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Trial record 1 of 1 for:    HMR-1002
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AAVCAGsCD59 for the Treatment of Wet AMD

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03585556
Recruitment Status : Active, not recruiting
First Posted : July 13, 2018
Last Update Posted : January 31, 2020
Sponsor:
Information provided by (Responsible Party):
Hemera Biosciences

Tracking Information
First Submitted Date  ICMJE July 1, 2018
First Posted Date  ICMJE July 13, 2018
Last Update Posted Date January 31, 2020
Actual Study Start Date  ICMJE September 13, 2018
Actual Primary Completion Date January 29, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 4, 2019)
Number of intravitreal anti-VEGF injections [ Time Frame: 12 months ]
Measure the number of intravitreal anti VEGF injections from Month 1 through Month 12 following an intravitreal injection of AAVCAGsCD59 at Day 7
Original Primary Outcome Measures  ICMJE
 (submitted: July 12, 2018)
Number of intravitreal anti-VEGF injections [ Time Frame: 12 months ]
from Month 1 through Month 12 following an intravitreal injection of AAVCAGsCD59 at Day 7
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 4, 2019)
  • Number of patients with change in vision of ≥ 15 letters [ Time Frame: 12 months ]
    Evaluate the change in vision in eyes receiving intravitreal AAVCAGsCD59 in conjunction with anti VEGF for new onset wet AMD
  • Number of patients with inflammation, endophthalmitis, IOP>30, retinal detachment, cataract, and systemic adverse events [ Time Frame: 12 months ]
    Evaluate SAEs associated with the use of intravitreal AAVCAGsCD59
Original Secondary Outcome Measures  ICMJE
 (submitted: July 12, 2018)
  • Number of patients with change in vision of ≥ 15 letters [ Time Frame: 12 months ]
  • Number of patients with inflammation, endophthalmitis, IOP>30, retinal detachment, cataract, and systemic adverse events [ Time Frame: 12 months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE AAVCAGsCD59 for the Treatment of Wet AMD
Official Title  ICMJE A Phase 1 Proof of Concept Study Evaluating Intravitreal AAVCAGsCD59 for the Treatment of Wet Age-Related Macular Degeneration (AMD)
Brief Summary Patients with treatment naive wet AMD will receive an intravitreal anti-VEGF injection at Day 0 followed by an intravitreal injection of AAVCAGsCD59 at Day 7. Patients will be followed monthly through Month 12 and receive an intravitreal anti-VEGF injection as needed based on an increase in central subfoveal thickness (CST) of >50 micrometers on OCT from Day 0, new subretinal hemorrhage on clinical exam, and/or loss of 10 or more ETDRS letters from the previous month exam.
Detailed Description

This is a Phase 1, multi-center, open label study to assess the efficacy and safety of two doses of the adeno-associated viral vector serotype 2 (AAVCAGsCD59) expressing sCD59 administered via intravitreal injection seven days after a single intravitreal injection of anti-VEGF. All patients considered for enrollment in this study must have treatment naive wet AMD, adequate pupillary dilation to permit a thorough ocular exam, and best corrected distance visual acuity in the study eye of 20/25 to 20/400 using the Snellen eye chart.

Written informed consent will be obtained from each study patient prior to his/her participation in any study related procedures. Screening will determine patient eligibility for the study according to written inclusion and exclusion criteria, which include both general medical and AMD-specific criteria. Patients will be enrolled into the study upon verification that they fulfill all eligibility criteria and after completion of all screening assessments.

This study consists of a screening and injection of anti-VEGF (Day 0), injection of AAVCAGsCD59 (Day 7), and a monthly follow-up period (Month 1 through Month 12) where enrolled patients are treated as needed with intravitreal anti-VEGF based on an increase in central subfoveal thickness of >50 micrometers on OCT from Day 0, new subretinal hemorrhage, a decrease in > or equal to 10 ETDRS letters from the previous exam, or as needed based on the treating clinician. The purpose of the study is to evaluate the number of anti-VEGF injections that are required after a single intravitreal injection of AAVCAGsCD59 at a dose of 3.56x10e11vg for subjects 1-22 and 1.071x10e12vg for subjects 23-25 is administered on Day 7. Anti-VEGF will be injected at Day 0 to treat the CNV per standard of care and enable the AAVCAGsCD59 adequate time (up to two weeks) to enter the ganglion cells in the retina and start producing the transgene product, sCD59. Up to twenty-five (25) patients will be enrolled at to 2 clinical sites in this study.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
All patients with treatment naive, new onset wet AMD will be treated with an intravitreal anti-VEGF at Day 0 followed by intravitreal AAVCAGsCD59 at Day 7. Patients will be followed for 12 months and treated with additional anti-VEGF monthly as needed if recurrent disease is identified.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Wet Age-related Macular Degeneration
Intervention  ICMJE
  • Drug: Intravitreal anti-VEGF
    Intravitreal injection of anti-VEGF at Day 0 then monthly as needed
    Other Names:
    • Bevacizumab (Avastin)
    • Ranibizumab (Lucentis)
    • Aflibercept (Eylea)
  • Biological: Intravitreal AAVCAGsCD59
    An intravitreal injection of AAVCAGsCD59 will occur at Day 7 once
    Other Name: HMR59
  • Drug: Oral prednisolone
    A 7 day tapering dose of oral prednisone will be taken at Day 30 by all patients
    Other Name: oral corticosteroid
Study Arms  ICMJE Experimental: AAVCAGsCD59 Treated Arm
An anti-VEGF injection will be given at Day 0 followed by an intravitreal injection of AAVCAGsCD59 at Day 7. All eyes will then be treated with intravitreal anti-VEGF monthly as needed based on disease activity.
Interventions:
  • Drug: Intravitreal anti-VEGF
  • Biological: Intravitreal AAVCAGsCD59
  • Drug: Oral prednisolone
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: January 6, 2019)
25
Original Estimated Enrollment  ICMJE
 (submitted: July 12, 2018)
10
Estimated Study Completion Date  ICMJE February 6, 2021
Actual Primary Completion Date January 29, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Men or women 50 years of age or older.
  2. Treatment naive Wet AMD with no evidence of subretinal fibrosis under the fovea.
  3. Presence of intraretinal and/or subretinal fluid on OCT.
  4. Best corrected visual acuity (BCVA) Snellen equivalent 20/25 to 20/400 in the study eye using ETDRS charts at a starting distance of 4m.
  5. Adequate pupillary dilation to permit ocular examination and testing.
  6. Ability and willingness to return for all scheduled visits and assessments.
  7. Understand and comply with the clinical protocol and provide written informed consent prior to any study-related procedure.
  8. All fertile men must be willing to use barrier contraception during the study.
  9. Women of childbearing potential must have a negative pregnancy test and agree to use effective contraception for the duration of the trial. A woman of childbearing potential is defined as any female who has had menses within the last two years or has not undergone a hysterectomy or surgical sterilization.

Exclusion Criteria:

  1. Wet AMD secondary to non-AMD etiologies.
  2. Subretinal hemorrhage that interferes with the ability to adequately measure visual acuity or follow retinal or subretinal fluid collection on OCT.
  3. Serous pigment epithelial detachment (PED) that is >50% of the CNV lesion, >400µm in any diameter, or presence of a RPE tear.
  4. Presence of polypoidal choroidal vasculopathy (PCV), retinal angiomatous proliferation (RAP), central serous retinopathy, or symptomatic vitreomacular adhesion.
  5. Previous macular laser photocoagulation for CNV, photodynamic therapy (PDT), ocular radiation, or subretinal surgery for CNV in the study eye.
  6. History of conditions in the study eye which might alter visual acuity or interfere with study testing including clinically significant macular edema, central retinal vein occlusion, macular branch retinal vein occlusion, and optic neuropathy.
  7. Active uncontrolled glaucoma with IOP>30 mmHg despite treatment with glaucoma medications, cup-to-disc ratio of >0.9, visual field defects secondary to glaucoma that involve the macula, and optic atrophy from glaucoma.
  8. Likely candidate for intraocular surgery (including cataract surgery) in the study eye during the clinical trial.
  9. Acute or chronic infection in the study eye.
  10. History of uveitis unrelated to eye surgery in the study eye or opposite eye requiring treatment with topical corticosteroids or systemic immunosuppression within 24 months of enrollment.
  11. Any contraindication to intravitreal injection.
  12. Use intravitreal (study eye) corticosteroids within 3 months prior to screening.
  13. Any of the following underlying systemic diseases:

    • Unstable or severe cardiovascular disease, e.g., congestive heart failure (New York Heart Association Functional class III or IV), myocardial infarction within 6 months, ventricular tachyarrhythmias requiring ongoing treatment, unstable angina, or critical limb ischemia;
    • Cerebrovascular disease within 12 months prior to Screening that impairs the patient's ability to participate in the clinical trial;
    • Dementia or neurodegenerative disease (e.g., Alzheimer's disease, Parkinson's disease) of a level that prevents adequate evaluation of the subject during the study;
    • Has an active malignancy or is currently undergoing treatment for an active malignancy at Screening, or has a history of malignancy that precludes completion of this 12-month study;
    • Immunocompromised conditions and/or need for immunosuppressive therapy
  14. Any significant poorly controlled illness that would preclude study compliance and follow-up
  15. Current or prior use of any medication known to be toxic to the retina or optic nerve including, but not limited, to chloroquine/hydrochloroquine, deferoxamine, phenothiazines and ethambutol
  16. Previous treatment with any ocular or systemic gene transfer product
  17. Received any investigational product within 120 days prior to screening
  18. Any psychological, familial, sociological, geographical, or other condition that would preclude study compliance and follow-up
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 50 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03585556
Other Study ID Numbers  ICMJE HMR-1002
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Hemera Biosciences
Study Sponsor  ICMJE Hemera Biosciences
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Hemera Biosciences
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP