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Study to Understand Safety Efficacy of Novel Stem Cell Formulation to Treat Late Stage Dry Age-related Macular Degeneration (d-AMD)

Phase 1
Conditions
Health Condition 1: H353- Degeneration of macula and posterior pole
Registration Number
CTRI/2024/04/065639
Lead Sponsor
Eyestem Research Pvt. Ltd.
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Open to Recruitment
Sex
Not specified
Target Recruitment
0
Inclusion Criteria

1. Men and women greater than or equal to 50 years of age at Screening.

2. Diagnosis of Geographic Atrophy secondary to d-AMD

3. Have Best Corrected Visual Acuity (BCVA) equal to or less than 20/200 Snellen (ETDRS letter score = 35) in the study eye at screening.

a. Phase 1 less than or equal to 20/200 and

b. Phase 2a less than or equal to 20/64 (ETDRS letter score 60) in the study eye at Screening.

4. Vision in the unoperated eye must be better or equal to vision in the study eye.

5. Willing, committed, and able to return for ALL clinic visits and complete all study related procedures.

6. Be medically suitable to undergo anesthesia, vitrectomy and subretinal injection in the opinion of the Investigator.

7. Be medically suitable for immunosuppression therapy in accordance with the requirements of this protocol in the opinion of the Investigator.

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 a family member) and understand, and willing to sign the informed consent form (ICF)

9. Willing to provide signed Informed Consent prior to any procedures being performed at visit 1, screening

10. Negative for HIV, HbsAg, HCV, TB

11.The GA lesion must meet the following criteria as determined by the central reading centers

assessment of Fundus Autofluorescence FAF imaging at screening

a. Total GA area must be greater than or equal to 1.25 and less than or equal to 17.5 mm2 (0.5 and 7 disk areas [DA] respectively)

b. The entire GA lesion must be completely visualized on the macula centered image

and must be able to be imaged in its entirety and not contiguous with any areas of

peripapillary atrophy.

c. At least one of the lesions has to be sub-foveal.

Exclusion Criteria

1. Have evidence of neovascular AMD in either eye by clinical examination, fluorescein angiography or optical coherence tomography.

2. Have GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like Chloroquine maculopathy in either eye.

3. Have any evidence of active or inactive choroidal neovascularization (CNV) due to other causes such as ocular histoplasmosis syndrome, angioid streaks, choroidal rupture, uveitis, punctate inner choroidopathy, or multifocal

choroiditis in the study eye.

4. Axial myopia greater than -6 diopters or axial length more than 26 mm.

5. Have a decrease in BCVA in the study eye due to causes other than GA (e.g., pigment abnormalities, dense sub foveal hard exudates, previous vitreoretinal surgery, retinal dystrophies, non-retinal conditions, visually

significant cataract, macular ischemia, etc.)

6. Have the presence of retinal pigment epithelial tears or rips involving the macula in the study eye at screening.

7. Have a history or evidence of vitreous hemorrhage in the study eye.

8. Have a history or clinical evidence of severe diabetic retinopathy, diabetic macular edema, retinal vein occlusion or any other vascular disease affecting

the retina in the study eye.

9. Have had a prior pars plana vitrectomy in the study eye.

10. Have a history of retinal detachment or surgery for retinal detachment in the study eye.

11. Have history of a macular hole in the study eye.

12. Have had any other ocular surgery (except cataract) within 2 months or Yttrium Aluminum Garnet (YAG) laser capsulotomy in the study eye in the past 4 weeks.

13. Have had a prior trabeculectomy or other filtration surgery in the study eye.

14. History of any form of glaucoma in the study eye.

15. Patients with ocular pathology, particularly that of retina (other than AMD).

16. Have active intraocular inflammation or a history or evidence of uveitis in either eye.

17. Have active ocular or periocular infection in either eye, or a history of any ocular or periocular infection within the 2 weeks prior to Visit 1, Screening in either eye.

18. Have a history of scleromalacia in either eye.

19. Have had previous therapeutic radiation in the study eye.

20. Have a history of corneal transplant or corneal dystrophy.

21. Have any concurrent ocular condition in the study eye which, in the opinion of the investigator, could either increase the risk to the subject beyond what is to be expected from standard procedures of intraocular injection, or which otherwise may interfere with the injection procedure or with evaluation of efficacy or safety.

22. Have a history of other diseases, physical examination findings, or clinical laboratory findings giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that might affect interpretation of the results of the study or render the subject at high risk for treatment complications.

23. Have participated as a subject in any clinical study within 6 months prior to the Screening visit.

24. Have a known serious allergy to fluorescein sodium for injection in angiography, Povidone Iodine or any of the other medications required for anesthesia or the subretinal injection procedure.

25. Be a female who is pregnant, breastfeedin

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Primary Efficacy Endpoint <br/ ><br>a Fundus AutofluorescenceTimepoint: At Screening (= 28 days), <br/ ><br> <br/ ><br>During Active Follow-up on the following visits <br/ ><br>Days- 1, 7, 14, 28, 42, 56, 70, 84, <br/ ><br>120, 150 and 180 <br/ ><br> <br/ ><br>During Safety Follow-up on the following visits <br/ ><br>Months- 12, 18, 24, 36, 48 and 60
Secondary Outcome Measures
NameTimeMethod
f. NEI VFQ-25 Quality of Life scoreTimepoint: Screening (= 28 days), <br/ ><br>Active Follow-up <br/ ><br>Day 180 <br/ ><br>Safety Follow-up <br/ ><br>Months- 12, 18, 24, 36, 48 and 60;Secondary Efficacy Endpoints <br/ ><br>b. BCVA ETDRS <br/ ><br>c. Fundus Photography <br/ ><br>d. SD-OCT <br/ ><br>e. Microperimetry <br/ ><br>Timepoint: b. BCVA ETDRS <br/ ><br>Screening (= 28 days), <br/ ><br>Active Follow-up <br/ ><br>Days- 1, 7, 14, 28, 42, 56, 70, 84, <br/ ><br>120, 150 and 180 <br/ ><br>Safety Follow-up <br/ ><br>Months- 12, 18, 24, 36, 48 and 60 <br/ ><br> <br/ ><br>c. Fundus Photography, d. SD-OCT and e. Microperimetry <br/ ><br>Screening (= 28 days), <br/ ><br>Active Follow-up <br/ ><br>Days- 1, 7, 14, 28, 42, 56, 70, 84, <br/ ><br>120, 150 and 180 <br/ ><br>Safety Follow-up <br/ ><br>Months- 12, 18, 24, 36, 48 and <br/ ><br>60 <br/ ><br> <br/ ><br> <br/ ><br> <br/ ><br> <br/ ><br>
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