Safety and Tolerability Study for Age-Related Macular Degeneration

Phase 1

Completed

Conditions: Neovascular Age-Related Macular Degeneration

Interventions: Drug: hI-con1™ 150µl
Drug: hI-con1™ 300µl
Drug: hI-con1™ 60µl

Registration Number: NCT01485588

Lead Sponsor: Iconic Therapeutics, Inc.

Brief Summary: Phase 1: The purpose of this study is to evaluate the safety and tolerability of single ascending doses of hI-con1™ for subjects with Age-Related Macular Degeneration.

Phase 2: The purpose of this study is to evaluate the safety of 3 injections of hI-con1™ at 2 different dose levels.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 18

Inclusion Criteria

Active choroidal neovascularization (CNV) associated with age-related macular degeneration, as evidenced on fluorescein angiography (FA) and Optical Coherence Tomography (OCT), with the following lesion characteristics:
Subretinal hemorrhage if present < 50% of total lesion size
During Phase 1, the 4th, 5th, and 6th subjects enrolled in each cohort must have total lesion area < 6 Disc Area (DA) (total area of detachment) (15.24 mm2), of which at least 50% must be actively leaking, and 30% should be classic on the angiography as determined by a reading center, and no more than 3 prior injections of any therapy for the treatment of CNV.
For Phase 2, total lesion area < 6 DA (total area of detachment) (15.24 mm2), of which at least 50% must be actively leaking, and 30% should be classic on the angiography as determined by a reading center and no more than 3 prior injections of any therapy for the treatment of CNV.
Best Corrected Visual Acuity (BCVA) for Phase 1: 20/ 80 - count fingers in the study eye; visual acuity in the fellow eye must be the same or better than the study eye
BCVA for Phase 2: 20/40 to 20/320 in the study eye; visual acuity in the fellow eye must be the same or better than the study eye
Only one eye of each subject will be treated in the study. If both eyes are eligible, the study eye will be the eye with the worst visual acuity. If visual acuity is the same in both eyes, the eye with the most active CNV will be selected to be the study eye
Clear ocular media and adequate pupillary dilation in the study eye to permit fundus photography for screening
Intraocular pressure of 21 mm Hg or less in the study eye.

General Inclusion Criteria

Subjects of either gender, > 50 years of age
Subjects who are informed of, and willing and able to comply with, the investigational nature of the study and are able to provide written informed consent
Ability to return for all study visits
Females must be of non-child bearing potential (surgically sterilized or at least 2 years post-menopausal) or if of child-bearing potential, the subject must have a negative serum pregnancy test within 14 days prior to the first injection and agree to use 2 forms of effective contraception during the trial and for at least 60 days following the last study injection.

Ocular

Exclusion Criteria

Any retinal vascular disease or retinal degeneration other than AMD in the study eye
Serous pigment epithelial detachment without the presence of choroidal neovascularization in the study eye
Pigment epithelial tears or rips in the study eye
Previous posterior vitrectomy or retinal surgery in the study eye
Any periocular infection in the past 4 weeks in the study eye
During the duration of the study, subjects cannot be on any concomitant therapy with anti-VEGF (Vascular Endothelial Growth Factor) agents, e.g., Lucentis® , Avastin®, or Macugen® in the study eye (unless identified as rescue therapy given according to protocol guidelines)
Concomitant therapy or use within 30 days of Baseline (Day 1) of systemic (e.g. intravenous, oral, intramuscular, rectal) corticosteroids in doses > 10 mg/ day prednisone or prednisone equivalent, or use of intravitreous or periocular steroids within 90 days of Baseline (Day 1) in the study eye
Any current or prior use of extended-release steroid implants (e.g., Retisert®, Posurdex®, Medidur®) in the study eye
Significant media opacities, including cataract, in the study eye which might interfere with visual acuity, assessment of toxicity, or fundus photography.
Cataract surgery in the study eye within three months of screening
Trabeculectomy or outflow-device glaucoma surgery in the study eye
Intraocular surgery in the study eye within three months of screening
Periocular or ocular infection in the study eye
Severe myopia (spherical equivalent -8 diopters or greater) in the study eye
History of vascular pigment epithelial detachment or submacular hemorrhage in the fellow eye.

General Exclusion Criteria:

Use of any investigational agent or participation in any clinical trial of an investigational agent or investigational therapy that has the potential to affect the disease process (neovascular AMD) in the study eye within sixty (60) days of Baseline (Day 1), or participation in any other clinical trial of an investigational agent or investigational therapy within thirty (30) days of Baseline (Day 1). Participation in clinical trials of oral supplements of vitamins and minerals for the prevention of neovascular AMD (e.g. AREDS2) are allowed, as are studies that do not involve the administration of an investigational agent and/or investigational therapy
Undiagnosed acute illness first observed during screening or between screening and baseline, or severe concurrent medical conditions that, in the investigator's judgment, represent a safety concern.
Allergy to or prior significant adverse reaction to fluorescein
Any major surgical procedure within one month of trial entry
Blood pressure >160/90 mmHg.

Study & Design

Study Type: INTERVENTIONAL

Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
hI-con1™ 150µl	hI-con1™ 150µl	Phase 1- This is a dose escalation study (60µl, 150µl, or 300 µl) given at baseline and then the subject is followed up to week 24.
hI-con1™ 300µl	hI-con1™ 300µl	Phase 1- This is a dose escalation study (60µl, 150µl, or 300 µl) given at baseline and then the subject is followed up to week 24.
hI-con1™ 60µl	hI-con1™ 60µl	Phase 1- This is a dose escalation study (60µl, 150µl, or 300 µl) given at baseline and then the subject is followed up to week 24.

Primary Outcome Measures

Name	Time	Method
Mean Change in Central Retinal Subfield Thickness as Measured by Optical Coherence Tomography (OCT) at Week 24 From Baseline	24 Weeks	The Mean Change in Central Retinal Subfield Thickness as Measured by OCT is part of the evaluation on the safety and tolerability of single ascending doses of hI-con1 and to assist in determining the Maximum Tolerated Dose (MTD) that can be administered by intravitreal injection.
Mean Change in Best Corrected Visual Acuity (BCVA) at Week 24 From Baseline	24 Weeks	The Mean Change in Best Corrected Visual Acuity (BCVA) is part of the evaluation on the safety and tolerability of single ascending doses of hI-con1 and to assist in determining the Maximum Tolerated Dose (MTD) that can be administered by intravitreal injection. BCVA is measured using the Early Diabetic Retinopathy Study (EDTRS) chart. More letters read result in a higher score.

Secondary Outcome Measures

Name	Time	Method

Trial Locations

Locations (5): Valley Retina Institute, PA
🇺🇸
McAllen, Texas, United States
Palmetto Retina Center
🇺🇸
West Columbia, South Carolina, United States
Rocky Mountain Eye Center, P.C.
🇺🇸
Missoula, Montana, United States
Retina Research Center
🇺🇸
Austin, Texas, United States
Retina & Vitreous Center of Southern Oregon, P.C.
🇺🇸
Ashland, Oregon, United States