Efficacy/Safety of Human Plasminogen Eye Drop in Ligneous Conjunctivitis Patients

NCT ID: NCT01554956

Last Updated: 2023-01-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-05-22
• Study Completion Date: 2020-12-04

Brief Summary

Kedrion Human Plasminogen, a sterile human plasma-derived plasminogen preparation for topical ocular use will be evaluated for the indication of treatment of ligneous conjunctivitis.

KB046 will be an open-label, historically controlled clinical trial. At least 10 subjects with ligneous conjunctivitis, for approximately 20 eyes, will be treated and assessed. All subjects will receive the investigational medicinal product (IMP) for 12 to 48 weeks, with a possibility for extended treatment (Continuation segment)

Detailed Description

Kedrion Human Plasminogen, a sterile human plasma-derived plasminogen preparation for topical ocular use will be evaluated for the indication of treatment of ligneous conjunctivitis.

KB046 will be an open-label, historically controlled clinical trial. At least 10 subjects with ligneous conjunctivitis, for approximately 20 eyes, will be treated and assessed. All subjects will receive the investigational medicinal product (IMP) for 12 to 48 weeks, with a possibility for extended treatment (Continuation segment).

The study will be divided into 3 segments: segments 1 and 2 for assessment of efficacy and safety and segment 3 (continuation segment) assessing long-term safety. For each enrolled patient, both eyes will be treated regardless of unilateral or bilateral involvement. Treatment of the unaffected eyes provided data for the safety assessment. To assess efficacy, comparisons will be made against individual patient historical data.

Conditions

Ligneous Conjunctivitis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Human Plasminogen

Human Plasminogen Eye Drop treatment

Group Type: EXPERIMENTAL

Human Plasminogen

Intervention Type: BIOLOGICAL

Eye Drops

Interventions

Human Plasminogen

Eye Drops

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Subjects should be diagnosed with ligneous conjunctivitis associated with Type I plasminogen deficiency, confirmed by the central laboratory and documented at pre-enrollment screening. The concomitant presence of other ligneous pseudomembranes at different sites will not constitute an exclusion criterion.
• Subjects should have documented historical records of disease course available for a period of at least 6 months surrounding an episode of LC, even if asymptomatic in the past for a newly diagnosed subject , including but not limited to age of LC onset, diagnosis of Plasminogen 1 deficiency, history of pseudomembrane lesions, disease duration, past treatment for LC, response to treatment and/or surgery (including regression and recurrence), before study entrance. If more history than 6 months surrounding an LC episode is available it will be included.
• Subjects, or their legally authorized representative, in the case of study participants < 18 years of age, should have been informed of the nature of the study, agreed to its provision, signed and dated the informed consent approved by the investigational review board (IRB) or ethics committee (EC).
• Subjects available for the duration of the study will be included. The Investigator will make sure that there is no plan for the subject to leave the area of the study site before the end of the study period. If they come from another center, they must agree to be compliant with the protocol mandated study visits and return for follow-up.

Exclusion Criteria

• Subjects presenting ligneous conjunctivitis not associated with Type 1 plasminogen deficiency.
• Subjects with no history of LC lesions for Group 2, for Group 1 the entry lesions could be the first and included as history.
• Subject presenting antibodies against plasminogen at screening.
• Subjects with any condition which, in the opinion of the Investigator, might interfere with the evaluation of the study objectives, or participation in this trial.
• Subjects unwilling to give written informed consent or assent to participation.
• Subjects who have participated in another clinical trial within 1 month before study initiation, i.e. they have received any test drug within 30 days prior the study.
• Females of childbearing potential who are either pregnant or not using an adequate method of birth control
• Females who are breastfeeding.
• Subjects being treated with FFP or Laboratory Grade Plasminogen will undergo a washout period of at least 15 days before being considered for this study. This information will be disseminated to subjects ahead of their Screening Visit and will only occur following signing of the Informed Consent

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kedrion S.p.A.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, United States

Meyer Children's Hospital

Florence, , Italy

AOU Padova

Padua, , Italy

Countries

United States; Italy

References

Caputo R, Shapiro AD, Sartori MT, Leonardi A, Jeng BH, Nakar C, Di Pasquale I, Price FW Jr, Thukral N, Suffredini AL, Pino L, Crea R, Mathew P, Calcinai M. Treatment of Ligneous Conjunctivitis with Plasminogen Eyedrops. Ophthalmology. 2022 Aug;129(8):955-957. doi: 10.1016/j.ophtha.2022.03.019. Epub 2022 Mar 26. No abstract available.

Reference Type: DERIVED

PMID: 35346720 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

KB046

Identifier Type: -

Identifier Source: org_study_id