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

Primary Purpose

Keratoconjunctivitis Sicca, in Sjogren's Syndrome

Status

Completed

Phase

Phase 2

Locations

Denmark

Study Type

Interventional

Intervention

ASCs

Cryostor CS10

About this trial

This is an interventional treatment trial for Keratoconjunctivitis Sicca, in Sjogren's Syndrome

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Rigshospitalet

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Adipose tissue-derived mesenchymal stem cells (ASCs)

Placebo (vehicle, Cryostor CS10)

Arm Description

One transconjunctival injection of allogeneic ASCs into the LG in one eye.

One transconjunctival injection of Cryostor CS10 into the LG in one eye.

Outcomes

Primary Outcome Measures

Ocular Surface Disease Index (OSDI)

The OSDI is a valid and reliable instrument for measuring dry eye disease severity

Secondary Outcome Measures

Non-invasive keratography tear break-up time (NIKBUT)

As measured with the Keratograph 5M (Oculus™)

Tear meniscus height (TMH)

As measured with the Keratograph 5M (Oculus™)

Schirmer's I test

Change in tear production as evaluated with the Schirmer's I test

Tear osmolarity

Change in tear osmolarity measured with TearLab™

Oxford scale

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

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

Full Information

NCT ID

NCT04615455

First Posted

November 3, 2020

Last Updated

March 7, 2023

Sponsor

Rigshospitalet, Denmark

1. Study Identification

Unique Protocol Identification Number

NCT04615455

Brief Title

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

Acronym

AMASS

Official Title

A Randomized Clinical Trial Evaluating Allogeneic Adipose-derived MesenchymAl Stem Cells as a Treatment of Dry Eye Disease in Patients With Sjögren's Syndrome

Study Type

Interventional

2. Study Status

Record Verification Date

March 2023

Overall Recruitment Status

Completed

Study Start Date

November 3, 2020 (Actual)

Primary Completion Date

December 1, 2022 (Actual)

Study Completion Date

January 1, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Rigshospitalet, Denmark

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Keratoconjunctivitis Sicca, in Sjogren's Syndrome

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantOutcomes Assessor

Allocation

Randomized

Enrollment

40 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Adipose tissue-derived mesenchymal stem cells (ASCs)

Arm Type

Experimental

Arm Description

One transconjunctival injection of allogeneic ASCs into the LG in one eye.

Arm Title

Placebo (vehicle, Cryostor CS10)

Arm Type

Placebo Comparator

Arm Description

One transconjunctival injection of Cryostor CS10 into the LG in one eye.

Intervention Type

Drug

Intervention Name(s)

ASCs

Other Intervention Name(s)

Allogeneic adipose-derived mesenchymal stem cells

Intervention Description

ASCs expanded from healthy donors. The ASC product contains 22 million ASCs/ml.

Intervention Type

Drug

Intervention Name(s)

Cryostor CS10

Intervention Description

CryoStor® CS10 freeze medium

Primary Outcome Measure Information:

Title

Ocular Surface Disease Index (OSDI)

Description

The OSDI is a valid and reliable instrument for measuring dry eye disease severity

Time Frame

4 months after treatment

Secondary Outcome Measure Information:

Title

Non-invasive keratography tear break-up time (NIKBUT)

Description

As measured with the Keratograph 5M (Oculus™)

Time Frame

4 months after treatment

Title

Tear meniscus height (TMH)

Description

As measured with the Keratograph 5M (Oculus™)

Time Frame

4 months after treatment

Title

Schirmer's I test

Description

Change in tear production as evaluated with the Schirmer's I test

Time Frame

4 months after treatment

Title

Tear osmolarity

Description

Change in tear osmolarity measured with TearLab™

Time Frame

4 months after treatment

Title

Oxford scale

Description

Change in staining of the ocular surface (grade 0-5 with 0 being absent corneal staining and 5 being severe corneal staining)

Time Frame

4 months after treatment

Title

HLA anti-bodies

Description

Time Frame

12 months after treatment

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Steffen Heegaard, MD, DMSc

Organizational Affiliation

Rigshospitalet, Denmark

Official's Role

Study Chair

Facility Information:

Facility Name

Rigshospitalet

City

Copenhagen

State/Province

ZIP/Postal Code

2200

Country

Denmark

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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