Recruiting

Plasminogen Deficiency

Sponsor:

University of Saskatchewan

Code:

NCT05404932

Conditions

Plasminogen Deficiency

Eligibility Criteria

Sex: All

Age: 0 - 18

Healthy Volunteers: Not accepted

Interventions

Aliquoted allogeneic donor plasma in patient's conjunctiva

Study Details

Brief summary:

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.

Conditions

Plasminogen Deficiency

Study ID

NCT05404932

Start date

Dec 15, 2022

Status verified date

Dec, 2023

Completion date

Dec 31, 2024

Anticipated

Primary completion date

Sep 30, 2024

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 18

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

1 participant

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Intervention arm (aliquoted plasma application into the conjunctiva)

This is the only arm of the study. The intervention is administration of aliquoted plasma provided by Canadian Blood Services in eye droppers (3 ml aliquots in 7 ml vials) in the patient's conjunctiva.

The duration of therapy can be 2 to 6 months depending on response The intra-ocular drops will be administered every 1-5 hours daily in the affected eye based on disease severity and may be repeated

Interventions

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

Primary outcome measure

  • 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 ) [ Time Frame: 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. [ Time Frame: 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. [ Time Frame: 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 [ Time Frame: 24 months. Information will be aggregated in data collection form ]

Central Contacts and Locations

Central contacts

Sarah Tehseen, MBBS MSc.

639-998-3972sarah.tehseen@saskhealthauthority.ca

Simona Meier, BSc, ACRP-CP.

306-978-8302simona.meier@usask.ca

Locations

University of Saskatchewan

Recruiting

Saskatoon, Saskatchewan, Canada, S7N0W8

Contacts

Sarah Tehseen, MBBS, MSc.

6399983972sarah.tehseen@saskhealthauthority.ca

Simona Meier

306-978-8302simona.meier@usask.ca

More Information

Sponsor

University of Saskatchewan

Last update posted

Dec 15, 2023

Last verified

Dec, 2023

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of Saskatchewan on 2023-12-15.