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Prescreening Study to Identify Potential Participants for ACDN-01 Clinical Trials

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ClinicalTrials.gov Identifier: NCT06445322
Recruitment Status : Recruiting
First Posted : June 6, 2024
Last Update Posted : June 6, 2024
Sponsor:
Information provided by (Responsible Party):
Ascidian Therapeutics, Inc

Brief Summary:
This is an observational prescreening study. Individuals who are eligible for prescreening will undergo testing procedures that may be used to determine eligibility in ACDN-01 clinical trials.

Condition or disease Intervention/treatment
Stargardt Disease Stargardt Disease 1 Cone Rod Dystrophy Juvenile Macular Degeneration Diagnostic Test: Prescreening Assessments

Detailed Description:
The prescreening process will be used to help determine the initial eligibility and interest of potential participants in ACDN-01 clinical trials by conducting assessments of key eligibility criteria before the clinical trial screening procedures are performed.

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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials
Estimated Study Start Date : May 30, 2024
Estimated Primary Completion Date : August 31, 2027
Estimated Study Completion Date : August 31, 2030


Group/Cohort Intervention/treatment
Prescreening Group
The prescreening study consists of genetic and visual assessments and will require at least 1 onsite visit. All clinical assessments performed are for the purpose of determining research eligibility for ACDN-01 clinical trials.
Diagnostic Test: Prescreening Assessments
Various genetic and visual assessments.




Primary Outcome Measures :
  1. Confirm mutations in the ABCA4 gene [ Time Frame: 12 months ]
    Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.

  2. Confirm the absence of pathogenic mutations in genes known to cause retinal disease other than ABCA4-related retinopathy [ Time Frame: 12 months ]
    Using a Clinical Laboratory Improved Amendments (CLIA)-certified laboratory.

  3. Measure BCVA and LLVA [ Time Frame: 12 months ]
    Measure best corrected visual acuity and low luminance visual acuity

  4. Measure the area of retinal atrophy [ Time Frame: 12 months ]
    Using FAF imaging

  5. Measure baseline retinal structure [ Time Frame: 12 months ]
    Using OCT (SD-OCT)

  6. Historical FAF or OCT images [ Time Frame: 4 years ]
    Confirm historical timepoint images

  7. Historical BCVA/LLVA measurements [ Time Frame: 4 years ]
    Collect past measurements



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Males and females with a diagnosis of ABCA4-related retinopathy will be invited to give informed consent prior to prescreening procedures. After appropriate informed consent/assent has been obtained, potential participants will then undergo the assessments to determine eligibility for ACDN-01 clinical trials.
Criteria

Key Inclusion Criteria:

  • Presence of mutations in the ABCA4 gene
  • ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)

Key Exclusion Criteria:

  • The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy
  • Retinal disease other than ABCA4-related retinopathy
  • Presence of a medical condition (systemic or ophthalmic), psychiatric condition, including substance abuse disorder, or physical examination or laboratory finding that may in the opinion of the principal investigator and sponsor preclude adherence to the scheduled study visits, safe participation in the study, or affect the results of the study.

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): NCT06445322


Contacts
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Contact: Associate Director, Clinical Operations 207-573-0412 researchtrials@ascidian-tx.com

Locations
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United States, Florida
Vitreo Retinal Associates Recruiting
Gainesville, Florida, United States, 32607
United States, Ohio
Cincinnati Eye Institute Recruiting
Cincinnati, Ohio, United States, 45245
United States, Texas
Retina Foundation of Texas Recruiting
Dallas, Texas, United States, 75382
Retina Consultants of Texas Not yet recruiting
Houston, Texas, United States, 77401
Sponsors and Collaborators
Ascidian Therapeutics, Inc
Investigators
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Study Director: Alia Rashid Ascidian Therapeutics
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Responsible Party: Ascidian Therapeutics, Inc
ClinicalTrials.gov Identifier: NCT06445322    
Other Study ID Numbers: ACDN-01-000
First Posted: June 6, 2024    Key Record Dates
Last Update Posted: June 6, 2024
Last Verified: May 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ascidian Therapeutics, Inc:
Gene Therapy
ABCA4
ABCA4-related retinopathy
Stargardt Disease
Stargardt macular dystrophy
Cone rod dystrophy
Gene editing
RNA
Exon editing
IRD
Inherited retinal disease
Inherited retinal dystrophy
Inherited retinal degeneration
Additional relevant MeSH terms:
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Macular Degeneration
Stargardt Disease
Cone-Rod Dystrophies
Retinal Degeneration
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Retinal Dystrophies