Mesenchymal Stem Cell Therapy of Dry Eye Disease in Patients With Sjögren's Syndrome

Phase 2

Completed

• Conditions: Keratoconjunctivitis Sicca, in Sjogren's Syndrome
• Interventions: Drug: ASCs; Drug: Cryostor CS10

• Registration Number: NCT04615455
• Lead Sponsor: Rigshospitalet, Denmark

Brief Summary

AMASS is a double-blinded randomized clinical trial with the purpose of investigating whether injection of allogeneic adipose-derived mesenchymal stem cells (ASCs) into the lacrimal gland (LG) results in increased ocular comfort compared to placebo.

Detailed Description

AMASS is a double-blinded randomized clinical trial which will be performed at the Department of Ophthalmology, University Hospital of Copenhagen, Denmark. 40 patients with severe aqueous deficient dry eye disease (ADDE) due to Sjögren's Syndrome (SS) will be recruited from the Dept. of Ophthalmology, Rigshospitalet, and allocated in ratio 1:1 to either injection of allogeneic adipose-derived mesenchymal stem cells (ASCs) or placebo (vehicle, Crystore CS10) into the lacrimal gland (LG) in one eye. We hypothesize that injection of allogeneic ASCs into the LG increases tear production and reduce inflammation resulting in increased ocular comfort compared to placebo.

Study Timeline

• Study Start: 2020-11-03
• Study First Submitted: 2020-11-03
• Study First Submitted QC: 2020-11-03
• Study First Posted: 2020-11-04
• Primary Completion: 2022-12-01
• Study Completion: 2023-01-01
• Status Verified: 2023-03
• Last Update Submitted: 2023-03-07
• Last Update Posted: 2023-03-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• Diagnosis of Sjögren's syndrome according to the 2016 American College of Rheumatology/European League Against Rheumatism classification criteria for primary Sjögren's syndrome
• OSDI-score ≥ 33
• Schirmer's test 1-5 mm/5 minutes
• NIKBUT < 10 sec

Exclusion Criteria

• LG volume on MRI < 0,2 cm3 in the study eye
• Previous treatment with ASCs or other stem cell products in the LG(s)
• Reduced immune response (e.g. HIV positive)
• Pregnancy or planned pregnancy within the next 2 years
• Breastfeeding
• Topical treatment with eye drops other than to treat dry eye disease (DED)
• Any other disease/condition judged by the investigator to be grounds for exclusion, such as infection in or around the eye

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Adipose tissue-derived mesenchymal stem cells (ASCs)	ASCs	One transconjunctival injection of allogeneic ASCs into the LG in one eye.
Placebo (vehicle, Cryostor CS10)	Cryostor CS10	One transconjunctival injection of Cryostor CS10 into the LG in one eye.

Primary Outcome Measures

Name	Time	Method
Ocular Surface Disease Index (OSDI)	4 months after treatment	The OSDI is a valid and reliable instrument for measuring dry eye disease severity

Secondary Outcome Measures

Name	Time	Method
Tear osmolarity	4 months after treatment	Change in tear osmolarity measured with TearLab™
Schirmer's I test	4 months after treatment	Change in tear production as evaluated with the Schirmer's I test
Non-invasive keratography tear break-up time (NIKBUT)	4 months after treatment	As measured with the Keratograph 5M (Oculus™)
Tear meniscus height (TMH)	4 months after treatment	As measured with the Keratograph 5M (Oculus™)
Oxford scale	4 months after treatment	Change in staining of the ocular surface (grade 0-5 with 0 being absent corneal staining and 5 being severe corneal staining)
HLA anti-bodies	12 months after treatment	Development of anti-human leucocyte antigen (HLA) anti-bodies evaluated with Luminex HLA anti-body screening; Development of anti-HLA anti-bodies evaluated with Luminex HLA anti-body screening; Development of donor-specific HLA-antibodies

Trial Locations

Locations (1)

Rigshospitalet; 🇩🇰 Copenhagen, DK, Denmark