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OPTIC: ADVM-022 Intravitreal Gene Therapy for Wet AMD

Sponsor
Adverum Biotechnologies, Inc. (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03748784
Collaborator
(none)
30
Enrollment
11
Locations
2
Arms
42.5
Anticipated Duration (Months)
2.7
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

ADVM-022 (AAV.7m8-aflibercept) is a gene therapy product developed for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is a serious condition and the leading cause of blindness in the elderly. The available therapies for treating wet AMD require life-long intravitreal (IVT) injections every 4-12 weeks to maintain efficacy. A one-time IVT administration of ADVM-022 has the potential to treat wet AMD by providing durable expression of therapeutic levels of intraocular anti-VEGF protein (aflibercept) and maintaining the vision of patients. ADVM-022 is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with wet AMD receiving anti-VEGF therapy in clinical practice.

Condition or Disease Intervention/Treatment Phase
  • Biological: ADVM-022
 Phase 1

Detailed Description

This open-label, multicenter, dose-ranging study will evaluate 2 dose levels in up to 30 subjects (15 per dose) with active choroidal neovascularization (CNV) secondary to AMD. Subjects who are under active anti-VEGF treatment and have demonstrated a meaningful response to anti-VEGF therapy will be considered for participation in this study. The primary endpoint for this study is safety and tolerability of ADVM-022. All subjects will continue to be assessed for 104 weeks following treatment with ADVM-022.

Study Design

Study Type:
Interventional
Actual Enrollment :
30 participants
Allocation:
Non-Randomized
Intervention Model:
Sequential Assignment
Intervention Model Description:
Open-label, dose-ranging clinical study to evaluate the safety and tolerability of ADVM-022 in subjects with wet AMD.Open-label, dose-ranging clinical study to evaluate the safety and tolerability of ADVM-022 in subjects with wet AMD.
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
An Open Label Phase 1 Study of ADVM-022 (AAV.7m8-aflibercept) in Neovascular (Wet) Age-Related Macular Degeneration
Actual Study Start Date :
Nov 14, 2018
Anticipated Primary Completion Date :
Jun 1, 2022
Anticipated Study Completion Date :
Jun 1, 2022

Arms and Interventions

Arm Intervention/Treatment
Experimental: Dose 1

6E11 vg of ADVM-022

Biological: ADVM-022
ADVM-022 (AAV.7m8-aflibercept) is a recombinant, replication-deficient adeno-associated virus (AAV.7m8) gene therapy vector carrying a coding sequence for aflibercept
Other Names:
  • AAV.7m8-aflibercept

    		•
    Experimental: Dose 2

    2E11 vg of ADVM-022

    Biological: ADVM-022
    ADVM-022 (AAV.7m8-aflibercept) is a recombinant, replication-deficient adeno-associated virus (AAV.7m8) gene therapy vector carrying a coding sequence for aflibercept
    Other Names:
  • AAV.7m8-aflibercept

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Type, severity, and incidence of ocular and systemic adverse events (AEs) [104 weeks]

      Type, severity, and incidence of ocular and systemic adverse events (AEs)

    Secondary Outcome Measures

    1. Change in best corrected visual acuity (BCVA) [104 weeks]

      Change in best corrected visual acuity (BCVA)

    2. Change in central subfield thickness (CST) and macular volume measured by SD-OCT [104 weeks]

      Change in central subfield thickness (CST) and macular volume measured by SD-OCT

    3. Percentage of subjects requiring anti-VEGF injections over time [104 weeks]

      Percentage of subjects requiring anti-VEGF injections over time

    4. Mean number of anti-VEGF injections over time [104 weeks]

      Mean number of anti-VEGF injections over time

    5. Percentage of subjects without intraretinal fluid over time [104 weeks]

      Percentage of subjects without intraretinal fluid over time

    6. Percentage of subjects without subretinal fluid over time [104 weeks]

      Percentage of subjects without subretinal fluid over time

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    50 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Age ≥ 50

    • Diagnosis of neovascular (wet) AMD

    • BCVA ETDRS Snellen equivalent between ≤20/32 and ≥20/320 for each cohort

    • Subjects must be under active anti-VEGF treatment for wAMD and received a minimum of 2 injections within 4 months prior to screening

    • Demonstrated a meaningful response to anti-VEGF therapy

    • Willing and able to provide consent

    Exclusion Criteria:

    • History of retinal disease in the study eye other than wet AMD

    • Fibrosis or atrophy, retinal epithelial tear in the center of the fovea in the study eye, or any condition preventing visual acuity improvement

    • History of retinal detachment (with or without repair) in the study eye

    • History of vitrectomy, trabeculectomy, or other filtration surgery in the study eye

    • Uncontrolled glaucoma in the study eye

    • Any prior treatment with photodynamic therapy or retinal laser for the treatment of wet AMD and any previous therapeutic radiation in the region of the study eye

    • Any previous intraocular or periocular surgery on the study eye within 6 months

    • Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months

    • Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Adverum Clinical Site Bakersfield California United States 93309
    2 Adverum Clinical Site Beverly Hills California United States 90211
    3 Adverum Clinical Site Golden Colorado United States 80401
    4 Adverum Clinical Site Deerfield Beach Florida United States 33064
    5 Adverum Clinical Site Reno Nevada United States 89502
    6 Adverum Clinical Site Philadelphia Pennsylvania United States 19107
    7 Adverum Clinical Site West Columbia South Carolina United States 29169
    8 Adverum Clinical Site Nashville Tennessee United States 37203
    9 Adverum Clinical Site Abilene Texas United States 79606
    10 Adverum Clinical Site Houston Texas United States 77030
    11 Adverum Clinical Site The Woodlands Texas United States 77384

    Sponsors and Collaborators

    • Adverum Biotechnologies, Inc.

    Investigators

    • Study Chair: OPTIC Medical Monitor, Adverum Biotechnologies, Inc.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Adverum Biotechnologies, Inc.
    ClinicalTrials.gov Identifier:
    NCT03748784
    Other Study ID Numbers:
    • ADVM-022-01
    First Posted:
    Nov 21, 2018
    Last Update Posted:
    Mar 22, 2022
    Last Verified:
    Mar 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Adverum Biotechnologies, Inc.
    Choroidal Neovascularizatio
    ADVM-022
    CNV
    ADVM-022-01
    AAV.7m8
    Anti-VEGF therapy
    Blindness
    Gene therapy
    Aflibercept (Eylea)
    Age-Related Macular Degeneration
    Wet Macular Degeneration
    Retinal Degeneration
    Retinal Diseases
    Eye Diseases
    AAV Vector
    Adverum
    wAMD
    AMD
    wet AMD
    Additional relevant MeSH terms:
    Macular Degeneration
    Aflibercept

    Study Results

    No Results Posted as of Mar 22, 2022