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Dry Age-related Macular DegenerationPhase 1NCT03144999

Treatment of Advanced Dry Age Related Macular Degeneration With AAVCAGsCD59

A Phase 1 study testing AAVCAGsCD59 in people with Dry Age-related Macular Degeneration, run by Janssen Research & Development, LLC at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Age related macular degeneration (AMD) is the leading cause of vision loss in individuals over age 60. AMD is classified as wet and dry. Wet AMD constitutes 10 to 15% of all cases of AMD and occurs when an abnormal blood vessel grows in or under the retina leading to central vision loss. Read more about this study

Who is it for?
Ages 50 years and older
Conditions the study is for
Dry Age-related Macular Degeneration
What is being tested?
AAVCAGsCD59
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
17 participants
Where
1 siteUnited States
Study dates
29 Mar 2017to 9 Dec 2019
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Men or women 50 years of age or older
  • Advanced dry AMD with GA in the study eye
  • BCVA Snellen equivalent of 20/80 or worse in the study eye using ETDRS charts at a starting distance of 4m after the first 3 patients are enrolled and demonstrate favorable safety data
  • Total GA lesion size 5mm2 (2 DA) to 20mm2 (8 DA) in the study eye; if multifocal, then at least one focus of GA needs to measure 1.27 mm2 (0.5 DA)
  • Fellow eye BCVA of 20/800 or better and must be the eye with the better visual acuity
  • Must be willing to undergo paracentesis of the anterior chamber

Who cannot join

  • GA secondary to non AMD etiologies
  • Prior or active choroidal neovascularization (CNV) in the study eye
  • History of conditions in the study eye during screening which might alter visual acuity or interfere with study testing
  • Active uncontrolled glaucoma
  • Intraocular surgery in the study eye within 3 months of enrollment or are known or likely candidate for intraocular surgery (including cataract surgery) in the study eye within 1 year of treatment
  • Acute or chronic infection in the study eye
Show all 24Show fewer
  • History of inflammation in the study eye or ongoing inflammation in either eye
  • History of uveitis in the study eye
  • Ongoing ocular inflammation in either eye
  • Any contraindication to intravitreal injection
  • Currently using or have used treatment for exudative AMD in the study eye only: laser photocoagulation, photodynamic therapy (PDT), ranibizumab (Lucentis®), pegaptanib sodium (Macugen®), bevacizumab (Avastin®) or aflibercept (Eylea®)
  • Currently using any periocular (study eye), intravitreal (study eye) or systemic (oral or intravenous) corticosteroids within 3 months prior to screening.
  • 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;
  • Poorly controlled diabetes;
  • Clinically significant impaired renal or hepatic function, for example:
  • Cerebrovascular disease within 12 months prior to Screening;
  • Dementia or neurodegenerative disease (e.g., Alzheimer's disease, Parkinson's disease);
  • Have a malignancy at Screening or a history of malignancy that precludes completion of this 2-year study, including presence of tumor or disease whose treatment may directly affect the ability to evaluate the safety and efficacy of AAVCAGsCD59, or currently undergoing treatment for a specific cancer;
  • Immunocompromised conditions and/or need for immunosuppressive therapy;
  • Any significant poorly controlled illness that would preclude study compliance and follow-up
  • 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
  • Previous treatment with any ocular or systemic gene transfer product
  • Received any investigational product within 120 days prior to screening

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Ophthalmic Consultants of Boston

    Boston, Massachusetts, 02114

Other trials in United States

About this study

Official title: A Phase 1, Open-Label, Multi-Center, Dose-Escalating, Safety and Tolerability Study of a Single Intravitreal Injection of AAVCAGsCD59 in Patients With Advanced Non-Exudative (Dry) Age-Related Macular Degeneration With Geographic Atrophy

Age related macular degeneration (AMD) is the leading cause of vision loss in individuals over age 60. AMD is classified as wet and dry. Wet AMD constitutes 10 to 15% of all cases of AMD and occurs when an abnormal blood vessel grows in or under the retina leading to central vision loss. Wet AMD is successfully treated with injections in the eye on a monthly basis that stop the blood vessel from growing and leaking. The most common form of AMD is the dry variant or dry AMD that affects 85 to 90% of all patients with AMD. In dry AMD, there is loss of retinal pigment, formation of deposits called drusen, and loss of the vessels in a layer of the retina called the choriocapillaris. In the most severe forms of dry AMD there is loss of retinal tissue called geographic atrophy. Over time retinal tissue degenerates in the area responsible for central vision leading to vision loss leading to legal blindness. Currently no treatment for dry AMD exists so that there is a significant unmet need in patients with this ocular disease.

Recently, evidence has implicated an overactive inflammatory cascade called the complement system as playing a pivotal role in the development of dry AMD. The complement cascade consists of 3 arms that converge to form a pore-like complex on the surface of cells called the membrane attack complex (MAC). Accumulation of MAC on cell surfaces leads to cell damage and death causing the clinical findings seen in AMD. Normal cells within the human body produce a protein on their cell surfaces called CD59 that blocks the MAC from forming. In AMD, the complement cascade is upregulated and leads to more MAC formation than the body can protect itself against leading to cell destruction.

AAVCAGsCD59, an ocular gene therapy product that is injected in to the eye in the physician's office, causes normal retinal cells to increase the expression of a soluble form of CD59 (sCD59). This soluble recombinant version of the naturally occurring CD59 is designed and intended to protect retinal cells that are responsible for central vision by inhibiting the formation of the membrane attack complex (MAC), the terminal step of complement-mediated cell lysis. In gene therapy the cells of the retina are potentially permanently altered to make sCD59 for the life of the patient. With gene therapy only one injection is needed for the drug to be effective for the patient's entire life. This study will evaluate the safety after a single injection of AAVCAGsCD59 administered in an office setting for patients whose enrolled eye has advanced dry AMD with geographic atrophy. The initial study is 26 weeks followed by an additional 18-month safety evaluation.

Being tested
AAVCAGsCD59
Keywords
Dry AMD, Dry Age-related Macular Degeneration, Gene Therapy, Complement, Intravitreal Injection, Geographic Atrophy, Drusen, Membrane Attack Complex, CD59
Medical subject headings
Geographic Atrophy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AAVCAGsCD59Biological

    Also called HMR59

    AAVCAGsCD59 is administered as a single intravitreal injection in an office setting

Study groups in NCT03144999
Study groupRoleWhat they receive
Low DoseExperimentalAAVCAGsCD59
Receives: Biological: AAVCAGsCD59
Mid DoseExperimentalAAVCAGsCD59
Receives: Biological: AAVCAGsCD59
High DoseExperimentalAAVCAGsCD59
Receives: Biological: AAVCAGsCD59

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants experiencing ocular and systemic adverse events as graded by CTCAE v4.0

    Measure intraocular inflammation, ocular changes and systemic side effects following a single intraocular injection of AAVCAGsCD59

    Measured: 26 Weeks

Secondary outcomes (5)

  1. Evaluate the change in area of GA in eyes with dry AMD

    Efficacy of AAVCAGsCD59

    Measured: 26 Weeks

  2. Evaluate the rate of growth of GA in eyes with dry AMD

    Efficacy of AAVCAGsCD59

    Measured: 26 Weeks

  3. Incidence of conversion of dry AMD to wet AMD

    Efficacy of AAVCAGsCD59

    Measured: 26 Weeks

  4. Change in drusen volume measured by spectral domain OCT

    Efficacy of AAVCAGsCD59

    Measured: 26 Weeks

  5. Prevention of loss of 15 or more letters on an ETDRS visual acuity chart

    Efficacy of AAVCAGsCD59

    Measured: 26 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 May 2017
  2. Study start29 Mar 2017
  3. Primary completion6 Dec 2017
  4. Study completion9 Dec 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Other
Allocation
Non-randomised
Intervention model
Sequential
Model description
This is a dose escalation study consisting of a low, mid, and high dose to evaluate the maximum tolerated dose (MTD). An additional 8 patients are enrolled at the MTD.
Masking
None (open label)
Enrolment
17 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03144999
Sponsor's protocol ID
HMR-1001
Status last verified
Mar 2025
First submitted
2 May 2017
Last updated
30 Mar 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing AAVCAGsCD59 in people with Dry Age-related Macular Degeneration, run by Janssen Research & Development, LLC at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants experiencing ocular and systemic adverse events as graded by CTCAE v4.0 (time frame: 26 Weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03144999.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2025.

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