E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
The trial will include participants with Aqueous Deficient Dry Eye Disease due to Sjögrens syndrom (International Classification of Diseases-10: DM 350A) |
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E.1.1.1 | Medical condition in easily understood language |
Sjögren's syndrome can cause Aqueous Deficient Dry Eye Disease which is a condition in which the tear production in the lacrimal gland is impaired. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Eye Diseases [C11] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10023350 |
E.1.2 | Term | Keratoconjunctivitis sicca |
E.1.2 | System Organ Class | 100000004853 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Our objective is to assess the efficacy of allogeneic adipose-tissue derived mesenchymal stem cells (ASCs) administered for lacrimal gland hypofunction in patients with ADDE due to Sjögren's syndrome |
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E.2.2 | Secondary objectives of the trial |
Our secondary objective is to assess whether injection of allogeneic ASCs results in any adverse reactions to the IMP |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Age > 18 years 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 |
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E.4 | Principal exclusion criteria |
Lacrimal gland volume on MRI < 0,2 cm3 in the study eye Previous treatment with ASCs or other stem cell products in the lacrimal gland(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 diease Any other disease/condition judged by the investigator to be grounds for exclusion, such as infection in or around the eye |
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E.5 End points |
E.5.1 | Primary end point(s) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1 week, 4 weeks, 4 months and 12 months after treatment |
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E.5.2 | Secondary end point(s) |
The secondary endpoints are change in TMH and NIKBUT-first using the K5M, change in tear production as evaluated with the Schirmer’s I test, change in tear osmolarity, and change in ocular surface staining evaluated with the Oxford score. All adverse events (AEs) and severe adverse events (SAEs) will be recorded and graded according to Common Terminology Criteria for Adverse Events (CTCAE). |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1 week, 4 weeks, 4 months and 12 months after treatment |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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Last participant's last visit 12 months after treatment |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |