Study to Evaluate OXERVATE™ in Patients With Stage 1 Neurotrophic Keratitis (DEFENDO)
Primary Purpose
Neurotrophic Keratitis
Status
Completed
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
cenegermin-bkbj
Sponsored by
About this trial
This is an interventional treatment trial for Neurotrophic Keratitis focused on measuring Stage 1 Neurotrophic Keratitis, Corneal Disease, Ocular Disease
Eligibility Criteria
Inclusion Criteria:
- Male or female aged ≥ 18 years.
- Patients with Stage 1 NK defined by the Mackie criteria
Exclusion Criteria:
- Evidence of an active ocular infection (bacterial, viral, protozoal) in either eye.
- Have current or history of conditions that may confound the study data including but not limited to Ocular Cicatricial Pemphigoid (OCP), Graft Versus Host Disease (GVHD), neuromyelitis optica, uncontrolled dry eye, and Steven Johnson's syndrome.
- History of severe systemic allergy or severe ocular allergy (including seasonal conjunctivitis expected during the subject's participation in the trial) or chronic conjunctivitis and/or keratitis other than dry eye disease.
- Patients with severe vision loss with no potential for visual improvement in the study eye, in the opinion of the investigator, or if the subject is deemed legally blind.
- Ocular surgery or elective ocular surgery expected during participation in the trial.
- Patients with eyelid abnormality that may alter eyelid function including but not limited to Blepharospasm, Cerebrovascular accident, entropion, ectropion, floppy lid syndrome.
Sites / Locations
- San Diego
- Edgewood
- Boston
- Saint Louis
- Philadelphia
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
OXERVATE™ 0.002% (20 mcg/mL) cenegermin-bkbj
Arm Description
Outcomes
Primary Outcome Measures
Corneal Epithelial Healing
Percentage of patients who experience corneal epithelial healing. Corneal epithelial healing was defined by the central reading center as the absence of persistent epithelial staining abnormalities related to disease.
Secondary Outcome Measures
Full Information
NCT ID
NCT04485546
First Posted
July 21, 2020
Last Updated
June 23, 2023
Sponsor
Dompé Farmaceutici S.p.A
Collaborators
Dompé US
1. Study Identification
Unique Protocol Identification Number
NCT04485546
Brief Title
Study to Evaluate OXERVATE™ in Patients With Stage 1 Neurotrophic Keratitis
Acronym
DEFENDO
Official Title
An 8-week, Multicenter, Open Label, Prospective Study With 24 Weeks of Follow-up to Evaluate Safety and Efficacy of OXERVATE™ 0.002% (20 mcg/mL) Cenegermin-bkbj Ophthalmic Solution in Patients With Stage 1 Neurotrophic Keratitis (NK)
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
September 9, 2020 (Actual)
Primary Completion Date
September 30, 2021 (Actual)
Study Completion Date
March 30, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Dompé Farmaceutici S.p.A
Collaborators
Dompé US
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study is to evaluate the safety and efficacy of OXERVATE™ 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution in patients with Stage 1 neurotrophic keratitis (NK).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neurotrophic Keratitis
Keywords
Stage 1 Neurotrophic Keratitis, Corneal Disease, Ocular Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
37 (Actual)
8. Arms, Groups, and Interventions
Arm Title
OXERVATE™ 0.002% (20 mcg/mL) cenegermin-bkbj
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
cenegermin-bkbj
Other Intervention Name(s)
OXERVATE™ 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution
Intervention Description
Cenegermin-bkbj ophthalmic solution administered as one drop in affected eye(s) every 2 hours 6 times daily for 8 weeks
Primary Outcome Measure Information:
Title
Corneal Epithelial Healing
Description
Percentage of patients who experience corneal epithelial healing. Corneal epithelial healing was defined by the central reading center as the absence of persistent epithelial staining abnormalities related to disease.
Time Frame
Week 8
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female aged ≥ 18 years.
Patients with Stage 1 NK defined by the Mackie criteria
Exclusion Criteria:
Evidence of an active ocular infection (bacterial, viral, protozoal) in either eye.
Have current or history of conditions that may confound the study data including but not limited to Ocular Cicatricial Pemphigoid (OCP), Graft Versus Host Disease (GVHD), neuromyelitis optica, uncontrolled dry eye, and Steven Johnson's syndrome.
History of severe systemic allergy or severe ocular allergy (including seasonal conjunctivitis expected during the subject's participation in the trial) or chronic conjunctivitis and/or keratitis other than dry eye disease.
Patients with severe vision loss with no potential for visual improvement in the study eye, in the opinion of the investigator, or if the subject is deemed legally blind.
Ocular surgery or elective ocular surgery expected during participation in the trial.
Patients with eyelid abnormality that may alter eyelid function including but not limited to Blepharospasm, Cerebrovascular accident, entropion, ectropion, floppy lid syndrome.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Flavio Mantelli, MD, PhD
Organizational Affiliation
Domp Farmaceutici SpA
Official's Role
Study Chair
Facility Information:
Facility Name
San Diego
City
San Diego
State/Province
California
ZIP/Postal Code
92122
Country
United States
Facility Name
Edgewood
City
Edgewood
State/Province
Kentucky
ZIP/Postal Code
41017
Country
United States
Facility Name
Boston
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02111
Country
United States
Facility Name
Saint Louis
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63131
Country
United States
Facility Name
Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
27683068
Citation
Mastropasqua L, Massaro-Giordano G, Nubile M, Sacchetti M. Understanding the Pathogenesis of Neurotrophic Keratitis: The Role of Corneal Nerves. J Cell Physiol. 2017 Apr;232(4):717-724. doi: 10.1002/jcp.25623. Epub 2016 Oct 17.
Results Reference
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Study to Evaluate OXERVATE™ in Patients With Stage 1 Neurotrophic Keratitis
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