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Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03364153
Recruitment Status : Active, not recruiting
First Posted : December 6, 2017
Last Update Posted : March 4, 2019
Sponsor:
Information provided by (Responsible Party):
Ophthotech Corporation

Tracking Information
First Submitted Date  ICMJE December 1, 2017
First Posted Date  ICMJE December 6, 2017
Last Update Posted Date March 4, 2019
Actual Study Start Date  ICMJE January 12, 2018
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 1, 2017)
Spectral Domain-Optical Coherence Tomography (SD-OCT) [ Time Frame: 18 months ]
Mean rate of change in the area of ellipsoid zone defect measured by en face SD-OCT
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03364153 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
Official Title  ICMJE A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects With Autosomal Recessive Stargardt Disease
Brief Summary To evaluate the safety and efficacy of Zimura™ (complement factor C5 inhibitor) compared to Sham in subjects with autosomal recessive Stargardt disease 1 (STGD1).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Stargardt Disease 1
Intervention  ICMJE
  • Drug: Zimura
    Zimura Intravitreal Injection
    Other Name: avacincaptad pegol
  • Other: Sham
    Sham Intravitreal Injection
Study Arms  ICMJE
  • Experimental: Cohort 1
    Zimura dose group
    Intervention: Drug: Zimura
  • Sham Comparator: Cohort 2
    Sham dose group
    Intervention: Other: Sham
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: February 28, 2019)
95
Original Estimated Enrollment  ICMJE
 (submitted: December 1, 2017)
120
Estimated Study Completion Date  ICMJE September 2020
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • At least two pathogenic mutations of ABCA4 gene confirmed by a CLIA-certified laboratory
  • Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen equivalent, inclusive

Exclusion Criteria:

  • Macular atrophy secondary to any condition other than STGD1 in either eye
  • Any prior treatment for STGD1 including gene therapy, stem cell therapy or any prior intravitreal treatment for any indication in either eye
  • Participation in an interventional study of a vitamin A derivative ≤ 3 months prior to screening
  • Presence of intraocular inflammation, macular hole, pathologic myopia, epiretinal membrane, evidence of significant vitreo-macular traction, vitreous hemorrhage or aphakia
  • Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region
  • Diabetes mellitus
  • HbA1c value of ≥6.5%
  • Stroke within 12 months of trial entry
  • Any major surgical procedure within one month of trial entry or anticipated during the trial
  • Any treatment with an investigational agent in the past 60 days for any condition
  • Women who are pregnant or nursing
  • Known serious allergies to the fluorescein dye used in angiography, povidone iodine, or to the components of the Zimura formulation
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 50 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Germany,   Hungary,   Israel,   Italy,   Spain,   United Kingdom,   United States
Removed Location Countries Canada
 
Administrative Information
NCT Number  ICMJE NCT03364153
Other Study ID Numbers  ICMJE OPH2005
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ophthotech Corporation
Study Sponsor  ICMJE Ophthotech Corporation
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Ophthotech Corporation
Verification Date August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP