    Key Record Dates
Last Update Posted: December 8, 2022
Last Verified: December 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by STZ eyetrial:
CNGA3-linked
Additional relevant MeSH terms:
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Color Vision Defects
Vision Disorders
Sensation Disorders
Neurologic Manifestations
 Nervous System Diseases
Cone Dystrophy
Eye Diseases, Hereditary
Eye Diseases