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A Study to Evaluate Efficacy, Safety & Pharmacokinetics of the Port Delivery System (PDS) With Ranibizumab in Participants With Diabetic Macular Edema (DME) Compared With Intravitreal Ranibizumab; A Subtudy to Evaluate the Safety of Re-Implanting the PDS With Ranibizumab in Participants With DME

Phase 3
Recruiting
Conditions
Diabetic Macular Edema
Interventions
Drug: PDS Implant Pre-Filled with 100 mg/mL Ranibizumab
Drug: Intravitreal Ranibizumab 0.5 mg Injection
Registration Number
NCT04108156
Lead Sponsor
Hoffmann-La Roche
Brief Summary

This study will evaluate the efficacy, safety, and pharmacokinetics of the PDS with Ranibizumab in participants with DME when treated every 24 weeks (Q24W) compared with intravitreal ranibizumab 0.5 mg every 4 weeks (Q4W).

The substudy will evaluate safety of re-implanting the updated PDS with ranibizumab and the refill-exchange procedures following re-implantation in participants with DME who were previously enrolled in the main Study, GR40550. Up to 100 participants from the main study will be enrolled and followed for a maximum of 72 weeks post-re-implantation in the substudy.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
634
Inclusion Criteria
  • Age ≥18 years at time of signing Informed Consent Form
  • Documented diagnosis of diabetes mellitus (Type 1 or Type 2)
  • HbA1c level of ≤10% within 2 months prior to screening or at screening

Study eye

  • Macular thickening secondary to DME involving the center of the fovea with CST ≥325 um on SD-OCT at screening
  • BCVA score of 78 to 25 letters (20/32 to 20/320 approximate Snellen equivalent)
Exclusion Criteria
  • High-risk proliferative diabetic retinopathy
  • Active intraocular inflammation (grade trace or above)
  • Suspected or active ocular or periocular infection of either eye
  • Uncontrolled ocular hypertension or glaucoma and any such condition the investigator determines may require a glaucoma-filtering surgery during a patient's participation in the study
  • Cerebrovascular accident or myocardial infarction within 6 months prior to randomization
  • Atrial fibrillation diagnosis or worsening within 6 months prior to randomization
  • Uncontrolled blood pressure

Substudy:

Inclusion Criteria:

  • Having experienced a septum dislodgement in the original implant while in the main study or after exiting the main study
  • Sufficiently clear ocular media and adequate pupillary dilation to allow for analysis and grading by central reading center

Exclusion Criteria (Cohort 1 Only):

  • Recent history (in the last 3 months prior to enrollment) of other disease, other non-diabetic metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a condition that contraindicates the use of ranibizumab or surgical placement of the PDS implant; that might affect interpretation of the results of the study; or that renders the participant at high risk for treatment complications
  • Active cancer within the last 12 months, except for appropriately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, or prostate cancer
  • Current systemic treatment for a confirmed active systemic infection
  • Participation in an investigational trial that involves treatment with any drug or device (with the exception of vitamins and minerals or enrollment in Study GR40550) within 6 months prior to enrollment
  • Use of antimitotic or antimetabolite therapy within 30 days

Ocular Exclusion Criteria for Study Eye:

  • Any ocular condition that may render the participant at high risk for surgical or treatment complications
  • Intraocular surgery (including cataract surgery) within 1 month preceding the enrollment visit
  • Any use of medicated intraocular implants (other than the PDS implant), at any time prior to enrollment
  • History of rhegmatogenous retinal tears or peripheral retinal breaks within 3 months prior to the enrollment visit
  • Any concurrent ocular condition that would require surgical intervention during the study to prevent or treat visual loss
  • Concurrent conjunctival, Tenon's capsule, and/or scleral condition in the supero-temporal quadrant of the eye (e.g., scarring, thinning, mass) that may affect the refill-exchange procedure of the PDS implant
  • Ongoing ocular complications that might affect participant safety Ocular Exclusion Criteria for Either Eye
  • Suspected or active ocular or periocular infection (e.g., infectious conjunctivitis or endophthalmitis)
  • Any history of uveitis
  • Active blepharitis

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PDS ArmRanibizumab refill exchangeParticipants randomized to the PDS arm will receive intravitreal ranibizumab injection every 4 weeks (loading phase) and will then have the PDS implant (pre-filled with ranibizumab) surgically inserted. PDS implant refill-exchange procedures will be performed on a fixed interval every 24-weeks (Q24W) thereafter
Intravitreal ArmRanibizumab refill exchangeParticipants randomized to the intravitreal arm will receive intravitreal ranibizumab injection every 4 weeks until they receive the PDS implant (pre-filled with ranibizumab). PDS implant refill-exchange procedures will be performed on a fixed interval Q24W thereafter.
Substudy: Cohort 1Ranibizumab refill exchangeParticipants will undergo re-implantation with the updated PDS implant (pre-filled with ranibizumab 100 mg/mL) on Day 1 (or enrollment visit) and then will have two refill-exchanges (ranibizumab 100 mg/mL) Q24W up to 48 weeks.
Substudy: Cohort 2aRanibizumab refill exchangeParticipants who received an updated PDS implant, have \< 24 weeks post-re-implantation follow-up and no refill exchange visit in the main study, will undergo two refill-exchange procedures (ranibizumab 100 mg/mL) Q24W, post main study re-implantation visit.
Substudy: Cohort 2bRanibizumab refill exchangeParticipants who received an updated PDS implant, have \< 48 weeks post-re-implantation follow-up and one refill exchange visit in the main study, will undergo one refill-exchange procedure (ranibizumab 100 mg/mL) Q24W, post main study re-implantation visit.
Substudy: Cohort 1PDS Implant Pre-Filled with 100 mg/mL RanibizumabParticipants will undergo re-implantation with the updated PDS implant (pre-filled with ranibizumab 100 mg/mL) on Day 1 (or enrollment visit) and then will have two refill-exchanges (ranibizumab 100 mg/mL) Q24W up to 48 weeks.
PDS ArmPDS Implant Pre-Filled with 100 mg/mL RanibizumabParticipants randomized to the PDS arm will receive intravitreal ranibizumab injection every 4 weeks (loading phase) and will then have the PDS implant (pre-filled with ranibizumab) surgically inserted. PDS implant refill-exchange procedures will be performed on a fixed interval every 24-weeks (Q24W) thereafter
Intravitreal ArmIntravitreal Ranibizumab 0.5 mg InjectionParticipants randomized to the intravitreal arm will receive intravitreal ranibizumab injection every 4 weeks until they receive the PDS implant (pre-filled with ranibizumab). PDS implant refill-exchange procedures will be performed on a fixed interval Q24W thereafter.
Primary Outcome Measures
NameTimeMethod
Change in BCVA Score From Baseline Averaged Over Weeks 60 and 64 as Measured Using the ETDRS Chart in the Efficacy Population Using a Treatment Policy Strategy for All Intercurrent EventsBaseline to Week 64

BCVA = Best-Corrected Visual Acuity

ETDRS = Early Treatment Diabetic Retinopathy Study

A vision score of 20/20 vision is considered normal. A score of 20/200 is considered being legally blind.

Substudy: Number of Participants with Ocular and Systemic (Non-ocular) Adverse Events (AEs) and Severity of Ocular and Systemic AEsBaseline to Week 72
Substudy: Number of Participants with Adverse Events of Special Interests (AESIs) and Severity of AESIsBaseline to Week 72
Substudy: Duration of AESIsBaseline to Week 72
Substudy: Number of Participants with Ocular AESIs and Severity of Ocular AESIs During the Post-operative PeriodUp to Day 37 post re-implantation
Substudy: Number of Participants with Ocular AESIs and Severity of Ocular AESIs During the Follow-up Period> 37 days post-implantation (up to approximately 72 weeks)
Substudy: Duration of Ocular AESIs During the Post-operative PeriodUp to Day 37 post re-implantation
Substudy: Duration of Ocular AESIs During the Follow-up Period> 37 days post-implantation (up to approximately 72 weeks)
Substudy: Number of Participants with Adverse Device Effects (ADEs) and Severity of ADEsBaseline to Week 72
Substudy: Number of Participants with Anticipated Serious ADEsBaseline to Week 72
Substudy: Duration of Serious ADEsBaseline to Week 72
Substudy: Number of Device DeficienciesBaseline to Week 72
Secondary Outcome Measures
NameTimeMethod
Change in BCVA Score From Baseline Averaged Over Weeks 60 And 64 as Measured with Use of the ETDRS Chart in the mITT Population Using a Hypothetical Strategy for All Intercurrent EventsBaseline to Week 64
Percentage of Participants with a ≥2-step Improvement from Baseline on the ETDRS-DRSS at Week 64 in the Efficacy PopulationBaseline to Week 64
Percentage of Participants with a ≥2-step Improvement from Baseline on the ETDRS-DRSS at Week 64 in the mITT populationBaseline to Week 64
Change from Baseline in BCVA as Measured on the ETDRS Chart Over TimeBaseline up to Week 120
Change in BCVA Score from Baseline Averaged Over Weeks 60 and 64 as Measured With Use of the ETDRS Chart in the Modified Intent-To-Treat (mITT) Population Using a Treatment Policy Strategy for All Intercurrent EventsBaseline to Week 64

ETDRS-DRSS = ETDRS Diabetic Retinopathy Severity Scale

Percentage of Participants Who Lose <15, <10, and <5 letters in BCVA from Baseline Over TimeBaseline up to Week 120
Percentage of Participants Who Gain ≥15, ≥10, ≥5, ≥0 Letters in BCVA from Baseline Over TimeBaseline up to Week 120
Percentage of Participants with a BCVA Snellen Equivalent of 20/40 or Better Over TimeBaseline up to Week 120
Percentage of Participants with a BCVA Snellen Equivalent of 20/200 or Worse Over TimeBaseline up to Week 120
Percentage of Participants with a ≥2-step Improvement from Baseline on the ETDRS-DRSS Over TimeBaseline up to Week 120
Percentage of Participants with a ≥3-step Improvement from Baseline on the ETDRS-DRSS Over TimeBaseline up to Week 120
Time to ≥2-step Worsening from Baseline on the ETDRS-DRSSBaseline up to Week 120
Time to ≥3-step Worsening from Baseline on the ETDRS-DRSSBaseline up to Week 120
Change from Baseline in ETDRS-DRSS Score Over TimeBaseline up to Week 120
Change from Baseline in CST as Measured on SD-OCT Over TimeBaseline up to Week 120
Change from Baseline in Total Macular Volume as Measured on SD-OCT Over TimeBaseline up to Week 120
Percentage of Participants with Absence of Intraretinal Fluid Over Time (Intraretinal Fluid as Measured in the Central 1 mm Subfield)Baseline up to Week 120
Percentage of Participants with Absence of Subretinal Fluid Over Time (Subretinal Fluid as Measured in the Central 1 mm Subfield)Baseline up to Week 120
Percentage of Participants with Absence of Intraretinal Fluid and Subretinal Fluid Over TimeBaseline up to Week 120
Percentage of Participants with Absence DME (Defined as CST ≥325 μm on SD-OCT) Over TimeBaseline up to Week 120

DME = diabetic macular edema

Time to PDR (Defined as a Score ≥60 on the ETDRS-DRSS)Baseline up to Week 120

PDR = proliferative diabetic retinopathy

Percentage of Participants Who do Not Undergo Supplemental Treatment with Intravitreal Ranibizumab Within Each Refill-Exchange IntervalBaseline up to Week 120
Percentage of Participants Who Report Preferring PDS Treatment Compared with Intravitreal Ranibizumab TreatmentBaseline to Week 64

As measured by the PDS Patient Preference Questionnaire at Week 64 among patients in the PDS arm efficacy population, mITT population

Percentage of Participants Who Report Preferring PDS Treatment Compared with Intravitreal Ranibizumab Treatment, as Measured by the PDS Patient Preference Questionnaire (PPPQ) at Week 64Baseline to Week 64

Participants in in a subset of patients with bilateral disease who are simultaneously receiving ranibizumab via study eye PDS implant and fellow eye intravitreal injection

Patient-reported Vision-Related Functioning and Health-Related Quality of Life (HRQoL) Among Patients in Both Treatment Arms, as Measured by Changes from Baseline, Baseline Week 48, Week 96

As measured by in the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) composite score and Near Activities, Distance Activities, and Driving subscale scores

Patient-reported Vision-related Functioning and HRQoL, as Measured by the Proportion of Patients with a ≥ 4-Point Improvement from Baseline in the NEI VFQ-25 Composite Score at Weeks 48 and 96 Among Patients in Both Treatment ArmsBaseline, Week 48, Week 96
Incidence and Severity of Ocular Adverse EventsBaseline up to Week 120
Incidence and Severity of Non-ocular Adverse EventsBaseline up to Week 120
Incidence, Severity, and Duration of Adverse Events of Special InterestBaseline up to Week 120
Serum Concentration of Ranibizumab Observed Over TimeBaseline up to Week 120
PK Parameter Value Area Under the Concentration- Time Curve Over 24 weeks (AUC24W)Baseline to Week 24
Pharmacokinetic (PK) Parameter Maximum Serum Concentration (Cmax)Baseline up to Week 120
PK Parameter Minimum Serum Concentration (Cmin)Baseline up to Week 120
Time of Maximum Observed Serum Concentration (Tmax) After PDS Implant InsertionBaseline up to Week 120
Prevalence of Anti-drug Antibodies (ADAs) at Baseline and Incidence of ADAs During the StudyBaseline up to Week 120
Prevalence of Neutralizing Antibodies at Baseline and Incidence of Neutralizing Antibodies During the StudyBaseline up to Week 120
Reported Incidence of Device DeficienciesBaseline up to Week 120
Incidence, Severity, and Duration of Ocular Adverse Events of Special Interest During the Postoperative Period (up to 37 Days After Initial Implantation) and Follow-up period (> 37 days After Implantation Surgery)Baseline up to Week 120
Incidence and Severity of ADEsBaseline up to Week 120
Incidence, Causality, Severity, and Duration of Anticipated Serious Adverse Device EffectsBaseline up to Week 120
Substudy: Number of Participants with Ocular AESIs and Severity of Ocular AESIs After Refill-exchange ProcedureUp to approximately 72 weeks
Substudy: Duration of AESIs After Refill-exchange ProcedureUp to approximately 72 weeks
Substudy: Number of Participants With ADEs and Severity of ADEs After Refill-exchange ProcedureUp to approximately 72 weeks
Substudy: Number of Participants with Anticipated Serious ADEs After Refill-exchange ProcedureUp to approximately 72 weeks
Substudy: Duration of Serious ADEs After Refill-exchange ProcedureUp to approximately 72 weeks
Substudy: Number of Device Deficiencies After Refill-exchange ProcedureUp to approximately 72 weeks

Trial Locations

Locations (90)

Retinal Consultants of Arizona;Opthalmology

🇺🇸

Phoenix, Arizona, United States

Barnet Dulaney Perkins Eye Center

🇺🇸

Mesa, Arizona, United States

Arizona Retina and Vitreous Consultants

🇺🇸

Phoenix, Arizona, United States

Jules Stein Eye Institute/ UCLA

🇺🇸

Los Angeles, California, United States

Northern California Retina-Vitreous Associates

🇺🇸

Mountain View, California, United States

Associated Retina Consultants

🇺🇸

Phoenix, Arizona, United States

California Retina Consultants

🇺🇸

Bakersfield, California, United States

The Retina Partners

🇺🇸

Encino, California, United States

Retina Consultants of Orange County;Clinical Research

🇺🇸

Fullerton, California, United States

East Bay Retina Consultants

🇺🇸

Oakland, California, United States

Doheny Eye Institute

🇺🇸

Pasadena, California, United States

Orange County Retina Medical Group

🇺🇸

Santa Ana, California, United States

Eye Center of Northern Colorado

🇺🇸

Fort Collins, Colorado, United States

Retina Group of New England

🇺🇸

Waterford, Connecticut, United States

Retina Group of Florida

🇺🇸

Fort Lauderdale, Florida, United States

National Ophthalmic Research Institute

🇺🇸

Fort Myers, Florida, United States

Southeast Retina Center

🇺🇸

Augusta, Georgia, United States

Georgia Retina

🇺🇸

Marietta, Georgia, United States

Northwestern Memorial Hospital

🇺🇸

Chicago, Illinois, United States

Illinois Retina Associates

🇺🇸

Joliet, Illinois, United States

Retina-Vitreous Associates Medical Group

🇺🇸

Beverly Hills, California, United States

California Eye Specialists Medical Group

🇺🇸

Pasadena, California, United States

Retina Consultants Medical Group

🇺🇸

Sacramento, California, United States

Zuckerberg San Francisco General Hospital and Trauma Center

🇺🇸

San Francisco, California, United States

California Retina Consultants;Research Department

🇺🇸

Santa Barbara, California, United States

Colorado Clinical Research

🇺🇸

Lakewood, Colorado, United States

Retina Specialty Institute

🇺🇸

Pensacola, Florida, United States

Fort Lauderdale Eye Institute

🇺🇸

Plantation, Florida, United States

Retina Vitreous Associates of Florida

🇺🇸

Saint Petersburg, Florida, United States

Southern Vitreoretinal Associates;Research

🇺🇸

Tallahassee, Florida, United States

Retina Associates of Florida;Retina Associates of Florida

🇺🇸

Tampa, Florida, United States

University Retina

🇺🇸

Lemont, Illinois, United States

Wolfe Eye Clinic

🇺🇸

West Des Moines, Iowa, United States

Retina Associates

🇺🇸

Lenexa, Kansas, United States

Retina & Vitreous Associates of Kentucky

🇺🇸

Lexington, Kentucky, United States

Maine Eye Center

🇺🇸

Portland, Maine, United States

The Retina Care Center

🇺🇸

Baltimore, Maryland, United States

Johns Hopkins Hospital;Johns Hopkins Med;Wilmer Eye Inst

🇺🇸

Baltimore, Maryland, United States

The Retina Group of Washington;Retinal Disease

🇺🇸

Chevy Chase, Maryland, United States

Cumberland Valley Retina Consultants;Clinical Research

🇺🇸

Hagerstown, Maryland, United States

Retina Specialist

🇺🇸

Towson, Maryland, United States

Tufts Medical Center

🇺🇸

Boston, Massachusetts, United States

Ophthalmic Consultants of Boston

🇺🇸

Boston, Massachusetts, United States

Vitreo Retinal Associates

🇺🇸

Worcester, Massachusetts, United States

Retina Specialists Of Michigan

🇺🇸

Grand Rapids, Michigan, United States

Associated Retinal Consultants - Royal Oak

🇺🇸

Royal Oak, Michigan, United States

VitreoRetinal Surgery PLLC;DBA Retina Consultants of Minnesota

🇺🇸

Minneapolis, Minnesota, United States

Pepose Vision Institute

🇺🇸

Chesterfield, Missouri, United States

Retina Institute

🇺🇸

Saint Louis, Missouri, United States

Sierra Eye Associates

🇺🇸

Reno, Nevada, United States

Envision Ocular, LLC

🇺🇸

Bloomfield, New Jersey, United States

Retina Associates of New Jersey

🇺🇸

Teaneck, New Jersey, United States

Long Island Vitreoretinal Consultants;Opthalmology

🇺🇸

Great Neck, New York, United States

Retina Vitreous Surgeons of Central New York

🇺🇸

Liverpool, New York, United States

New York University (NYU)

🇺🇸

New York, New York, United States

Ophthalmic Consultants of Long Island

🇺🇸

Oceanside, New York, United States

Asheville Eye Associates Western Carolina Retinal Associates;Clinical Research

🇺🇸

Asheville, North Carolina, United States

Charlotte Eye Ear Nose and Throat Associates- SouthPark;Retina

🇺🇸

Charlotte, North Carolina, United States

Duke Eye Center

🇺🇸

Durham, North Carolina, United States

Graystone Eye;Clinical Research

🇺🇸

Hickory, North Carolina, United States

Cape Fear Retinal Associates

🇺🇸

Wilmington, North Carolina, United States

Cincinnati Eye Institute;Retina

🇺🇸

Cincinnati, Ohio, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

Ohio State Havener Eye Institute;Ophthalmology Research

🇺🇸

Columbus, Ohio, United States

Midwest Retina;Retina/Vitreous

🇺🇸

Dublin, Ohio, United States

Retina Vitreous Center - Glen Eagles

🇺🇸

Edmond, Oklahoma, United States

Retina Northwest;Research Department

🇺🇸

Portland, Oregon, United States

Cumberland Valley Retina Consultants

🇺🇸

Chambersburg, Pennsylvania, United States

Mid Atlantic Retina

🇺🇸

Philadelphia, Pennsylvania, United States

Charleston Neuroscience

🇺🇸

Ladson, South Carolina, United States

Carolina Eyecare Physicians

🇺🇸

Mount Pleasant, South Carolina, United States

Palmetto Retina Center

🇺🇸

West Columbia, South Carolina, United States

Charles Retina Institution;Retina surgery

🇺🇸

Germantown, Tennessee, United States

Tennessee Retina

🇺🇸

Nashville, Tennessee, United States

Retina Research Institute of Texas

🇺🇸

Abilene, Texas, United States

Texas Retina Associates

🇺🇸

Fort Worth, Texas, United States

Austin Research Center for Retina

🇺🇸

Austin, Texas, United States

Austin Retina Associates;Opthalmology

🇺🇸

Austin, Texas, United States

Austin Clinical Research, LLC

🇺🇸

Austin, Texas, United States

Retina & Vitreous of Texas

🇺🇸

Bellaire, Texas, United States

Texas Retina Associates;Research

🇺🇸

Dallas, Texas, United States

Retina Consultants of Texas

🇺🇸

Houston, Texas, United States

Medical Center Ophthalmology Associates

🇺🇸

San Antonio, Texas, United States

Rocky Mountain Retina Consultants

🇺🇸

Murray, Utah, United States

Retina Associates of Utah, PLLC;Clinical Research

🇺🇸

Salt Lake City, Utah, United States

Piedmont Eye Center

🇺🇸

Lynchburg, Virginia, United States

Wagner Kapoor Institute;Opthalmology

🇺🇸

Norfolk, Virginia, United States

Retina Institute of Virginia

🇺🇸

Richmond, Virginia, United States

Pacific Northwest Retina

🇺🇸

Silverdale, Washington, United States

Spokane Eye Clinical Research;Spokane Eye Surgery Center

🇺🇸

Spokane, Washington, United States

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