Clinical trial NCT05552261: DEFENDO Long Term Follow-up Study in Stage 1 NK Patients
Official title: A Long-term Extension Study to Evaluate the Safety and Efficacy of OXERVATE 0.002% (20 mcg/mL) Cenegermin-bkbj Ophthalmic Solution in Patients With Stage 1 Neurotrophic Keratitis Who Enrolled in the DEFENDO Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Neurotrophic Keratitis
Keywords: Stage 1 NK
Oxervate
Cenegermin
NK
DEFENDO
Lead sponsor: Dompé Farmaceutici S.p.A
Sponsor class: INDUSTRY
First posted: 2022-09-23
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Stage 1 NK

Brief summary: The primary objectives are to evaluate the long-term safety and efficacy of OXERVATE™ 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution in Stage 1 Neurotrophic Keratitis (NK) patients who enrolled in the DEFENDO Study.

Detailed description: This clinical study will be a multi-center, open label, long term follow-up study of the patients who were enrolled in the DEFENDO Study who had Stage 1 Neurotrophic Keratitis (NK) who were treated with OXERVATE™ 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution.

The DEFENDO Long-Term Follow-up Study will have follow-up with the patients at month 24-30 post-treatment in the DEFENDO Study: All patients will be evaluated in a real-world setting post the enrollment in the DEFENDO Study through 30 months. Patients will be treated per standard of care as determined by the Investigator and will document topical ophthalmic medications.

The original DEFENDO Study duration was for a total of 34 weeks: a screening period of 2 weeks, followed by enrollment in 8 weeks of OXERVATE™ treatment and an Off- Treatment Follow-Up of 6 months.

After completing enrollment in the original DEFENDO Study, patients will be invited to enter the DEFENDO Long- Term Follow-up Study (all standard of care is permitted).

Two additional long-term follow-up visits will occur at 24- and 30-months to evaluate long-term clinical outcomes.

Eligibility criteria: Inclusion Criteria:

1. Previously enrolled in the DEFENDO Study.
2. Satisfy all Informed Consent requirements. The patient and/or his/her legal representative has read, signed, and dated the IRB approved Informed Consent document before any study-related procedures are performed.
3. Must have the ability and willingness to comply with study procedures.

Exclusion Criteria:

Participating in another study that involves treating the study eye.

a. Participation in non-ocular studies is acceptable provided that the treatment is not considered to be confounding with the DEFENDO Long-Term Follow-up Study, in the opinion of the Investigator.

Primary outcomes:
measure: Corneal Epithelial Healing
timeFrame: From baseline to 24 months
measure: Corneal Epithelial Healing
timeFrame: From baseline to 30 months
Secondary outcomes:
measure: Corneal Sensitivity
timeFrame: 24 months and 30 months
measure: Change from Baseline in BCDVA at 24 months and 30 months
timeFrame: 24 months and 30 months
measure: Change from Baseline in TFBUT at 24 months and 30 months
timeFrame: 24 months and 30 months
measure: Percentage of patients that achieve a 15- letter gain in BCDVA
timeFrame: 24 months and 30 months
measure: Change from baseline in Quality of life (QoL) change from baseline
timeFrame: 24 months and 30 months
measure: Change from baseline in Quality of life (QoL) change from baseline
timeFrame: 24 months and 30 months
measure: Change from baseline in corneal nerve structure (IVCM) at months 24 and 30
timeFrame: 24 months and 30 months
measure: Change from baseline in Optical Coherence Tomography (OCT) at months 24 and 30
timeFrame: 24 months and 30 months
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: Gordon Schanzlin New Eye Institute
state: California
city: Edgewood
country: United States
facility: Cincinnati Eye Institute
state: Kentucky
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Philadelphia
country: United States
facility: Scheie Eye Institute
state: Pennsylvania
Study officials:
affiliation: Dompé
name: Flavio Mantelli, MD, PhD
role: STUDY_CHAIR
References: