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The trial is taking place at:
O

Ophthalmic Consultants of the Capital Region | Troy, NY

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Evaluation of OLX10212 in Patients With Neovascular Age-related Macular Degeneration

O

OliX Pharmaceuticals

Status and phase

Enrolling
Phase 1

Conditions

Neovascular Age-related Macular Degeneration

Treatments

Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)

Study type

Interventional

Funder types

Other
Industry

Identifiers

NCT05643118
OLX10212-01

Details and patient eligibility

About

This is a Phase 1, multicenter, open-label, single- and multi-dose, dose-escalating study of OLX10212 in patients with neovascular age-related macular degeneration (AMD). This study is composed of 2 parts: Part A and Part B. Part A is a single ascending dose study, and Part B is a multiple ascending dose study. The primary objective is to evaluate the safety and tolerability of single and multiple intravitreal injection(s) of OLX10212 in patients with neovascular AMD. The exploratory objectives are to evaluate the preliminary efficacy of single and multiple intravitreal injection(s) of OLX10212 in patients with neovascular AMD, and to evaluate the pharmacokinetics (PK) of single and multiple intravitreal injection(s) of OLX10212 in patients with neovascular AMD.

Full description

This is a Phase 1, multicenter, open-label, single- and multi-dose, dose escalation study to evaluate the safety, tolerability, and preliminary efficacy of OLX10212 in the treatment of age-related macular degeneration (AMD). This study is composed of 2 parts: Part A and Part B. Part A is a single ascending dose study, i.e. participants will receive one intravitreal injection of OLX10212 at different dose levels and Part B is a multiple ascending dose study, i.e. participants will receive up to three intravitreal injections of OLX10212. Up to 42 individuals with AMD will be invited to participate in this study. The mechanism of action of OLX10212 holds promise to treat AMD by improving inflammation in the retina which is typically observed in patients with AMD. This is the first time OLX10212 is used in patients with AMD. Safety and tolerability of OLX10212 will be assessed via detailed ophthalmologic evaluations, vital signs, and clinical laboratory testing. In addition, plasma concentrations of OLX10212 will be measured and evaluations of the therapeutic effects of OLX10212 will be performed.

Part A uses a dose-ascending, sequential design to evaluate up to five doses of OLX10212, starting with the lowest dose of OLX10212 in a 50-μL injection. Up to six patients will be enrolled at each dose level. Each of the enrolled patients will receive a single intravitreal administration of OLX10212. The safety and tolerability evaluation period will encompass the first 14 days following OLX10212 administration. The effects of OLX10212 will be observed up to 24-weeks after injection. Based on the safety and tolerability evaluation, a decision will be made whether or not to increase the dose to the next higher dose levels for the subsequent patient cohorts. Therefore, a total of up to 30 patients (up to 5 dose levels and up to 6 patients/dose level) will be enrolled in Part A of this study.

Part B of this study uses a dose-ascending, sequential design to evaluate 2 dose levels of OLX10212 (750 and 950 μg/eye/50 μL), starting with the 750 μg/eye/50 μL dose. Three patients will initially be enrolled in each cohort. Each of the enrolled patients will receive a total of up to three intravitreal injections of OLX10212, each four weeks apart (Week 0, Week 4, and Week 8). The DLT evaluation period will encompass the first 10 weeks following the first OLX10212 administration (ending 2 weeks following the third OLX10212 administration), during which safety and tolerability will be assessed at each visit. In addition, the plasma concentrations of OLX10212 will be measured and therapeutic effects will be evaluated. A total of up to 12 patients with AMD (3 dose levels and up to 6 patients/dose level) will be invited to participate in Part B of this study.

Enrollment

42 estimated patients

Sex

All

Ages

50+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  1. Men and women ≥50 years of age
  2. Primary subfoveal CNV lesions secondary to AMD, including juxtafoveal lesions that affect the fovea, as evidenced by FA in the study eye
  3. CNV must be ≥50% of the total lesion size in the study eye
  4. ETDRS BCVA score ranging from 20/60 to 20/400 in the study eye
  5. Clear ocular media and adequate pupillary dilation (able to dilate pupil to ≥4 mm using standard mydriatics) in the study eye to permit good stereoscopic fundus photography
  6. Retinal thickness ≥200 μm in the macular region of the study eye as measured by SD-OCT, and active neovascular AMD, in the opinion of the Investigator
  7. Willing, committed, and able to return for all clinic visits and complete all study-related procedures
  8. Able to read (or if unable to read due to visual impairment, be read to verbatim by the person administering the informed consent or by a family member), understand, and willing to sign the informed consent form

Exclusion criteria

  1. Any prior systemic treatment for neovascular AMD in either eye, except dietary supplements or vitamins or systemic anti-VEGF therapy, or planned use at any time during the study

  2. Any prior treatment in the study eye with another investigational agent to treat neovascular AMD within 6 months prior to Day 0 or planned use at any time during the study

  3. Prior treatment with anti-VEGF agents as follows:

    1. Anti-VEGF therapy in the study eye within 4 weeks prior to Day 0
    2. Anti-VEGF therapy in the study eye at any time to which there was no response, as defined by the presence of at least 1 of the following conditions: (1) persistent (plasma) fluid exudation, (2) unresolved or new hemorrhage, and (3) progressive lesion fibrosis
    3. Anti-VEGF therapy in the fellow eye with an investigational agent (not FDA approved, unless it is bevacizumab) within 3 months prior to Day 0 (prior treatment with an FDA approved anti-VEGF therapy in the fellow eye is allowed at any time)
    4. Systemic anti-VEGF therapy, investigational or FDA approved, within 3 months prior to Day 0 or planned use at any time during the study
  4. Scar or fibrosis in the study eye involving >50% of the total lesion size

  5. Retinal pigment epithelial tears or rips in the study eye involving the macula within 6 months prior to Day 0

  6. History of any vitreous hemorrhage in the study eye within 4 weeks prior to Day 0

  7. Presence of other causes of CNV in the study eye, including pathologic myopia, ocular histoplasmosis syndrome, angioid streaks, choroidal rupture, or multifocal choroiditis

  8. Clinical evidence of moderate or severe diabetic retinopathy, diabetic macular edema, or any other inflammatory or occlusive vascular disease affecting the retina (other than AMD) in either eye

  9. History of stage ≥2 macular hole in the study eye

  10. Any prior intraocular or periocular surgery on the study eye within 3 months prior to Day 0 (lid surgery is allowed if it took place at least 1 month prior to Day 0 and is unlikely to interfere with OLX10212 injection). Prior vitrectomy in the study eye, surgery for retinal detachment in the study eye, and prior trabeculectomy or other filtration surgery in the study eye are not permitted at any time

  11. Uncontrolled glaucoma (defined as IOP ≥25 mmHg despite treatment with antiglaucoma medication) in the study eye

  12. Glaucoma in the study eye requiring treatment with 3 or more antiglaucoma medications

  13. Active intraocular inflammation or history of uveitis in either eye

  14. Presence or history of ocular or periocular infection in either eye within 2 weeks prior to Day 0

  15. Presence of scleromalacia in the study eye

  16. Aphakia or absence of posterior capsule in the study eye (unless due to yttrium aluminum garnet [YAG] posterior capsulotomy)

  17. Prior therapeutic radiation in the region of the study eye or planned use at any time during the study

  18. Significant media opacities, including cataract, in the study eye that, in the opinion of the Investigator, could interfere with visual acuity, assessment of safety, or fundus photography

  19. Any concurrent intraocular condition in the study eye (eg, cataract) that, in the opinion of the Investigator, could (1) require either medical or surgical intervention during the 24- or 32-week study period (Part A or Part B, respectively), (2) increase the risk to the patient beyond what is to be expected from standard intraocular injection procedures, or (3) otherwise interfere with the injection procedure or efficacy or safety evaluation

  20. History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that might affect interpretation of the results of the study or renders the patient at high risk for treatment complications

  21. Participation as a patient in any clinical study or prior systemic or ocular treatment with an investigational agent within 12 weeks prior to Day 0

  22. Prior systemic or intraocular treatment with long-acting steroids within 6 months prior to Day 0 or planned use at any time during the study

  23. History of allergy to povidone iodine

  24. Known allergy to fluorescein sodium for injection in angiography

  25. Unwillingness among females who are pregnant, breastfeeding, or of childbearing potential to practice adequate contraception throughout the study. Adequate contraceptive measures include oral contraceptives (stable use for ≥2 cycles prior to Day 0), intrauterine device, Depo-Provera® (Pfizer, Inc., New York) or Norplant System® (Pfizer, Inc., New York) implants, bilateral tubal ligation, vasectomy, and condom or diaphragm plus contraceptive sponge, foam, or jelly. A female is considered to be of childbearing potential unless she is premenstrual, 1 year postmenopausal, or 3 months post-surgical sterilization. All females of childbearing potential, including those with post-tubal ligation, must have a negative urine pregnancy test result at Day 0 and every 4 weeks as outlined in the Schedule of Activities. A negative serum pregnancy test must be obtained at Screening.

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

42 participants in 7 patient groups

Part A 100 μg/eye/50 μL
Experimental group
Description:
study eye treated with 100 μg (94.3 μg free acid) of OLX10212
Treatment:
Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)
Part A 250 μg/eye/50 μL
Experimental group
Description:
study eye treated with 250 μg (235.8 μg free acid) of OLX10212
Treatment:
Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)
Part A 500 μg/eye/50 μL
Experimental group
Description:
study eye treated with 500 μg (471.5 μg free acid) of OLX10212
Treatment:
Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)
Part A 750 μg/eye/50 μL
Experimental group
Description:
study eye treated with 750 μg (707.3 μg free acid) of OLX10212
Treatment:
Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)
Part A 950 μg/eye/50 μL
Experimental group
Description:
study eye treated with 950 μg (895.9 μg free acid) of OLX10212
Treatment:
Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)
Part B 750 μg/eye/50 μL
Experimental group
Description:
study eye treated with a total of 3 intravitreal injections of 750 μg (707.3 μg free acid) of OLX10212 each 28 days apart
Treatment:
Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)
Part B 950 μg/eye/50 μL
Experimental group
Description:
study eye treated with a total of 3 intravitreal injections of 950 μg (895.9 μg free acid) of OLX10212 each 28 days apart
Treatment:
Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)

Trial contacts and locations

5

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Central trial contact

Saehong Min, MS; Eunah Park, MS

Data sourced from clinicaltrials.gov

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