Clinical trial

Management of Recurrent and Progressive Ligneous Conjunctivitis Due to Plasminogen Deficiency. An N of One Clinical Trial of Topical Administration of Allogenic Plasma to Affected Eye

Name

USASK0511ST

Description

Congenital plasminogen deficiency causes impaired wound healing and growth of pseudomembranous lesions over multiple parts of the body. The most common lesions involve eyes and are known as Ligneous conjunctivitis. These can cause scarring of the sclera, vision loss and even blindness. These pseudomembranous lesions are recur after surgical excisions, administration of intra-ocular cyclosporine, autologous serum drops or corticosteroids. Clinical data shows that these growths do not worsen and do not recur after administration of plasminogen (either as concentrate or as plasma) in the eyes, locally or intravenously. As plasminogen is not available as concentrate, we are using aliquoted allogenic plasma provided by Canadian Blood Services for intra-ocular application. These will be applied to eyes multiple times a day for a period of 2 to 6 months depending on disease severity and patient response. These may be used again if ligneous conjunctivitis recurs. The patient will be followed for a period of 2 years at least. All serious adverse events will be reported to Canadian Blood Services and Health Canada as appropriate.

Trial arms

Trial start

2022-12-15

Estimated PCD

2024-09-30

Trial end

2024-12-31

Status

Recruiting

Treatment

Aliquoted allogeneic donor plasma in patient's conjunctiva

1-2 drops of allogenic aliquoted plasma to affected eye every 1-5 hours per day based on disease severity to be weaned down based on clinical response Administration period: Two to Six months. This may be repeated based on recurrence/worsening of conjunctivitis

Arms:

Intervention arm (aliquoted plasma application into the conjunctiva)

Size

Primary endpoint

Mean difference in the size of pseudomembranous conjunctival lesion after 2 months of local administration of aliquoted plasma (reported as mean difference and percent difference from pre-administration size of pseudomembranous lesions )

24 months

Time to change in the size of pseudomembranous lesions by 50% or greater with topical administration of aliquoted allogenic plasma in the affected conjunctiva.

24 months. This knowledge will be aggregated in the patient data collection form

Difference in the visual acuity of affected eye after topical administration of aliquoted allogenic plasma into affected conjunctiva.

24 months. This information will be aggregated in data collection form

Number of participants with development of strabismus or other visual defects in affected eye

24 months. Information will be aggregated in data collection form

Eligibility criteria

Inclusion Criteria: * Patients under the age of 18 years * Diagnosis of plasminogen deficiency (Definition: clinical presence of pseudomembranous lesions and serum plasminogen level \< 50%) * Ocular involvement (Ligneous Conjunctivitis) due to plasminogen deficiency Exclusion Criteria: * Patient is 18 years or older * Patients with no plasminogen deficiency * Patients with no ocular manifestations of plasminogen deficiency

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['NA'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Only one patient (and their family) with laboratory diagnosis of plasminogen deficiency and ligneous conjunctivitis will be enrolled in this study.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 1, 'type': 'ESTIMATED'}}

Updated at

2023-12-15

1 organization

Organization

University of Saskatchewan