Keratoconjunctivitis Sicca, in Sjogren's SyndromePhase 2AMASSNCT04615455

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

A Phase 2 study testing ASCs and Cryostor CS10 in people with Keratoconjunctivitis Sicca, in Sjogren's Syndrome, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Keratoconjunctivitis Sicca, in Sjogren's Syndrome
What is being tested?: ASCs and Cryostor CS10
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 40 participants
Where: 1 siteDenmark
Study dates: 3 Nov 2020to 1 Jan 2023
Phase: Phase 2
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

1 site in 1 country.

Denmark1 site

Rigshospitalet
Copenhagen, DK, 2200

Other trials in Denmark

About this study

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

Read the 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.

Conditions: Keratoconjunctivitis Sicca, in Sjogren's Syndrome
Being tested: ASCs, Cryostor CS10
Medical subject headings: Keratoconjunctivitis Sicca

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ASCsDrug
Also called Allogeneic adipose-derived mesenchymal stem cells
ASCs expanded from healthy donors. The ASC product contains 22 million ASCs/ml.
Cryostor CS10Drug
CryoStor® CS10 freeze medium

Study groups in NCT04615455
Study group	Role	What they receive
Adipose tissue-derived mesenchymal stem cells (ASCs)	Experimental	One transconjunctival injection of allogeneic ASCs into the LG in one eye. Receives: Drug: ASCs
Placebo (vehicle, Cryostor CS10)	Placebo comparator	One transconjunctival injection of Cryostor CS10 into the LG in one eye. Receives: Drug: Cryostor CS10

What is being measured

The primary outcome is the main question the study is designed to answer.

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

Secondary outcomes (6)

Non-invasive keratography tear break-up time (NIKBUT)
As measured with the Keratograph 5M (Oculus™)
Measured: 4 months after treatment
Tear meniscus height (TMH)
As measured with the Keratograph 5M (Oculus™)
Measured: 4 months after treatment
Schirmer's I test
Change in tear production as evaluated with the Schirmer's I test
Measured: 4 months after treatment
Tear osmolarity
Change in tear osmolarity measured with TearLab™
Measured: 4 months after treatment
Oxford scale
Change in staining of the ocular surface (grade 0-5 with 0 being absent corneal staining and 5 being severe corneal staining)
Measured: 4 months after treatment
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
Measured: 12 months after treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

3 Nov 20201 Jan 2023

First posted4 Nov 2020
Study start3 Nov 2020
Primary completion1 Dec 2022
Study completion1 Jan 2023

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Keratoconjunctivitis Sicca, in Sjogren's SyndromeStudies of the same condition, open and under way.
More trials run by Copenhagen University Hospital, RigshospitaletThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Outcomes assessor
Enrolment: 40 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04615455
Sponsor's protocol ID: 2020-002804-38
Acronym: AMASS
Status last verified: Mar 2023
First submitted: 3 Nov 2020
Last updated: 8 Mar 2023
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

Larsen AC, Moller-Hansen M, Wiencke AK, Terslev L, Torp-Pedersen S, Heegaard S. Ultrasound-Guided Transcutaneous Injection in the Lacrimal Gland: A Description of Sonoanatomy and Technique. J Ocul Pharmacol Ther. 2023 May;39(4):275-278. doi: 10.1089/jop.2022.0156. Epub 2023 Mar 21.
PubMed 36944128

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Active, not recruitingPhase 3
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All Keratoconjunctivitis Sicca, in Sjogren's Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT04615455
Acronym / sponsor ID: AMASS / 2020-002804-38
Conditions: Keratoconjunctivitis Sicca, in Sjogren's Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 40 participants (actual)
Lead sponsor: Copenhagen University Hospital, RigshospitaletUniversity, hospital or other
Study start: 3 Nov 2020 (actual)
Primary completion: 1 Dec 2022 (actual)
Study completion: 1 Jan 2023 (actual)
First posted: 4 Nov 2020 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
Adipose tissue-derived mesenchymal stem cells (ASCs)	Experimental	Drug: ASCsOne transconjunctival injection of allogeneic ASCs into the LG in one eye.
Placebo (vehicle, Cryostor CS10)	Placebo comparator	Drug: Cryostor CS10One transconjunctival injection of Cryostor CS10 into the LG in one eye.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

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

Key exclusion criteria (7 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
ASCsAlso: Allogeneic adipose-derived mesenchymal stem cellsASCs expanded from healthy donors. The ASC product contains 22 million ASCs/ml.	Drug	Adipose tissue-derived mesenchymal stem cells (ASCs)
Cryostor CS10CryoStor® CS10 freeze medium	Drug	Placebo (vehicle, Cryostor CS10)

Primary outcome

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

6 secondary outcomes

Non-invasive keratography tear break-up time (NIKBUT)
Time frame: 4 months after treatment
Tear meniscus height (TMH)
Time frame: 4 months after treatment
Schirmer's I test
Time frame: 4 months after treatment
Tear osmolarity
Time frame: 4 months after treatment
Oxford scale
Time frame: 4 months after treatment
HLA anti-bodies
Time frame: 12 months after treatment

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Mar 2023.
Derived from this study: PubMed 36944128Larsen AC, Moller-Hansen M, Wiencke AK, Terslev L, Torp-Pedersen S, Heegaard S. Ultrasound-Guided Transcutaneous Injection in the Lacrimal Gland: A Description of Sonoanatomy and Technique. J Ocul Pharmacol Ther. 2023 May;39(4):275-278. doi: 10.1089/jop.2022.0156. Epub 2023 Mar 21.
Individual participant dataSharing planned: No

Related trials for Keratoconjunctivitis Sicca, in Sjogren's Syndrome

Not yet recruitingPhase 1
R-2487 in Patients With Sjogren's Syndrome (SjD)
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RecruitingPhase 1
A Trial in Healthy Adult Participants and Adults With Autoimmune Diseases to Test How HBM7020 is Tolerated and How it Behaves in the Body
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Recruiting
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Active, not recruitingPhase 3
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RecruitingPhase 2
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Steffen Heegaard, MD, DMSc study chair

Copenhagen University Hospital, Rigshospitalet

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ASCs and Cryostor CS10 in people with Keratoconjunctivitis Sicca, in Sjogren's Syndrome, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 8 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Ocular Surface Disease Index (OSDI) (time frame: 4 months after treatment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04615455, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Mar 2023.