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Will Be Recruiting
Astellas Study ID
2020-CL-0101

A study about the safety of a single ASP2020 eye injection and if it helps people with vision loss from Stargardt-type eye conditions (DEVOTE)

Astellas Study ID
2020-CL-0101
Clinicaltrials.gov ID
NCT07734064
EUCTIS ID
N/A
jRCT ID
N/A
CN CDE ID
N/A
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Trial Purpose

To evaluate the safety, tolerability, and potential to improve vision (efficacy) of transplanted ASP2020 cells for people with STGD-type macular dystrophies.

Additional Study Details

Phase
Phase 1
Product
ASP2020
Product
ASP2020
Type
Interventional
Masking
None (Open Label)
Enrollment number
30
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Who May Participate:

Inclusion Criteria:

  • 6 years of age or older
  • Documented clinical diagnosis of macular dystrophy with a STGD-type clinical presentation and molecular confirmation, defined as:
    • ABCA4-associated STGD disease
    • Mutations in other genes and a STGD-type clinical presentation

Exclusion Criteria:

  • Serious health problems like liver disease
  • Autoimmune condition treated with immune-suppressing medication
  • Received gene therapy, stem cell therapy, or artificial retinal implants

Additional study criteria will be reviewed by the study team.

What to Expect

The study includes four stages (screening, baseline, treatment, and follow-up) and a total of 12 study visits. Each visit will last several hours and include health assessments such as vision tests, eye imaging, blood draws, questionnaires, and other study procedures.

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Length of study treatment
Approximately 1 year
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Number of study visits
12 visits

Dose-Escalation of ASP2020 in Vision,Outcomes,Tolerability, and Efficacy in Stargardt-type Macular Dystrophies (DEVOTE)

A Phase 1b, Open-label, Multicenter Dose-escalation Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Subretinal Dose of ASP2020 in Participants with Macular Dystrophies with a Stargardt-type Clinical Presentation

Background:

  •  Macular dystrophies are a group of inherited eye conditions that affect the macula.
  • The macula is in the center of the retina, the light-sensitive part at the back of the eye.
  • In people with macular dystrophies, some of the cells in the macula gradually stop working and may die over time.
  • This leads to loss of central vision, which can make it harder to read, recognize faces, or see fine details. The peripheral vision (what is seen out of the corner of the eye) is mostly unaffected.
  • Stargardt disease (STGD) is a type of macular dystrophy which is caused by 1 faulty gene (ABCA4).
  • There are other macular dystrophies that look very similar to STGD and are called STGD-like macular dystrophies. These conditions are caused by changes in other genes but are not limited to PRPH2, ELOVL4, PROM1, CDHR1, MT-TL1, CTINNA1  
  • Together, STGD and STGD-like conditions can be called STGD-type macular dystrophies.

Study Overview:

  • This study is an early development study of ASP2020 in adults, teenagers, and children with STGD-type macular dystrophies. ASP2020 will be given to people for the first time.
  • ASP2020 are human stem cells which have been changed into cells found in the macula.
  • The main aim of the study is to check the safety, tolerability, and efficacy of ASP2020 and how well people tolerate it.
  • Other aims of the study are to evaluate vision improvement, see if ASP2020 cells replace damaged retinal cells, and monitor for immune reactions.
  • ASP2020 will be given as a single injection into the eye, under the retina.
  • This injection into the eye requires a surgical procedure where the person is put to sleep by a general anesthetic.
  • At the end of surgery, a steroid will be injected into the eye to reduce any swelling.     
  • The study has two parts. In Part 1, different small groups will receive a lower to higher dose of ASP2020. This is done to find a suitable dose to use in Part 2. The adults will receive the lower dose and higher dose before the teenagers. Children are not included in Part 1.
  • In Part 2, different groups of adults, teenagers, and children will receive the most suitable dose of ASP2020 determined from Part 1.

Locations

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    Trial info
    Follow Trial
    Age
    6+ years
    Condition
  • Stargardt's Disease
    • Show more
  • Other hereditary macular dystrophies
    • Sex
    Female & Male
    Trial Dates
    Jul 2026 - Sep 2029
    Additional Study Details
    Inclusion Criteria
    • Documented clinical diagnosis of macular dystrophy with a STGD-type clinical presentation and molecular confirmation, defined as either:
      • STGD: presence of biallelic (pathogenic or likely pathogenic) ABCA4 variants, or one definite disease-causing ABCA4 variant together with a typical phenotype consistent with STGD.
      • STGD-like macular dystrophy: presence of one or more pathogenic variants in a gene known to cause macular dystrophy, as appropriate for its expected inheritance mode.
    • Sufficiently clear ocular media and adequate pupillary dilation to allow for all imaging procedures.
    • Intraocular pressure (IOP) of ≤ 21 mmHg
    • Participant has a spherical equivalent refractive error between +8.00 D and -10.00 D.
    • BCVA ranging from 20/500 to 20/40 (equivalent to 15 to 70 ETDRS letters)
      • For participants in the > 20/80 to ≤ 20/40 BCVA range (moderate visual impairment [MVI]): presence of a visible definite or probable residual ellipsoid zone (EZ) on SD-OCT, and a total retinal SD-OCT central subfield thickness ≥ 150 micrometers (µm)
      • For participants in the ≥ 20/500 to ≤ 20/80 BCVA range (severe visual impairment): Presence of a residual ONL within the macular optical coherence tomography (OCT) scan area and Evidence of RPE disease/damage by means of SD-OCT (hypertransmission defect) and/or FAF imaging (questionably decreased autofluorescence/definitely decreased autofluorescence).
    Exclusion Criteria
    • Participant has a known history of significant systemic disease that could impact ocular health or confound study assessments, based on medical history or prior clinical documentation.
    • Participant has an autoimmune condition that requires treatment with immunomodulatory therapy and/or biologics that cause immunosuppression.
    • Participant has known diagnosis of diabetes mellitus with a documented glycated hemoglobin (HbA1c) value ≥ 7% 3 months prior to screening and based on available medical records.
    • Participant has a history or evidence of severe cardiac disease, cardiovascular or cerebrovascular disease, including a history of stroke within 12 months prior to screening.
    • Participant has any complicating systemic disease or active malignancy.
    • Participant has a known history of any systemic or metabolic condition, or physical examination finding that may significantly affect ocular health or interfere with the interpretation of study assessments.
    • Participant has presence of another known or suspected molecular diagnosis of macular or retinal disease that could confound interpretation of study outcomes, indicate a second concomitant retinal condition, or suggest a different etiology for the macular disease.
    • Participant has macular atrophy due to any cause other than a genetically or clinically confirmed diagnosis of STGD or STGD-like macular dystrophy.
    • Participant has evidence or history of choroidal neovascularization.
    • Participant has diagnosis of any form of uncontrolled glaucoma (for high-tension glaucoma IOP > 25 mmHg).
    • Participant has a history of steroid-induced IOP elevation or known steroid responder status.
    • Participant has and/or is receiving treatment for thyroid eye disease.
    • Participant has diabetic retinopathy in excess of mild nonproliferative diabetic retinopathy
    • Participant has any other disease(s) affecting the optic nerve.
    • Participant has a history of anterior or posterior uveitis and/or presence of intraocular inflammation (trace anterior chamber cell or flare), or history of idiopathic or autoimmune-associated uveitis in either eye.
    • Participant has media opacities impeding the visualization of the fundus and/or the reliable performance of the visual function tests required by the protocol.
    • Participant has aphakia.
    • Participant has a clinically significant epiretinal membrane or evidence of clinically significant vitreomacular traction syndrome.
    • Participant has any other disorders which could interfere with or confound visual acuity and other ocular assessments, including OCT or FAF.
    • Participant has history of any of the following procedures: posterior vitrectomy, retinal detachment surgery, glaucoma filtering surgery, glaucoma drainage device implantation, selective laser trabeculoplasty, full-thickness or partial- thickness corneal transplant.
    • Participant has had any intraocular surgery within 3 months of screening.
    • Participant has a history of intraocular metallic foreign bodies.
    • Participant has received any treatment including gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or any prior intravitreal treatment for any indication in either eye that may be considered to potentially interfere with the study participation or its conduct.
    • Participant has received within 1 month prior to screening or is receiving concomitant treatment with any ocular or systemic medication known to be toxic to the lens, retina, or optic nerve.
    • Participant has received any investigational therapy within 3 months prior to screening.
    • Participant has any condition, which makes the participant unsuitable for study participation.

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