Clinical Trials Register

Summary
EudraCT Number:	2017-003923-31
Sponsor's Protocol Code Number:	OPH2007
National Competent Authority:	Latvia - SAM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2017-10-24
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Latvia - SAM

A.2

EudraCT number

2017-003923-31

A.3

Full title of the trial

A Phase 2A Open-Label Trial to Assess the Safety of Zimura™ (Anti-C5) Administered in Combination With Lucentis® 0.5 mg in Treatment Naïve Subjects with Neovascular Age Related Macular Degeneration

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A Clinical Trial to Assess the Safety of Zimura™ Administered in Combination With Lucentis® 0.5 mg in Subjects with Neovascular Age Related Macular Degeneration

A.4.1

Sponsor's protocol code number

OPH2007

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	OPHTHOTECH CORPORATION
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	OPHTHOTECH CORPORATION
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	OPHTHOTECH CORPORATION
B.5.2	Functional name of contact point	Patricia Johnson
B.5.3	Address:
B.5.3.1	Street Address	One University Square Drive, Suite 280
B.5.3.2	Town/ city	Princeton
B.5.3.3	Post code	NJ 08540
B.5.3.4	Country	United States
B.5.6	E-mail	patricia.johnson@ophthotech.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Zimura
D.3.2	Product code	ARC1905
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravitreal use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	avacincaptad pegol
D.3.9.3	Other descriptive name	ARC1905 20 MG/ML
D.3.9.4	EV Substance Code	SUB30770
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	20
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Lucentis 10 mg/ml solution for injection
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Europharm Limited
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravitreal use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	RANIBIZUMAB
D.3.9.1	CAS number	347396-82-1
D.3.9.4	EV Substance Code	SUB22314
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Neovascular Age Related Macular Degeneration

E.1.1.1

Medical condition in easily understood language

Neovascular Age Related Macular Degeneration

E.1.1.2

Therapeutic area

Diseases [C] - Eye Diseases [C11]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10071129
E.1.2	Term	Neovascular age-related macular degeneration
E.1.2	System Organ Class	10015919 - Eye disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the safety of intravitreal Zimura™ administered in combination with Lucentis® in treatment naïve subjects with NVAMD

E.2.2

Secondary objectives of the trial

Not applicable

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Ophthalmic Inclusion Criteria
The following inclusion criteria apply to the study eye:

1. Active subfoveal NVAMD as documented by fluorescein angiography.
2. Presence of intraretinal and/or subretinal fluid on OCT within the central 1 mm subfield.
3. Best corrected visual acuity in the study eye between 20/63 and 20/200, inclusive at Screening and reconfirmed at Day 1 according to Reconfirmation of Eligibility Criteria
4. Total area of the lesion (including blood, neovascularization, and scar/atrophy) must be ≤ 5 disc areas (DA), of which at least 50% must be active CNV. Active CNV is defined as the neovascular component of the lesion as defined by the fluorescein angiography.
5. Clear ocular media and adequate pupillary dilatation to permit good quality stereoscopic fundus photography.
6. Intraocular pressure (IOP) of 21 mmHg or less.

General Inclusion Criteria

7. Subjects of either gender aged ≥ 50 years.
8. Performance Status ≤ 2 according to Eastern Cooperative Oncology Group (ECOG) / World Health Organization (WHO) scale (Appendix 17.6)
9. Normal electrocardiogram (ECG) or clinically non-significant changes.
10. Women must be using two forms of effective contraception, be post-menopausal for at least 12 months prior to trial entry, or surgically sterile; if of child-bearing potential, a serum pregnancy test must be performed within 14 days prior to the first injection with a negative result. The two forms of effective contraception must be implemented during the trial and for at least 60 days following the last dose of test medication.
11. Provide written informed consent.
12. Ability to return for all trial visits

E.4

Principal exclusion criteria

Ophthalmic Exclusion Criteria

1. Any prior treatment for AMD or any intravitreal treatment for any indication in the study eye prior to the Baseline visit, except oral supplements of vitamins and minerals.
2. Presence of significant serous pigment epithelial detachments (PEDs), such as large PEDs constitute greater than 50% of the total lesion or have a vertical height of > 400 μm. Presence of pure PED without CNV.
3. Presence of pigment epithelial tears or rips.
4. Presence of retinal angiomatous proliferation (RAP).
5. More than 25% of the total lesion size made up of scarring or atrophy. Subjects with subfoveal scar or subfoveal atrophy are excluded.
6. More than 50% of the total lesion size consisting of subretinal hemorrhage.
7. Presence of intraocular inflammation (≥ trace cell or flare), macular hole, epiretinal membrane, vitreous hemorrhage or aphakia (pseudophakia with or without an intact capsule is not an exclusion criteria).
8. Presence of idiopathic or autoimmune-associated uveitis in either eye.
9. Significant media opacities, including cataract, which might interfere with visual acuity, assessment of toxicity, or fundus photography. Subjects should not be entered if there is likelihood that they will require cataract surgery in the study eye in the following year.
10. Presence of other causes of choroidal neovascularization, including pathologic myopia (spherical equivalent of -8 diopters or more negative, or axial length of 25mm or more), the ocular histoplasmosis syndrome, angioid streaks, choroidal rupture, and multifocal choroiditis.
11. Any intraocular surgery or thermal laser within three (3) months of trial entry. Any prior thermal laser in the macular region, regardless of indication.
12. History of any of the following procedures: Posterior vitrectomy, filtering surgery (e.g. trabeculectomy), glaucoma drainage device, corneal transplant, or retinal detachment.
13. Previous or concomitant therapy with intravitreal corticosteroids.
14. A diagnosis of diabetic retinopathy (presence of microaneurysms or any vasculopathy and/or leakage from retinal vasculature in a subject with diabetes mellitus).
15. Previous history of any vasculopathy and/or leakage from retinal or choroidal vasculature.

General Exclusion Criteria

16. Any of the following underlying diseases including:
- History or evidence of severe cardiac disease (e.g., NYHA Functional Class III or IV - see Appendix 17.5), history or clinical evidence of unstable angina, acute coronary syndrome, myocardial infarction or revascularization within last 6 months, ventricular tachyarrhythmia requiring ongoing treatment.
- History or evidence of clinically significant peripheral vascular disease, such as intermittent claudication or prior amputation.
- Stroke within 12 months of trial entry.
- Any major surgical procedure within one month of trial entry.
17. Subjects with a clinically significant laboratory value. Laboratory tests may be repeated once before randomization.
18. Previous therapeutic radiation in the region of the study eye.
19. Any treatment with an investigational agent in the past 60 days for any condition.
20. Women who are pregnant or nursing.
21. Known serious allergies to the fluorescein dye used in angiography, povidone iodine, to the components of the ranibizumab formulation, or to the components of the Zimura™ formulation

E.5 End points

E.5.1

Primary end point(s)

Safety endpoints include:
- Adverse events (AEs)
- Vital signs (respiration, heart rate, systolic and diastolic blood pressure, etc.)
- ECG recordings
- Ophthalmic variables (visual acuity, IOP, ophthalmologic examination, stereoscopic fundus photography, fluorescein angiography, and spectral domain optical coherence tomography)
- Laboratory variables (blood: hematology, renal function, hepatic function, and electrolytes; urine: urinalysis)

E.5.1.1

Timepoint(s) of evaluation of this end point

Adverse events: All visits after injection
Vital signs and laboratory tests: Screening, day 9 (only Cohort 1), month 6
ECG: Screening, month 6
Ophthalimc examination, IOP, Visual acuty: Each visit
Color fundus photography and Fluorescein angiography: Screening, baseline, month 3+6
SD-optical coherence tomography: Screening, baseline, day 9 (only Cohort 1), month 1-6

E.5.2

Secondary end point(s)

Not applicable

E.5.2.1

Timepoint(s) of evaluation of this end point

Not applicable

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Hungary

Latvia

United States

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2018-01-30

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-10-31

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2018-10-18

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