RecruitingPhase 1interventionalRules rewrite

Phase 1: testing ASP2020 for Stargardt Disease

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.

Official registry: https://clinicaltrials.gov/study/NCT07734064 · Astellas Institute for Regenerative Medicine · enrollment listed as 30

What this phase means

Phase 1: the first tests in people. The main question is safety and dose, not whether it cures the condition. Groups are small. Side effects are watched closely.

Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

What the registry says happens

If you join, the listed intervention(s) are ASP2020. The registry lists allocation as randomized. Macular dystrophies are a group of inherited eye conditions that affect the macula.

They plan to enroll about 30 people at 4 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.

Official title (unrewritten)

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

Interventions: ASP2020

Primary outcome (registry wording): Number of participants with treatment-emergent adverse events (TEAEs)

Who it is for (from inclusion criteria)

Ages 6 Years to any
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)

Who the registry excludes

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

Public study contact (from the registry)

Astellas Institute for Regenerative Medicine

800-888-7704

Astellas.registration@astellas.com

Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.

Sites listed on ClinicalTrials.gov

4 listed

Associated Retina Consultants — Phoenix, Arizona (recruiting)
Deep Blue Retina — Southaven, Mississippi (recruiting)
Cincinnati Eye Institute — Cincinnati, Ohio (recruiting)
Retina Foundation of Southwest — Dallas, Texas (recruiting)

Related recruiting trials in this snapshot

RecruitingNot a traditional drug phaseBethesda, Maryland

Von Hippel-Lindau (VHL): Clinical Manifestations, Diagnosis, Management and Molecular Bases of Inherited Re…

We will investigate the clinical manifestations and molecular genetic defects of heritable urologic malignant disorders. Families with urologic malignancy with known or suspected genetic basis will be enrolled.

NCT00001238 · Official ClinicalTrials.gov record · National Cancer Institute (NCI)

RecruitingNot a traditional drug phaseBethesda, Maryland

Study of Systemic Lupus Erythematosus

This protocol will evaluate patients with systemic lupus erythematosus (SLE) and their relatives to learn more about how the disease develops and changes over time. It will also study genetic factors that make a person susceptible to SLE.

NCT00001372 · Official ClinicalTrials.gov record · National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

RecruitingNot a traditional drug phaseWashington D.C., District of Columbia

Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics

This study is designed to explore the genetics and pathophysiology of diseases presenting with intermittent fever, including familial Mediterranean fever, TRAPS, hyper-IgD syndrome, and related diseases. The following individuals may be eligible for this natural history study: 1) patients with known or suspected familial Mediterranean fever, TRAPS, hyper-IgD syndrome or related disorders; 2) relatives of these patients; 3) healthy, normal volunteers 7 years of age or older.

NCT00001373 · Official ClinicalTrials.gov record · National Human Genome Research Institute (NHGRI)

FAQ about this NCT record

Is NCT07734064 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

What phase is NCT07734064?

Phase 1. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Can PlainTrials tell me if I qualify for NCT07734064?

No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.

How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary