A Study to Establish the Safety and Tolerability of Zimura® (Anti-C5 Aptamer) in Combination With Anti-VEGF Therapy in Subjects With Idiopathic Polypoidal Choroidal Vasculopathy (IPCV)

• Sponsor: Ophthotech Corporation (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02397954
• Collaborator: (none)
• Enrollment: 4
• Location: 1
• Arm: 1
• Duration (Months): 7
• Patients Per Site Per Month: 0.6

Study Details

Study Description

Brief Summary

The objectives of this study are to evaluate the safety and tolerability of Zimura™ intravitreous injection in combination with anti-vascular endothelial growth factor (VEGF) therapy in subjects with Idiopathic Polypoidal Choroidal Vasculopathy (IPCV).

Condition or Disease	Intervention/Treatment	Phase
Idiopathic Polypoidal Choroidal Vasculopathy	Drug: Zimura	Phase 2

Detailed Description

Treatment experienced (Prior treatment with anti-VEGF monotherapy of ≥8 injections in the previous twelve (12) months) subjects of either gender aged 50 years or above with a diagnosis of IPCV, will receive 3 monthly (Q4W) intravitreal injections of Zimura™ (1 mg/eye) in combination with anti-VEGF therapy (Avastin® 1.25 mg/eye or Lucentis® 0.5 mg/eye or Eylea® 2 mg/eye).

Safety endpoints include visual acuity loss (proportion of subjects with >15 letter loss at Month 3), ophthalmic adverse events (AEs), systemic adverse events (AEs), change in total retinal thickness (SD-OCT) at Month 3, regression and/or elimination of polyps at Month 3 compared to screening as measured by indocyanine green angiography (ICGA), and laboratory values.

Study Design

• Study Type: Interventional
• Actual Enrollment: 4 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 2a Study to Establish the Safety and Tolerability of Zimura® (Anti-C5 Aptamer) in Combination With Anti-VEGF Therapy in Subjects With Idiopathic Polypoidal Choroidal Vasculopathy (IPCV)
• Study Start Date: Mar 1, 2015
• Actual Primary Completion Date: Oct 1, 2015
• Actual Study Completion Date: Oct 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Zimura + Anti-VEGF Subjects will receive monthly intravitreous injections of Zimura in combination with either Lucentis, Avastin or Eylea.	Drug: Zimura; Subjects will receive monthly intravitreous injections of Zimura in combination with Lucentis, Avastin or Eylea.; Other Names: Avastin Eylea Lucentis

Outcome Measures

Primary Outcome Measures

1. Number of Participants With >15 ETDRS Letter Loss at Month 3 [3 Months]

   Number of participants with >15 ETDRS letter loss (with calculated percentage)

2. Number of Participants With Ophthalmic Adverse Events [3 months]

   Number of Participants with Ophthalmic Adverse Events (with calculated percentage)

3. Number of Participants With Systemic Adverse Events [3 months]

   Number of Participants with Systemic Adverse Events (with calculated percentage)

Other Outcome Measures

1. Mean Change in Central Subfield Retinal Thickness (SD-OCT) From Baseline at Month 3 [Baseline and Month 3]

   Mean change in central subfield retinal thickness from baseline at Month 3 as measured by SD-OCT

2. Regression and/or Elimination of Polyps at Month 3 [Baseline and Month 3]

   Regression and/or Elimination of Polyps from baseline to Month 3

Eligibility Criteria

Criteria

• Ages Eligible for Study: 50 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Subjects of either gender aged ≥ 50 years

• Diagnosis of IPCV

• Treatment-experienced defined as prior treatment with anti-VEGF mono therapy of ≥ 8 injections in the previous twelve (12) months

Exclusion Criteria:

• Any intraocular surgery or thermal laser within three (3) months of trial entry

• Any prior thermal laser in the macular region, regardless of indication

• Any ocular or periocular infection in the twelve (12) weeks prior to entry

• History of any of the following conditions or procedures in the study eye: Rhegmatogenous retinal detachment, pars plana vitrectomy, filtering surgery (e.g. trabeculectomy), glaucoma drainage device, corneal transplant

• Previous therapeutic radiation in the region of the study eye

• A diagnosis of diabetic retinopathy (presence of microaneurysms or any vasculopathy and/or leakage from retinal vasculature in a subject with diabetes mellitus)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1		Phoenix	Arizona	United States	85014

Sponsors and Collaborators

• Ophthotech Corporation

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Ophthotech Corporation
• ClinicalTrials.gov Identifier: NCT02397954
• Other Study ID Numbers: OPH2002
• First Posted: Mar 25, 2015
• Last Update Posted: Jun 19, 2019
• Last Verified: Apr 1, 2017

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Ophthotech Corporation: idiopathic polypoidal choroidal vasculopathy; IPCV; Zimura™; Avastin®; Eylea®; Lucentis®
• Additional relevant MeSH terms: Vascular Diseases; Ranibizumab

Study Results

Participant Flow

Arm/Group Title	Zimura + Anti-VEGF
Arm/Group Description	Zimura 1mg/eye intravitreal injection + Anti-vascular endothelial growth factor (VEGF) (Avastin 1.25mg/eye or Lucentis 0.5mg/eye or Eylea 2mg/eye) intravitreal injection
Period Title: Overall Study
STARTED	4
COMPLETED	4
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Zimura + Anti-VEGF
Arm/Group Description	Zimura 1mg/eye intravitreal injection + Anti-VEGF (Avastin 1.25mg/eye or Lucentis 0.5mg/eye or Eylea 2mg/eye) intravitreal injection
Overall Participants	4
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	74.5 (6.03)
Age, Customized (Count of Participants)
Adults 50-65	0 0%
Adults >65-75	2 50%
Adults >75	2 50%
Sex: Female, Male (Count of Participants)
Female	1 25%
Male	3 75%
Region of Enrollment (participants) [Number]
United States	4 100%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With >15 ETDRS Letter Loss at Month 3
Description	Number of participants with >15 ETDRS letter loss (with calculated percentage)
Time Frame	3 Months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Zimura + Anti-VEGF
Arm/Group Description	Zimura 1mg/eye intravitreal injection + Anti-VEGF (Avastin 1.25mg/eye or Lucentis 0.5mg/eye or Eylea 2mg/eye) intravitreal injection
Measure Participants	4
Count of Participants [Participants]	0 0%

2. Primary Outcome

Title	Number of Participants With Ophthalmic Adverse Events
Description	Number of Participants with Ophthalmic Adverse Events (with calculated percentage)
Time Frame	3 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Zimura + Anti-VEGF
Arm/Group Description	Zimura 1mg/eye intravitreal injection + Anti-VEGF (Avastin 1.25mg/eye or Lucentis 0.5mg/eye or Eylea 2mg/eye) intravitreal injection
Measure Participants	4
Count of Participants [Participants]	3 75%

3. Primary Outcome

Title	Number of Participants With Systemic Adverse Events
Description	Number of Participants with Systemic Adverse Events (with calculated percentage)
Time Frame	3 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Zimura + Anti-VEGF
Arm/Group Description	Zimura 1mg/eye intravitreal injection + Anti-VEGF (Avastin 1.25mg/eye or Lucentis 0.5mg/eye or Eylea 2mg/eye) intravitreal injection
Measure Participants	4
Count of Participants [Participants]	1 25%

4. Other Pre-specified Outcome

Title	Mean Change in Central Subfield Retinal Thickness (SD-OCT) From Baseline at Month 3
Description	Mean change in central subfield retinal thickness from baseline at Month 3 as measured by SD-OCT
Time Frame	Baseline and Month 3

Outcome Measure Data

Analysis Population Description

Limited sample size; all study participants were included in the analysis.

Arm/Group Title	Zimura + Anti-VEGF
Arm/Group Description	Zimura 1mg/eye intravitreal injection + Anti-VEGF (Avastin 1.25mg/eye or Lucentis 0.5mg/eye or Eylea 2mg/eye) intravitreal injection
Measure Participants	4
Mean (Full Range) [μm]	-2.75

5. Other Pre-specified Outcome

Title	Regression and/or Elimination of Polyps at Month 3
Description	Regression and/or Elimination of Polyps from baseline to Month 3
Time Frame	Baseline and Month 3

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Zimura + Anti-VEGF
Arm/Group Description	Zimura 1mg/eye intravitreal injection + Anti-VEGF (Avastin 1.25mg/eye or Lucentis 0.5mg/eye or Eylea 2mg/eye) intravitreal injection
Measure Participants	4
Number [Polyps]	NA

Adverse Events

Time Frame	3 Months
Arm/Group Title	Zimura + Anti-VEGF
Arm/Group Description	Zimura 1mg/eye intravitreal injection + Anti-VEGF (Avastin 1.25mg/eye or Lucentis 0.5mg/eye or Eylea 2mg/eye) intravitreal injection
All Cause Mortality
	Zimura + Anti-VEGF
	Affected / at Risk (%)	# Events
Total	0/4 (0%)
Serious Adverse Events
	Zimura + Anti-VEGF
	Affected / at Risk (%)	# Events
Total	1/4 (25%)
Infections and infestations
Endophthalmitis	1/4 (25%)	1
Other (Not Including Serious) Adverse Events
	Zimura + Anti-VEGF
	Affected / at Risk (%)	# Events
Total	3/4 (75%)
Eye disorders
Conjunctival haemorrhage	3/4 (75%)	4
Gastrointestinal disorders
Gastroesophageal reflux disease	1/4 (25%)	1

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

Institution agrees not to individually publish the results of the Study without Ophthotech's prior written consent. Institution may participate in a joint, multi-center publication of the Study results with other investigators and/or institutions only, upon the prior written consent of Ophthotech.