Clinical trial NCT05456828: A Study of ASKG712 in Patients With Neovascular Age-Related Macular Degeneration
Official title: A Multi-Center, Open-label, Single Ascending-Dose and Multiple Ascending-Dose Phase 1 Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Efficacy of ASKG712 Following Intravitreal Administration in Patients With Neovascular Age-related Macular Degeneration
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Neovascular Age-related Macular Degeneration
Lead sponsor: AskGene Pharma, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Suzhou Aosaikang Biopharmaceutical Co., Ltd.
First posted: 2022-07-13
Enrollment: 36
Sex: ALL
Minimum age (years): 50
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of the Phase 1 study is comprised of single ascending-dose component (Part 1) and multiple ascending-dose component (Part 2) to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ASKG712 in patients with neovascular age-related macular degeneration (nAMD).

Detailed description: The Part 1 of study is a multicenter, open-label, sequentially, single ascending-dose study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ASKG712 in subjects with nAMD.

The Part 2 of study is a multicenter, open-label, sequentially, multiple ascending-dose (3 doses) study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ASKG712 in subjects with nAMD.

Subjects will be sequentially enrolled into different dose-level cohorts following the "3+3" design to determine the maximum tolerated dose (MTD) or the maximum administered dose has been reached.

Eligibility criteria: Inclusion Criteria:

* 1. Signed the informed consent form;
* 2. Male or female subjects with 50\~80 years of age;
* 3. Active sub-foveal or juxta-foveal choroidal neovascularization(CNV) lesions secondary to neovascular age-related macular degeneration(nAMD);
* 4. Total lesion area ≤ 12 disc area(DA);
* 5. BCVA letter score measured at screening of 19\~78 letters.

Exclusion Criteria:

* 1. History of uveitis in either eye；
* 2. Current active inflammation or infection in the study eye;
* 3. Central foveal scar, fibrosis or atrophy of macular in the study eye;
* 4. Subretinal hemorrhage area in the study eye ≥ 50% of total lesion size;
* 5. Scar or fibrosis area in study eyes ≥ 50% of total lesion size;
* 6. History or any concurrent ocular condition which, in opinion of investigator, could either confound interpretation of efficacy and safety of ASKG712 or require medical or surgical intervention.
* 7. Presence of retinal pigment epithelial tear;
* 8. Previous intraocular operations in the study eye;
* 9. Uncontrolled previous or current glaucoma in either eye, or previous glaucoma filtering operation in the study eye;
* 10. Previous anti-VEGF drug treatment within 30 days prior to screening；
* 11. Diseases that affect intravenous injection and venous blood sampling;
* 12. Systemic autoimmune diseases;
* 13. Any uncontrolled clinical disorders;
* 14. History of allergy or current allergic response to ASKG712 or fluorescein;
* 15. Pregnant or nursing women;
* 16. Subjects should be excluded in the opinion of investigators.

Primary outcomes:
measure: Incidence of ocular adverse events (AEs) of the study eyes
timeFrame: Part 1: 6 weeks; Part 2: 20 weeks
measure: Incidence of non-ocular adverse events (AEs)
timeFrame: Part 1: 6 weeks; Part 2: 20 weeks
Secondary outcomes:
measure: Area under the concentration time curve (AUC)
timeFrame: Part 1: 6 weeks; Part 2: 20 weeks
measure: Maximum plasma concentration (Cmax)
timeFrame: Part 1: 6 weeks; Part 2: 20 weeks
measure: Anti-Drug Antibody
timeFrame: Part 1: 6 weeks; Part 2: 20 weeks
measure: Mean change from baseline in best corrected visual acuity (BCVA) as measured by Early Treatment of Diabetic Retinopathy Study (ETDRS) letter score
timeFrame: Part 1: 6 weeks; Part 2: 20 weeks
measure: Mean change from baseline in central subfield thickness (CST) of macula measured by optical coherence tomography (OCT)
timeFrame: Part 1: 6 weeks; Part 2: 20 weeks
measure: Mean change from baseline in choroidal neovascularization area measured by fundus angiography
timeFrame: Part 1: 6 weeks; Part 2: 20 weeks
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Shanghai General Hospital
state: Shanghai
Study officials:
affiliation: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
name: Kun Liu, MD
role: PRINCIPAL_INVESTIGATOR
References: